Current Stem Cell Clinical Trials

UCLA

X-linked Chronic Granulomatous Disease
This study is a prospective non-controlled, non-randomized Phase I/II clinical trial to assess the safety, feasibility and efficacy of cellular gene therapy in patients with chronic granulomatous disease using transplantation of autologous bone marrow CD34+ cells transduced ex vivo by the G1XCGD lentiviral vector containing the human CGD gene.

Ages Eligible for Study: 23 Months and older
Genders Eligible for Study: Male

Contacts
Donald B Kohn, MD (310) 794-1964 dkohn1@mednet.ucla.edu
Caroline Kuo, MD (310) 825-6481 ckuo@mednet.ucla.edu


Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency
The study is open to twenty (20) infants and children diagnosed with ADA-deficient SCID who do not have a medically eligible, HLA-identical sibling donor for bone marrow transplantation.

Ages Eligible for Study: 1 Month and older
Genders Eligible for Study: Both

Contacts
Donald B Kohn, MD (310) 794-1964 dkohn1@mednet.ucla.edu
Kit L Shaw, PhD (310) 267-0584 kshaw@mednet.ucla.edu


Sickle Cell Disease
This Phase I clinical trial assesses the safety and initial evidence for efficacy of an autologous transplant of lentiviral vector modified bone marrow for adults with severe sickle cell disease.

Ages Eligible for Study: 18 Years and older
Genders Eligible for Study: Both

Contacts
Gary Schiller, MD 310-206-5755 gschiller@mednet.ucla.edu
Fabrizia Urbinati, PhD 310-794-1884 furbinati@ucla.edu


HIV
This Phase I clinical trial assesses the safety of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection. It is an early phase research study looking at whether an experimental gene transfer, LVsh5/C46 (also known as Cal-1), is safe and if it can protect the immune system from the effects of HIV without the use of antiretroviral drugs.

Ages Eligible for Study: 18 Years to 65 Years
Genders Eligible for Study: Both

Contacts
Maricela Gonzalez, 310-557-3729 mmgonzalez@mednet.ucla.edu
Principal Investigator: Ronald Mitsuyasu, M.D.


UC Irvine

Retinitis Pigmentosa (RP) Disease 
A Prospective, Multicenter, Open-Label, Single-Arm Study of the Safety and Tolerability of a Single, Intravitreal Injection of Human Retinal Progenitor Cells (jCell) in Adult Subjects with Retinitis Pigmentosa (RP).

This Phase 1/2a study evaluates the safety and potential activity of a single dose of live human retinal progenitor cells (jCell) administered to adults with retinitis pigmentosa. Two different dose levels of cells will be assessed in each of two groups of patients.

Ages Eligible for Study: 18 Years and older
Genders Eligible for Study: Both

Contact
Baruch D. Kuppermann, MD, PhD (949)824-3990 stemcell@uci.edu


Ischemic Stroke
A Study of Allogeneic Mesenchymal Bone Marrow Cells in Subjects With Ischemic Stroke

***Please Note: As of 05/03/2016 this study is no longer recruiting patients. It is an ongoing clinical trial but no new patients are being recruited to participate in the study at this time***

This Phase I/II study evaluates the safety and potential of a single intravenous dose of allogeneic mesenchymal bone marrow cells to subjects with Ischemic Stroke

Ages Eligible for Study: 18 years and older
Genders Eligible for Study: Both

Contact
Steven Cramer, MD 949-824-3990 or stemcell@uci.edu


Glioblastoma Multiforme

Phase 3 Randomized, Double-blind, Controlled Study of ICT-107 in Glioblastoma

ICT-107 consists of dendritic cells, prepared from autologous mononuclear cells that are pulsed with six synthetic peptides that were derived from tumor associated antigens (TAA) present on glioblastoma tumor cells. This is a Phase 3 study to evaluate ICT-107 in patients with newly diagnosed glioblastoma. Subjects will be randomized to receive standard of care chemoradiation (temozolomide (TMZ) with either ICT-107 or a blinded control. Reinfusion with the pulsed dendritic cells should stimulate cytotoxic T cells to specifically target glioblastoma tumour cells.

Ages Eligible for Study: 18 Years and older
Genders Eligible for Study: Both

Contacts
Daniela Bota, MD, PhD, 949-824-3990 or stemcell@uci.edu