Safety and efficacy assessments

OTHER11 trials

Sponsors

Genetix Biotherapeutics Inc., Celgene, IRCCS San Raffaele, Magenta Therapeutics, Inc., Rocket Pharmaceuticals Inc.

Conditions

Beta ThalassaemiaBeta-ThalassemiaCystinosisFabry DiseaseFanconi AnemiaFanconi Anemia Complementation Group AHemoglobinopathiesInherited Metabolic Disorders (IMD)

Unknown Phase

Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia (TDT) Treated With Ex Vivo Gene Therapy

Active, not recruitingNCT02633943

Genetix Biotherapeutics Inc.Transfusion-dependent Beta-Thalassemia

Start: 2014-01-31End: 2035-11-30Updated: 2025-04-09

Longterm Follow-up of Subjects Treated With bb2121

CompletedNCT02786511

CelgeneMultiple Myeloma

Start: 2016-04-28End: 2019-10-11Target: 50Updated: 2020-01-22

Long-term Follow-up of Subjects Treated With OTL-300 for Transfusion Dependent Beta-thalassemia Study (TIGET-BTHAL)

Active, not recruitingNCT03275051

IRCCS San RaffaeleBeta Thalassaemia

Start: 2017-10-04End: 2026-06-01Updated: 2022-11-23

A Follow-up Study in Patients With Inherited Metabolic Disorders (IMD) Who Underwent Hematopoietic Stem Cell Transplantation (HSCT) With MGTA-456

TerminatedNCT04008849

Magenta Therapeutics, Inc.Inherited Metabolic Disorders (IMD)

Start: 2019-05-21End: 2020-10-08Updated: 2021-03-15

Long-Term Follow-up of Subjects With Fanconi Anaemia Subtype A Treated With ex Vivo Gene Therapy

Active, not recruitingNCT04437771

Rocket Pharmaceuticals Inc.Fanconi Anemia, Fanconi Anemia Complementation Group A

Start: 2020-06-01End: 2034-01-17Target: 9Updated: 2026-04-13

Long-term Follow-up of Subjects with Sickle Cell Disease Treated with Ex Vivo Gene Therapy

Enrolling by invitationNCT04628585

Genetix Biotherapeutics Inc.Sickle Cell Disease

Start: 2020-10-21End: 2038-01-31Target: 85Updated: 2025-03-20

A Long-Term Follow-up Study of Subjects With Gaucher Disease Who Previously Received AVR-RD-02

TerminatedNCT04836377

AVROBIOType 1 Gaucher Disease

Start: 2021-07-06End: 2023-08-21Updated: 2023-08-29

Long-Term Follow-up Study of Subjects With Fabry Disease Who Received Lentiviral Gene Therapy in Study AVRO-RD-01-201

TerminatedNCT04999059

AVROBIOFabry Disease

Start: 2019-05-08End: 2023-08-16Updated: 2023-08-31

A Long-Term Follow-Up Study of Participants With Cystinosis Who Previously Received CTNS-RD-04

Enrolling by invitationNCT05146830

Stephanie CherquiCystinosis

Start: 2022-11-14End: 2036-11-30Target: 50Updated: 2024-07-03

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Enrolling by invitationNCT06363760

Editas Medicine, Inc.Hemoglobinopathies, Sickle Cell Disease, Transfusion-dependent Beta-Thalassemia

Start: 2024-06-17End: 2040-08-31Target: 54Updated: 2025-04-02

A Long-term Follow-up Study in Participants Who Received CS-101

Enrolling by invitationNCT06685536

CorrectSequence Therapeutics Co., LtdBeta-Thalassemia

Start: 2025-01-18End: 2039-07-01Target: 5Updated: 2026-02-10

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