| 1 |
NCT02926963 |
Terminated |
Generation of Powerful Biological Tools for Understanding the Pathophysiology of Chronic Granulomatous Disease. |
- Chronic Granulomatous Disease
|
- Other: samples collection : hair and skin biopsy
|
Interventional
|
Not Applicable |
- University Hospital, Grenoble
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- to study the impact of protein deficits Nox2 and p22phox, in the physiopathology of neurons from inducible pluripotent bone marrow cells (iPSC)
- To study the impact of protein deficits Nox2 and p22phox on cytochrome b558 synthesis process of phagocytes from inducible pluripotent bone marrow cells ( iPSC ).
- To study the impact of protein deficits Nox2 and p22phox , at the physiology of fibroblasts and their transformation into myofibroblasts
- Constitute cellular models of different types of CGD for future physiopathological studies and therapeutic trials
|
3 |
All |
6 Months and older (Child, Adult, Older Adult) |
NCT02926963 |
38RC09.018
2009-A00944-53 |
FIBRO CGD |
October 2010 |
June 2017 |
June 2017 |
October 6, 2016 |
September 29, 2017 |
|
- University Hospital, Grenoble Alpes
Grenoble, Cs10217, France
|
|
| 2 |
NCT03080480 |
Recruiting |
Pioglitazone Therapy for Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
|
|
Interventional
|
Phase 1
Phase 2 |
- Children's Hospital of Fudan University
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- efficiency of Pioglitazone
- Incidence of Treatment-Emergent Adverse Events
|
100 |
All |
1 Month to 18 Years (Child, Adult) |
NCT03080480 |
PTSICGD |
|
September 1, 2017 |
August 31, 2020 |
August 31, 2020 |
March 15, 2017 |
December 6, 2018 |
|
- Children's Hospital of Fudan University
Shanghai, Shanghai, China
|
|
| 3 |
NCT00927134 |
Completed |
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children |
- Chronic Granulomatous Disease
|
- Genetic: retroviral SF71-gp91phox transduced CD34+ cells
|
Interventional
|
Phase 1
Phase 2 |
- University of Zurich
- Goethe University
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- eradication of pre-existing therapy refractory bacterial and/or fungal infections
- Reconstitution of ROS production by peripheral blood cells
|
2 |
Male |
1 Year to 18 Years (Child, Adult) |
NCT00927134 |
PedsZürich_GT05 |
XCGDinChildren |
June 2004 |
December 2010 |
September 2011 |
June 24, 2009 |
September 27, 2011 |
|
- University Children's Hospital
Zürich, Switzerland
|
|
| 4 |
NCT03548818 |
Recruiting |
Role of Interferon-gamma 1-b (IFN-γ) on Cells of the Innate Immune System: Functional, Biochemical and Gene Expression Studies in Patients With Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
|
- Drug: Interferon Gamma-1B
|
Observational
|
|
- University of Colorado, Denver
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Changes in Neutrophil bacterialcidal activity in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ.
- Changes in Gene expression as measured with RNA and affimetrics gene chips in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ.
- Differences in Neutrophil biochemical studies in patients with Chronic Granulomatous Disease (CGD) on standard treatment schedule and dose of IFN-γ in comparison to results for the patients off IFN-γ.
|
20 |
All |
5 Years to 60 Years (Child, Adult) |
NCT03548818 |
17-1676 |
|
May 16, 2018 |
December 2018 |
June 2019 |
June 7, 2018 |
June 14, 2018 |
|
- University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
|
|
| 5 |
NCT00778882 |
Active, not recruiting |
Gene Therapy for Chronic Granulomatous Disease in Korea |
- Chronic Granulomatous Disease
|
|
Interventional
|
Phase 1
Phase 2 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The incidence of adverse events through 1 year
- RCR, insertional mutagenesis, immune response against normal gp91 protein
- Safety and efficacy of fludarabine/busulfan conditioning
- (and 2 more...)
|
2 |
Male |
Child, Adult, Older Adult |
NCT00778882 |
VM106-KR-01 |
|
January 2007 |
October 2008 |
October 2022 |
October 24, 2008 |
February 11, 2016 |
|
- Seoul National University Hospital
Seoul, Korea, Republic of
|
|
| 6 |
NCT02116764 |
Recruiting |
Analysis of Patients Treated for Chronic Granulomatous Disease Since January 1, 1995 |
- Chronic Granulomatous Disease
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- The primary endpoint in this study is overall survival
- Survival of HCT subjects vs. conventional therapy subjects, immune reconstitution, engraftment, andclinical outcomes such as infection, autoimmune disease and inflammatory complications, GvHD, growth, and quality of life.
|
140 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT02116764 |
140091
14-I-0091 |
|
April 15, 2014 |
December 1, 2019 |
December 1, 2019 |
April 17, 2014 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 7 |
NCT02282904 |
Active, not recruiting |
Haploidentical Transplant for People With Chronic Granulomatous Disease Using Post Transplant Cyclophosphamide |
- Chronic Granulomatous Disease
|
- Drug: Pre-Transplant Conditioning Medications
- Other: Haploidentical Cellular Infusion
- Drug: Post-Trasnplant Medications
- Radiation: Total Body Irradiation
|
Interventional
|
Early Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To use a subablative conditioning regimen followed by post-transplant cyclophosphamide to attain an engraftment rate of 100% with no occurrence of acute Grade 3 or higher GvHD (graft versus host disease).
- Stable Chimerism as indicated by 30-50% myeloid engraftment and 50% lymphoid engraftment as assessed by 1 year post transplant
- Immune reconstitution levels with DHR as a marker of normal neutrophil function by 1 year post transplant.
|
12 |
All |
2 Years to 65 Years (Child, Adult, Older Adult) |
NCT02282904 |
150007
15-I-0007 |
|
November 4, 2014 |
March 1, 2022 |
March 1, 2023 |
November 5, 2014 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 8 |
NCT00394316 |
Terminated |
Gene Therapy for Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
|
- Drug: Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells
|
Interventional
|
Early Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Can conditioning with busulfan improve gene therapy outcomes
- 1) To evaluate further the safety of gene therapy2) To monitor long-term results of gene therapy with conditioning
|
3 |
Male |
3 Years to 55 Years (Child, Adult) |
NCT00394316 |
070017
07-I-0017 |
|
October 30, 2006 |
April 8, 2014 |
April 8, 2014 |
November 1, 2006 |
July 4, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 9 |
NCT00578643 |
Completed
Has Results |
Matched Unrelated or Non-Genotype Identical Related Donor Transplantation For Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
|
- Drug: Busulfan
- Biological: Alemtuzumab
- Drug: Cyclophosphamide
- (and 3 more...)
|
Interventional
|
Phase 2 |
- Baylor College of Medicine
- Texas Children's Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage of Participants With Engraftment
- Number of Patients That Have Complete Donor Chimerism After Transplant.
- Number of Patients That Have Acute GVHD and Regimen Related Morbidity/Mortality Post Transplant.
- Number of Patients That Have Chronic GVHD and Regimen Related Morbidity/Mortality Post Transplant.
|
15 |
All |
Child, Adult, Older Adult |
NCT00578643 |
14771-MUNCHR |
MUNCHR |
March 2004 |
July 31, 2017 |
November 24, 2017 |
December 21, 2007 |
November 9, 2018 |
November 9, 2018 |
- Texas Children's Hospital
Houston, Texas, United States
|
|
| 10 |
NCT02757911 |
Recruiting |
Gene Therapy for X-linked Chronic Granulomatous Disease |
- X-Linked Chronic Granulomatous Disease
|
- Genetic: X vivo gene therapy
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety as measured by the incidence of adverse events
- Restoration and stability over time of the NADPH functioning granulocytes assessed by a Dihydrorhodamine (DHR) flow cytometry test
- Clinical improvement
- (and 2 more...)
|
5 |
Male |
24 Months and older (Child, Adult, Older Adult) |
NCT02757911 |
G1XCGD.02 |
|
March 2016 |
December 2023 |
December 2023 |
May 2, 2016 |
May 30, 2018 |
|
- Hôpital Necker Enfants Malades
Paris, France
|
|
| 11 |
NCT00799071 |
Completed |
Pharmacokinetics of Posaconazole in Children With Chronic Granulomatous Disease (CGD) |
- Chronic Granulomatous Disease
|
|
Interventional
|
Phase 2 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Posaconazole trough levels
- adverse events monitoring
|
12 |
All |
2 Years to 16 Years (Child) |
NCT00799071 |
UMCN-AKF 08.01 |
iPOD |
February 2009 |
July 2010 |
August 2010 |
November 27, 2008 |
May 31, 2012 |
|
- Radboud University Medical Centre Nijmegen
Nijmegen, Gelderland, Netherlands - AMC
Amsterdam, Netherlands
|
|
| 12 |
NCT01855685 |
Recruiting |
Gene Therapy for X-linked Chronic Granulomatous Disease (X-CGD) |
- X-Linked Chronic Granulomatous Disease
|
- Genetic: X vivo gene therapy
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety of the procedure as measured by the incidence of adverse events
- Restoration and stability over time of the NADPH functioning granulocytes assessed by a DHR test
- Normalisation of nutritional status, growth, development, severe infection and/or inflammatory complication which recommended patient's inclusion
- (and 2 more...)
|
5 |
Male |
6 Months and older (Child, Adult, Older Adult) |
NCT01855685 |
G1XCGD.01 |
CGD |
February 2013 |
March 2020 |
March 2020 |
May 16, 2013 |
May 21, 2018 |
|
- University Hospital Frankfurt and Institute for Biomedical Research, Georg-Speyer-Haus
Frankfurt, Germany - University Children's Hospital Zürich
Zurich, Switzerland - University College London Hospital (UCLH)
London, United Kingdom - (and 2 more...)
|
|
| 13 |
NCT00001317 |
Completed |
A Phase IV Study of Recombinant Human Gamma Interferon in Patients With Chronic Granulomatous Diseases of Childhood |
- Chronic Granulomatous Disease
|
|
Interventional
|
Phase 4 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
100 |
All |
Child, Adult, Older Adult |
NCT00001317 |
920186
92-I-0186 |
|
May 1992 |
|
July 2001 |
December 10, 2002 |
March 4, 2008 |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
|
|
| 14 |
NCT00023192 |
Completed |
Treatment of Chronic Granulomatous Disease With Allogeneic Stem Cell Transplantation Versus Standard of Care |
- Chronic Granulomatous Disease
|
- Drug: T-Cell Depleted & CD34+Select/w/StemCell Enriched Product
|
Interventional
|
Phase 3 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
60 |
All |
Child, Adult, Older Adult |
NCT00023192 |
010242
01-I-0242 |
|
August 2001 |
|
June 2004 |
August 30, 2001 |
March 4, 2008 |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
|
|
| 15 |
NCT01851460 |
Recruiting |
Radiofrequency Ablation for Liver Abscesses From Chronic Granulomatous Disease |
- Chronic Granulomatous Disease (CGD)
|
- Device: Cool-tip RF Ablation System
|
Interventional
|
Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary objective of this study is to determine the safety ofradiofrequency ablation (RFA) in the treatment of liver abscesses insubjects with chronic granulomatous disease (CGD).
