1 |
NCT01410019 |
Unknown † |
Gene Therapy for X-linked Severe Combined Immunodeficiency |
- X-linked Severe Combined Immunodeficiency
|
|
Interventional |
Phase 1 Phase 2 |
- Assistance Publique - Hôpitaux de Paris
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Assessment of immunological reconstitution at short term
- Molecular characterization of gene transfer
- Analysis of activated proto-oncogene s expression
|
5 |
Male |
up to 12 Months (Child) |
NCT01410019 |
P071204 2008-002380-14 |
SCID2 |
December 2010 |
July 2015 |
October 2016 |
August 4, 2011 |
August 26, 2016 |
|
- Hopital Necker
Paris, France
|
2 |
NCT01175239 |
Active, not recruiting |
Gene Therapy for X-linked Severe Combined Immunodeficiency (SCID-X1) |
- X-linked Severe Combined Immunodeficiency
|
- Genetic: Single infusion of autologous CD34+ cells transduced with the self-inactivating (SIN) gammaretroviral vector pSRS11.EFS.IL2RG.pre
|
Interventional |
Not Applicable |
- Great Ormond Street Hospital for Children NHS Foundation Trust
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Immunological reconstitution
- Incidence of adverse reactions
- Molecular characterisation of gene transfer
- Normalisation of nutritional status, growth, and development
|
1 |
Male |
up to 16 Years (Child) |
NCT01175239 |
06MI10 |
|
April 2011 |
December 2018 |
December 2018 |
August 4, 2010 |
August 1, 2017 |
|
- Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom
|
3 |
NCT01512888 |
Recruiting |
Gene Transfer for X-Linked Severe Combined Immunodeficiency in Newly Diagnosed Infants |
- Severe Combined Immunodeficiency Disease, X-linked
|
- Genetic: CL20-4i-EF/a-hyc-OPT
- Drug: Busulfan
- Device: CliniMacs
|
Interventional |
Phase 1 Phase 2 |
- St. Jude Children's Research Hospital
- National Heart, Lung, and Blood Institute (NHLBI)
- Assisi Foundation
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of patients with adequate cell collection and processing
- Number of patients with adequate neutrophil count recovery after busulfan conditioning
- Number of patients without Grade 4 adverse event (AE)
- (and 2 more...)
|
28 |
Male |
up to 24 Months (Child) |
NCT01512888 |
LVXSCID-ND P01HL053749 |
LVXSCID-ND |
August 17, 2016 |
August 2025 |
August 2034 |
January 19, 2012 |
January 23, 2018 |
|
- University of California-San Francisco
San Francisco, California, United States - St. Jude Children's Research Hospital
Memphis, Tennessee, United States - Seattle Children's Research Institute
Seattle, Washington, United States
|
4 |
NCT03315078 |
Recruiting |
Lentiviral Gene Transfer for Treatment of Children Older Than 2 Years of Age With X-Linked Severe Combined Immunodeficiency |
- X-Linked Combined Immunodeficiency Diseases
|
- Biological: CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1α-hγc-OPT
- Drug: Palifermin
- Drug: Busulfan
|
Interventional |
Phase 1 Phase 2 |
- National Institute of Allergy and Infectious Diseases (NIAID)
|
NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Level of autologous transduced T-lymphocytes with functional γc
- Incidence of serious side effects due to lentiviral gene transfer
- Distribution of integration sites of the lentiviral vector in reconstituted peripheral blood cells
|
13 |
All |
2 Years to 40 Years (Child, Adult) |
NCT03315078 |
11-I-0007 |
LVXSCID-OC |
April 2012 |
December 2022 |
December 2022 |
October 19, 2017 |
October 19, 2017 |
|
- Laboratory of Host Defenses (LHD), National Institute of Allergy and Infectious Diseases (NIAID), National Institutes of Health (NIH)
Bethesda, Maryland, United States
|
5 |
NCT01306019 |
Recruiting |
Lentiviral Gene Transfer for Treatment of Children Older Than Two Years of Age With X-Linked Severe Combined Immunodeficiency (XSCID) |
- X-linked Severe Combined Immunodeficiency
- XSCID
- SCID-X1
- Gamma C-Deficient SCID
|
- Other: Gene-modified CD34+ Hematopoietic stem cells
- Drug: Busulfan
|
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
|
- The primary objective is to assess the efficacy of immune reconstitution in XSCID patients transplanted with autologous CD34 plus cells that have been transduced with a self-inactivating lentiviral vector expressing a Gamma C gene.
- To determine the incidence of serious side effects due to lentiviral gene transfer.
