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Clinical Trials at the UC San Diego Health Alpha Stem Cell Clinic

At the UC San Diego CIRM Alpha Stem Cell Clinic, our goal is to bring stem cell-based therapies to patients with unmet medical needs. There are over 50 clinical trials in California's CIRM Alpha Stem Cell Clinics Network. The trials at the UC San Diego Alpha Clinic span many diseases and include UC San Diego-developed cancer treatment, Cirmtuzumab.
We invite you to browse our clinical trials below. If you believe you may be a candidate for an Active & Enrolling study, contact the person listed for that study. Some studies are no longer open to new patients, so those are listed as "Not Enrolling."

Crohn's Disease

Study to Assess Efficacy and Safety of Cx601, Adult Allogeneic Expanded Adipose-derived Stem Cells (eASC) for the Treatment of Complex Perianal Fistula(s) in Participants With Crohn's Disease (CD) (ADMIRE-CD-II) 

This study is to assess the efficacy and safety of Cx601, eASC, for the treatment of complex perianal fistulas in participants with Crohn's disease.

The study will randomize approximately 554 participants.

  • Cx601 eASCs intralesional injection
  • Placebo - Cx601 placebo-matching eASCs intralesional injection

Study treatments will be allocated, on a 1:1 ratio, by central randomization through interactive web response system (IWRS). The study will follow an add-on design, participants receiving any ongoing concomitant medical treatment, at stable doses at the time of screening, for the CD will be allowed to continue it throughout the study.

The primary efficacy analysis, will be conducted at Week 24 timepoint. The double blind design will be maintained up to Week 52 (both participant and investigator) by a specific blinding for study treatment administration and for evaluating its efficacy.

This multicenter trial will be conducted globally across 150 centers. The overall time to participate in this study is approximately 5 years.

STATUS: Active / Enrolling

PI: Samuel Eisenstein
Site Contact: 858-657-5284
Trial Sponsor: Takeda

Knee Osteoarthritis

Bone Marrow Aspirate Concentrate (BMAC)Treatment for Knee Osteoarthritis (BMAC)                               

This is a pilot open-label, non-randomized, single institution study of BMAC administration in patient's with moderate to severe osteoarthritis of the knee who will undergo total knee replacement. Several weeks prior to total knee replacement, patients will have a sample of bone marrow taken from their pelvic region and concentrated using an investigational device called the Arthrex Angel Concentrated Platelet Rich Plasma (cPRP) System also known as the Angel System. The concentrated bone marrow will be immediately delivered back to the patient arthroscopically to the knee of interest. Blood and tissues samples will be collected at scheduled visits for molecular and histological analysis. Adverse events will be monitored throughout the trial. Assessment of healing will be performed by physical exam and standardized questionnaires related to the health of the patient.

STATUS: Active / Enrolling

PI: Kenneth Kalunian
Contact Number: 858-249-3015
Trial Sponsor: UC San Diego

Alcoholic Steatohepatitis

Guselkumab (Anti-IL 23 Monoclonal Antibody) for Alcohol Associated Liver Disease                                                 

This is a phase I study of guselkumab, a humanized anti-IL23 monoclonal antibody, for patients with alcoholic liver disease. This drug is approved for the use in psoriatic arthritis but not for alcoholic liver disease. The investigators will be using a standard 3+3 phase I dose escalation trial design, the dose levels will start from 30 mg, 70 mg and to 100 mg, a maximum total of 24 patients will be evaluable. In this study the investigators propose to establish safety of the product in those with alcoholic liver disease and efficacy (secondary endpoint) will be determined by biomarkers for liver inflammation and fibrosis surrogate biomarkers.                                                                                                                    