- To determine if RFA is a reasonable treatment option for patients with liver abscesses who are not suitable candidates for completely curative hepatic surgery
- To compare the recovery outcomes of patients undergoing RFA versus historical controls for patients undergoing surgery for treatment of liver abscesses
|
30 |
All |
18 Years to 75 Years (Adult, Older Adult) |
NCT01851460 |
130117
13-I-0117 |
|
May 8, 2013 |
January 1, 2020 |
January 1, 2021 |
May 10, 2013 |
August 15, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 16 |
NCT03645486 |
Recruiting |
Lentiviral Gene Therapy for CGD |
- Chronic Granulomatous Disease
|
- Genetic: Infusion of lentiviral TYF-CGD-modified autologous stem cells
|
Interventional
|
Not Applicable |
- Shenzhen Geno-Immune Medical Institute
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall survival
- Gene marking in bone marrow cells
- Change in infection frequency
- Recovery of immune function
|
10 |
All |
Child, Adult, Older Adult |
NCT03645486 |
GIMI-IRB-18004 |
|
July 1, 2018 |
June 30, 2021 |
December 31, 2021 |
August 24, 2018 |
August 24, 2018 |
|
- Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China
|
|
| 17 |
NCT00001765 |
Completed |
Stem Cell Transplant Following Low-Intensity Chemotherapy to Treat Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
|
- Device: Nexell Isolex with T-cell Depletion
- Device: Baxter isolex 300i
|
Interventional
|
Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
60 |
All |
Child, Adult, Older Adult |
NCT00001765 |
980104
98-I-0104 |
|
April 1998 |
|
February 2005 |
November 4, 1999 |
March 4, 2008 |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
|
|
| 18 |
NCT00006417 |
Completed |
Modified Stem Cell Transplantation Procedure for Treating Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
|
- Procedure: Stem cell transplantation
|
Interventional
|
Phase 2 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
10 |
All |
Child, Adult, Older Adult |
NCT00006417 |
010013
01-I-0013 |
|
October 2000 |
|
November 2004 |
October 25, 2000 |
March 4, 2008 |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
|
|
| 19 |
NCT01338675 |
Unknown † |
Targeted Busulfan, Fludarabine Conditioning Regimen for Hematopoietic Stem Cell Transplantation in Chronic Granulomatous Disease(CGD) |
- Chronic Granulomatous Disease
|
|
Interventional
|
Phase 1
Phase 2 |
- Seoul National University Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Engraftment rate
- Transplantation-related mortality and toxicities
|
5 |
All |
Child, Adult, Older Adult |
NCT01338675 |
SNUCH-SCT -1002 |
|
January 2011 |
December 2013 |
December 2013 |
April 19, 2011 |
November 19, 2013 |
|
- Seoul National University Hospital
Seoul, Daehangno, Jongno-gu, Korea, Republic of
|
|
| 20 |
NCT03547830 |
Recruiting |
Plerixafor/G-CSF as Additional Agents for Conditioning Before HSCT in CGD Patients |
- Chronic Granulomatous Disease
|
- Drug: Plerixafor
- Drug: Gcsf
|
Interventional
|
Phase 2 |
- Federal Research Institute of Pediatric Hematology, Oncology and Immunology
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Event free survival
- 1. Overall survival
- Proportion of patients with full/mixed donor chimerism
- (and 3 more...)
|
17 |
All |
1 Month to 24 Years (Child, Adult) |
NCT03547830 |
NCPHOI-2018-02 |
|
May 29, 2018 |
January 1, 2023 |
January 1, 2023 |
June 6, 2018 |
June 11, 2018 |
|
- Dmitry Rogachev National Research Center of Pediatric Hematology, Oncology and Immunology
Moscow, Russian Federation
|
|
| 21 |
NCT02082353 |
Recruiting |
Patients Treated for Chronic Granulomatous Disease (CGD) Since 1995 |
- Granulomatous Disease, Chronic
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- Rare Diseases Clinical Research Network
- Primary Immune Deficiency Treatment Consortium (PIDTC)
|
NIH / Other |
- Observational Model: Cohort
- Time Perspective: Other
|
- Death
- Engraftment
- Quality of Life Measures
- (and 2 more...)
|
1480 |
All |
Child, Adult, Older Adult |
NCT02082353 |
DAIT RDCRN PIDTC-6903 |
|
June 2014 |
August 2019 |
August 2019 |
March 10, 2014 |
November 21, 2018 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Phoenix Children's Hospital
Phoenix, Arizona, United States - Children's Hospital Los Angeles
Los Angeles, California, United States - (and 41 more...)
|
|
| 22 |
NCT02234934 |
Recruiting |
Study of Gene Therapy Using a Lentiviral Vector to Treat X-linked Chronic Granulomatous Disease |
- Granulomatous Disease, Chronic, X-linked
|
- Biological: Lentiviral G1XCGD Gene Therapy
|
Interventional
|
Phase 1
Phase 2 |
- University of California, Los Angeles
- Boston Children’s Hospital
- National Institute of Allergy and Infectious Diseases (NIAID)
- (and 2 more...)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
|
10 |
Male |
23 Months and older (Child, Adult, Older Adult) |
NCT02234934 |
G1XCGD |
|
January 2015 |
December 2018 |
December 2020 |
September 9, 2014 |
February 5, 2018 |
|
- University of California, Los Angeles (UCLA)
Los Angeles, California, United States - National Institutes of Health
Bethesda, Maryland, United States - Children's Hospital Boston
Boston, Massachusetts, United States
|
|
| 23 |
NCT00564759 |
Unknown † |
Gene Therapy for Chronic Granulomatous Disease |
- Granulomatous Disease, Chronic
|
- Drug: retroviral SF71-gp91phox transduced CD34+ cells
|
Interventional
|
Phase 1
Phase 2 |
- Johann Wolfgang Goethe University Hospital
- German Federal Ministry of Education and Research
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
|
- safety, toxicity and feasibility
- Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
