2 active not recruiting, 2 completed, 1 recruiting, 1 temporarily not available. 4 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 4 | Completed 2, Active not recruiting 1, Recruiting 1 |
| Phase 2 | 4 | Completed 2, Active not recruiting 1, Recruiting 1 |
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Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a - Sickle Cell Disease | Phase 2 | Active not recruiting | David Williams | 25 | 2022-07-12 | 2026-08-25 |
| Phase I/II Trial of Lentiviral Gene Transfer for SCID-X1 With Low Dose Targeted Busulfan Conditioning autologous CD34+ cell transduced with G2SCID vector - Severe Combined Immunodeficiency, X Linked, Gene Therapy | Phase 1 / Phase 2 | Recruiting | David Williams | 12 | 2018-02-26 | 2025-12-11 |
| Gene Transfer for Sickle Cell Disease single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a - Sickle Cell Disease | Phase 1 | Active not recruiting | David Williams | 10 | 2018-02-13 | 2026-01-09 |
| Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome Retrovirus-mediated gene transfer - Wiskott-Aldrich Syndrome | Phase 1 / Phase 2 | Completed | David Williams | 5 | 2011-07-01 | 2025-08-14 |
| Gene Transfer for Severe Combined Immunodeficiency, X-linked (SCID-X1) Using a Self-inactivating (SIN) Gammaretroviral Vector Gene transfer - Severe Combined Immunodeficiency | Phase 1 / Phase 2 | Completed | David Williams | 8 | 2010-04-01 | 2026-04-21 |
| Expanded Access Protocol for Gene Therapy Utilizing shmiR Lentivirus Vector to Induce Fetal Hemoglobin in Sickle Cell Disease Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a - Sickle Cell Disease | Temporarily not available | David Williams | 2026-06-11 |
Anemia, Sickle Cell (3) · beta-Thalassemia (3) · X-Linked Combined Immunodeficiency Diseases (2) · Severe Combined Immunodeficiency (1) · Wiskott-Aldrich Syndrome (1)
United States (6)
Erica Esrick, MD (1) · Jennifer Whangbo, M.D. (1) · Jennifer Whangbo, MD (1) · Sung-Yun Pai, MD (1)
PMID 42233421, PMID 25295500, PMID 30261899, PMID 33283990, PMID 37478401
1OT2HL154815 (1)