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
|
NIH / Other |
- Observational Model: Cohort
- Time Perspective: Other
|
- Death
- Engraftment
- Quality of Life Measures
- (and 2 more...)
|
1480 |
All |
Child, Adult, Senior |
NCT02082353 |
DAIT RDCRN PIDTC-6903 |
|
June 2014 |
August 2019 |
August 2019 |
March 10, 2014 |
October 17, 2017 |
|
- 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 42 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, Senior) |
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 |
November 17, 2017 |
|
- Children's Hospital of Fudan University
Shanghai, Shanghai, China
|
4 |
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.
|
120 |
All |
2 Years and older (Child, Adult, Senior) |
NCT02116764 |
140091 14-I-0091 |
|
April 15, 2014 |
July 1, 2018 |
July 1, 2019 |
April 17, 2014 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
5 |
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, Senior) |
NCT02757911 |
G1XCGD.02 |
|
March 2016 |
December 2019 |
December 2019 |
May 2, 2016 |
May 2, 2016 |
|
- Hôpital Necker Enfants Malades
Paris, France
|
6 |
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, Senior) |
NCT01855685 |
G1XCGD.01 |
CGD |
February 2013 |
March 2020 |
March 2020 |
May 16, 2013 |
February 9, 2017 |
|
- 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...)
|
7 |
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, Senior) |
NCT01851460 |
130117 13-I-0117 |
|
May 8, 2013 |
January 1, 2020 |
January 1, 2021 |
May 10, 2013 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
8 |
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, Senior) |
NCT01906541 |
X-CGD |
|
July 2013 |
December 2013 |
December 2019 |
July 24, 2013 |
August 2, 2013 |
|
- University Hospital Frankfurt
Frankfurt am Main, Germany
|
9 |
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
|
10 |
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, Senior) |
NCT00001405 |
940073 94-I-0073 |
|
February 9, 1994 |
|
|
November 4, 1999 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
11 |
NCT02629120 |
Recruiting |
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.
|
50 |
All |
4 Years to 65 Years (Child, Adult) |
NCT02629120 |
160032 16-I-0032 |
|
December 10, 2015 |
December 31, 2025 |
December 1, 2030 |
December 14, 2015 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
12 |
NCT01063309 |
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...
|
550 |
All |
18 Years and older (Adult, Senior) |
NCT01063309 |
100029 10-I-0029 |
|
December 11, 2009 |
|
|
February 5, 2010 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
13 |
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, Senior) |
NCT03278912 |
170163 17-I-0163 |
|
April 30, 2018 |
July 1, 2030 |
April 30, 2031 |
September 12, 2017 |
April 25, 2018 |
|
- National Institutes of Health Clinical Center
Bethesda, Maryland, United States
|
14 |
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, Senior) |
NCT02108028 |
140079 14-C-0079 |
|
April 5, 2014 |
December 1, 2018 |
December 18, 2018 |
April 9, 2014 |
April 19, 2018 |
|
- Children's Hospital of Orange County
Orange, California, United States - Childrens National Medical Center
Washington, District of Columbia, United States - National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States - (and 3 more...)
|
15 |
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, Senior) |
NCT00471250 |
070142 07-H-0142 |
|
May 7, 2007 |
|
|
May 9, 2007 |
April 4, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
16 |
NCT01953016 |
Recruiting |
Participation in a Research Registry for Immune Disorders |
- Primary Immunodeficiencies
- GATA2
- 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 |
|
- We will add NHGRI patients enrolled in 00-HG-0209 into a national registry of individuals with primary immune deficiency disorders to discover basic outcome data, ethnic and racial characteristics, kinds of complications etc. of these immune def...
|
1000 |
All |
up to 99 Years (Child, Adult, Senior) |
NCT01953016 |
130199 13-HG-0199 |
|
September 25, 2013 |
May 1, 2020 |
May 1, 2020 |
September 30, 2013 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
17 |
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
|
18 |
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, Senior) |
NCT03333486 |
I 40916 NCI-2017-01949 P30CA016056 |
|
December 7, 2017 |
September 6, 2021 |
September 6, 2022 |
November 7, 2017 |
December 11, 2017 |
|
- Roswell Park Cancer Institute
Buffalo, New York, United States
|
19 |
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 2018 |
December 2020 |
July 27, 2012 |
December 5, 2017 |
|
- Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States
|
20 |
NCT01917708 |
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
- (and 4 more...)
|
20 |
All |
up to 21 Years (Child, Adult) |
NCT01917708 |
IRB00069836 |
|
January 2014 |
September 2018 |
January 2020 |
August 7, 2013 |
March 30, 2018 |
|
- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
|
21 |
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, Senior) |
NCT00404560 |
070033 07-I-0033 |
|
November 27, 2006 |
|
|
November 29, 2006 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
22 |
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, Senior |
NCT01222741 |
100216 10-I-0216 |
|
September 24, 2010 |
|
|
October 18, 2010 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
23 |
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, Senior) |
NCT00128973 |
050213 05-I-0213 |
|
August 8, 2005 |
|
|
August 10, 2005 |
April 25, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
24 |
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...)
|
30 |
All |
2 Months to 35 Years (Child, Adult) |
NCT01962415 |
PRO13100018 |
HSCT+RIC |
February 4, 2014 |
November 2019 |
November 2020 |
October 14, 2013 |
April 5, 2018 |
|
- Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, United States
|