- To determine the integration site distribution of the lentiviral vector in reconstituted peripheral blood cells.
|
20 |
All |
2 Years to 40 Years (Child, Adult) |
NCT01306019 |
110007 11-I-0007 |
|
February 26, 2011 |
December 31, 2022 |
December 31, 2025 |
March 1, 2011 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
6 |
NCT00028236 |
Completed |
Stem Cell Gene Therapy to Treat X-Linked Severe Combined Immunodeficiency (XSCID) |
- Severe Combined Immunodeficiency
|
- Drug: Gene-Transduced Autologous CD34+ Stem Cells
|
Interventional |
Phase 1 |
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
3 |
All |
18 Months to 20 Years (Child, Adult) |
NCT00028236 |
020057 02-I-0057 |
|
December 10, 2001 |
July 25, 2011 |
July 25, 2011 |
December 18, 2001 |
July 2, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
7 |
NCT00490100 |
Terminated Has Results |
Treatment for Growth Failure in Patients With X-Linked Severe Combined Immunodeficiency: Phase 2 Study of Insulin-Like Growth Factor-1 |
- Growth Failure
- X-linked Severe Combined Immunodeficiency (XSCID)
- Growth Hormone Resistence
|
|
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
|
- Safety of Study Drug
- Change in Growth Rate on Study Drug
|
6 |
All |
2 Years to 20 Years (Child, Adult) |
NCT00490100 |
070171 07-I-0171 |
|
June 2007 |
December 2012 |
December 2012 |
June 22, 2007 |
July 3, 2015 |
July 3, 2015 |
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
8 |
NCT02860559 |
Not yet recruiting |
Safety and Early Efficacy Study of TBX-1400 in Patients With Severe Combined Immunodeficiency |
- Severe Combined Immunodeficiency
|
|
Interventional |
Phase 1 |
- Taiga Biotechnologies, Inc.
|
Industry |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Adverse Events following transplant with TBX-1400
- Transplant Engraftment
- Chimerism
- (and 9 more...)
|
8 |
All |
1 Month to 4 Years (Child) |
NCT02860559 |
TBX-1400-001 |
|
April 2018 |
February 2022 |
March 2022 |
August 9, 2016 |
October 23, 2017 |
|
- Hadassah Medical Center (Ein Kerem site)
Jerusalem, Israel - Schneider Children's Medical Center
Petach Tikva, Israel
|
9 |
NCT01129544 |
Active, not recruiting |
Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector |
- Severe Combined Immunodeficiency
|
- Biological: Gene transfer
|
Interventional |
Phase 1 Phase 2 |
- David Williams
- Boston Children’s Hospital
- Children's Hospital Medical Center, Cincinnati
- University of California, Los Angeles
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- CD3 cell count post transfusion
- Incidence of life-threatening adverse reactions related to the gene therapy procedure.
- Molecular characterization of gene transfer.
- (and 2 more...)
|
8 |
Male |
Child, Adult, Senior |
NCT01129544 |
IND14067 |
|
April 2010 |
March 2018 |
March 2033 |
May 24, 2010 |
January 2, 2018 |
|
- Mattel Children's Hospital - UCLA
Los Angeles, California, United States - Children's Hospital Boston
Boston, Massachusetts, United States - Cincinnati Children's Medical Center
Cincinnati, Ohio, United States
|
10 |
NCT02244450 |
Active, not recruiting |
Generalized Neonatal Screening of Severe Combined Immunodeficiencies |
- Severe Combined Immunodeficiency, Atypical
|
- Biological: SCID screening
|
Interventional |
Not Applicable |
- Nantes University Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Screening
|
- cost / efficiency ratio of the implementation of the generalized neonatal screening of SCID at birth
- Cost / efficiency ratio of the implementation of the generalized neonatal screening of SCID at birth
- The cost of care during the first 18 months of life per child enjoying an early curative treatment in the first 4 months of life.
- (and 5 more...)
|
190539 |
All |
up to 18 Months (Child) |
NCT02244450 |
RC14_0030 |
DEPISTREC |
December 2014 |
July 2018 |
July 2018 |
September 19, 2014 |
August 29, 2017 |
|
- Angers Private Hospital Clinique de l'Anjou
Angers, France - Angers University Hospital
Angers, France - Argenteuil Hospital
Argenteuil, France - (and 48 more...)
