Current Stem Cell Clinical Trials

UCLA

Beta-Thalassemia

A Study to Assess the Safety, Tolerability, and Efficacy of ST-400 for Treatment of Transfusion-Dependent Beta-thalassemia (TDT) [NCT03432364]
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).

Ages Eligible for Study: 18 Years to 40 Years
Genders Eligible for Study: Both

Contacts
Principal Investigator: Donald B Kohn, MD (310) 794-1964 dkohn1@mednet.ucla.edu
Principal Investigator: Gary Schiller, MD (310) 206-5755 gschiller@mednet.ucla.edu

Contact: Bruck Habtemariam (310) 794-0242 BHabtemariam@mednet.ucla.edu


Duchenne Muscular Dystrophy

A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy (HOPE-2) [NCT03406780]
HOPE-2 is a double-blind clinical trial evaluating the safety and efficacy of a cell therapy called CAP-1002 in study participants with Duchenne muscular dystrophy (DMD). Non-ambulatory and ambulatory boys and young men who meet eligibility criteria will be randomly assigned to receive either CAP-1002 or placebo every 3 months for a total of 4 doses during a 12-month period.

Ages Eligible for Study: 10 Years and older
Genders Eligible for Study: Male

Contacts
Principal Investigator: Nancy Halnon, MD (310) 267-7667 nhalnon@mednet.ucla.edu
Contact: Michael Mapel (310) 206-4479 MMapel@mednet.ucla.edu


HIV

Safety Study of a Dual Anti-HIV Gene Transfer Construct to Treat HIV-1 Infection [NCT01734850]
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
Principal Investigator: Ronald Mitsuyasu, MD (310) 825-6689 rmitsuya@mednet.ucla.edu
Contact: Maricela Gonzalez (310) 557-3729 mmgonzalez@mednet.ucla.edu


HIV

Safety Study of Zinc Finger Nuclease CCR5-modified Hematopoietic Stem/Progenitor Cells in HIV-1 Infected Patients [NCT02500849]
The purpose of the study is to evaluate the safety and feasibility of administering SB-728mR-HSPC after conditioning with busulfan.

Ages Eligible for Study: 18 Years to 75 Years
Genders Eligible for Study: Both

Contacts
Principal Investigator: Ronald Mitsuyasu, MD (310) 825-6689 rmitsuya@mednet.ucla.edu
Contact: Maricela Gonzalez (310) 557-3729 mmgonzalez@mednet.ucla.edu


Lung Cancer

Intratumoral Administration of CCL21-gene Modified Dendritic Cell With Intravenous Pembrolizumab for Advanced NSCLC [NCT03546361]
This is a phase 1, non-randomized, dose-escalating, multi-cohort trial of intratumoral administration of CCL21-gene modified dendritic cells combined with intravenous pembrolizumab for advanced non-small cell lung cancer. All enrolled patients will continue to be followed by a physician, and undergo a history and physical examination every 3 months until progressive disease (PD) or withdrawal from the study. Eligible patients will be assigned to a cohort and will receive intratumoral injections of autologous Ad-CCL21-DC and intravenous pembrolizumab in conjunction with tumor sampling and patient monitoring.

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

Contacts
Principal Investigator: Steven M Dubinett, MD (310) 267-2725 sdubinett@mednet.ucla.edu
Principal Investigator: Edward B Garon, MD (310) 586-2098 egaron@mednet.ucla.edu

Contact: Lia Etheridge (310) 825-7174 letheridge@mednet.ucla.edu


Sarcoma

Genetically Engineered PBMC and PBSC Expressing NY-ESO-1 TCR After a Myeloablative Conditioning Regimen to Treat Patients With Advanced Cancer [NCT03240861]
This Phase I clinical trial evaluates the safety and feasibility of administering NY-ESO-1 TCR (T cell receptor) engineered peripheral blood mononuclear cells (PBMC) and peripheral blood stem cells (PBSC) after a myeloablative conditioning regimen to treat patients with cancer that has spread to other parts of the body. 


