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AAV Gene Therapy for Menkes Syndrome
NCT05507996Phase 1Terminated
- Sponsor
- Kunming Hope of Health Hospital
- Start
- Aug 2022
- Primary completion
- Nov 2022
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of AAV9-ATP7A in Menkes syndrome (MNK), sponsored by Kunming Hope of Health Hospital. Primary endpoints: Monitoring the disease course or vital signs to assess the efficacy of the investigational medical product and Incidence of adverse events (AE) after the investigational medical product infusion at 12 months. Started Aug 2022; status: terminated.
02
Drugs
AAV9-ATP7A
03
Conditions
Menkes syndrome (MNK)
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