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First-in-Human, Dose Escalation Trial of AXV-101 in BBS1-Related Retinal Degeneration

NCT07269665 · Status: NOT_YET_RECRUITING · Phase: EARLY_PHASE1 · Type: INTERVENTIONAL · Enrollment: 12

Last updated 2026-03-31

No results posted yet for this study

Summary

The goal of this first in human study is to evaluate the preliminary safety and tolerability of AXV-101 in participants with BBS1. The main questions it aims to answer are:

* Is AXV-101 safe and tolerable to use in participants with BBS1?
* To determine the therapeutic dose of AXV-101 in participants with BBS1
* To investigate the concentration of AXV-101 in blood, urine and tears (both eyes)

Participants will undergo comprehensive ophthalmic assessments to evaluate functional and structural changes from baseline to one year in the treated eye compared with the untreated eye. Additional evaluations will include blood, urine, and tear testing for safety and pharmacokinetics, and quality of life questionnaires completed by both participants and caregivers. Safety will also be assessed by monitoring the frequency and severity of adverse events, including serious adverse events, through medical history, physical examinations, and laboratory testing.

Conditions

  • Bardet-Biedl Syndrome 1
  • Retinal Degeneration

Interventions

GENETIC

AXV-101

This is a gene therapy named AXV-101, being injected into the eye in participants with BBS1 bi-allelic mutations and retinal degeneration. The treated eye will be compared with the eye not injected with AXV-101.

Sponsors & Collaborators

  • Axovia Therapeutics

    lead INDUSTRY

Principal Investigators

  • Prof Mariya Moosajee · Moorfield Eye Hospital

Study Design

Allocation
NON_RANDOMIZED
Purpose
PREVENTION
Masking
NONE
Model
PARALLEL

Eligibility

Min Age
4 Years
Max Age
17 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-05-31
Primary Completion
2027-02-28
Completion
2032-02-29

Countries

  • United Kingdom

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07269665 on ClinicalTrials.gov