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Gene Transfer Clinical Trial for Krabbe Disease

Study acronym: RESKUE
StatusActive, Not Recruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Study Statusv17
  2. Study Statusv16
  3. Study Status, Contacts/Locationsv15
  4. Study Statusv14
  5. Study Statusv13
Show 11 earlier versionsHide 11 earlier versions
  1. Trial status: Recruiting to Active, Not Recruitingv12
  2. Study Status, Outcome Measures, Eligibility, Contacts/Locationsv11
  3. Trial status: Active, Not Recruiting to Recruitingv10
  4. Study Status, Study Description, Outcome Measures, Eligibility, Contacts/Locationsv9
  5. Trial status: Recruiting to Active, Not Recruitingv8
  6. Study Status, Contacts/Locationsv7
  7. Study Status, Contacts/Locationsv6
  8. Trial status: Not Yet Recruiting to Recruitingv5
  9. Study Status, Outcome Measuresv4
  10. Study Status, Outcome Measures, Contacts/Locationsv3
  11. Study Status, Study Design, Outcome Measuresv2
  1. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
This is a nonblinded, non-randomized dose escalation study of intravenous AAVrh10 after hematopoietic stem cell transplantation (HSCT) in which subjects will receive standard of care hematopoietic cell transplantation for Krabbe disease, followed by a single infusion of an adeno-associated virus gene therapy product. Extensive natural history subjects will be used to compare as control group.
Trial Details
NCT Number NCT04693598
Lead Sponsor Forge Biologics, Inc
Conditions
(from sponsor)		 Krabbe Disease
Related terms Leukodystrophy, Globoid Cell, Lysosomal Storage Diseases
Enrollment 6 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov