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Hematology/Oncology Department
Research Studies Open for Enrollment
Date: 10/22/2018

Thalassemia:

Title ID/Protocol NCT/Phase Sponsor P.I. Contact
Evaluate the Efficacy and Safety of RBCs Derived from Mirasol-treated Whole Blood Compared with Conventional RBCs in Patients Requiring Chronic Transfusion SupportPRAISE NCT03329404
TerumoAshutosh Lal, MDKacie Smith (510-428-3885 x2752)
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-thalassemiaHEPCIDIN NCT03381833
Phase 2
La Jolla Pharmaceutical CompanyAshutosh Lal, MDJennifer Ferguson, RN, MSN, FNP (510-428-3885 x5324)
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 MajorIUHCT NCT02986698
Phase 1
UC San FranciscoElliott Vichinsky, MDElliott Vichinsky, MD (510-428-3885 x3651)
Allogeneic Stem Cell Transplantation of CordIn (Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells) in Patients with HemoglobinopathiesCordIn NCT02504619
Phase 1/2
Gamida Cell LtdMark Walters, MDMarci Moriarty, RN (510-428-3385 x5396)
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 CellsNiCord N/A
Gamida Cell LtdMark Walters, MDJulie Decaris (510-428-3385 x2739)
Sample Collection of Bone Marrow, Blood, Peripheral Blood Stem Cells or Umbilical Cord Blood from Patients with Genetic Diseases Affecting the Blood CellsBMT-repository N/A
California Institute for Regenerative MedicineMark Walters, MDMarci Moriarty, RN (510-428-3385 x5396)
Evaluating Efficacy and Safety of Gene Therapy in Transfusion-dependent Beta-Thalassemia Beta0/Beta0 Genotype by Autologous Transplantation of CD34+ Stem Cells Transduced Ex Vivo with Lentiviral BA-T87Q Globin VectorHgb-212 NCT02906202
Phase 3
Bluebird BioAshutosh Lal, MDMarci Moriarty, RN (510-428-3385 x5396)
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 (TDT)ST-400-01 NCT03432364
Phase 1/2
Sangamo Therapeutics, Inc.Mark Walters, MDMarci Moriarty, RN (510-428-3385 x5396)

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