|
11 |
NCT00006335 |
Completed |
Influences on Female Adolescents' Decisions Regarding Testing for Carrier Status of XSCID |
- Severe Combined Immunodeficiency
|
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
40 |
Female |
13 Years to 17 Years (Child) |
NCT00006335 |
000223 00-HG-0223 |
|
September 26, 2000 |
September 25, 2008 |
|
October 5, 2000 |
July 2, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
12 |
NCT02231983 |
Unknown † |
Clinical Characteristics and Genetic Profiles of Severe Combined Immunodeficiency in China |
- Severe Combined Immunodeficiency
|
|
Observational |
|
- Shanghai Children's Medical Center
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
|
50 |
All |
2 Months to 2 Years (Child) |
NCT02231983 |
SCID-20140901 |
|
September 2014 |
September 2016 |
September 2016 |
September 4, 2014 |
September 4, 2014 |
|
- Shanghai children's medical center
Shanghai, Shanghai, China
|
13 |
NCT00152100 |
Completed |
Transplantation of Hematopoietic Cells in Children With Severe Combined Immunodeficiency Syndrome |
- Severe Combined Immunodeficiency
|
- Procedure: Stem cell transplant
- Drug: Filgrastim, Alemtuzumab
- Device: Miltenyi CliniMACS
|
Interventional |
Phase 1 |
- St. Jude Children's Research Hospital
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To investigate safety issues related to use of haploidentical highly purified CD133+ hematopoietic cells in patients with SCID
- To study the effects (good and bad) of this procedure
- To learn if this procedure will result in normal immune function in children with SCID
|
4 |
All |
up to 2 Years (Child) |
NCT00152100 |
ALSCID |
|
February 2004 |
August 2007 |
August 2007 |
September 9, 2005 |
May 20, 2009 |
|
- St. Jude Children's Research Hospital
Memphis, Tennessee, United States
|
14 |
NCT03217617 |
Recruiting |
Gene Transfer for SCID-X1 Using a Self-inactivating Lentiviral Vector |
|
- Biological: Gene-modified autologous stem cells
|
Interventional |
Phase 1 Phase 2 |
- Shenzhen Geno-Immune Medical Institute
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- percentage of treated patients with adverse events
- Efficacy of immune reconstitution
- Number of patients with successful immune system reconstitution
|
30 |
Male |
1 Month to 1 Year (Child) |
NCT03217617 |
GIMI-IRB-17014 |
|
July 15, 2017 |
December 31, 2019 |
December 31, 2020 |
July 14, 2017 |
October 3, 2017 |
|
- Capital Institute of Pediatrics affiliated Children's hospital
Beijing, Beijing, China - Beijing Children's Hospital
Beijing, Beijing, China - Shenzhen Geno-immune Medical Institute
Shenzhen, Guangdong, China
|
15 |
NCT02590328 |
Recruiting |
Neonatal Screening of Severe Combined Immunodeficiencies |
- Severe Combined Immunodeficiency
- Neonatal Screening
|
|
Observational |
|
- Children's Hospital of Fudan University
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- prevalence of SCID
- Number of detected SCID patients
|
200000 |
All |
up to 28 Days (Child) |
NCT02590328 |
NSSCID |
|
December 2015 |
December 2020 |
December 2020 |
October 29, 2015 |
March 30, 2018 |
|
- Children's Hospital of Fudan University
Shanghai, Shanghai, China
|
16 |
NCT00794508 |
Completed Has Results |
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID |
- Severe Combined Immunodeficiency
|
- Biological: ADA gene transfer
|
Interventional |
Phase 2 |
- Donald B. Kohn, M.D.
- FDA Office of Orphan Products Development
- National Institutes of Health (NIH)
- University of California, Los Angeles
|
Other / U.S. Fed / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Adverse Events
- Number of Participants With Greater Than 1% of Gene-Modified Cells in the Peripheral Blood
- Number of Participants Reaching the Normal Range of ADA Enzyme Activity
|
10 |
All |
1 Month to 18 Years (Child, Adult) |
NCT00794508 |
ADA Gene Therapy 1R01FD003005-01 9908-337 |
|
November 2008 |
December 2014 |
January 2015 |
November 20, 2008 |
March 1, 2018 |
May 30, 2016 |
- University of California, Los Angeles
Los Angeles, California, United States
|
17 |
NCT01182675 |
Terminated Has Results |
Hematopoietic Stem Cell Transplantation (HSCT) for Children With SCID Utilizing Alemtuzumab, Plerixafor & Filgrastim |
- Severe Combined Immunodeficiency
|
- Drug: Transplant Conditioning with Mobilization Only
- Drug: Transplant Conditioning with Mobilization and Alemtuzumab
|
Interventional |
Phase 2 |
- University of California, San Francisco
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Engraftment of Donor B-cells in Blood by STR Testing
- Incidence of Acute GVHD
- Incidence of Chronic GVHD
- (and 3 more...)
|
7 |
All |
up to 3 Years (Child) |
NCT01182675 |
UCSF10-00701 |
|
August 2010 |
September 2013 |
September 2013 |
August 17, 2010 |
December 2, 2017 |
November 10, 2014 |
- UCSF Benioff Children's Hospital
San Francisco, California, United States
|
18 |
NCT02127892 |
Terminated Has Results |
SCID Bu/Flu/ATG Study With T Cell Depletion |
- Severe Combined Immunodeficiency
|
- Biological: unrelated BM with T cell depletion
- Biological: unrelated cord blood
- Biological: haplo BM with T cell depletion
- Device: unrelated PBSC with T cell depletion
|
Interventional |
Phase 1 Phase 2 |
- Neena Kapoor, M.D.
