- Conditions
- Severe Combined Immunodeficiency Disease, X-linked
- Interventions
- CL20-i4-EF1α-hγc-OPT, Busulfan, CliniMacs
- Genetic · Drug · Device
- Lead sponsor
- St. Jude Children's Research Hospital
- Other
- Eligibility
- Up to 24 Months · Male only
- Enrollment
- 28 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2034
- U.S. locations
- 3
- States / cities
- San Francisco, California • Memphis, Tennessee • Seattle, Washington
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results come from ClinicalTrials.gov. When the live registry is unavailable, a clearly labeled stored copy may be shown.
These results were retrieved from the live ClinicalTrials.gov registry. Filters and sort order apply to this result list.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Showing 25–36
of 36
matching trials from the live ClinicalTrials.gov search.
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- ADA gene transfer
- Biological
- Lead sponsor
- Donald B. Kohn, M.D.
- Other
- Eligibility
- 1 Month to 18 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2008 – 2015
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Sickle Cell Disease
- Interventions
- lovo-cel
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 12 Years to 50 Years
- Enrollment
- 54 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 11
- States / cities
- Birmingham, Alabama • Oakland, California • Atlanta, Georgia + 8 more
- Conditions
- Hemophilia A
- Interventions
- Auto CD34+PBSC, transduced with a lentiviral vector encoding the B domain deleted from of human coagulation factor VIII
- Biological
- Lead sponsor
- Medical College of Wisconsin
- Other
- Eligibility
- 18 Years and older · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2033
- U.S. locations
- 1
- States / cities
- Milwaukee, Wisconsin
- Conditions
- Severe Combined Immunodeficiency Due to ADA Deficiency
- Interventions
- Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101), busulfan, PEG-ADA ERT
- Genetic · Drug
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 30 Days to 17 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Beta-Thalassemia
- Interventions
- LentiGlobin BB305 Drug Product
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 0 Years to 50 Years
- Enrollment
- 24 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2022
- U.S. locations
- 3
- States / cities
- Oakland, California • Chicago, Illinois • Philadelphia, Pennsylvania
- Conditions
- ADA-SCID
- Interventions
- Infusion of autologous EFS-ADA LV CD34+ (OTL-101), busulfan, PEG-ADA ERT
- Genetic · Drug
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 1 Month to 17 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Bethesda, Maryland
- Conditions
- Cerebral Adrenoleukodystrophy (CALD)
- Interventions
- Lenti-D Drug Product (eli-cel)
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- Up to 17 Years · Male only
- Enrollment
- 32 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2021
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Boston, Massachusetts • Minneapolis, Minnesota
- Conditions
- Glioblastoma Multiforme (WHO Grade IV), Anaplastic Astrocytoma (WHO Grade III)
- Interventions
- 06Benzylguanine, MGMT P140K, Meltenyi CliniMacs
- Drug · Genetic · Device
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 5 Years to 55 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2012
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Anemia, Sickle Cell
- Interventions
- Autologous enriched CD34+ cell fraction that contains CD34+ cells transduced with lentiviral vector encoding human γ-globinG16D and short-hairpin RNA734
- Biological
- Lead sponsor
- CSL Behring
- Industry
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Duarte, California
- Conditions
- Sickle Cell Disease
- Interventions
- bb1111
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 2 Years to 50 Years
- Enrollment
- 35 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2027
- U.S. locations
- 9
- States / cities
- Birmingham, Alabama • Washington D.C., District of Columbia • Boston, Massachusetts + 6 more
- Conditions
- Sickle Cell Disease
- Interventions
- βAS3-FB vector transduced peripheral blood CD34+ cells
- Biological
- Lead sponsor
- Donald B. Kohn, M.D.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2025
- U.S. locations
- 1
- States / cities
- Los Angeles, California