Testing sepofarsen against a placebo for leber congenital amaurosis 10
Official title: Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)
A Double-Masked, Randomized, Placebo-Controlled, Paired-Eye Study to Evaluate the Efficacy, Safety and Tolerability of Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Due to the c.2991+1655A>G (p.Cys998X) Mutation in the CEP290 Gene
- Phase 3
- 7 groups
- Sites in Edmonton and Toronto
- Recruiting
Interventions
- Medication
sepofarsen
RNA antisense oligonucleotide for intravitreal injection
- Other intervention
Placebo IVT
Placebo with identical appearance to sepofarsen
Canadian Sites (2)
2 of 2 recruiting
- Recruiting
University of Alberta
Edmonton, Alberta
- Recruiting
The Hospital for Sick Children - SickKids
Toronto, Ontario
Eligibility Criteria
See who this study is looking for10 criteria
The trial’s own eligibility text, word for word from the registry. Only the trial site can say who takes part.
Inclusion
- +Confirmed clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation in CEP290.
- +Adults: \>=18 years / Minors: 6 to \<18 years.
- +BCVA (FrACT) equal to or worse than logMAR +0.4 (approximate Snellen equivalent 20/50) to +2.9 logMAR based on quantifiable, reliable FrACT. LP subjects with documented evidence of prior better vision eligible.
- +Symmetrical disease between the two eyes as defined by a BCVA (FrACT) within 0.2 logMAR at baselineBaselineYour starting measurements, taken before treatment begins.Read more →.
- +Detectable ONL in the macular area as determined by the CRC at ScreeningScreeningThe checks done before joining, to see whether a study fits.Read more →.
Exclusion
- −Mutations in genes other than the CEP290 gene associated with other IRD diseases or syndromes.
- −Presence of any ocular pathology in either eye that may make comparison of the eyes not feasible.
- −Presence of unstable concurrent CME, or subject started on (or changed dose of) topical or systemic carbonic anhydrase inhibitor treatment in the 3 months prior to enrollmentEnrolmentThe number of participants a study plans to include, or has included.Read more →. CME is allowed if stable for 3 months (with or without treatment).
- −Presence of any clinically significant lens opacities/cataracts based on the AREDS lens grading scale.
- −Any prior receipt of genetic (RNA or DNA therapy) or stem-cell therapy for ocular or non-ocular disease, including sepofarsen.
In plain language
Assembled directly from this trial’s registry record. Every sentence traces to a field below — nothing here is generated or interpreted.
What this study is
This study is testing a treatment for a condition.
Phase 3Phase 3A large study comparing a treatment against the current standard.Read more → — a large study comparing this against the current standard, usually across many hospitals.
From the trial registry
Built from these fields:
- designModule.designInfo.primaryPurpose
- designModule.phases
Who receives what
There are 7 groups in this study.
Groups A, C, E and F, the comparison group, receive sepofarsen.
Registry label: A: Sepofarsen - Treatment Eye - up to Month 12 · C: Continued - Treatment Eye - up to Month 24 · E: Mixed - Treatment Eye - up to Month 24 · F: Mixed - Fellow Eye - Month 12 to Month 24
Groups B, D and G, the placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group, receive Placebo IVT.
Registry label: B: Placebo - Fellow Eye - up to Month 12 · D: Continued - Fellow Eye - up to Month 24 · G: Mixed - Fellow Eye - up to Month 12
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].label
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
How the study is run
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
From the trial registry
Built from these fields:
- designModule.designInfo.allocation
- designModule.designInfo.maskingInfo.masking
Is there a placebo?
One group receives a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → — a dummy treatment with no active medicine in it.
One group receives an existing treatment, so the two can be compared.
From the trial registry
Built from these fields:
- armsInterventionsModule.armGroups[].type
- armsInterventionsModule.armGroups[].interventionNames
Who the study is looking for
The study lists a minimum age of 6 years, with no upper limit given.
The study is open to people of any sex.
The study does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
These are the criteria the study lists. Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part.
From the trial registry
Built from these fields:
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How big and how long
The study aims to enrol about 32 people.
The study is currently expected to finish around October 2028.
The main measurement is taken over: 12 Months.