STATUS: Active / Enrolling

PI: Rohit Loomba
Contact Number: 858-246-5333
Trial Sponsor: UC San Diego

Nephropathic Cystinosis

Phase 1/2 Trial for Hematopoietic Stem Cell Gene Therapy for Cystinosis                                                                     

Cystinosis is a rare inherited recessive disease belonging to the family of Lysosomal Storage Disorders and is characterized by lysosomal accumulation of cystine in all the cells of the body leading to multi-organ failure. Cystinosis has a devastating impact on the affected individuals, primarily children, and young adults, even with cysteamine treatment. The prevalence of cystinosis is 1 in 100,000 to 1 in 200,000. The gene involved in cystinosis is the gene CTNS that encodes for the transmembrane lysosomal cystine transporter - cystinosin. The current standard of care does not prevent the progression of the disease and significantly impacts the quality of life of patients with cystinosis.

For this study, up to 6 subjects meeting eligibility criteria will be transplanted following a 3-cohort staggered treatment design with 2 subjects per cohort. The first 2 cohorts will consist of 4 adults (18 years or older), potentially followed by a cohort consisting of 2 adolescents or adults (> 14 years old). Following the informed consent process, enrolled subjects will be screened to confirm full eligibility for participation. Eligible subjects will undergo hematopoietic stem cell (HSC) mobilization and collection (leukapheresis). A portion of cells will be kept as "back-up" for rescue purpose if necessary, and a portion will be ex vivo gene-modified with a lentiviral vector, pCCL-CTNS, to express CTNS gene (product name: CTNS-RD-04). The subjects will receive marrow cytoreduction with busulfan prior to infusion of CTNS-RD-04. Subjects will discontinue cysteamine treatment during the assessment period. The assessment follow-up period will include an initial 2 years of active end-point evaluations, where the subjects will be evaluated at 3-, 6-, 9-, 12-, 18- and 24-months post-transplantation. A Long-Term Follow-Up study (LTFU) for a total 15-year follow-up period will be offered to all subjects.

The objectives of this Phase 1/2 clinical study are to assess the safety/tolerability of CTNS-RD-04, and its efficacy through a number of clinical, molecular and biochemical assessments.

STATUS: Active / Enrolling

PI: Stephanie Cherqui 
Contact Number: 844-317-7836 
Trial Sponsor: UC San Diego

Spinal Cord Injury

Study to Evaluate the Safety and Preliminary Efficacy of IDCT, a Treatment for Symptomatic Lumbar Intervertebral Disc Degeneration

This is a Phase I, first-in-human, randomized, double-blind, vehicle and placebo-controlled, parallel-group, multi-center study in subjects with single-level, symptomatic lumbar intervertebral disc degeneration (>6 months) and unresponsive to conservative therapy for at least 3 months. The study will compare single intradiscal injections of high and low dose IDCT with two control groups (saline, Sodium Hyaluronate).

8 study visits will be completed by all subjects; screening, day 1 (injection day), week 4, week 12, week 26, week 52, week 72 and week 104. The subject will be assessed for safety and efficacy utilizing VAS and ODI questionnaires alongside radiographic evaluations. The study will have a 1 year follow-up and a 1 year extension period (total 2 years).

STATUS: Active / Not Enrolling

PI: Joseph Ciacci
Contact Number: 844-317-7836 
Trial Sponsor: DiscGenics


Safety Study of Human Spinal Cord-derived Neural Stem Cell Transplantation for the Treatment of Chronic SCI (SCI)

Phase I, open-label, single-site, safety study of HSSC transplantation for the treatment of chronic spinal cord injury (SCI). Group A enrolled 4 subjects with a cord injury at T2-T12; Group B will enroll 4 subjects with a C5-C7 cord injury. Study period will be 6 months post-operative. Post-study, subjects will be followed for an additional 54 months.