- Children's Hospital Los Angeles
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Participants With Engraftment
- Number of Participants With Donor-derived CD3+ T Lymphocytes >/= 100/mm3
|
9 |
All |
up to 21 Years (Child, Adult) |
NCT02127892 |
CCI-06-00243 |
|
January 2, 2007 |
August 1, 2016 |
August 1, 2016 |
May 1, 2014 |
September 18, 2017 |
September 18, 2017 |
- Children's Hospital Los Angeles
Los Angeles, California, United States
|
19 |
NCT00001255 |
Completed |
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency: A Natural History Study |
- Severe Combined Immunodeficiency
|
- Drug: ADA PBSC
- Drug: ADA Umbilical Cord Blood Cells
- Drug: Transduced Lymphocytes
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
10 |
All |
Child, Adult, Senior |
NCT00001255 |
900195 90-HG-0195 |
|
September 1990 |
|
July 2002 |
November 4, 1999 |
March 4, 2008 |
|
- National Human Genome Research Institute (NHGRI)
Bethesda, Maryland, United States
|
20 |
NCT02999984 |
Active, not recruiting |
Autologous Cryopreserved CD34+ Hematopoietic Cells Transduced With EFS-ADA Lentivirus for ADA SCID |
- Severe Combined Immunodeficiency Due to ADA Deficiency
|
- Biological: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells
|
Interventional |
Phase 1 Phase 2 |
- University of California, Los Angeles
- Orchard Therapeutics Limited
- University College, London
- California Institute for Regenerative Medicine
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Overall survival
- Event-free survival
|
10 |
All |
up to 17 Years (Child) |
NCT02999984 |
UCLA IRB#15-001678 |
|
December 2016 |
October 2019 |
June 2020 |
December 21, 2016 |
February 5, 2018 |
|
- University of California, Los Angeles
Los Angeles, California, United States
|
21 |
NCT03311503 |
Recruiting |
Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning |
- Severe Combined Immunodeficiency, X Linked
- Gene Therapy
|
- Biological: autologous CD34+ cell transduced with G2SCID vector
|
Interventional |
Phase 1 Phase 2 |
- David Williams
- University of California, Los Angeles
- Boston Children’s Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- The primary objective is to measure event free survival
- T cell reconstitution
|
10 |
Male |
up to 18 Years (Child, Adult) |
NCT03311503 |
P00023098 |
|
January 19, 2018 |
January 1, 2021 |
January 1, 2023 |
October 17, 2017 |
February 8, 2018 |
|
- Mattel Children's Hospital - UCLA
Los Angeles, California, United States - Boston Childrens Hospital
Boston, Massachusetts, United States
|
22 |
NCT00845416 |
Completed |
Newborn Screening for Severe Combined Immunodeficiency (SCID) in a High-Risk Population |
- Severe Combined Immunodeficiency
- T Cell Lymphocytopenia
|
|
Observational |
|
- University of California, San Francisco
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
|
1800 |
All |
up to 30 Days (Child) |
NCT00845416 |
H55235-32562-01 R03HD060311 |
|
March 2009 |
November 2011 |
November 2011 |
February 18, 2009 |
July 16, 2012 |
|
- Chinle Hospital
Chinle, Arizona, United States - Tuba City Hospital
Tuba City, Arizona, United States
|
23 |
NCT02177760 |
Withdrawn |
Sirolimus Prophylaxis for aGVHD in TME SCID |
- Severe Combined Immunodeficiency
- Transplacental Maternal Engraftment
- Stem Cell Transplant
|
|
Interventional |
Phase 2 |
- University of California, San Francisco
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Prevention
|
- Incidence of aGVHD
- T-regulatory cell enumeration
- Sirolimus therapeutic drug monitoring
|
0 |
All |
up to 1 Year (Child) |
NCT02177760 |
14-13024 |
|
July 2014 |
November 2015 |
November 2015 |
June 30, 2014 |
December 3, 2015 |
|
- Benioff Children's Hospital at UCSF Medical Center
San Francisco, California, United States
|
24 |
NCT00055172 |
Recruiting |
Genetic Basis of Immunodeficiency |
- Severe Combined Immunodeficiency
|
|
Observational |
|
- National Heart, Lung, and Blood Institute (NHLBI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
99999999 |
All |
Child, Adult, Senior |
NCT00055172 |
030105 03-H-0105 |
|
February 19, 2003 |
|
|
February 20, 2003 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States - Universidad de Immunologia
