LUNA
Research type
Research Study
Full title
A Two-Year Double-masked, Randomized, Sham-Controlled Study to Evaluate the Efficacy, Safety and Tolerability of Ultevursen in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene
IRAS ID
1010743
Contact name
Daniel Sea
Contact email
Sponsor organisation
Laboratoires THEA
Research summary
Retinitis pigmentosa (RP) is caused by one or more mutations (“defects") in a gene in the DNA. This mutation disrupts production of an important protein in the eyes, called usherin, which is believed to cause vision loss. Ultevursen is the study medication which will be investigated in this trial. Ultevursen is designed to overcome this genetic defect and to restore this important protein, which may slow or stop some effects of RP.
This study involves injection of the study medication, ultevursen, or a sham injection procedure. Participants who are assigned to study medication (2 in 3 chance), will have the medication ultevursen injected into the eye. Those assigned to the sham injection procedure (1 in 3 chance) will have nothing injected, only the tip of the syringe (no needle) will touch the study eye.
Patients with RP aged 8 years and above may be eligible to take part in the study. The study will be conducted at NHS sites in England and Scotland and will last 3 years and 3 months in total. Participants who take part will be included in the study for up to 2 years.REC name
London - Chelsea Research Ethics Committee
REC reference
24/LO/0835
Date of REC Opinion
13 Jan 2025
REC opinion
Further Information Favourable Opinion