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ADAPT: Long-term Safety Study of INZ-701 in ENPP1 and ABCC6 Deficiency
2024-512715-42-00Phase 2Completed
- Sponsor
- Inozyme Pharma
- Start
- Oct 2024
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes, Fc-Fusion Proteins → Carrier/Scaffold Proteins → Recombinant Proteins and Enzymes
- Countries
- 4
01
Overview
Phase 2, open-label, single-arm trial of INZ-701 in ENPP1 deficiency, pseudoxanthoma elasticum (PXE) and generalized arterial calcification of infancy (GACI), sponsored by Inozyme Pharma. Primary endpoints: Incidence, frequency, and severity of adverse events, Changes in clinical laboratory tests resulting in an adverse event and 2 more. Started Oct 2024; status: completed.
02
Drugs
INZ-701
03
Conditions
ENPP1 deficiencypseudoxanthoma elasticum (PXE)generalized arterial calcification of infancy (GACI)
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