Thalassemia at UCSF Benioff Children's Hospital Oakland
Children’s Hospital has a renowned thalassemia research program. Our research allows us
to offer patients and families the most up-to-date and innovative therapies. Research done at Children’s
has led to many advances in thalassemia treatment. As part of each thalassemia patient’s care, we
consult with your family about studies your child may be qualified to participate in. Patients are never
obligated to join any study; if a family chooses not to participate in a study, their child will still receive
high quality healthcare. Our research and clinical teams work together to provide the highest quality,
integrated healthcare possible. We look forward to talking to you and your family about research
studies available for patients.
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 |
NCT03381833 |
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 |
NCT02986698 |
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 |
NCT02504619 |
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 |
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 |
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 |
NCT02906202 |
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 |
NCT03432364 |
Sangamo Therapeutics, Inc. |
Mark Walters, MD
|