From the trial registry
Built from these fields:
- designModule.enrollmentInfo.count
- statusModule.completionDateStruct.date
- outcomesModule.primaryOutcomes[].timeFrame
What the study measures
Change from baselineBaselineYour starting measurements, taken before treatment begins.Read more → in Best-Corrected Visual Acuity (BCVA) — measured over 12 Months.
From the trial registry
Built from these fields:
- outcomesModule.primaryOutcomes[].measure
- outcomesModule.primaryOutcomes[].timeFrame
Source: NCT06891443 on ClinicalTrials.gov. The full registry text is further down this page — this summary never replaces it.
Not medical advice. What do these terms mean?
What is being tested — in plain terms
About sepofarsenDrug
RNA antisense oligonucleotide for intravitreal injection
From the trial registry — its own words, unedited.
What a drug is here: A medicine — a chemical or small-molecule treatment — given as the thing being studied.
Read the full explanation → · in clinical review
About Placebo IVTOther
Placebo with identical appearance to sepofarsen
From the trial registry — its own words, unedited.
What a other is here: An intervention the registry did not place in another category.
Read the full explanation → · in clinical review
Common questions
Answered from this trial’s registry record. Where the record doesn’t say, these answers say so rather than filling the gap.
Am I eligible for this trial?
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can decide whether someone can take part. Concord cannot make that determination, and neither can any tool that has not examined you.
What the study lists: a minimum age of 6 years.
The full inclusionInclusion criteriaThe things you must have or be for a study to consider you.Read more → and exclusion criteriaExclusion criteriaThe things that would prevent someone from taking part.Read more → are published on this page, exactly as the study team wrote them.
The useful next step is to bring this trial to your doctor. Answering a few questions first gives them something concrete to review.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.eligibilityCriteria
Is there a placebo?
Yes. This study includes a placeboPlaceboA dummy treatment with no active medicine, used for comparison.Read more → group — a dummy treatment with no active medicine in it.
One group receives an existing treatment so the two can be compared.
From the trial registry
- armsInterventionsModule.armGroups[].type
Would I know which treatment I am getting?
You, the study team, the people giving the treatment, and the people analysing the results would all be kept unaware of which group you are in until the study ends.
Which group you would be placed in is decided by chance, like a coin flip — not by you and not by your doctor.
From the trial registry
- designModule.designInfo.maskingInfo.masking
- designModule.designInfo.allocation
Who can join?
The study lists a minimum age of 6 years, with no upper limit given.
It is open to people of any sex.
It does not accept healthy volunteersHealthy volunteerSomeone without the condition being studied who takes part anyway.Read more →.
Only the trial siteTrial siteA hospital or clinic where a study is actually run.Read more → can confirm whether a particular person can take part.
From the trial registry
- eligibilityModule.minimumAge
- eligibilityModule.sex
- eligibilityModule.healthyVolunteers
How long would this take?
The study's main measurement is taken over: 12 Months.
The study as a whole is currently expected to finish around 2028-10.
How long any one person takes part can differ from the study length. The study team can tell you what the schedule looks like in practice.
From the trial registry
- outcomesModule.primaryOutcomes[].timeFrame
- statusModule.completionDateStruct.date
How many people are taking part?
The study aims to enrol about 32 people.
From the trial registry
- designModule.enrollmentInfo.count
Where is this happening?
This study lists 3 locations, including: Edmonton, Alberta, Canada; Toronto, Ontario, Canada; Minneapolis, Minnesota, United States.
Sites can open and close during a study, so confirm with the team before travelling.
From the trial registry
- trial_locations
About This Trial
The purpose of this double-masked, randomized, placebo-controlled, paired-eye study is to evaluate the efficacy, safety and tolerability of Sepofarsen in subjects with Leber Congenital Amaurosis (LCA) due to the c.2991+1655A\>G (p.Cys998X) mutation in the CEP290.
Other Sites (1)
University of Minnesota Medical School
Minneapolis, Minnesota, United States
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See if this trial could fit youThis page is for informational purposes only and does not constitute medical advice. Clinical trial eligibility can only be determined by the trial site after proper screening. Trial information is sourced from ClinicalTrials.gov and may not reflect the most current status. Always consult your healthcare provider before making decisions about clinical trial participation.