Santiago, Chile
|
25 |
NCT00599781 |
Completed |
Gene Therapy for ADA-SCID |
- Severe Combined Immunodeficiency Syndrome
|
- Genetic: gene transduced PBL and/or gene transduced HSC
|
Interventional |
Phase 1 Phase 2 |
- IRCCS San Raffaele
- Fondazione Telethon
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Evaluation of safety of the administration of the autologous PBL and/or autologous HSC transduced with the normal human ADA gene
- Evaluation of extent, kinetic and duration of the engraftment of transduced cells and the potential selective advantage of ADA positive cells
- Evaluation of efficacy of the administration of autologous PBL/HSC(Clinical, immunological, hematological, microbiological, ADA activity and purine metabolism)
- To identify the relative role of peripheral blood lymphocytes and hematopoietic stem cells and progenitor cells in the long-term reconstitution of immune functions after gene therapy
|
8 |
All |
Child, Adult, Senior |
NCT00599781 |
150291 |
|
March 1992 |
July 2006 |
January 2007 |
January 24, 2008 |
January 24, 2008 |
|
|
26 |
NCT00018018 |
Completed |
Gene Transfer Therapy for Severe Combined Immunodeficieny Disease (SCID) Due to Adenosine Deaminase (ADA) Deficiency |
- Severe Combined Immunodeficiency Syndrome
|
- Drug: CD34+ cells transduced with ADA retrovir
|
Interventional |
Phase 1 |
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Primary Purpose: Treatment
|
|
8 |
All |
1 Month and older (Child, Adult, Senior) |
NCT00018018 |
010189 01-HG-0189 |
|
June 20, 2001 |
|
September 17, 2014 |
June 28, 2001 |
April 17, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
27 |
NCT00008450 |
Active, not recruiting |
Total-Body Irradiation Followed By Cyclosporine and Mycophenolate Mofetil in Treating Patients With Severe Combined Immunodeficiency Undergoing Donor Bone Marrow Transplant |
- Adenosine Deaminase Deficiency
- Autosomal Recessive Disorder
- Immune System Disorder
- (and 4 more...)
|
- Procedure: Allogeneic Bone Marrow Transplantation
- Drug: Cyclosporine
- Other: Laboratory Biomarker Analysis
- (and 3 more...)
|
Interventional |
Phase 1 |
- Fred Hutchinson Cancer Research Center
- National Cancer Institute (NCI)
- National Heart, Lung, and Blood Institute (NHLBI)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Mixed hematopoietic chimerism in a population of pediatric patients with immunodeficiency diseases
|
6 |
All |
Child, Adult, Senior |
NCT00008450 |
1227.00 NCI-2010-02045 P01HL036444 P30CA015704 |
|
August 11, 1997 |
October 25, 2011 |
|
January 8, 2001 |
July 14, 2017 |
|
- Fred Hutch/University of Washington Cancer Consortium
Seattle, Washington, United States
|
28 |
NCT03478670 |
Recruiting |
Phase IV, GSK2696273, Registry Study to Follow-up Patients With Adenosine Deaminase Severe Combined Immunodeficiency (ADA-SCID) |
- Immunologic Deficiency Syndromes
|
- Genetic: Strimvelis (or GSK2696273)
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Overall survival
- Intervention free survival
- Number of subjects with the use of medications/treatments of interest
- (and 20 more...)
|
50 |
All |
Child, Adult, Senior |
NCT03478670 |
200195 |
|
March 31, 2017 |
May 31, 2037 |
May 31, 2037 |
March 27, 2018 |
March 27, 2018 |
|
- GSK Investigational Site
Milano, Lombardia, Italy
|
29 |
NCT01852071 |
Active, not recruiting |
Autologous Transplant of EFS-ADA Modified Bone Marrow Cells for ADA-Deficient Severe Combined Immunodeficiency (SCID) |
|
- Genetic: EFS-ADA transduced CD34+ cells from the bone marrow
|
Interventional |
Phase 1 Phase 2 |
- Donald B. Kohn, M.D.
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Human Genome Research Institute (NHGRI)
- (and 2 more...)
|
Other / NIH |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Assess safety by recording clinical toxicities.
- Assess safety by determining absence or presence of exposure to replication-competent lentivirus (RCL)
- Assess safety by evaluating the absence or development of monoclonal expansion or leukoproliferative complications from vector insertional effects
- (and 12 more...)
|
20 |
All |
1 Month and older (Child, Adult, Senior) |
NCT01852071 |
IND 15440 U01AI100801 2P01HL073104 0910-1006 |
|
May 2013 |
July 2018 |
September 2018 |
May 13, 2013 |
September 27, 2017 |
|
- Mattel Children's Hospital, UCLA
Los Angeles, California, United States - Mark O. Hatfield Clinical Research Center, NIH
Bethesda, Maryland, United States
|
30 |
NCT03232203 |
Not yet recruiting |
Evaluating the Effectiveness of STRIMVELIS Risk Minimization Measures (RMMs) |
- Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency
|
|
Observational |
|
|
Industry |
- Observational Model: Cohort
- Time Perspective: Cross-Sectional
|
- Proportion of HCPs providing the correct response
- Proportion of Parents/Carers providing the correct response
|
1 |
All |
18 Years and older (Adult, Senior) |
NCT03232203 |
205881 |
|
January 31, 2018 |
September 30, 2020 |
September 30, 2020 |
July 27, 2017 |
December 27, 2017 |
|
|
31 |
NCT02915406 |
No longer available |
cliniMACs HUD for T Cell Depletion |
- x Linked Combined Immunodeficiency
|
|
Expanded Access |
|
- University of Miami
- Jackson Health System
|
Other |
|
|
|
All |
up to 65 Years (Child, Adult) |
NCT02915406 |
20160472 |
|
|
|
|
September 27, 2016 |
August 16, 2017 |
|
- University of Miami
Miami, Florida, United States
|
32 |
NCT00579137 |
Terminated Has Results |
Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders |
- Severe Combined Immunodeficiency Disease
- Severe Primary Immunodeficiency Disorder
- Undefined T Cell Deficiency Disorder
- Wiskott-Aldrick Syndrome
|
- Biological: Campath -1H
- Drug: Fludarabine
- Biological: Anti-CD45
- Procedure: Stem cell infusion
|
Interventional |
Phase 1 Phase 2 |
- Baylor College of Medicine
- Center for Cell and Gene Therapy, Baylor College of Medicine
- Texas Children's Hospital
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Number of Patients With Donor Engraftment
- Patients Alive at 1 Year
- Number of Patients With Grade III or IV Toxicity
- Number of Patients With Grade III to IV Acute GVHD
|
3 |
All |
Child, Adult, Senior |
NCT00579137 |
21123-MASCI |
MASCI |
October 2007 |
October 2009 |
October 2009 |
December 21, 2007 |
July 2, 2013 |
July 2, 2013 |
- Texas Children's Hospital
Houston, Texas, United States
|
33 |
NCT00228852 |
Completed |
IMM 0212: Busulfan With Fludarabine and Antithymocyte Globulin as Preparative Therapy for Hematopoietic Stem Cell Transplant for the Treatment of Severe Congenital T-Cell Immunodeficiency |
- T-Cell Immune Deficiency Diseases
- Severe Combined Immunodeficiency
|
- Drug: Busulfan, Fludarabine and ATG
|
Interventional |
Phase 1 Phase 2 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
|
|
|
All |
Child, Adult, Senior |
NCT00228852 |
296-2003 |
|
|
|
November 2006 |
September 29, 2005 |
June 30, 2009 |
|
- Children's Healthcare of Atlanta
Atlanta, Georgia, United States
|
34 |
NCT01380990 |
Recruiting |
Lentiviral Gene Therapy for Adenosine Deaminase (ADA) Deficiency |
- Adenosine Deaminase Deficiency
- Severe Combined Immunodeficiencies (SCID)
|
- Genetic: EF1αS-ADA lentiviral vector transduced patient Cd34+ cells
|
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 as assessed by sustained improvement in thymic function
|
10 |
Male |
up to 5 Years (Child) |
NCT01380990 |
10-MI-29 |
|
November 2012 |
December 2018 |
December 2018 |
June 27, 2011 |
September 14, 2015 |
|
- Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom
|
35 |
NCT01420627 |
Recruiting |
EZN-2279 in Patients With ADA-SCID |
- ADA-SCID
- Adenosine Deaminase Deficiency
- Severe Combined Immunodeficiency
|
- Biological: EZN-2279
- Biological: Adagen
|
Interventional |
Phase 3 |
- Leadiant Biosciences, Inc.
|
Industry |
- Allocation: Non-Randomized
- Intervention Model: Crossover Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- total erythrocyte dAXP concentration from a trough blood sample
- plasma ADA activity
- Immune status
- (and 2 more...)
|
6 |
All |
Child, Adult, Senior |
NCT01420627 |
STP-2279-002 |
|
December 2013 |
May 2018 |
March 2019 |
August 19, 2011 |
October 31, 2017 |
|
- Children's Hospital Los Angeles
Los Angeles, California, United States - University of California San Francisco
San Francisco, California, United States - National Jewish Health
Denver, Colorado, United States - (and 3 more...)
|
36 |
NCT00895271 |
Recruiting |
Establishing Fibroblast-Derived Cell Lines From Skin Biopsies of Patients With Immunodeficiency or Immunodysregulation Disorders |
- Lymphohistiocytosis, Hemophagocytic
- Common Variable Immunodeficiency
- Severe Combined Immunodeficiency
|
|
Observational |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Obtain skin punch bx to generate fibroblast, dermal, or other skin-resident cell lines in pts who previously underwent HSCT.
- A second objective of this study is to generate induced pluripotent stem cells (iPS) from skin cells of patients or healthy volunteers, serving as controls.
|
200 |
All |
2 Years to 85 Years (Child, Adult, Senior) |
NCT00895271 |
090133 09-I-0133 |
|
May 6, 2009 |
|
|
May 8, 2009 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
37 |
NCT01346150 |
Recruiting |
Patients Treated for SCID (1968-Present) |
- SCID
- ADA-SCID
- XSCID
- (and 3 more...)
|
|
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: Retrospective
|
- Retrospective Study - Part 1
- Cross-Sectional Study - Part 2
- Retrospective Study Part 1
|
1146 |
All |
Child, Adult, Senior |
NCT01346150 |
DAIT RDCRN PIDTC-6902 |
|
May 2011 |
August 2019 |
August 2019 |
May 2, 2011 |
November 9, 2017 |
|
- University of Alabama at Birmingham
Birmingham, Alabama, United States - Children's Hospital Los Angeles
Los Angeles, California, United States - University of California, Los Angeles
Los Angeles, California, United States - (and 33 more...)
|
38 |
NCT01186913 |
Recruiting |
Natural History Study of SCID Disorders |
- SCID
- Leaky SCID
- Omenn Syndrome
- (and 3 more...)
|
|
Observational |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- Office of Rare Diseases (ORD)
|
NIH |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Overall survival following HCT
- Full T cell reconstitution
- Full B cell reconstitution
- (and 8 more...)
|
209 |
All |
Child, Adult, Senior |
NCT01186913 |
DAIT RDCRN PIDTC-6901 |
|
August 2010 |
August 2019 |
August 2019 |
August 23, 2010 |
October 12, 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...)
|
39 |
NCT00001467 |
Enrolling by invitation |
Genetic Analysis of Immune Disorders |
- Healthy
- Immunologic Deficiency Syndrome
- Job's Syndrome
- Severe Combined Immunodeficiency
|
|
Observational |
|
- National Institute of Allergy and Infectious Diseases (NIAID)
- National Institutes of Health Clinical Center (CC)
|
NIH |
|
|
3546 |
All |
Child, Adult, Senior |
NCT00001467 |
950066 95-I-0066 |
|
February 3, 1995 |
|
|
November 4, 1999 |
March 29, 2018 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
40 |
NCT01019876 |
Unknown † |
Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases |
- Bone Marrow Failure
- Osteopetrosis
- Fanconi Anemia
- Severe Combined Immunodeficiency
|
- Drug: Fludarabine
- Drug: Cyclophosphamide
- Drug: Cyclophosphamide 40
- Drug: Cyclophosphamide 30
|
Interventional |
Phase 2 Phase 3 |
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Parallel Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- This study is to determine the toxicity of administering a fludarabine/cyclophosphamide (Flu/CY) or busulfan (Bu)/Flu based conditioning regimen followed by allogeneic stem cell transplant.
- To determine the risk of disease progression (including neuropsychological deterioration in patients with metabolic non-malignant diseases) following a Flu/CY or Bu/Flu based conditioning regimen.
- To measure immune reconstitution following a Flu/CY or Bu/Flu based conditioning regimen and AlloSCT in patients with selected non-malignant diseases.
- (and 2 more...)
|
50 |
All |
up to 30 Years (Child, Adult) |
NCT01019876 |
AAAB0170 CHNY-01-509 |
|
June 2002 |
May 2012 |
May 2013 |
November 25, 2009 |
October 19, 2011 |
|
- Columbia University Medical Center
New York, New York, United States
|
41 |
NCT01182857 |
Withdrawn |
Quality of Life and Neuropsychiatric Sequelae in Patients Treated With Gene Therapy for ADA-SCID and in Their Parents |
|
|
Observational |
|
- National Human Genome Research Institute (NHGRI)
- National Institutes of Health Clinical Center (CC)
|
NIH |
- Time Perspective: Prospective
|
- Quality of life and neuropsychiatric status of patients being treated with gene therapy for ADA-SCID.
- Parental stress in parents of children being treated with gene therapy for ADA-SCID.
|
0 |
All |
5 Months to 50 Years (Child, Adult) |
NCT01182857 |
100151 10-HG-0151 |
|
August 5, 2010 |
|
September 25, 2014 |
August 17, 2010 |
April 23, 2018 |
|
|
42 |
NCT00695279 |
Recruiting |
Long Term Follow Up Of Patients Who Have Received Gene Therapy Or Gene Marked Products |
- Severe Combined Immunodeficiency
- Malignancy, Hematologic
- Neuroblastoma
- (and 2 more...)
|
|
Observational |
|
- St. Jude Children's Research Hospital
|
Other |
- Observational Model: Cohort
- Time Perspective: Prospective
|
- Obtain histories for detection of significant delayed medical events in research participants who have received an integrating vector-based gene therapy/gene marked product at St. Jude Children's Research Hospital (SJCRH).
|
100 |
All |
Child, Adult, Senior |
NCT00695279 |
GENEFU |
|
January 4, 2007 |
December 2036 |
December 2036 |
June 11, 2008 |
November 20, 2017 |
|
- St. Jude Children's Research Hospital
Memphis, Tennessee, United States
|
43 |
NCT02022696 |
Completed |
Treatment of SCID Due to ADA Deficiency With Autologous Transplantation of Cord Blood or Hematopoietic CD 34+ Cells After Addition of a Normal Human ADA cDNA by the EFS-ADA Lentiviral Vector |
- Adenosine Deaminase Deficiency
- ADA-SCID
|
- Genetic: Lentiviral Gene Transfer
|
Interventional |
Phase 1 |
- National Human Genome Research Institute (NHGRI)
- UCLA@@@Duke University Medical Center
- Duke Univ. Medical Center
- (and 2 more...)
