Health TrialsbyHealthPanel

Study of Subretinally Injected AAVB-081 in Patients With Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa

ConditionUsher Syndrome, Type 1B
Trial statusRecruiting
Trial phasePhase 1, Phase 2
Trial typeInterventional
Biological sexAll
Age18-50
SponsorAAVantgarde Bio Srl

About this trial

The purpose of the 081-101 study is to evaluate the safety and tolerability of a single subretinal injection of AAVB-081 in USH1B patients with retinitis pigmentosa due to a mutation in the MYO7A gene. The study will also assess the initial efficacy following AAVB-081 administration.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Molecular diagnosis of USB1B due to MYO7A mutation

Willingness to adhere to protocol per informed consent

Disqualifiers

Unwillingness to meet the requirements of the study

Participation in a clinical study with an Investigation Product in the past 6 months

Previous participation in another Gene Therapy trial

Any condition that would preclude subretinal surgery

Trial design

Design model

Sequential

Treatments tested in this trial

  • AAVB-081

    Biological/Vaccine

    Single subretinal administration

Treatment groups

15 Participants
are divided into 3 treatment groups
Group A: Cohort 1Experimental treatment 1 intervention
Group B: Cohort 2Experimental treatment 1 intervention
Group C: Cohort 3Experimental treatment 1 intervention

Trial outcomes

Primary outcomes

1

To measure the number and severity of treatment related adverse events following treatment with AAVB-081

Time frame
61 months

Secondary outcomes

1

Change from baseline in microperimetry following treatment with AAVB-081

Time frame
61 months
2

Change from baseline in static perimetry following treatment with AAVB-081

Time frame
61 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

AAVantgarde Bio Srl

Lead sponsor

Source ClinicalTrials.gov
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