Clinical Trials

As part of the CIRM Alpha Stem Cell Clinics Network, the UCSF Alpha Stem Cell Clinic is pursuing its goal to accelerate the development and delivery of stem cell therapies to patients through high quality clinical trials. Ongoing trials include:

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Alpha Thalassemia Major

A 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

This is a phase 1 clinical trial to demonstrate the safety, feasibility and efficacy of performing in utero stem cell transplantation on fetuses affected with ATM. The investigators aim to recruit ten participants with a prenatal diagnosis of ATM. Participants will undergo bone marrow harvest and an in utero transfusion combined with maternal stem cells. Transplanting maternal cells into the fetus takes advantage of existing maternal-fetal tolerance during pregnancy. Hematopoietic stem cell (HSC) transplantation into the fetus takes advantage of the developing fetal immune system to induce tolerance to the transplanted cells without using conditioning or immunosuppression. Performing stem cell transplantation at the same time as IUT minimizes any additional procedural risk to the fetus.

The investigators hope to demonstrate that it is safe and feasible to perform in utero stem cell transplantation. Additionally, the investigators want to demonstrate postnatal chimerism of maternal cells so that, if a bone marrow transplant remains necessary after delivery, conditioning and immune suppression will not be required.

Status of enrollment:

Open to accrual

Ages Eligible for Study:

18 Weeks to 25 Weeks

Genders Eligible for Study:

All

Disease indication:

• Alpha Thalassemia Major

• Hemoglobinopathy; With Thalassemia

• Hemoglobinopathies

• Fetal Anemia

• Fetal Hydrops

• Alpha; Thalassemia

• Thalassemia Major

• Thalassemia Alpha

• A-Thalassemia

Principal Investigator:

Tippi MacKenzie, MD, [email protected]

Contact:

Romobia Hutchinson, MPA
415-476-9326
[email protected]

For more information on this study and Alpha Thalassemia Major please visit:

UCSF Center for Maternal-Fetal Precision Medicine

The Fetal Treatment Center

Study details on clinicaltrials.gov

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Beta-Thalassemia

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT)

This is a single-arm, multi-site, single-dose, phase 1/2 study to assess ST-400 in subjects with transfusion-dependent β-thalassemia (TDT). ST-400 is a type of investigational therapy that consists of gene edited cells. ST-400 is composed of the patient's own blood stem cells which are genetically modified in the laboratory using Sangamo's zinc finger nuclease (ZFN) technology to disrupt a precise and specific sequence of the enhancer of the BCL11A gene (which normally suppresses fetal hemoglobin production in erythrocytes).

Status of enrollment:

open to accrual

Ages Eligible for Study:

18 Years to 40 Years

Genders Eligible for Study:

All

Disease indication:

• Transfusion Dependent Beta-thalassemia

Principal Investigator:

Mark Walters, MD, 510-428-3774, [email protected]

Contact:

Julie DeCaris, 510-428-3885 ext 2739, jdecari[email protected]

Study Details on ClinicalTrials.gov

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Leukemia

Pediatric and Young Adult Leukemia Adoptive Therapy (PLAT)-02: A Phase 1/2 Feasibility and Safety Study of CD19-CAR T Cell Immunotherapy for CD19+ Leukemia

Patients with relapsed or refractory leukemia often develop resistance to chemotherapy. For this reason, we are attempting to use T cells obtained directly from the patient, which can be genetically modified to express a chimeric antigen receptor (CAR). The CAR enables the T cell to recognize and kill the leukemic cell through the recognition of CD19, a protein expressed of the surface of the leukemic cell in patients with CD19+ leukemia. This is a phase 1/2 study designed to determine the maximum tolerated dose of the CAR+ T cells as well as to determine the efficacy.

Status of enrollment:

open to accrual

Ages Eligible for Study:

1 to 26 years

Genders Eligible for Study:

All

Disease indication:

• CD19+ Acute Leukemia

Principal Investigator:

Anurag Agrawal, MD, 510-428-3539, [email protected]

Contact:

Julie DeCaris, 510-428-3885 ext 2739, [email protected]

Study Details of ClinicalTrials.gov

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Severe Combined Immune Deficiency Syndrome (SCID)

A Phase 1 study to evaluate the safety and tolerability of tandemly-purified allogeneic CD34+CD90+ HSC administered following conditioning with AMG 191 to achieve engraftment and immune reconstitution in patients with SCID This is a single-arm, open label, Phase 1 study to assess the safety and tolerability of CD34+CD90+ hematopoietic stem cell (HSC) allografts infused into patients with SCID who are conditioned for transplantation with AMG 191, an antibody that targets CD117 present on endogenous HSC. The target dose of CD34+CD90+ HSC will be >1 x 10^6 cells/kg and the optimal conditioning dose of AMG 191 will be determined during dose escalation.

Status of enrollment:

open to accrual

Ages Eligible for Study:

3 months and older

Genders Eligible for Study:

All

Disease indication:

SCID

Principal Investigator:

Christopher Dvorak, MD,

Contact:

Carol Fraser-Brown, 415-476-2188, [email protected]

Study Details of ClinicalTrials.gov

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Sickle Cell Disease

A Phase 1 Study Evaluating Gene Therapy by Transplantation of Autologous CD34+ Stem Cells Transduced Ex Vivo with the LentiGlobin BB305 Lentiviral Vector in Subjects with Severe Sickle Cell Disease. Subject participation for this study will be 2 years post-transplant. Subjects who enroll in this study will be asked to participate in a subsequent long-term follow up study that will monitor the safety and efficacy of the treatment they receive for up to 13 years post-transplant.

Status of enrollment:

Open to accrual

Ages Eligible for Study:

18 Years and older

Genders Eligible for Study:

All

Disease indication:

• Sickle Cell Disease

Principal Investigator:

Mark Walters, MD, 510-428-3774, [email protected]

Contact:

Cyrus Bascon, 510-428-3885 ext 5396, [email protected]

Study details on clinicaltrials.gov