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Study of Subretinally Injected AAVB-081 in Patients with Usher Syndrome Type IB (USH1B) Retinitis Pigmentosa
EU CTIS ID: 2024-518489-29-00
What this study is testing
- To assess the safety and tolerability of subretinal administration of AAVB-081 in participants with USH1B retinitis pigmentosa - To determine a well-tolerated dose with optimal risk-benefit profile (RBP)
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- All participants: Informed consent signed by the participant or participant’s legally authorized representative (if applicable).
- All participants: Male and female adults diagnosed with USH1B; ≥ 18 to 50 ≤ 60 years of age
- All participants: Molecular diagnosis of USH1B due to MYO7A mutations (homozygotes or compound heterozygotes) by a Sponsor approved Clinical Laboratory Improvement Amendments (CLIA) laboratory.
- All participants: Residual central visual function evidenced by LLVA of ≥10 letters
- All participants: Female participants of childbearing potential must use a highly effective method of contraception (see Appendix 3 for examples) for 1 year after treatment (from Day 0). Male participants who have a partner of childbearing potential must use condoms (barrier contraception) for 1 year after treatment. For the purpose of this protocol, a female participant is considered of childbearing potential (FPOCBP), i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause.
You likely can't join if
- Unable or unwilling to meet the requirements of the study.
- Previous participation in any other gene therapy trial.
- Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints (including but not limited to glaucoma, steroid response, corneal or significant lenticular or media opacities, cystoid macular oedema, macular hole, uveitis, epiretinal membrane).
- Any systemic condition that would preclude subretinal surgery.
- Profound vision loss in one eye with visual acuity of counting fingers or worse on semiquantitative scale.
- Complicating ocular and systemic diseases, medications, or clinically significant abnormal baseline laboratory values.
See the full eligibility criteria
Who can join
- All participants: Informed consent signed by the participant or participant’s legally authorized representative (if applicable).
- All participants: Male and female adults diagnosed with USH1B; ≥ 18 to 50 ≤ 60 years of age
- All participants: Molecular diagnosis of USH1B due to MYO7A mutations (homozygotes or compound heterozygotes) by a Sponsor approved Clinical Laboratory Improvement Amendments (CLIA) laboratory.
- All participants: Residual central visual function evidenced by LLVA of ≥10 letters
- All participants: Female participants of childbearing potential must use a highly effective method of contraception (see Appendix 3 for examples) for 1 year after treatment (from Day 0). Male participants who have a partner of childbearing potential must use condoms (barrier contraception) for 1 year after treatment. For the purpose of this protocol, a female participant is considered of childbearing potential (FPOCBP), i.e., fertile, following menarche and until becoming post-menopausal unless permanently sterile. Permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy. A postmenopausal state is defined as no menses for 12 months without an alternative medical cause.
What rules you out
- Unable or unwilling to meet the requirements of the study.
- Previous participation in any other gene therapy trial.
- Pre-existing eye conditions that would preclude the planned surgery or interfere with the interpretation of study endpoints (including but not limited to glaucoma, steroid response, corneal or significant lenticular or media opacities, cystoid macular oedema, macular hole, uveitis, epiretinal membrane).
- Any systemic condition that would preclude subretinal surgery.
- Profound vision loss in one eye with visual acuity of counting fingers or worse on semiquantitative scale.
- Complicating ocular and systemic diseases, medications, or clinically significant abnormal baseline laboratory values.
- Prior ocular non-macular laser within 3 months, prior cataract surgery within 3 months, use of post-operative anti-inflammatory drops in the past month, presence of inflammatory complications of cataract surgery in the past month, any other non-retinal intra-ocular surgery in the past 6 months prior to Day 0; and prior macular laser or retinal surgery at any time.
- Use of high dose vitamin A (>7500 retinol equivalent units or >3300 IU per day), tretinoin-containing skin crème (e.g., Retin-A), or isotretinoin within 3 months prior to Day 0, or intended use during the study.
- Chronic use of Viagra (sildenafil) or any other phosphodiesterase type 5 inhibitors used to treat erectile dysfunction, defined as at least once per month over the 12 months prior to Screening and until completion of study participation.
- Participants who are positive for hepatitis B, hepatitis C, HIV, tuberculosis (TB), or syphilis infection.
- Suspected or laboratory confirmed SARS-CoV-2 infection within 14 days prior to Day 0.
- History of retinal detachment.
- Has received a live, attenuated vaccine within 30 days prior to Day 0. Examples of live vaccines include, but are not limited, to the following: measles, mumps, rubella, varicella/zoster (chicken pox), yellow fever, rabies, Bacillus Calmette-Guerin (BCG), typhoid vaccine, and covid vaccine.
- Poorly controlled diabetes mellitus (of any type), defined as HbA1c ≥7, in the 6 months prior to subretinal injection.
- Presence of moderate or severe non-proliferative diabetic retinopathy or worse, diabetic macular edema (DME), retinal tumors, high axial myopia (>-6 Diopters), and micro/nanophthalmos.
- Known sensitivity to medications planned for use in the peri- operative period.
- Participants who are pregnant and/or breastfeeding at Screening.
- Any other condition that would not allow the potential participant to complete follow-up examinations during the course of the study and in the opinion of the investigator, makes the potential participant unsuitable for the study.
- Unable to communicate with suitable verbal/auditory and/or tactile sign language (in the opinion of the investigator).
- Participation in a clinical study with an investigational drug in the past six months, or 5 half-lives, whichever is longer.
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.