|
NIH / Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- To examine the safety of autologous transplantation of hematopoietic CD34+ cells transduced with the EFS-ADA lentiviral vector after non-myeloablative conditioning with busulfan and while withholding of PEG-ADA enzyme replacement therapy@@...
|
1 |
All |
1 Year to 65 Years (Child, Adult) |
NCT02022696 |
140038 14-HG-0038 |
|
December 16, 2013 |
December 16, 2013 |
September 21, 2017 |
December 30, 2013 |
September 27, 2017 |
|
- National Institutes of Health Clinical Center, 9000 Rockville Pike
Bethesda, Maryland, United States
|
44 |
NCT01279720 |
Completed |
Gene Therapy ADA Deficiency |
- Adenosine Deaminase Deficiency
|
- Biological: Intravenous infusion of transduced cells
|
Interventional |
Phase 1 Phase 2 |
- Great Ormond Street Hospital for Children NHS Foundation Trust
|
Other |
- Allocation: Non-Randomized
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Immunological reconstitution
- Incidence of adverse reactions
- Molecular characterisation of gene transfer
- Normalisation of nutritional status, growth, and development
|
8 |
All |
up to 18 Years (Child, Adult) |
NCT01279720 |
03MI14 |
|
October 2003 |
November 2013 |
November 2013 |
January 19, 2011 |
September 14, 2015 |
|
- Great Ormond Street Hospital for Children NHS Trust
London, United Kingdom
|
45 |
NCT00220766 |
Completed |
Rapid Infusion of Immune Globulin Intravenous (Human) In Primary Immunodeficiency Patients |
- Immunologic Deficiency Syndrome
- Agammaglobulinemia
- Severe Combined Immunodeficiency
- (and 2 more...)
|
- Drug: Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified
- Drug: Dextrose, 5% in Water
|
Interventional |
Phase 3 |
|
Industry |
- Allocation: Randomized
- Intervention Model: Crossover Assignment
- Masking: Single (Participant)
- Primary Purpose: Treatment
|
- Infusion related adverse events
- All adverse events
|
100 |
All |
18 Years to 75 Years (Adult, Senior) |
NCT00220766 |
100348 |
|
August 2002 |
August 2002 |
March 2004 |
September 22, 2005 |
September 25, 2009 |
|
- Departments of Medicine and Microbiology
Birmingham, Alabama, United States - National Jewish Medical and Researach Center
Denver, Colorado, United States - International Center for Interdisciplinary Studies of Immunology
Washington, District of Columbia, United States - (and 10 more...)
|
46 |
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
|
47 |
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
|
48 |
NCT01529827 |
Active, not recruiting Has Results |
Fludarabine Phosphate, Melphalan, and Low-Dose Total-Body Irradiation Followed by Donor Peripheral Blood Stem Cell Transplant in Treating Patients With Hematologic Malignancies |
- Accelerated Phase Chronic Myelogenous Leukemia
- Adult Acute Lymphoblastic Leukemia in Remission
- Adult Acute Myeloid Leukemia in Remission
- (and 75 more...)
|
- Drug: fludarabine phosphate
- Drug: melphalan
- Radiation: total-body irradiation
- (and 6 more...)
|
Interventional |
Phase 2 |
- Roswell Park Cancer Institute
|
Other |
- Intervention Model: Single Group Assignment
- Masking: None (Open Label)
- Primary Purpose: Treatment
|
- Transplant Related Mortality (TRM)
- Clinical Response
- Progression Free Survival (PFS) at One Year
- Median Time to Neutrophil Engraftment
|
94 |
All |
3 Years to 75 Years (Child, Adult, Senior) |
NCT01529827 |
I 177110 NCI-2011-03563 |
|
February 28, 2012 |
May 28, 2015 |
May 1, 2018 |
February 9, 2012 |
September 12, 2017 |
July 2, 2017 |
- Roswell Park Cancer Institute
Buffalo, New York, United States
|
49 |
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
|
50 |
NCT00000603 |
Completed |
Cord Blood Stem Cell Transplantation Study (COBLT) |
- Anemia, Aplastic
- Fanconi Anemia
- Hematologic Diseases
- (and 5 more...)
|
- Procedure: stem cell transplantation
|
Interventional |
Phase 2 |
- National Heart, Lung, and Blood Institute (NHLBI)
|
NIH |
- Primary Purpose: Treatment
|
|
|
All |
up to 55 Years (Child, Adult) |
NCT00000603 |
316 |
|
September 1996 |
October 2007 |
October 2007 |
October 28, 1999 |
April 14, 2016 |
|
|