Retinitis Pigmentosa (RP)Phase 2CAPELLANCT07796646
Study to Evaluate the Long-Term Safety of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
A Phase 2 study testing Intravitreal Injection of Ultevursen in people with Retinitis Pigmentosa (RP) and Usher Syndrome Type 2, run by Laboratoires Thea.
Trial 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. Read more about this study
- Who is it for?
- Ages 8 years and older
- Conditions the study is for
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind
- What is being tested?
- Intravitreal Injection of Ultevursen
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 81 participants
- Where
- No sites listed yet
- Study dates
- 5 Oct 2026to Mar 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 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
- Be adults (≥18 years old) who are able to provide informed consent OR minors (8 to <18 years old) providing assent with parental permission
- Participant (and/or parent/caregiver) willing and able to comply with the 24-month protocol, attend all study visits, and complete all assessments
- Treatment Cohort only: Non-pregnant and non-breastfeeding. Women of childbearing potential (WOCBP) and fertile males must use highly effective contraception
Who cannot join
- 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.
- Treatment Cohort only: Known hypersensitivity to antisense oligonucleotides or any constituents of the injection
- 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).
- 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
- Both cohorts: Participation in any other interventional clinical trial involving an investigational drug or device during the course of the CAPELLA study
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
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.
Read the 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.
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital
- Being tested
- No intervention, will not receive any active study intervention, Intravitreal Injection of Ultevursen
- Keywords
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, USH2A, RP, Exon 13, RNA Therapies, antisense oligonucleotide, exon skipping, LUNA, Capella, IVT, NSRP
- Medical subject headings
- Retinitis Pigmentosa, Usher Syndromes, Retinal Diseases, Eye Diseases, Hereditary, Eye Abnormalities, Vision Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
No intervention, will not receive any active study interventionOther
Observation only (no experimental drug administered)
Intravitreal Injection of UltevursenDrug
Participants will receive up to 4 doses over a 24-month period
| Study group | Role | What they receive |
|---|---|---|
| Ultevursen 180/60 μg | Experimental | Participants will receive an intravitreal injection (IVT) of Ultevursen with concentrations of 3.6 mg/mL for initial dose and 1.2 mg/mL for maintenance doses every 6 months thereafter through Month 18 (up to 4 doses). The dose concentrations will be determined by the participant's randomized cohort assignment during the Phase 2 LUNA study. Receives: Drug: Intravitreal Injection of Ultevursen |
| Observation Only | Other | Participants who are assigned to the Observation Only Cohort will not receive active study treatment via intravitreal injection (IVT), but will be asked to visit the trial location at Screening/Baseline, Months 12 and 24 for safety follow up and other study-related tests. Receives: Other: No intervention, will not receive any active study intervention |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
Measured: 24 Months
Incidence and severity of non-ocular AEs, including SAEs.
Measured: 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Sep 2026
- Study start5 Oct 2026 (estimated)
- Primary completion28 Feb 2030 (estimated)
- Study completionMar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Retinitis Pigmentosa (RP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- 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.
- Masking
- None (open label)
- Enrolment
- 81 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Laboratoires Thea
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Sepul Bio
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07796646
- Sponsor's protocol ID
- SB-421a-005
- Other IDs
- 2026-527242-45-00
- Acronym
- CAPELLA
- Status last verified
- Jun 2026
- First submitted
- 20 Aug 2026
- Last updated
- 1 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Extension Study to Evaluate the Long-Term Safety and Tolerability of Ultevursen in Participants With Retinitis Pigmentosa (RP) Due to Mutations in Exon 13 of the USH2A Gene
- ClinicalTrials.gov ID
- NCT07796646
- Acronym / sponsor ID
- CAPELLA / SB-421a-005
- Conditions
- Retinitis Pigmentosa (RP), Usher Syndrome Type 2, Deaf Blind, Retinal Disease, Eye Diseases, Hereditary, Eye Disorders Congenital
- Keywords
- Retinitis Pigmentosa, Usher Syndrome Type 2, Deaf Blind, USH2A, RP, Exon 13, RNA Therapies, antisense oligonucleotide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 81 participants (estimated)
- Lead sponsor
- Laboratoires TheaIndustry (pharma, biotech, devices)
- Collaborators
- Sepul Bio
- Study start
- 5 Oct 2026 (estimated)
- Primary completion
- 28 Feb 2030 (estimated)
- Study completion
- Mar 2030 (estimated)
- First posted
- 1 Sep 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- 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.
| Arm | Type | Receives |
|---|---|---|
| Ultevursen 180/60 μg | Experimental | Drug: Intravitreal Injection of UltevursenParticipants will receive an intravitreal injection (IVT) of Ultevursen with concentrations of 3.6 mg/mL for initial dose and 1.2 mg/mL for maintenance doses every 6 months thereafter through Month 18 (up to 4 doses). The dose concentrations will be determined by the participant's randomized cohort assignment during the Phase 2 LUNA study. |
| Observation Only | Other | Other: No intervention, will not receive any active study interventionParticipants who are assigned to the Observation Only Cohort will not receive active study treatment via intravitreal injection (IVT), but will be asked to visit the trial location at Screening/Baseline, Months 12 and 24 for safety follow up and other study-related tests. |
Who may qualify
- Ages
- 8 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 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
- Be adults (≥18 years old) who are able to provide informed consent OR minors (8 to <18 years old) providing assent with parental permission
- Participant (and/or parent/caregiver) willing and able to comply with the 24-month protocol, attend all study visits, and complete all assessments
- Treatment Cohort only: Non-pregnant and non-breastfeeding. Women of childbearing potential (WOCBP) and fertile males must use highly effective contraception
Key exclusion criteria (6 listed)
- 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
- Treatment Cohort only: Known hypersensitivity to antisense oligonucleotides or any constituents of the injection
- 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).
- 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
- Both cohorts: Participation in any other interventional clinical trial involving an investigational drug or device during the course of the CAPELLA study
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| No intervention, will not receive any active study interventionObservation only (no experimental drug administered) | Other | Observation Only |
| Intravitreal Injection of UltevursenParticipants will receive up to 4 doses over a 24-month period | Drug | Ultevursen 180/60 μg |
Primary outcome
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI)
Time frame: 24 Months
Incidence and severity of non-ocular AEs, including SAEs.
Time frame: 24 Months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Sep 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Intravitreal Injection of Ultevursen in people with Retinitis Pigmentosa (RP) and Usher Syndrome Type 2, run by Laboratoires Thea.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 1 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Sepul Bio Advocacy Director (+31 617060791, contact@sepulbio.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence and severity of ocular adverse events (AEs), including serious AEs (SAEs) and adverse events of special interest (AESI) (time frame: 24 Months). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Sep 2026.