|
2 |
Male |
18 Years and older (Adult, Older Adult) |
NCT00564759 |
58/59
DeReG 31
KSG 31 |
CGD |
January 2004 |
|
December 2008 |
November 28, 2007 |
November 28, 2007 |
|
- University Hospital, Hematology
Frankfurt, Germany
|
|
| 24 |
NCT01381003 |
Withdrawn |
Lentiviral Gene Therapy for X-Linked Chronic Granulomatous Disease (X-CGD) |
- Granulomatous Disease, Chronic, X-linked, Variant
|
- Genetic: pCCLchimGp91s lentiviral vector transduced CD34+ cells infusion
|
Interventional
|
Phase 1
Phase 2 |
- Great Ormond Street Hospital for Children NHS Foundation Trust
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall survival following gene therapy
- Reduction in frequency of infections
- Long term immune reconstitution
|
0 |
Male |
up to 30 Years (Child, Adult) |
NCT01381003 |
11-MI-03 |
|
November 2011 |
November 2016 |
November 2016 |
June 27, 2011 |
June 4, 2012 |
|
- Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom
|
|
| 25 |
NCT00001476 |
Completed |
Gene Therapy for Chronic Granulomatous Diseases - Long-term Follow-up |
- Chronic Granulomatous Disease
- Communicable Disease
|
- Drug: Gene Therapy Method for CGD
- Device: Isolex 300i Magnetic Cell Selector
|
Interventional
|
Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
14 |
All |
5 Years and older (Child, Adult, Older Adult) |
NCT00001476 |
950134
95-I-0134 |
|
June 1, 1995 |
December 13, 2010 |
December 13, 2010 |
November 4, 1999 |
July 2, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 26 |
NCT00325078 |
Terminated
Has Results |
Infliximab to Treat Crohn'S-like Inflammatory Bowel Disease in Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
- Crohn'S-like IBD
- Inflammatory Bowel Disease (IBD)
|
|
Interventional
|
Phase 1
Phase 2 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety of Study Drug
- Efficacy of Treatment With Study Drug
|
40 |
All |
10 Years and older (Child, Adult, Older Adult) |
NCT00325078 |
060160
06-I-0160 |
|
May 2006 |
June 2012 |
June 2012 |
May 12, 2006 |
December 29, 2015 |
December 29, 2015 |
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 27 |
NCT02609932 |
Active, not recruiting |
Effect of IFN-γ on Innate Immune Cells |
- Chronic Granulomatous Disease
|
- Drug: Administration of drug (Interferon-gamma 1-b) subcutaneously
|
Interventional
|
Phase 1 |
- University of Colorado, Denver
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Change in Neutrophil Nox2 activity.
- Change in Plasma IL-10 and Neuropterin concentration.
- Change in Neutrophil Gene Expression Analysis.
- (and 5 more...)
|
20 |
All |
18 Years to 60 Years (Adult) |
NCT02609932 |
15-1643
UL1TR001082 |
|
July 2016 |
August 2018 |
August 2019 |
November 20, 2015 |
September 6, 2018 |
|
- University of Colorado Denver, Anschutz Medical Campus
Aurora, Colorado, United States
|
|
| 28 |
NCT02231996 |
Unknown † |
Chronic Granulomatous Disease Study in China |
- Granulomatous Disease, Chronic
|
|
Observational
|
|
- Shanghai Children's Medical Center
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
|
50 |
All |
up to 18 Years (Child, Adult) |
NCT02231996 |
CGD-20140829 |
|
September 2014 |
August 2016 |
|
September 4, 2014 |
September 5, 2014 |
|
- Shanghai children's medical center
Shanghai, Shanghai, China
|
|
| 29 |
NCT01906541 |
Recruiting |
Gene Therapy for X-CGD |
- X-linked Chronic Granulomatous Disease
|
- Genetic: ex-vivo gene-therapy
|
Interventional
|
Phase 1
Phase 2 |
- Hubert Serve, Prof., MD
- Johann Wolfgang Goethe University Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Transduction rate of granulocyte colony-stimulating factor (G-CSF) mobilized peripheral CD34+ cells from CGD patients with a SIN gamma retroviral vector
- Engraftment rate of the transduced CD34+ cells in the patients
- Long-term expression of the transgene (rate of gp91phox positive cells) in circulating cells in the peripheral blood
- (and 6 more...)
|
5 |
All |
18 Years and older (Adult, Older Adult) |
NCT01906541 |
X-CGD |
|
July 2013 |
December 2013 |
December 2019 |
July 24, 2013 |
August 2, 2013 |
|
- University Hospital Frankfurt
Frankfurt am Main, Germany
|
|
| 30 |
NCT01147042 |
Terminated
Has Results |
Biochemical Response to Interferon-Gamma in Subjects With Specific Gene Mutation in Chronic Granulomatous Disease |
- IFN-Gamma Therapy
- CGD Gene Mutation
- CGD Response to IFNg
- (and 2 more...)
|
|
Interventional
|
Phase 4 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Allocation: Non-Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Basal and PMA-stimulated O2 Production Detected by Ferricytochrome c Reduction in Neutrophils
|
2 |
All |
Child, Adult, Older Adult |
NCT01147042 |
100123
10-I-0123 |
|
May 18, 2010 |
November 14, 2014 |
November 14, 2014 |
June 22, 2010 |
September 20, 2017 |
September 20, 2017 |
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 31 |
NCT00001405 |
Recruiting |
Use of G-CSF to Obtain Blood Cell Precursors |
- Chronic Granulomatous Disease
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- 1. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC patients with any inherited primary immune deficiency (PID) where PBSC from these patients may be designated entirely or in part for future clinical t...
- 2. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC from healthy volunteers, which will be designated entirely for laboratory research.
- 3. To efficiently and safely mobilize to the peripheral blood and apheresis collect CD34+ PBSC from healthy sibling or other related donor of patients with PID in need of an allogeneic stem cell transplant where PBSC from these subjects are desi...
