4 enrolling by invitation, 3 terminated, 2 active not recruiting, 1 completed. 1 Not applicable.
| Phase | Trials | Status |
|---|---|---|
| Not applicable | 1 | Unknown 1 |
Follow Safety and efficacy assessments: new trials by email Literature Review of Safety and efficacy assessments trials Research Report on Safety and efficacy assessments
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| A Long-term Follow-up Study in Participants Who Received CS-101 Safety and efficacy assessments - Beta-Thalassemia | Enrolling by invitation | CorrectSequence Therapeutics Co., Ltd | 5 | 2025-01-18 | 2026-02-10 | |
| A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301 Safety and efficacy assessments - Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia | Enrolling by invitation | Editas Medicine, Inc. | 54 | 2024-06-17 | 2025-04-02 | |
| A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04 Safety and Efficacy Assessments - Cystinosis | Enrolling by invitation | Stephanie Cherqui | 50 | 2022-11-14 | 2024-07-03 | |
| A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02 Safety and efficacy assessments - Type 1 Gaucher Disease | Terminated | AVROBIO | 4 | 2021-07-06 | 2023-08-29 | |
| Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy Safety and efficacy assessments - Sickle Cell Disease | Enrolling by invitation | Genetix Biotherapeutics Inc. | 85 | 2020-10-21 | 2025-03-20 | |
| Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy Safety and efficacy assessments - Fanconi Anemia Complementation Group A, Fanconi Anemia | Active not recruiting | Rocket Pharmaceuticals Inc. | 9 | 2020-06-01 | 2026-04-13 | |
| A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456 Safety and efficacy assessments - Inherited Metabolic Disorders (IMD) | Terminated | Magenta Therapeutics, Inc. | 3 | 2019-05-21 | 2021-03-15 | |
| Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201 Safety and Efficacy Assessments - Fabry Disease | Terminated | AVROBIO | 5 | 2019-05-08 | 2023-08-31 | |
| Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL) Safety and Efficacy assessments - Beta Thalassaemia | Not applicable | Unknown | IRCCS San Raffaele | 9 | 2017-10-04 | 2022-11-23 |
| Longterm Follow-up of Subjects Treated With bb2121 Safety and efficacy assessments - Multiple Myeloma | Completed | Celgene | 50 | 2016-04-28 | 2020-01-22 | |
| Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy Safety and efficacy assessments - Transfusion-dependent Beta-Thalassemia | Active not recruiting | Genetix Biotherapeutics Inc. | 66 | 2014-01-01 | 2025-04-09 |
beta-Thalassemia (3) · Anemia (2) · Anemia, Sickle Cell (2) · Genetic Diseases, Inborn (2) · Anemia, Hemolytic (1) · Anemia, Hemolytic, Congenital (1) · Bone Marrow Failure Disorders (1) · Cystinosis (1) · Fabry Disease (1) · Fanconi Anemia (1) · Fanconi Syndrome (1) · Gaucher Disease (1) · Hematologic Diseases (1) · Hemoglobinopathies (1) · Leukodystrophy, Globoid Cell (1)
AVROBIO (2) · Genetix Biotherapeutics Inc. (2) · Celgene (1) · CorrectSequence Therapeutics Co., Ltd (1) · Editas Medicine, Inc. (1) · IRCCS San Raffaele (1) · Magenta Therapeutics, Inc. (1) · Rocket Pharmaceuticals Inc. (1) · Stephanie Cherqui (1)
United States (7) · Australia (2) · Canada (2) · France (2) · Italy (2) · Brazil (1) · China (1) · Germany (1) · Greece (1) · Spain (1) · Thailand (1) · United Kingdom (1)
Anjulika Chawla, MD (1) · Himal L Thakar, MD (1) · Inderpal Panesar, MRPharmS (1) · Kristen Hege (1) · Milena Veselinovic, MD (1) · Paul J Orchard, MD (1) · Stephanie Cherqui, PhD (1) · Yongrong Lai, M.D. (1)
PMID 35075288, PMID 23089735, PMID 25186209, PMID 39527960, PMID 39642902, PMID 41525466
2017 (1), 2019 (1)