At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- Intravitreal Injection of Ultevursen (drug), No intervention, will not receive any active study intervention (other)
- How long the study runs
- Study runs about 43 months (dates as stated)
- About the drug or intervention
- Intravitreal Injection of Ultevursen — drug: Up to 4 doses over a 24-month period · No intervention, will not receive any active study intervention — other: Sham-procedure (no experimental drug administered)
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 8 Years and over
- Who
- All
- Number of participants
- 81
- Started
- 2024-12-11
- Last checked
- 2025-07
Plain English Summary
What is this study?
- • Testing a new treatment for retinitis pigmentosa (rp)
- • Phase2 - 81 participants
- • The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene
Who can take part?
- • Ages 8 Years and over
- • Diagnosed with retinitis pigmentosa (rp)
Where?
- • Headington - Oxford Eye Hospital
- • Edinburgh - University of Edinburgh / NHS Lothian
- • London - Moorfields Eye Hosptial
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
The purpose of this Phase 2b study is to evaluate the safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. This is a multicenter Double-masked, Randomized, Sham-controlled study which will enroll 81 subjects.
More detail
A total of eighty-one (81) RP subjects will be enrolled in this study, randomized in a 2:1 ratio to either ultevursen or sham procedure, respectively. Subjects randomized to the active treatment group will receive therapy with ultevursen administered via intravitreal (IVT) injection to the treatment eye (TE) on Day 1 and at Months 6, 12, and 18. Subjects randomized to sham will undergo a sham procedure in the TE at the corresponding timepoints.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 8 Years and over
- Who can join: All genders
Treatment history
Treatments you must have had:
- ✓ and willing and able to complete all study assessments, in the opinion of the Investigator
What the study is looking for
- ✓An adult (≥18 years) willing and able to provide agreement to take part for participation prior to performing any study...
- ✓Clearly visible and measurable SD-OCT horizontal EZ width of ≥2.2 mm in both eyes based on the assessment of the CRC.
- ✓BCVA ≥55 letters based on ETDRS (equivalent to 20/80 based on Snellen notation, or logarithm of the minimum angle of...
- ✓Impairment of VF as assessed by SP with a mean sensitivity greater than 4 decibels (dB) and less than 25 dB measured...
- ✓Mean sensitivity greater than 2 dB as determined by MP in the TE at screening.
Who cannot take part
- ✗Presence of additional non-exon 13 USH2A pathogenic mutation(s) on both USH2A alleles in subjects who have biallelic...
- ✗At screening, the EZ horizontal or vertical width are outside the field of the SD-OCT scan based on the assessment...
- ✗Receipt of any IVT injection prior to study entry.
See the full criteria
Where Is This Study? (3 UK sites)
Oxford Eye Hospital
Headington OX3 9DU, United Kingdom
University of Edinburgh / NHS Lothian
Edinburgh EH39HA, United Kingdom
Moorfields Eye Hosptial
London EC1V 2PD, United Kingdom
Research Portfolio Managers- Daniela Narvaez, Anika Kadchha, Abi Chandrakumar, Xin Liu
moorfields.resadmin@nhs.net0207 566 2036