Deaf Blind, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease, Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Vision Disorders
Conditions
Keywords
Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, USH2A, RP, Exon 13, RNA Therapies, antisense oligonucleotide, exon skipping, LUNA, Capella, IVT, NSRP
Brief summary
The purpose of this Phase 2 Open-label study is to evaluate the long-term safety and tolerability of Ultevursen administered via intravitreal injection (IVT) in participants with Retinitis Pigmentosa due to mutations in exon 13 of the USH2A gene. This is a multicenter study which will enroll approximately 81 participants, however, the exact number enrolled will depend on the final number completing the 24-month LUNA trial and are eligible and willing to enroll in CAPELLA.
Detailed description
Approximately Eighty-One (81) RP participants will be enrolled in this study. These participants would have completed the Phase 2b study, LUNA, and are deemed eligible to participate in the open-label study, CAPELLA. The end of study (EOS) visit for LUNA will also serve as the Screening/Baseline visit for CAPELLA. All eligible participants enrolled in the Treatment Cohort will be given the study medication (Ultevursen) via intravitreal injection (IVT) every 6 months at Screening/Baseline, Month 6, Month 12 and Month 18. The starting dosage of Ultevursen will be determined by the participant's treatment cohort they were assigned in the LUNA study (active or sham cohort). Participants not participating in the CAPELLA Treatment Cohort, either due to Investigator discretion, or participant withdrawal, will be asked to enter the "Observation Only" Cohort. These participants will not receive IVT injection of Ultevursen, but will visit the trial location at Screening/Baseline, Month 12 and Month 24 for safety follow-up and other study related tests.
Interventions
Observation only (no experimental drug administered)
Participants will receive up to 4 doses over a 24-month period
Sponsors
Laboratoires Thea
Sepul Bio
Study design
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
Participants will be grouped into either Treatment Cohort (IVT injection) or Observation Only Cohort (no IVT injection), depending on a participant's eligibility for CAPELLA and/or a participant's willingness to continue into CAPELLA after completing the Phase 2 LUNA study.
Eligibility
Sex/Gender
ALL
Age
8 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Participants who have completed the LUNA study through Month 24 and, in the Investigator's judgment, may benefit from continued treatment or observational follow-up 2. Be adults (≥18 years old) who are able to provide informed consent OR minors (8 to \<18 years old) providing assent with parental permission 3. Participant (and/or parent/caregiver) willing and able to comply with the 24-month protocol, attend all study visits, and complete all assessments 4. Treatment Cohort only: Non-pregnant and non-breastfeeding. Women of childbearing potential (WOCBP) and fertile males must use highly effective contraception
Exclusion criteria
1. Treatment Cohort only: New ocular condition in the SE (e.g., severe infection, uncontrolled glaucoma, or retinal detachment) that, in the Investigator's opinion, makes the SE unsafe for IVT injection * Additionally, any ocular condition or AE in the fellow eye that, in the opinion of the Investigator, may impact participant safety or confound the assessment of the SE. Eligibility in such cases will be evaluated on a case-by-case basis. 2. Treatment Cohort only: Known hypersensitivity to antisense oligonucleotides or any constituents of the injection 3. Treatment Cohort only: Any clinically significant safety finding observed during the LUNA trial that, in the Investigator's opinion, compromises the participant's safety with continued dosing. Participants that have had study drug temporarily withdrawn or elective ocular surgery or AESI's during the LUNA trial may be reviewed on a case by case basis for eligibility into the Treatment Cohort (Note: These participants may still be eligible for the Observation Only Cohort at the Investigator's discretion and following discussion with medical monitor). 4. Both cohorts: Presence of any significant ocular or systemic disease, or laboratory abnormality, that would increase participant risk irrespective of treatment or interfere with study assessments 5. Both cohorts: Participation in any other interventional clinical trial involving an investigational drug or device during the course of the CAPELLA study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI) | 24 Months |
| Incidence and severity of non-ocular AEs, including SAEs. | 24 Months |
Contacts
CONTACTSepul Bio Advocacy Director
Outcome results
None listed