Open for Enrollment
| Title | ID/Protocol | NCT/Phase | Sponsor | P.I. |
|---|---|---|---|---|
| Multi-center, randomized, open-label, parallel-group study with LPC-401 for the treatment of myocardial iron overload in adult patients with transfusion-dependent beta-thalassemia | HEPCIDIN | NCT03381833 Phase 2 |
La Jolla Pharmaceutical Company | Ashutosh Lal, MD |
| Single-Center, Non-Randomized Study of the Safety and Efficacy of In Utero Hematopoietic Stem Cell Transplantation for the Treatment of Fetuses with Alpha Thalassemia Major | IUHCT | NCT02986698 Phase 1 |
UC San Francisco | Elliott Vichinsky, MD |
| Allogeneic Stem Cell Transplantation of CordIn (Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells) in Patients with Hemoglobinopathies | CordIn | NCT02504619 Phase 1/2 |
Gamida Cell Ltd | Mark Walters, MD |
| Long Term Follow Up for Patients who have received Allogeneic Stem Cell Transplantation of NiCord/CordIn, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells | NiCord | N/A | Gamida Cell Ltd | Mark Walters, MD |
| Sample Collection of Bone Marrow, Blood, Peripheral Blood Stem Cells or Umbilical Cord Blood from Patients with Genetic Diseases Affecting the Blood Cells | BMT-repository | N/A | California Institute for Regenerative Medicine | Mark Walters, MD |
| Evaluating Efficacy and Safety of Gene Therapy in Transfusion-dependent Beta-Thal Beta0/Beta0 Genotype by Autologous Transplantation of CD34+ Stem Cells Transduced Ex Vivo with Lentiviral BA-T87Q Globin Vector in Subjects < 50 Years | Hgb-212 | NCT02906202 Phase 3 |
Bluebird Bio | Ashutosh Lal, MD |
| Open-label, Single-arm Study to Assess Safety, Tolerability, and Efficacy of ST-400 Autologous Hematopoietic Stem Cell Transplant for Treatment of Transfusion-dependent Beta thalassemia | ST-400-01 | NCT03432364 Phase 1/2 |
Sangamo Therapeutics, Inc. | Mark Walters, MD |