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Clinical trial

A First-In-Human, Open Label, Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacodynamics of a Single Dose of AXV-101 in Patients With Bardet-Biedl Syndrome 1 (BBS1) Bi-Allelic Mutations and Retinal Degeneration

NCT07269665 is registered in ClinicalTrials.gov with Axovia Therapeutics as lead sponsor.

NCT NCT07269665
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Registry record

As filed in ClinicalTrials.gov.

Title
A First-In-Human, Open Label, Dose Escalation Trial to Evaluate the Safety, Tolerability and Pharmacodynamics of a Single Dose of AXV-101 in Patients With Bardet-Biedl Syndrome 1 (BBS1) Bi-Allelic Mutations and Retinal Degeneration
Lead sponsor
Axovia Therapeutics
Phase
Phase 1
Overall status
Recruiting
Start date
June 26, 2026
Completion date
February 29, 2032
Enrollment
12 (estimated)
Sites
1

Conditions

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

bardet-biedl syndrome 1, retinal degeneration