- (and 3 more...)
|
450 |
All |
2 Years to 70 Years (Child, Adult, Older Adult) |
NCT00001405 |
940073
94-I-0073 |
|
February 9, 1994 |
|
|
November 4, 1999 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 32 |
NCT03055247 |
Recruiting |
Combination of Ibuprofen, G-CSF and Plerixafor as Stem Cells Mobilization Regimen in Patients Affected by X-CGD |
- Chronic Granulomatous Disease X-linked (X-CGD)
|
- Drug: Ibuprofen
- Drug: Myelostim
- Drug: Mozobil
|
Interventional
|
Phase 2 |
- IRCCS San Raffaele
- Fondazione Telethon
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Basic Science
|
- Percentage of patients experiencing adverse events
- Number of CD34+ collected per body weight after the last LP
- Change in number of CD34+ cells in PB before and after administration of Ibuprofen
- (and 3 more...)
|
3 |
Male |
18 Years to 45 Years (Adult) |
NCT03055247 |
2015-002356-27 |
XCGD-MOBI |
November 2015 |
September 2017 |
August 2018 |
February 16, 2017 |
May 30, 2017 |
|
- Ospedale Pediatrico Bambino Gesù
Rome, Lazio, Italy - Ospedale San Raffaele
Milan, Lombardia, Italy
|
|
| 33 |
NCT01063309 |
Active, not recruiting |
Non-Invasive Assessment of Atherosclerosis in Patients With CGD and Other Disorders of the Immune System |
- Chronic Granulomatous Disease (CGD)
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- To assess the prevalence of atherosclerotic disease in patients with disorders of the immune system compared to the general population. The amount of atherosclerotic disease and its degree of inflammation will be assessed.
- To assess circulating biomarkers (including lipid profile, MPO, CRP, ESR, TNF alpha, IL-8, and IFN gamma) and demographic factors (e.g., age, gender, smoking, family history) to determine if they are correlated with the development of atheroscle...
|
156 |
All |
18 Years and older (Adult, Older Adult) |
NCT01063309 |
100029
10-I-0029 |
|
December 11, 2009 |
|
|
February 5, 2010 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 34 |
NCT02233036 |
Completed |
Evaluating the Transition From Pediatric to Adult Care Among Adolescents With Chronic Granulomatous Disease |
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Retrospective
|
- Successful transition to adult care
- To explore strategies that young adult patients believe mayenhance the process of transition from pediatric to adult care
|
33 |
All |
18 Years to 26 Years (Adult) |
NCT02233036 |
999914088
14-I-N088 |
|
March 26, 2014 |
January 11, 2016 |
January 10, 2017 |
September 8, 2014 |
April 5, 2018 |
|
- National Institute of Allergy and Infectious Diseases (NIAID), 9000 Rockville Pi
Bethesda, Maryland, United States
|
|
| 35 |
NCT02629120 |
Suspended |
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease |
- X-Linked Chronic Granulomatious Disease
|
- Drug: Campath
- Drug: Busulfan
- Other: allogeneic peripheral blood allograft infusion
- Drug: cyclophosphamide
|
Interventional
|
Early Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Reduced incidence of graft failure or rejection (Engraftment as defined by >20% engraftment by oxidase positive neutrophils in at least 95% of participants by Day 100)
- Same or reduced rate of acute Graft versus Host Disease of <20% in subjects who will be receiving a high stem cell dose in combination with post cyclophosphamide.
- Establish stable mixed chimerism.
- Improve rapidity of immune reconstitution.
|
12 |
All |
4 Years to 65 Years (Child, Adult, Older Adult) |
NCT02629120 |
160032
16-I-0032 |
|
December 10, 2015 |
December 31, 2025 |
December 31, 2030 |
December 14, 2015 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 36 |
NCT00001280 |
Completed |
Itraconazole for the Prevention of Fungal Infections in Chronic Granulomatous Disease |
|
|
Interventional
|
Phase 2 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
100 |
All |
Child, Adult, Older Adult |
NCT00001280 |
910064
91-I-0064 |
|
January 1991 |
|
March 2001 |
December 10, 2002 |
March 4, 2008 |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
|
|
| 37 |
NCT03278912 |
Recruiting |
Natural History of Intestinal Inflammation in People With Primary Immune Dysregulations |
- Chronic Granulomatous Disease
- Inflammatory Bowel Disease
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Disease specific intestinal microbiome signatures, and related localized and systemic immune responses.
- Changes in microbiome signatures.
- Changes in systemic and tissue-specific markers of innate and adaptive immunity.
- (and 2 more...)
|
339 |
All |
3 Years and older (Child, Adult, Older Adult) |
NCT03278912 |
170163
17-I-0163 |
|
January 24, 2019 |
July 1, 2030 |
April 30, 2031 |
September 12, 2017 |
January 21, 2019 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
|
| 38 |
NCT00005933 |
Completed |
Learning and Behavior Problems in Children With Chronic Granulomatous Disease and Related Disorders |
- Chediak Higashi Syndrome
- Chronic Granulomatous Disease
- Job's Syndrome
- Leukocyte Disorder
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
150 |
All |
Child, Adult, Older Adult |
NCT00005933 |
000164
00-I-0164 |
|
June 2000 |
|
July 2005 |
July 5, 2000 |
March 4, 2008 |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
Bethesda, Maryland, United States
|
|
| 39 |
NCT02108028 |
Recruiting |
Study of Voicing My CHOiCES as a Tool for Advanced Care Planning in Young Adults With Cancer |
- HIV
- Cancer
- Chronic Granulomatous Disease
- Dock 8 Deficiency
|
|
Observational
|
|
- National Cancer Institute (NCI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Preceived helpfulness
- Helpfulness vs living & parental status
- Benefit vs Burden
- Feasibility
|
230 |
All |
18 Years to 100 Years (Adult, Older Adult) |
NCT02108028 |
140079
14-C-0079 |
|
April 3, 2014 |
December 1, 2019 |
December 31, 2020 |
April 9, 2014 |
December 17, 2018 |
|
- Children's Hospital of Orange County
Orange, California, United States - Childrens National Medical Center
Washington, District of Columbia, United States - Moffitt Cancer Center
Tampa, Florida, United States - (and 5 more...)
