Active Trials
Active Trials at UCLA Health Alpha Clinic
| Trial Sponsor | Disease Indication | Clinical Trial Name | ClinicalTrials.gov Identifier | IRB # | Phase | STATUS CT.GOV |
|---|---|---|---|---|---|---|
| CIRM | Blood Disorders | Clinical Research Study of Autologous Bone Marrow Transplantation for Sickle Cell Disease (SCD) Using Bone Marrow CD34+ Cells Modified With the Lenti/G-βAS3-FB Lentiviral Vector | NCT02247843 | 13-000288 | Phase 1/2 | Recruiting |
| Merck; CIRM | Cancer | A Phase I Trial of Intratumoral Administration of CCL21-gene Modified Dendritic Cell (DC) Combined With Intravenous Pembrolizumab For advanced NSCLC | NCT03546361 | 17-000174 | Phase 1 | Recruiting |
| CIRM; NEI | Vision Loss | Stem Cell-Based Therapy for Limbal Stem Cell Deficiency | NCT03957954 | 19-001056 | Phase 1 | Recruiting |
| Astellas Pharma, Inc. | Vision Loss | A Phase 1b, Multicenter, Dose Escalation, Evaluation of Safety and Tolerability of ASP7317 for Geographic Atrophy Secondary to Age-related Macular Degeneration | NCT03178149 | 17-000835 | Phase 1 | Recruiting |
| CIRM, UCLA | Immune Disease | Efficacy And Safety Of Cryopreserved Autologous Mobilized Peripheral Blood CD34+ Hematopoietic Stem And Progenitor Cells Transduced Ex Vivo With The EFS-ADA Lentiviral Vector In Patients With Severe Combined Immune Deficiency Due To Adenosine Deaminase Deficiency | NCT05432310 | 21-001814 | Phase 1/2 | Recruiting |
| Neurona Therapeutics Inc. | Neurological Disorders | A First-In-Human (FIH) Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE) | NCT05135091 | 21-001132 | Phase 1/2 | Recruiting |
| CIRM, NHLBI, UCSF | Blood Disorders | Transplantation of CRISPR-CAS9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patient with Severe Sickle Cell Disease | NCT04774536 | 21-001649 | Phase 1/2 | Recruiting |
| COH, Metastatic Melanoma Alliance | Cancer | Gene Modified Immune Cells (IL13Ralpha2 CAR T Cells) After Conditioning Regimen for the Treatment of Stage IIIC or IV Melanoma or Metastatic Solid Tumors | NCT04119024 | 19-001145 | Phase 1 | Recruiting |
| CIRM, NHLBI, Children's Hospital Boston | Blood Disorders | A Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell Disease (GRASP, BMT CTN 2001) (GRASP) | NCT05353647 | 22-000665 | Phase 1 | Active, not recruiting |
| Roche Holding AG | Kidney | A Phase III, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of RO7434656, An Antisense Inhibitor of Complement Factor B, in Patients with Primary IgA Nephropathy at High Risk of Progression | NCT05797610 | 23-5133 | Phase 3 | Open to Accrual |
| IECURE, INC | Genetic | A Phase I/II First-in-Human, Open-Label, Dose-Escalation Study to Evaluate the Safety and Efficacy of a Single Intravenous (IV) Administration of ECUR-506 in Males Less than 9 Months of Age with Genetically Confirmed Neonatal Onset Ornithine Transcarbamylase (OTC) Deficiency | NCT06255782 | 23-5203, 24-5243 | Phase 1/2 | Open to Accrual |
| Takeda Development Center Americas, Inc. | Genetic | A Randomized, Double-blind, Placebo-Controlled, Phase 3 Study to Evaluate the Efficacy and Safety of Fazirsiran in the Treatment of Alpha-1 Antitrypsin Deficiency Associated Liver Disease With METAVIR Stage F2 to F4 Fibrosis | NCT05677971 | 23-000093 | Phase 3 | Open to Accrual |
| Ultragenyx Pharmaceutical Inc. | Genetic | A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Seamless, Adaptive, Safety, Dose-Finding, and Phase 3 Clinical Study of UX701 AAV-Mediated Gene Transfer for the Treatment of Wilson Disease Protocol Number: UX701-CL301 | NCT04884815 | 21-000496 | Phase 3 | Open to Accrual |
| Dyne Therapeutics, Inc. | Genetic | A Randomized, Double-Blind, Placebo-Controlled, Multiple Ascending Dose Study Assessing Safety, Tolerability, Pharmacodynamics, Efficacy, and Pharmacokinetics of DYNE-251 Administered to Participants with Duchenne Muscular Dystrophy Amenable to Exon 51 Skipping | NCT05524883 | 22-001705 | Open to Accrual | |