Ages Eligible for Study: 16 Years and older
Genders Eligible for Study: Both

Contacts
Principal Investigator: Antoni Ribas, MD, PhD (310) 206-3928 ARibas@mednet.ucla.edu 
Contact: Justin Tran (310) 206-2090 justintran@mednet.ucla.edu


Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency

Autologous Cryopreserved CD34+ Hematopoeitic Cells Transduced With EFS-ADA Lentivirus for ADA SCID [NCT02999984]  
This is a prospective, non-randomized, single-cohort, longitudinal, single-center, clinical study designed to assess the efficacy and safety of a cryopreserved formulation of OTL-101 (autologous CD34+ hematopoietic stem/progenitor cells transduced ex vivo with EFS LV encoding for the human ADA gene) administered to ADA-SCID subjects between the ages of 30 days and 17 years of age, who are not eligible for an HLA-matched sibling/family donor and meeting the inclusion/exclusion criteria.


Ages Eligible for Study: Up to 17 Years
Genders Eligible for Study: Both

Contacts
Principal Investigator: Donald B Kohn, MD (310) 794-1964 dkohn1@mednet.ucla.edu

Principal Investigator: Theodore B Moore, MD (310) 825-6708 tbmoore@mednet.ucla.edu
Contact: Kit L Shaw, PhD (310) 825-6188 kshaw@mednet.ucla.edu


Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency

Autologous Transplant of EFS-ADA Modified Bone Marrow Cells for ADA-Deficient Severe Combined Immunodeficiency (SCID) [NCT01852071]
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
Principal Investigator: Donald B Kohn, MD (310) 794-1964 dkohn1@mednet.ucla.edu
Contact: Kit L Shaw, PhD (310) 825-6188 kshaw@mednet.ucla.edu


Sickle Cell Disease

Stem Cell Gene Therapy for Sickly Cell Disease [NCT02247843]
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
Principal Investigator: Gary Schiller, MD (310) 206-5755 gschiller@mednet.ucla.edu
Contact: Fabrizia Urbinati, PhD (310) 794-1884 furbinati@ucla.edu


Traumatic Brain Injury

A study of Modified Stem Cells in Traumatic Brain Injury (TBI) [NCT02416492]
The primary purpose of the clinical study is to evaluate the clinical efficacy of intracranial administration of SB623 cells on patients with chronic motor deficit from Traumatic Brain Injury. A secondary purpose of the study is 1) to evaluate the effect of intracranial administration of SB623 cells on disability parameters and 2) to evaluate the safety and tolerability of intracranial administration of SB623 cells. 

Ages Eligible for Study: 18 Years to 75 Years
Genders Eligible for Study: Both

Contacts
Principal Investigator: Daniel C Lu, MD, PhD (310) 267-2975 DCLu@mednet.ucla.edu
Contact: Courtney Real, RN (310) 267-7433 creal@mednet.ucla.edu


X-linked Chronic Granulomatous Disease

Study of Gene Therapy Using a Lentiviral Vector to Treat X-Linked Chronic Granulomatous Disease [NCT02234934]
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
Principal Investigator: Donald B Kohn, MD (310) 794-1964 dkohn1@mednet.ucla.edu
Contact: Caroline Kuo, MD (310) 825-6481 ckuo@mednet.ucla.edu


 

 

 

UC Irvine

A Double-Blind, Controlled Phase 2 Study of the Safety and Efficacy of Modified Stem Cells (SB623) in Patients with Chronic Motor Deficit from Traumatic Brain Injury (TBI), PI Leonid Groysman, MD


A Phase 3, Randomized, Double-Blind, Placebo-Controlled Multicenter Study to Evaluate Efficacy and Safety of Repeated Administrations of Nurown® (Autologous Mesenchymal Stem Cells Secreting Neurotrophic Factors) in Participants with Amyotrophic Lateral Sclerosis (ALS)


A Phase 3 Study to Compare the Efficacy and Safety of Humacytes Human Acellular Vessel with that of an Autologous Arteriovenous Fistula in Subjects with End-Stage Renal Disease


A Phase 2b, Randomized, Blinded and Placebo-Controlled Trial to Evaluate the Safety and Efficacy of Longeveron Allogeneic Human Mesenchymal Stem Cells (LMSCs) Infusion in Patients with Aging Frailty


Active trials at UCI: http://clinic.stemcell.uci.edu/search-clinical-trials.asp

Contact for all open studies at UCI is stemcell@uci.edu or (949) 824-3990