|
|
| 40 |
NCT00010361 |
Completed |
Study of Total Body Irradiation and Fludarabine Followed By Allogeneic Peripheral Blood Stem Cell or Bone Marrow Transplantation in Combination With Cyclosporine and Mycophenolate Mofetil in Patients With Inherited Disorders |
- Metabolism, Inborn Errors
- Granulomatous Disease, Chronic
|
- Drug: cyclosporine
- Drug: fludarabine
- Drug: mycophenolate mofetil
|
Interventional
|
Not Applicable |
- Fred Hutchinson Cancer Research Center
- Office of Rare Diseases (ORD)
|
Other |
- Primary Purpose: Treatment
|
|
20 |
All |
up to 55 Years (Child, Adult) |
NCT00010361 |
199/15577
FHCRC-1475.00 |
|
November 2000 |
April 2007 |
|
February 2, 2001 |
December 9, 2014 |
|
- Fred Hutchinson Cancer Research Center
Seattle, Washington, United States
|
|
| 41 |
NCT00033982 |
Completed |
Posaconazole to Treat Invasive Fungal Infections |
- Granulomatous Disease, Chronic
- Job's Syndrome
- Infection
|
|
Interventional
|
Phase 3 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
50 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT00033982 |
020171
02-I-0171 |
|
April 11, 2002 |
|
February 20, 2007 |
April 19, 2002 |
July 2, 2017 |
|
- Schering-Plough Research Institute
Kenilworth, New Jersey, United States
|
|
| 42 |
NCT02909244 |
Completed |
Study of Gut Microbiota in Primary Immune Deficiency, Possibly Associated With Inflammatory Bowel Disease |
- Primary Immune Deficiencies
|
- Biological: Biological sampling
|
Observational
|
|
- Imagine Institute
- Saint Antoine University Hospital
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Observational Model: Other
- Time Perspective: Prospective
|
- Number of factors associated with the onset of an inflammatory colitis in patients with three Primary Immuno-Deficiencies, identified in the study population
|
51 |
All |
Child, Adult, Older Adult |
NCT02909244 |
IMIS2014-02 |
DIPobiota |
February 2015 |
February 2017 |
February 2017 |
September 21, 2016 |
August 25, 2017 |
|
- CHRU
Lille, France - Saint-Louis Hospital
Paris, France - Necker - Enfants Malades Hospital
Paris, France
|
|
| 43 |
NCT01953016 |
Recruiting |
Participation in a Research Registry for Immune Disorders |
- Primary Immunodeficiencies
- APECED
- CGD (Chronic Granulomatous Disease)
- (and 2 more...)
|
|
Observational
|
|
- National Human Genome Research Institute (NHGRI)
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Other
|
|
1000 |
All |
up to 150 Years (Child, Adult, Older Adult) |
NCT01953016 |
130199
13-HG-0199 |
|
September 30, 2013 |
May 1, 2020 |
May 1, 2020 |
September 30, 2013 |
January 4, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 44 |
NCT00001515 |
Completed |
Diagnostic Effectiveness of Virtual Bronchoscopy |
- Bronchogenic Carcinoma
- Chronic Granulomatous Disease
- Job's Syndrome
- (and 2 more...)
|
- Device: virtual bronchoscopy
|
Interventional
|
Phase 1 |
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
120 |
All |
Child, Adult, Older Adult |
NCT00001515 |
960021
96-CC-0021 |
|
December 1995 |
|
November 2001 |
November 4, 1999 |
March 4, 2008 |
|
- Warren G. Magnuson Clinical Center (CC)
Bethesda, Maryland, United States
|
|
| 45 |
NCT00471250 |
Recruiting |
Collection of Lung Fluid and Tissue Samples for Research |
- Mycobacterium Infections, Atypical
- Granulomatous Disease, Chronic
- Job's Syndrome
- Influenza, Human
|
|
Observational
|
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
|
550 |
All |
10 Years to 75 Years (Child, Adult, Older Adult) |
NCT00471250 |
070142
07-H-0142 |
|
May 7, 2007 |
|
|
May 9, 2007 |
August 9, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 46 |
NCT01998633 |
Completed
Has Results |
Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204) |
- Hemophagocytic Lymphohistiocytosis
- Chronic Active Epstein-Barr Virus Infection
- Chronic Granulomatous Disease
- (and 3 more...)
|
- Biological: Hematopoietic Stem Cell Transplant
|
Interventional
|
Phase 2 |
- Medical College of Wisconsin
- National Heart, Lung, and Blood Institute (NHLBI)
- Blood and Marrow Transplant Clinical Trials Network
- (and 2 more...)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Percentage of Participants With Overall Survival (OS)
- Percentage of Participants With Overall Survival (OS) by Disease Type
- Percentage of HLH Participants With HLH Reactivation Post-Transplant
- (and 8 more...)
|
47 |
All |
4 Months to 45 Years (Child, Adult) |
NCT01998633 |
BMTCTN1204
2U10HL069294-11
5U24CA076518 |
RICHI |
December 2013 |
September 23, 2016 |
December 2016 |
December 2, 2013 |
June 1, 2018 |
June 1, 2018 |
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Children's National Medical Center
Washington, District of Columbia, United States - Children's Healthcare of Atlanta
Atlanta, Georgia, United States - (and 19 more...)
|
|
| 47 |
NCT02512679 |
Terminated
Has Results |
Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells |
- Stem Cell Transplantation
- Bone Marrow Transplantation
- Peripheral Blood Stem Cell Transplantation
- (and 10 more...)
|
- Drug: Cyclophosphamide Dose Level 1
- Drug: Cyclophosphamide Dose Level 2
- Drug: Cyclophosphamide Dose Level 3
- Drug: Cyclophosphamide Dose Level 4
|
Interventional
|
Phase 2 |
- Children's Hospital Los Angeles
- Lucile Packard Children's Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Neutrophil Engraftment (=/>500 Cells/uL) and Platelet Engraftment (>20K Cell/uL) at 30 Days
- Number of Participants With Disease Recurrence at 1 Year Post-transplant
- Number of Participants Who Developed Severe Mucositis, Veno-occlusive Disease (VOD), Toxicity of the Kidney, Liver, or Gastrointestinal (GI) Tract up to 1 Year Post-transplant
- (and 2 more...)
