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Clinical trial

A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency

NCT06199531 is registered in ClinicalTrials.gov with Grace Science, LLC as lead sponsor.

NCT NCT06199531
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Registry record

As filed in ClinicalTrials.gov.

Title
A Phase 1/2/3 Open-label, Single Arm, Dose-finding Study to Investigate Long-term Safety, Tolerability and Efficacy of GS-100, an Adeno-associated Virus Serotype 9 (AAV9) Vector-mediated Gene Transfer of Human NGLY1, in Patients With NGLY1 Deficiency
Lead sponsor
Grace Science, LLC
Phase
Phase 3
Overall status
Active, not recruiting
Start date
February 13, 2024
Completion date
March 31, 2031
Enrollment
10 (actual)
Sites
2

Conditions

As the registry lists them; a condition with its own page is linked.

ngly1 deficiency