STATUS: Active / Enrolling

PI: Joseph Ciacci
Contact Number: 844-317-7836 
Trial Sponsor: Seneca Biopharma


Study of Probable Benefit of the Neuro-Spinal Scaffold™ in Subjects With Complete Thoracic AIS A Spinal Cord Injury as Compared to Standard of Care (INSPIRE 2)                                                                                   

This is a randomized, controlled, single blind, two-arm, multicenter Humanitarian Device Exemption (HDE) study to evaluate the safety and probable benefit of the poly(lactic-co-glycolic acid)-b-poly(L-lysine) Scaffold ("Scaffold") in subjects with thoracic AIS A traumatic spinal cord injury at neurological level of injury of T2-T12 as compared to standard of care open spine surgery. Subjects will be randomized in a blinded manner to one of two study arms, the Treatment or "Scaffold" Arm and the Standard of Care or "Comparator" Arm. Subjects in the Scaffold Arm will have the Scaffold implantation immediately following standard of care open spine surgery. Subjects in the Comparator Arm will have standard of care open spine surgery and will not receive the Scaffold. The subjects will be blinded to their study arm for the duration of the study.

Primary Objective: To evaluate whether the Scaffold is safe and demonstrates probable benefit for the treatment of complete T2-T12 spinal cord injury as compared to standard of care open spine surgery.

Regulatory Objective: To enhance the clinical evidence for the Scaffold in the treatment of complete thoracic spinal cord injuries.

Intended Use: The Scaffold is intended for use in patients age 16-70 years diagnosed with a T2-T12 neurological level of injury functionally complete (AIS A) spinal cord injury for whom open spine surgery, (e.g., laminectomy, spine stabilization) which allows access to the dura of the injured spinal cord, is recommended as an option. The Scaffold is intended to be implanted in a cavity at the epicenter of the spinal cord contusion during open spine surgery. The Scaffold is intended for use in recent (≤7 days) spinal cord injuries that do not involve penetrating injury to the cord or complete severing of the cord.

STATUS: Active / Enrolling

PI: Joseph Ciacci
Contact Number: 844-317-7836 
Trial Sponsor: InVivo Therapeutics

B-Cell Malignancies

Open label, First in human study Evaluating the Combination of CLBR001, an Engineered Autologous T-Cell product and SWI019, an Antibody-based Biologic in Patients with Relapsed/Refractory B-cell Malignancies 

CLBR001 + SWI019 is a two-component therapy comprising an autologous chimeric antigen receptor T (CAR-T) cell product (CLBR001, the switchable CAR-T cell (sCAR-T)) and an anti-CD19 (cluster of differentiation antigen 19) antibody (SWI019, the switch, a biologic). In combination, SWI019 acts as an adapter molecule that controls the activity of the CLBR001 CAR-T cell product.

STATUS: Active / Enrolling

PI: Carolyn Mulroney
Contact Number:  858-822-5364 or 858-822-3614
Trial Sponsor: CALIBR


Long-term Follow-up Study for Patients Treated With CLBR001 CAR-T                                                               

Patients will be enrolled following either the completion or early termination/discontinuation from Study NCT04450069 or any protocol in which patients were administered CLBR001. Patients will begin the long-term follow-up period regardless of whether they responded to treatment or progressed on treatment. Patients will be followed for up to 15 years post CLBR001 infusion and will continue to be monitored for safety, immunogenicity, and efficacy. 

STATUS: Active / Enrolling

PI: Carolyn Mulroney
Contact Number:  858-822-5364 or 858-822-3614
Trial Sponsor: CALIBR

Advanced Tumors

Dose-escalation study of CRX100 in patients with Advanced Solid Tumors                                                   

This clinical study is an open-label, phase 1, dose-escalation study to determine the safety, tolerability, and pharmacokinetic (PK) properties of CRX100 in adult subjects with advanced solid tumors. Patients will be screened and evaluated to determine whether or not they meet stated inclusion criteria. Enrolled subjects will undergo leukapheresis to enable the ex vivo generation of autologous cytokine induced killer (CIK) cells. Patients with triple-negative breast cancer, colorectal cancer, hepatocellular carcinoma, osteosarcoma, epithelial ovarian cancer, and gastric cancer will be considered.

STATUS: Active / Enrolling

PI: Sandip Patel
Contact Number: 858-822-5354
Trial Sponsor: BioEclipse Therapeutics

Chronic Lymphocytic Leukemia (CLL)

Cirmtuzumab Consolidation for Treatment of Patients With Detectable CLL on Venetoclax (Venetoclax)                 

This is a phase 2 study to test whether cirmtuzumab in combination with venetoclax given as consolidation therapy can decrease the number of cancer cells that may be left in the bone marrow or in the blood in patients who have been treated with venetoclax for at least one year. Consolidation therapy is given after initial cancer treatment to further reduce the number of cancer cells that may be left in the body. Cirmtuzumab, a monoclonal antibody that inhibits receptor tyrosine kinase like orphan receptor (ROR1) signaling and stemness, may be effective in reducing the risk of disease progression in patients with detectable minimal residual disease (MRD) after treatment with venetoclax.

STATUS: Active / Enrolling

PI: Benjamin Heyman
Contact Number: 858-249-3000 
Trial Sponsor: Oncternal Therapeutics

B-Cell Lymphoid Malignancies

A Study of Cirmtuzumab and Ibrutinib in Patients With B-Cell Lymphoid Malignancies

This is a Phase 1b/2 study to investigate the safety and effectiveness of the investigational drug, cirmtuzumab, when given in combination with ibrutinib in patients with B-cell lymphoid malignancies. The Phase 1b will be conducted in two parts (Part 1 and Part 2). Part 1 is a dose-finding evaluation of the sequential administration of cirmtuzumab monotherapy followed by cirmtuzumab and ibrutinib combination therapy in chronic lymphocytic leukemia /small lymphocytic leukemia (CLL/SLL) or previously treated mantle cell lymphoma (MCL) subjects who have not received prior Bruton tyrosine kinase (BTK) inhibitor therapy. Up to 48 subjects will be enrolled in Part 1 to determine the recommended dosing regimen (RDR). In Part 2, up to 18 subjects will be enrolled to further evaluate the safety and pharmacology of the cirmtuzumab and ibrutinib combination given at the RDR determined in Part 1 of the study. In the Phase 2 (Part 3) portion of the study, approximately 90 subjects with CLL/SLL who may have received minimal prior BTK inhibitor therapy will be randomized to either Arm 1 (cirmtuzumab and ibrutinib) at the RDR or Arm 2 (ibrutinib alone) to evaluate the clinical activity and safety of the two arms.

STATUS: Active / Enrolling

PI: Michael Choi
Contact Number: 844-317-7836 
Trial Sponsor: Oncternal Therapeutics

Ovarian Cancer

Autologous Dendritic Cells Loaded With Autologous Tumor Associated Antigens for Treatment of Advanced Epithelial Ovarian Carcinomas                                                                                                                                 

This is a double-blind study in which approximately 99 study patients will be randomized in a 2:1 ratio to receive either AVOVA-1 or MC. Patients eligible for randomization and treatment will be those (1) who have undergone debulking surgery, (2) for whom a cell line has been established, (3) who have undergone leukapheresis from which sufficient PMBC were obtained, and (4) have an ECOG performance grade of 0 or 1 (Karnofsky score of 70-100%).

The primary endpoint of this trial is death from any cause with the metric of OS from the date of randomization. PFS will be a secondary endpoint and will be calculated as the time from the date of randomization for treatment until subjective tumor progression or death. Progression will be subjectively defined by the treating physician, and is expected to be based on tumor marker levels (e.g. CA-125) and/or imaging. Secondarily, we will also define PFS and OS from the date of debulking surgery.

Patients will be stratified into (1) no evidence of disease (NED) (no measurable or non-measurable disease per RECIST and normal CA-125 levels) or (2) non-NED (measurable or non-measurable disease per RECIST or elevated CA-125 levels).

STATUS: Active / Not Enrolling

PI: Ramez Eskander
Contact Number: 858-822-6100
Trial Sponsor: AVITA