Clinical Trials

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