| SIngle-Stage, Open-Label, Safety and Efficacy Study of Adeno-Associated Virus Encoding Human Aromatic L-Amino Acid Decarboxylase by Magnetic Resonance MR-guided Infusion Into Midbrain in Pediatric Patients With AADC Deficiency |
|
Recruiting |
| A Phase 1/2a, Open Label, Dose Escalation Study to Evaluate the Safety and Preliminary Efficacy of TRX103 in Subjects with Moderate to Severe Treatment-Refractory Crohn’s Disease |
Disease Name
Adult Refractory Crohn's Disease
|
Recruiting |
| 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 |
|
Enrolling by invitation |
| Autologous Gene Therapy for Artemis-Deficient SCID |
|
Recruiting |
| A Phase 1 Multicenter Study Evaluating the Safety and Tolerability of GCC19CART for Subjects With Relapsed or Refractory Metastatic Colorectal Cancer |
|
Recruiting |
| PNOC018 A Phase 1 Clinical Trial of Autologous T Cells Expressing a TCR Specific for H3.3K27M With Inhibition of Endogenous TCR (KIND T Cells) in HLA-A*0201-positive Participants With Newly Diagnosed H3.3K27M-positive Diffuse Midline Gliomas |
|
Recruiting |
| A First-in-human (FIH) Study of Inhibitory Interneurons (NRTX-1001) in Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy (MTLE) |
Disease Name
Drug-resistant unilateral mesial temporal lobe epilepsy (MTLE)
|
Recruiting |
| A Study of Inhibitory Interneurons (NRTX-1001) for the Treatment of Drug-resistant Bilateral Temporal Lobe Focal Seizures |
Disease Name
Epilepsy - Bilateral Temporal Focal Seizures
|
Recruiting |
| A Two-Stage, Randomized, Controlled Trial Comparing the Safety and Efficacy of Iltamiocel with Placebo in the Treatment of Female Participants with Chronic Fecal Incontinence and a History of Obstetric Anal Sphincter Injury |
Disease Name
Female Fecal Incontinence
|
Recruiting |
| Phase 1 Study of Autologous anti-EGFRvIII synNotch Receptor Induced anti-EphA2/IL-13Rα2 CAR (E-SYNC) T Cells in Adult Participants with EGFRvIII+ Glioblastoma |
Disease Name
Glioblastoma, Brain Tumor
|
Recruiting |
| Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Subjects With Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis |
|
Active, not recruiting |
| A Feasibility Study Using CLINIMACS® for Alpha/Beta T-Cell Depletion in Stem Cell Transplant |
|
Recruiting |
| An Open-Label, Dose Escalation, Multi-Center Phase I/II Clinical Trial of ET140203 T Cells in Pediatric Subjects with Relapsed/Refractory Hepatoblastoma (HB), Hepatocellular Neoplasm-Not Otherwise Specified (HCN-NOS), or Hepatocellular Carcinoma (HCC) |
Disease Name
Hepatoblastoma, Hepatocellular Carcinoma
|
Recruiting |
| A Phase I/II Study to Evaluate AZD5851 in GPC3+ Advanced/Recurrent Hepatocellular Carcinoma |
Disease Name
Hepatocellular Carcinoma, Liver Cancer
|
Suspended |
| Safety and Anti-HIV Activity of Autologous CD4+ and CD8+ T Cells Transduced With a Lentiviral Vector Encoding Bi-specific Anti-gp120 CAR Molecules (LVgp120duoCAR-T) in Anti-retroviral Drug-treated HIV-1 Infection |
|
Recruiting |
| Pancreatic Islets and Parathyroid Gland Co-transplantation for Treatment of Diabetes in the Intra-Muscular Site |
Disease Name
Human pancreatic islets in intramuscular site
|
Recruiting |
| A Phase 1, Open-Label, Dose-Finding Study of BMS-986453, Dual Targeting BCMAxGPRC5D Chimeric Antigen Receptor T Cells, in Participants With Relapsed and/or Refractory Multiple Myeloma |
|
Recruiting |
| A Phase 1b/2 Study of GC012F (AZD0120), a Chimeric Antigen Receptor T-cell (CAR T) Therapy Targeting CD19 and B-cell Maturation Antigen (BCMA) in Subjects With Relapsed/Refractory Multiple Myeloma (RRMM) |
|
Recruiting |
| A Phase 1 Clinical Trial of Anti-CD19 Chimeric Antigen Receptor T Cells for Treatment of Relapsed or Refractory Non-Hodgkin Lymphoma |
|
Recruiting |
| A Phase 1/2 Study to Evaluate the Safety, Tolerability, and Efficacy of JSP191 for Hematopoietic Cell Transplantation Conditioning to Achieve Engraftment and Immune Reconstitution in Subjects With SCID |
Disease Name
Severe Combined Immune Deficiency Syndrome (SCID)
|
Recruiting |
| A Multi-Center, Phase 2 Gene Transfer Study Inducing Fetal Hemoglobin in Sickle Cell (GRASP, BMT CTN 2001) |
|
Active, not recruiting |
| A Study Evaluating the Safety and Efficacy of EDIT-301 in Participants With Severe Sickle Cell Disease (RUBY) |
|
Active, not recruiting |
| Transplantation of CRISPR-Cas9 Corrected Hematopoietic Stem Cells (CRISPR_SCD001) in Patients With Severe Sickle Cell Disease |
|
Recruiting |
| GUARDIAN-101: A Phase 1 Dose Escalation and Expansion Study of CLSP-1025 in Adult Patients with Solid Tumors that Harbor the p53 R175H Mutation |
|
Recruiting |
| A Phase 2, adaptive, randomized, open-label, assessor-blinded active-controlled study to evaluate the efficacy and safety of rapcabtagene autoleucel versus Standard of Care in patients suffering from systemic lupus erythematosus (SLE) with active, refract |
Disease Name
Systemic Lupus Erythematosus (SLE) with Lupus Nephritis (LN)
|
Recruiting |
| Muscle Cell Mediated Therapy for Tongue Dysphagia: An Investigation of Cook MyoSite Autologous Muscle Derived Cells |
|
Recruiting |
| EDIT-301 for Autologous Hematopoietic Stem Cell Transplant (HSCT) in Participants With Transfusion-Dependent Beta Thalassemia (TDT) |
Disease Name
Transfusion-Dependent Beta Thalassemia (TDT)
|
Active, not recruiting |