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drugset / Trial / NCT05507996

Recombinant Adeno-associated Virus Administration for Patients With Menkes Syndrome

NCT05507996 ↗

Phase 0 Terminated 1 enrolled Kunming Hope of Health Hospital
NaSingle-groupOpen-labelTreatment

Summary

It is a single-center, open, single-arm, non-randomized investigator-initiated trial evaluating the efficacy and safety of recombinant adeno-associated virus administration for patients with Menkes syndrome.

Timeline

Start
2022-08-18
Primary completion
2022-11-23
Completion
2022-11-23

Outcome

Outcome not reported

Stopped: “The PI decides to stop.”

Drugs

EvaluationDrugModalityDoseRoute
Subject BEVUFENOGENE NOFEPARVOVEC Unknown 1e+12 unknown —
Subject BEVUFENOGENE NOFEPARVOVEC Unknown 1e+14 unknown —

Indications

Menkes disease