| 1 |
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...)
|
|
| 2 |
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
|
|
| 3 |
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
|
|
| 4 |
NCT03910452 |
Recruiting |
Haploidentical Transplant for People With Chronic Granulomatous Disease (CGD) Using Alemtuzumab, Busulfan and TBI With Post-Transplant Cyclophosphamide |
- Chronic Granulomatous Disease
|
- Drug: Busulfan
- Drug: Alemtuzumab
- Drug: Cyclophosphamide
- (and 3 more...)
|
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
|
- Engraftment - Chimerism
- EBV/CMV/VZV PCR
- Overall Survival/Event-Free survival
- Severe GvHD
|
30 |
All |
4 Years to 65 Years (Child, Adult, Older Adult) |
NCT03910452 |
190080
19-I-0080 |
|
July 22, 2019 |
June 15, 2034 |
June 15, 2034 |
April 10, 2019 |
July 17, 2019 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
|
| 5 |
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 |
September 2019 |
September 2019 |
June 7, 2018 |
June 24, 2019 |
|
- University of Colorado Anschutz Medical Campus
Aurora, Colorado, United States
|
|
| 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 |
July 11, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 7 |
NCT02629120 |
Recruiting |
High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease |
- Chronic Granulomatous Disease Transplant
|
- Drug: Campath
- Drug: Busulfan
- Other: allogeneic peripheral blood allograft infusion
- Drug: cyclophosphamide
|
Interventional
|
Phase 1
Phase 2 |
- 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 determine engraftment rates with the use of high cell doses, without increasing the risk of GvHD by using post- transplant cyclophosphamide and sirolimus in conjunction with a busulfan- based conditioning regimen.
- To measure the engraftment rate and the engraftment kinetics using such a regimen
- To assess the level and kinetics of immune reconstitution (via chimerism) when using post- transplant cyclophosphamide
- To further elucidate the factors involved in th
|
50 |
All |
4 Years to 65 Years (Child, Adult, Older Adult) |
NCT02629120 |
160032
16-I-0032 |
|
December 10, 2015 |
December 31, 2025 |
December 31, 2025 |
December 14, 2015 |
July 16, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 8 |
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
|
|
| 9 |
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 |
- 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 |
June 24, 2013 |
March 2020 |
March 2020 |
May 16, 2013 |
April 15, 2019 |
|
- 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...)
|
|
| 10 |
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
|
|
| 11 |
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
|
10 |
All |
18 Years to 75 Years (Adult, Older Adult) |
NCT01851460 |
130117
13-I-0117 |
|
May 8, 2013 |
January 1, 2020 |
December 31, 2020 |
May 10, 2013 |
January 28, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 12 |
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
|
|
| 13 |
NCT03983837 |
Not yet recruiting |
Elemental Diet for Treatment of Inflammatory Bowel Disease in Patients With Chronic Granulomatous Disease |
- Chronic Granulomatous Disease
- Inflammatory Bowel Disease
|
|
Interventional
|
Phase 2 |
- 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
|
- Change in the relative abundance of proteobacteria in stool samples obtained after elemental diet.
- Unexpected AEs possibly, probably, or definitely related to the elemental diet.
- Ability to tolerate 4 weeks of elemental diet as measured by > 75% of caloric intake via elemental diet over the 4-week period.
- (and 5 more...)
|
10 |
All |
8 Years to 40 Years (Child, Adult) |
NCT03983837 |
190108
19-I-0108 |
|
July 22, 2019 |
May 31, 2022 |
May 31, 2022 |
June 12, 2019 |
July 17, 2019 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
|
| 14 |
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
|
|
| 15 |
NCT03984890 |
Not yet recruiting |
Vitamin D3 For CGD Patients With BCGosis/Itis |
- Vitamin D3
- Chronic-granulomatous Disease
- BCG
|
- Drug: Vitamin D3
- Drug: Traditional treatment of CGD and TB
|
Interventional
|
Phase 2
Phase 3 |
- Children's Hospital of Fudan University
|
Other |
- Allocation: Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Mortality
- Rate of Sputum Culture acid-fast bacilli microscopy Conversion
- Duration of Fever
- (and 7 more...)
|
50 |
All |
up to 18 Years (Child, Adult) |
NCT03984890 |
VDBCG |
|
August 1, 2019 |
December 31, 2020 |
December 31, 2022 |
June 13, 2019 |
June 13, 2019 |
|
- Children's Hospital of Fudan University
Shanghai, Shanghai, China - Children's Hospital of Fudan University
Shanghai, Shanghai, China
|
|
| 16 |
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 |
|
July 22, 2019 |
July 1, 2030 |
July 1, 2030 |
September 12, 2017 |
July 17, 2019 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
|
| 17 |
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...)
|
|
| 18 |
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 |
February 7, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 19 |
NCT00001405 |
Recruiting |
Use of G-CSF to Obtain Blood Cell Precursors |
- Granuloma
- Granulomatous Disease, Chronic
- Leukocyte Disease
- (and 2 more...)
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Other
|
- Mobilize to peripheral blood and apheresis collect CD34+PBSC of PID patients for clinical treatment
- Mobilize to the peripheral blood and apheresis collect CD34+PBSC from healthy volunteers for research
- Mobilize to peripheral blood and apherisis collect CD34+PBSC of healthy sibling or other related donor of patients with PID for stem cell transplant
- Use of G-CSF alone or with periflexafor to stimulate CD34+PBSC
|
450 |
All |
2 Years to 70 Years (Child, Adult, Older Adult) |
NCT00001405 |
940073
94-I-0073 |
|
February 9, 1994 |
|
|
November 4, 1999 |
July 11, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 20 |
NCT00128973 |
Recruiting |
Evaluation of Patients With Immune Function Abnormalities |
- Immune System Diseases
- Chronic Granulomatous Disease (CGD)
- X-Linked Severe Combined Immune Deficiency (XSCID)
- (and 2 more...)
|
|
Observational
|
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Other
- Time Perspective: Cross-Sectional
|
- To characterize the physiologic, biochemical or genetic basis of the abnormality of immunity.
- To establish the extent of organ involvement (infection and/or inflammation) and organ damage or dysfunction resulting from the abnormality of immune function.
- To establish the pattern and pace of change of disease (frequency, distribution, type and extent of infections, inflammatory lesions and abnormalities of immune function) during a period of up
- (and 5 more...)
|
2400 |
All |
Child, Adult, Older Adult |
NCT00128973 |
050213
05-I-0213 |
|
October 19, 2005 |
|
|
August 10, 2005 |
July 16, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 21 |
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 2022 |
March 2024 |
April 1, 2013 |
March 26, 2019 |
|
- Washington University
Saint Louis, Missouri, United States - Methodist Heathcare
San Antonio, Texas, United States
|
|
| 22 |
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 |
- University of Florida
- Live Like Bella Pediatric Cancer Research
|
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
AWD05935 |
|
June 15, 2018 |
June 2021 |
June 2022 |
May 1, 2018 |
May 21, 2019 |
|
- UF Health Shands Children's Hospital
Gainesville, Florida, United States
|
|
| 23 |
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
|
|
| 24 |
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
|
|
| 25 |
NCT03921515 |
Not yet recruiting |
Skin Immunity Sample Collection Involving Blisters and Biopsies |
- Hyper-Immunoglobulin E. Syndrome (HEIS)
- Chronic Granulomatous (GGD)
|
- Other: Blister induction
- Other: Skin Biopsies
|
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: Basic Science
|
- Tumor necrosis factor alpha (TNFa) production by keratinocytes from patients with HIES versus healthy volunteers
- T-cell infiltration as percent of total cell infiltration in patients with CGD versus healthy volunteers.
- Fold induction in genes related to wound healing.
|
70 |
All |
7 Years to 65 Years (Child, Adult, Older Adult) |
NCT03921515 |
190084
19-I-0084 |
|
July 22, 2019 |
September 30, 2024 |
September 30, 2024 |
April 19, 2019 |
July 17, 2019 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
|
| 26 |
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 29, 2019 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
|
| 27 |
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 2020 |
November 2021 |
October 14, 2013 |
April 5, 2019 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|
|
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