A Phase 1/2 Clinical Study of Intravenous Gene Transfer With an AAVrh10 Vector Expressing GALC in Krabbe Subjects Receiving Hematopoietic Stem Cell Transplantation (RESKUE)
Latest Information Update: 10 Jun 2024
At a glance
- Drugs FBX-101 (Primary)
- Indications Globoid cell leukodystrophy
- Focus Adverse reactions; First in man
- Acronyms RESKUE
- Sponsors Forge Biologics
- 29 Jan 2024 According to a Forge Biologics media release, data of clinical update on five patients with Krabbe disease for which the comments during the open period at the public meeting of the Advisory Committee on Heritable Disorders in Newborns and Children (ACHDNC) on Tuesday, January 30, 2024, in support of adding Krabbe disease to the Recommended Uniform Screening Panel (RUSP).
- 08 Jan 2024 Planned End Date changed from 1 Dec 2024 to 1 Jul 2024.
- 08 Jan 2024 Planned primary completion date changed from 1 Dec 2024 to 1 Jul 2024.