Health TrialsbyHealthPanel

Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION)

ConditionsLeber Congenital Amaurosis 10BlindnessLeber Congenital AmaurosisSensation DisordersVision DisorderNeurological ManifestationsEye Diseases, HereditaryEye DiseasesEye Disorders CongenitalRetinal Disease
Trial statusRecruiting
Trial phasePhase 3
Trial typeInterventional
Biological sexAll
Age6+
SponsorLaboratoires Thea

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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 baseline.

Disqualifiers

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 enrollment. 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.

Trial design

Design model

Parallel

Treatments tested in this trial

  • sepofarsen

    Drug

    RNA antisense oligonucleotide for intravitreal injection

  • Placebo IVT

    Other intervention

    Placebo with identical appearance to sepofarsen

Treatment groups

32 Participants
are divided into 7 treatment groups

7

Treatment groups

See each treatment group below.

Group A: Sepofarsen - Treatment Eye - up to Month 12Active comparator 1 intervention
Group B: Placebo - Fellow Eye - up to Month 12Placebo comparator 1 intervention
Group C: Continued - Treatment Eye - up to Month 24Active comparator 1 intervention
Group D: Continued - Fellow Eye - up to Month 24Placebo comparator 1 intervention
Group E: Mixed - Treatment Eye - up to Month 24Active comparator 1 intervention
Group F: Mixed - Fellow Eye - Month 12 to Month 24Active comparator 1 intervention
Group G: Mixed - Fellow Eye - up to Month 12Placebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

Change from baseline in Best-Corrected Visual Acuity (BCVA)

Change from baseline in BCVA based on the Freiburg Acuity and Contrast Test (FrACT) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)

Time frame
12 Months

Secondary outcomes

1

Eye-specific Patient Global Impression of Change (PGI-C) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)

PGI-C (PATIENT GLOBAL IMPRESSION OF CHANGE ) assesses a patient's perception of overall change in their vision in the respective eye over time. The minimum score (best outcome) is 1 ("Very Much Better") and the maximum score (worst outcome) is 7 ("Very Much Worse"). Higher scores indicate a worse health outcome (greater worsening).

Time frame
Month 12
2

Change from baseline in Low Luminance Visual Acuity (LLVA) based on FrACT between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)

Time frame
Month 12
3

Change from baseline in retinal sensitivity as measured by dark-adapted Full-Field Stimulus Test (FST) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs)

Time frame
Month 12
4

Change from baseline in Contrast Sensitivity (CS) between Treatment Eyes (TEs) and Placebo Control Eyes (PCEs) based on qCSF

Time frame
Month 12

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Laboratoires Thea

Lead sponsor

Sepul Bio

Collaborator

Source ClinicalTrials.gov
Apply now