|
20 |
All |
3 Months and older (Child, Adult, Older Adult) |
NCT02512679 |
CCI-06-00177 |
|
February 2007 |
September 2013 |
February 2014 |
July 31, 2015 |
February 27, 2017 |
June 17, 2016 |
|
|
| 48 |
NCT01319851 |
Terminated
Has Results |
Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation |
- Thalassemia
- Sickle Cell Disease
- Glanzmann Thrombasthenia
- (and 10 more...)
|
|
Interventional
|
Not Applicable |
- Emory University
- Children's Healthcare of Atlanta
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Feasibility of Alefacept Pre-conditioning, Measured by Number of Subjects With Full Donor Engraftment
- Number of Participants That Expressed Grade 2 or 3 Regimen-Related Toxicity
- Number of Participants That Expressed Successful Neutrophil Engraftment
- (and 4 more...)
|
3 |
All |
up to 21 Years (Child, Adult) |
NCT01319851 |
IRB00039680
BMT Alefacept |
|
September 2010 |
September 2013 |
September 2013 |
March 22, 2011 |
July 27, 2017 |
March 9, 2015 |
- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
|
|
| 49 |
NCT00006054 |
Terminated |
Allogeneic Bone Marrow Transplantation in Patients With Primary Immunodeficiencies |
- Immunologic Deficiency Syndromes
- Chediak-Higashi Syndrome
- Common Variable Immunodeficiency
- (and 11 more...)
|
- Drug: anti-thymocyte globulin
- Drug: busulfan
- Drug: cyclophosphamide
- (and 6 more...)
|
Interventional
|
Not Applicable |
- Fairview University Medical Center
- Office of Rare Diseases (ORD)
|
Other |
- Primary Purpose: Treatment
|
|
|
All |
up to 35 Years (Child, Adult) |
NCT00006054 |
199/15104
UMN-MT-1995-26
UMN-MT-9526 |
|
March 2000 |
December 2002 |
December 2002 |
July 6, 2000 |
October 15, 2009 |
|
- Fairview University Medical Center
Minneapolis, Minnesota, United States
|
|
| 50 |
NCT01821781 |
Recruiting |
Immune Disorder HSCT Protocol |
- Immune Deficiency Disorders
- Severe Combined Immunodeficiency
- Chronic Granulomatous Disease
- (and 11 more...)
|
- Drug: Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan
|
Interventional
|
Phase 2 |
- Washington University School of Medicine
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of participants with donor engraftment
- Major Transplant Related Toxicities
- Time to neutrophil recovery
- (and 7 more...)
|
20 |
All |
up to 21 Years (Child, Adult) |
NCT01821781 |
201301135 |
|
March 2013 |
March 2019 |
March 2021 |
April 1, 2013 |
November 21, 2017 |
|
- Washington University
Saint Louis, Missouri, United States - Methodist Heathcare
San Antonio, Texas, United States
|
|
| 51 |
NCT03513328 |
Recruiting |
Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation |
- Bone Marrow Failure Syndrome
- Thalassemia
- Sickle Cell Disease
- (and 13 more...)
|
- Drug: Thiotepa--single daily dose
- Drug: Thiotepa--escalated dose
|
Interventional
|
Phase 1
Phase 2 |
|
Other |
- Allocation: Randomized
- Intervention Model: Sequential Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Assessment of minimum effective dose (MED) of Thiotepa
- Evaluation of risk of graft rejection/failure.
- Evaluation of disease free survival (DFS)
- (and 5 more...)
|
40 |
All |
6 Months to 38 Years (Child, Adult) |
NCT03513328 |
IRB201800798
PEDS024
OCR17838 |
|
June 15, 2018 |
June 2021 |
June 2022 |
May 1, 2018 |
January 16, 2019 |
|
- UF Health Shands Children's Hospital
Gainesville, Florida, United States
|
|
| 52 |
NCT03333486 |
Recruiting |
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer |
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive
- Acute Leukemia in Remission
- Acute Lymphoblastic Leukemia
- (and 34 more...)
|
- Drug: Cyclophosphamide
- Drug: Fludarabine Phosphate
- Other: Laboratory Biomarker Analysis
- (and 2 more...)
|
Interventional
|
Phase 2 |
- Roswell Park Cancer Institute
- National Cancer Institute (NCI)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Relapse rate
- Engraftment rate
- Incidence of acute graft versus host disease (GVHD)
- (and 4 more...)
|
58 |
All |
1 Year to 75 Years (Child, Adult, Older Adult) |
NCT03333486 |
I 40916
NCI-2017-01949
P30CA016056 |
|
December 7, 2017 |
September 6, 2021 |
September 6, 2022 |
November 7, 2017 |
December 20, 2018 |
|
- Roswell Park Cancer Institute
Buffalo, New York, United States
|
|
| 53 |
NCT00404560 |
Recruiting |
Detection and Characterization of Infections and Infection Susceptibility |
- Immune Disorders
- Chronic Granulomatous Disease
- Genetic Immunological Deficiencies
- (and 9 more...)
