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Clinical Trials and Research

Research -- Open Studies:
▶ Fetal Transplant Study
▶ Erythroferrone Study
Research -- Closed Studies:
▶ Celgene Believe Phase 3
Research -- Other:
▶ TCRN
▶ SQUID Update
▶ DEXA Update
▶ Published articles
Open Clinical Research Trials at UCSF Benioff Children's Hospital Oakland
Department of Hematology/Oncology

Thalassemia Research and Care:

Studies Currently Being Conducted

(These studies have patients who are enrolled and/or are actively looking for new patients to enroll.)


Title Abstract
2002-075 Natural History of Iron Burden and Risk of Organ Injury as Assessed and Predicted by Non-Invasive Measurement Techniques Longitudinal assessment of whole body iron burden is essential for managing chelation and phlebotomy therapies and may be effective in predicting risk of organ injury. Biomagnetic susceptibility measurement of liver iron concentration using SQUID technology. We will assess iron burden by biosusceptometry and serum ferritin at CHRCO and evaluate the clinical evidence of cardiac, hepatic, endocrine and orthopedic dysfuntion, and relate it to total iron burden as assessed by biosusceptometry and other non-invasive techniques.
2016-073 Evaluate the Efficacy and Safety of RBCs Derived from Mirasol-treated Whole Blood Compared with Conventional RBCs in Patients Requiring Chronic Transfusion Support (PRAISE Trial) Thalassemia is the most transfused syndrome worldwide. The risk of transmitting pathogens is reduced by pre-screening of blood donors and testing of the blood. The Mirasol System offers a means to make transfusions significantly safer by targeting unscreened and undetected pathogens. This is a prospective, multi-center, randomized, crossover trial to evaluate the clinical effectiveness of RBCs derived from Mirasol-treated WB versus conventional RBCs in transfusion dependent thalassemia patients.
2016-082 Towards the Development of a Noninvasive Prenatal Testing for Beta-Hemaglobinopathies The goal of this project is to show proof of concept for a non-invasive prenatal test (NIPT) for beta-hemoglobinopathies utilizing a novel DNA probe capture assay and next generation sequencing (NGS). Our preliminary data have shown that our probe capture/NGS system can overcome the challenges implicit in the analysis of cfFDNA for NIPT: low DNA amount. The final proof of principle for this NIPT assay requires blood samples from pregnant couples, confirmed to have mutations in the beta-globin gene. For this work we are collaborating with our Indian colleagues at the Postgraduate Institute of Medical Education and Research, Chandigarh.
2017-002 Evaluation of Human Erythroferrone in Thalassemia The purpose of this research study is to learn more about proteins that regulate iron in the blood in patients with thalassemia. Blood transfusions are used to treat thalassemia which leads to excess iron in the body. Studies have shown that individuals with thalassemia can develop iron overload even if they are not receiving transfusions. Protein signals in the blood affect the way iron moves through, and how iron is stored in, the body.
2017-065 Standardizing techniques to measure pancreas volume and identify head, body and tail with MRI in transfusion dependent thalassemia Patients with thalassemia require frequent blood transfusions, putting them at high risk for loading iron into their pancreas. As a result, patients can develop endocrine and exocrine aberrations, abnormal pancreatic growth patterns, and the onset of diabetes mellitus. In contrast to non-iron overloaded patients with diabetes, the excessive amounts of iron and diabetes mellitus cause significant variations throughout the pancreatic geometries of our patients, especially after a splenectomy. The purpose of this study is to develop standardized measurement techniques that will define the geometry of the pancreas in our patients with thalassemia. Using MRI technology, we retrospectively analyze scans to develop and standardize techniques to measure pancreatic volume and length in this population.
2016-032 A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent -Thalassemia, who do not have 0/0 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral A-T87Q-Globin Vector in Subjects 12 - 50 Years of Age This gene therapy study is a single-arm, multi-site, single dose, phase 3 study to evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product in patients with ß-thalassemia major. Patients must be at least 12 years of age with transfusion dependent ß-thalassemia who do not have the ß0/ß0 mutation and are clinically stable to undergo transplantation but who lack a suitable matched family member donor.
2017-050 A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent B-Thalassemia, who have a 0/00 Genotype, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with a Lentiviral Globin Vector in Subjects 12 - 50 Years of Age This gene therapy study is a single-arm, multi-site, single dose, phase 3 study to evaluate the safety and efficacy of autologous hematopoietic stem cell transplantation (HSCT) using LentiGlobin® BB305 Drug Product in patients with transfusion dependent ß-thalassemia. Patients must be at least 12 years of age with the ß0/ß0 mutation and be clinically stable to undergo transplantation but who lack a suitable matched family member donor.
2017-075 Longterm Follow-up of Subjects with Hemoglobinopathies Treated With Ex Vivo Gene Therapy Using Autologous Hematopoietic Stem Cells Transduced With a Lentiviral Vector This gene therapy follow up study is a multi-center trial aimed at providing continued review and reporting on patients with transfusion dependent ß-thalassemia or sickle cell disease who have received the bluebird bio LentiGlobin® BB305 Drug Product and who have completed that therapeutic study. This long-term follow up study is designed to monitor patients for 15 years post gene therapy product administration to evaluate long-term safety of the gene therapy drug product used and to monitor long-term efficacy of the therapeutic effects.

Video: Gene Therapy -- The time is now -- Nick Leschly, BluebirdBio
Gene Therapy Fact Sheet Gene Therapy Fact Sheet

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Updated 1/3/2018


Northern California Comprehensive Thalassemia Center
UCSF Benioff Children's Hospital Oakland
747 52nd Street, Oakland CA 94609   •   Phone: (510) 428-3651   •   Fax: (510) 450-5647
© 2003-2012 Children's Hospital & Research Center Oakland
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