- Conditions
- Fanconi Anemia Complementation Group A
- Interventions
- RP-L102
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 1 Year and older
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2026
- U.S. locations
- 2
- States / cities
- Stanford, California • Minneapolis, Minnesota
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Showing 1–24
of 50
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- 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
- 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
- Conditions
- Granulomatous Disease, Chronic, X-linked
- Interventions
- Lentiviral G1XCGD Gene Therapy
- Biological
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 23 Months and older · Male only
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2024
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Bethesda, Maryland • Boston, Massachusetts
- Conditions
- β-thalassemia Major
- Interventions
- LentiGlobin BB305 Drug Product
- Genetic
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 12 Years to 35 Years
- Enrollment
- 19 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2018
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Oakland, California • Chicago, Illinois + 1 more
- Conditions
- Fabry Disease
- Interventions
- AVR-RD-01
- Drug
- Lead sponsor
- AVROBIO
- Industry
- Eligibility
- 16 Years to 50 Years · Male only
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2022
- U.S. locations
- 2
- States / cities
- Hackensack, New Jersey • Pittsburgh, Pennsylvania
- 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
- 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
- Pyruvate Kinase Deficiency
- Interventions
- RP-L301
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years to 50 Years
- Enrollment
- 4 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2025
- U.S. locations
- 1
- States / cities
- Stanford, California
- Conditions
- Leukocyte Adhesion Defect - Type I
- Interventions
- RP-L201
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 3 Months and older
- Enrollment
- 9 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2023
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Severe Combined Immunodeficiency, X Linked, Gene Therapy
- Interventions
- autologous CD34+ cell transduced with G2SCID vector
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 0 Years to 5 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2028
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
- Conditions
- Sickle Cell Disease
- Interventions
- single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 3 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Boston, Massachusetts
- Conditions
- Severe Combined Immunodeficiency Due to ADA Deficiency
- Interventions
- OTL-101
- Biological
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- 30 Days to 17 Years
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2021
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- HIV
- Interventions
- Gene modified therapy
- Biological
- Lead sponsor
- American Gene Technologies International Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2038
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Glioblastoma, Malignant Glioma, Recurrent Glioblastoma, Recurrent Glioma
- Interventions
- CARv3-TEAM-E T cells
- Drug
- Lead sponsor
- Marcela V. Maus, M.D.,Ph.D.
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 21 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2027
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Long Term Follow-Up
- Interventions
- Long Term Follow-Up
- Other
- Lead sponsor
- Calibr, a division of Scripps Research
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 40 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2044
- U.S. locations
- 3
- States / cities
- Indianapolis, Indiana • Morrisville, North Carolina • Charlottesville, Virginia
- Conditions
- Anemia, Sickle Cell
- Interventions
- ARU-1801
- Genetic
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Other
- Eligibility
- 18 Years to 45 Years
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2022
- U.S. locations
- 1
- States / cities
- Cincinnati, Ohio
- Conditions
- Lysosomal Storage Diseases, Cystinosis
- Interventions
- CTNS-RD-04 (including CTNS-RD-04-LB manufactured with LentiBOOST)
- Genetic
- Lead sponsor
- University of California, San Diego
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2024
- U.S. locations
- 1
- States / cities
- La Jolla, California
- Conditions
- Pyruvate Kinase Deficiency
- Interventions
- RP-L301
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 8 Years to 55 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2029
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Transfusion-dependent Beta-Thalassemia
- Interventions
- Safety and efficacy assessments
- Other
- Lead sponsor
- Genetix Biotherapeutics Inc.
- Industry
- Eligibility
- 0 Years to 50 Years
- Enrollment
- 66 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2035
- U.S. locations
- 6
- States / cities
- Oakland, California • Chicago, Illinois • Bethesda, Maryland + 3 more
- Conditions
- MPS-IH (Hurler Syndrome)
- Interventions
- Experimental: OTL-203, Active Comparator: Allo-HSCT
- Genetic
- Lead sponsor
- Orchard Therapeutics
- Industry
- Eligibility
- 28 Days to 30 Months
- Enrollment
- 41 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2023 – 2031
- U.S. locations
- 1
- States / cities
- Minneapolis, Minnesota
- 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
- Conditions
- Infantile Malignant Osteopetrosis
- Interventions
- RP-L401
- Biological
- Lead sponsor
- Rocket Pharmaceuticals Inc.
- Industry
- Eligibility
- 1 Month and older
- Enrollment
- 1 participant
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Wiskott-Aldrich Syndrome
- Interventions
- OTL-103
- Genetic
- Lead sponsor
- Fondazione Telethon
- Other
- Eligibility
- Up to 65 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2027
- U.S. locations
- 1
- States / cities
- Atlanta, Georgia