| Avidity Biosciences, Inc | Genetic | A Randomized, Double-blind, Placebo-controlled, Phase 1/2 Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Exploratory Efficacy of AOC 1020 Administered Intravenously to Adult Participants with Facioscapulohumeral Muscular Dystrophy (FSHD) | NCT05747924 | 23-000872 | Phase 1/2 | Open to Accrual |
| Solid Biosciences LLC | Genetic | A Phase 1/2, Multicenter, Open-Label Study to Investigate the Safety, Tolerability, and Efficacy of a Single Intravenous Dose of SGT-003 in Ambulant Males with Duchenne Muscular Dystrophy | NCT06138639 | 23-001795 | Phase 1/2 | Open to Accrual |
| Lexeo Therapeutics, Inc | Neurological Disorders | A Phase 1/2 Study of the Safety and Efficacy of LX2006 Gene Therapy in Participants with Cardiomyopathy Associated with Friedreich's Ataxia | NCT05445323 | 22-000494 | Phase 1/2 | Open to Accrual |
| UCLA, Merck, NIH-NCI, Phase One Foundation, Brain Tumor Funder's Collaborative, Oncovir, Inc. | Cancer | A phase 1 randomized blinded clinical trial to evaluate early immunologic pharmacodynamic parameters for the PD-1 antibody pembrolizumab/placebo with autologous tumor lysate-pulsed dendritic cell vaccination in patients with surgically accessible recurrent/progressive glioblastoma | NCT04201873 | 19-001090 | Phase 1 | Open to Accrual |
| Moderna Therapeutics | Genetic | A Global, Phase 1/2, Open-label, Dose Escalation Study to Evaluate the Safety, Pharmacodynamics, and Pharmacokinetics of mRNA-3927 in Participants with Propionic Acidemia. | NCT04159103 | 19-002009 | Phase 1/2 | Open to Accrual |
| Regeneron Pharmaceuticals, Inc, Decibel Therapeutics, Inc. | Genetic | A Phase 1/2, Open-Label, Multicenter Trial with A Single Ascending Dose Cohort with Unilateral Intracochlear Injection Followed by A Bilateral Injection Expansion Cohort to Evaluate the Safety, Tolerability and Efficacy of DB-OTO in Children and Infants with Biallelic hOTOF Mutations | NCT05788536 | 22-5129 | Phase 1/2 | Open to Accrual |
| UCLA | Cancer | A pilot study to evaluate the safety and feasibility of neoantigen-targeted dendritic cell vaccination in diffuse hemispheric glioma, H3 G34-mutant | NCT06342908 | 23-001700 | Phase 1 | Open to Accrual |
| Moderna Therapeutics | Genetic | A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency | NCT04899310 | 22-000935 | Phase 1/2 | Open to Accrual |
| Ultragenyx Pharmaceutical Inc. | Genetic | A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8 (AAV8)-mediated Gene Transfer of Human Ornithine Transcarbamylase (OTC) in Patients with Late-onset OTC Deficiency | NCT05345171 | 22-5038 | Phase 3 | Open to Accrual |
| Avidity Biosciences, Inc | Genetic | A Phase 3 Randomized, Double-Blind, Placebo-Controlled, Global Study to Evaluate the Efficacy and Safety of Intravenous AOC 1001 for the Treatment of Myotonic Dystrophy Type 1 | NCT06411288 | 24-5160 | Phase 3 | Open to Accrual |
| Janssen Research & Development, LLC | Vision Loss | A Phase 2b, Randomized, Double-masked, Multicenter, Dose-ranging, Sham-controlled Clinical Trial to Evaluate Intravitreal JNJ-81201887(AAVCAGsCD59) Compared to Sham Procedure for the Treatment of Geographic Atrophy (GA) Secondary to Age-related Macular Degeneration (AMD) | NCT05811351 | 22-5125 | Phase 2b | Open to Accrual |
Other Active Clinical Trials at UCLA
To view more active clinical trials at UCLA, please visit our Clinical Trials Opportunities Page. On the page, you can see specific information for each trial, including a link to each trial's ClinicalTrials.gov page (via the NCT number). ClinicalTrials.gov is a registry and results database of publicly and privately supported clinical studies of human participants conducted around the world.