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Other
- Time Perspective: Retrospective
|
- The primary endpoint of this study will be determination of a discretediagnosis of an infecting agent, an underlying susceptibility trait, or both.
|
2000 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT00404560 |
070033
07-I-0033 |
|
November 27, 2006 |
|
|
November 29, 2006 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 54 |
NCT01917708 |
Active, not recruiting |
BMT Abatacept for Non-Malignant Diseases |
- Hurler Syndrome
- Fanconi Anemia
- Glanzmann Thrombasthenia
- (and 12 more...)
|
|
Interventional
|
Phase 1 |
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Supportive Care
|
- Tolerability of abatacept
- Proportion of Participants experiencing Immunological effects
- Proportion of Participants experiencing regimen-related toxicity (RRT)
- (and 4 more...)
|
10 |
All |
up to 21 Years (Child, Adult) |
NCT01917708 |
IRB00069836 |
|
January 2014 |
September 29, 2019 |
September 29, 2019 |
August 7, 2013 |
November 6, 2018 |
|
- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
|
|
| 55 |
NCT01652092 |
Recruiting |
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies |
- SCID
- Omenn's Syndrome
- Reticular Dysgenesis
- (and 11 more...)
|
- Drug: Alemtuzumab 0.3 mg
- Drug: Cyclophosphamide
- Drug: Busulfan
- (and 6 more...)
|
Interventional
|
Not Applicable |
- Masonic Cancer Center, University of Minnesota
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Neutrophil Engraftment
- Incidence of Graft Failure
- Incidence of Chimerism
- (and 5 more...)
|
30 |
All |
up to 50 Years (Child, Adult) |
NCT01652092 |
2012OC055
MT2012-10C |
|
September 4, 2012 |
December 2019 |
December 2021 |
July 27, 2012 |
January 15, 2019 |
|
- Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
|
|
| 56 |
NCT01852370 |
Enrolling by invitation |
Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases |
- Severe Combined Immunodeficiency (SCID)
- Immunodeficiency With Predominant T-cell Defect, Unspecified
- Severe Chronic Neutropenia
- (and 6 more...)
|
- Biological: CD3/CD19 negative allogeneic hematopoietic stem cells
|
Interventional
|
Phase 1
Phase 2 |
- Paul Szabolcs
- University of Pittsburgh
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Safety: Death
- Safety: Engraftment syndrome
- Safety: Engraftment failure
- (and 19 more...)
|
16 |
All |
5 Years to 45 Years (Child, Adult) |
NCT01852370 |
PRO16010311 |
BOLT+BMT |
June 20, 2013 |
December 2019 |
December 2021 |
May 13, 2013 |
February 8, 2018 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
|
| 57 |
NCT01222741 |
Recruiting |
Studies of Disorders With Increased Susceptibility to Fungal Infections |
- Primary Immune Deficiency
- Candida
- Autoimmune Polyendocrinopathy Candidiasis Ectodermal
- (and 2 more...)
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
|
850 |
All |
Child, Adult, Older Adult |
NCT01222741 |
100216
10-I-0216 |
|
September 24, 2010 |
|
|
October 18, 2010 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 58 |
NCT00092937 |
Completed |
Use of Busulfan as Conditioning Agent for a Second Stem Cell Transplant |
- Granulomatous Disease
- Chronic
|
|
Interventional
|
Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
2 |
All |
Child, Adult, Older Adult |
NCT00092937 |
040289
04-I-0289 |
|
September 23, 2004 |
April 6, 2010 |
April 6, 2010 |
September 27, 2004 |
July 2, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 59 |
NCT02231710 |
Terminated |
Safety Study of Gene Modified Donor T Cell Infusion After Stem Cell Transplant for Non-Malignant Diseases |
- Primary Immune Deficiency Disorders
- Hemophagocytic Lymphohistiocytosis
- Inherited Bone Marrow Failure Syndrome
- (and 2 more...)
|
- Biological: BPX-501 and AP1903
|
Interventional
|
Phase 1 |
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- GVHD
- Transplant related mortality
|
1 |
All |
4 Months to 55 Years (Child, Adult) |
NCT02231710 |
BP-003 |
|
February 2015 |
January 15, 2018 |
January 15, 2018 |
September 4, 2014 |
November 5, 2018 |
|
- Fred Hutchinson Cancer ResearchCenter
Seattle, Washington, United States
|
|
| 60 |
NCT00368446 |
Completed |
Genetic Disorders of Mucociliary Clearance in Nontuberculous Mycobacterial Lung Disease |
- Mycobacteria, Atypical
- Cystic Fibrosis
- Ciliary Disorders
- (and 2 more...)
|
|
Observational
|
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
|
87 |
All |
2 Years and older (Child, Adult, Older Adult) |
NCT00368446 |
060217
06-H-0217 |
|
August 1, 2006 |
|
|
August 24, 2006 |
August 9, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 61 |
NCT00730314 |
Completed |
Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells |
- Sickle Cell Disease
- Thalassemia
- Anemia
- (and 9 more...)
|
- Procedure: Hematopoietic stem cell transplantation
|
Interventional
|
Phase 1
Phase 2 |
- Children's Hospital Los Angeles
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- toxicities
- adverse events
- engraftment
- (and 2 more...)
|
25 |
All |
up to 21 Years (Child, Adult) |
NCT00730314 |
CCI #07-00119
CHLA-#07-00119 |
|
August 2008 |
August 2015 |
August 2015 |
August 8, 2008 |
June 23, 2016 |
|
- Children Hospital Los Angeles
Los Angeles, California, United States
|
|
| 62 |
NCT00128973 |
Recruiting |
Evaluation of Patients With Immune Function Abnormalities |
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
2400 |
All |
up to 90 Years (Child, Adult, Older Adult) |
NCT00128973 |
050213
05-I-0213 |
|
August 8, 2005 |
|
|
August 10, 2005 |
January 15, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 63 |
NCT01962415 |
Recruiting |
Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT |
- Primary Immunodeficiency (PID)
- Congenital Bone Marrow Failure Syndromes
- Inherited Metabolic Disorders (IMD)
- (and 2 more...)
|
- Drug: Hydroxyurea
- Drug: Alemtuzumab
- Drug: Fludarabine
- (and 2 more...)
|
Interventional
|
Phase 2 |
- Paul Szabolcs
- University of Pittsburgh
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Post-transplant treatment-related mortality (TRM)
- Neurodevelopmental milestones
- Immune Reconstitution
- (and 9 more...)
|
100 |
All |
2 Months to 40 Years (Child, Adult) |
NCT01962415 |
PRO13100018 |
HSCT+RIC |
February 4, 2014 |
November 2019 |
November 2020 |
October 14, 2013 |
May 25, 2018 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
|
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