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About this data Sourced primarily from ClinicalTrials.gov — a listing isn't proof of safety or FDA approval.

Pipeline and clinical trial information here comes primarily from ClinicalTrials.gov. A listing on ClinicalTrials.gov means a study has been registered — not that a treatment is proven safe, effective, or FDA-approved. Investigational treatments may never be approved. Who can join a trial is decided only by that trial's own study team.

Not yet reported

sepofarsen

Company
Laboratoires Thea
Mechanism
Mechanism not yet reported
Route
Route not yet reported
Recruiting trials
1 recruiting

Efficacy results: Not yet reported

Find a Clinical Trial Near Me

1 recruiting study currently listed on ClinicalTrials.gov.

Data source: ClinicalTrials.gov · trial data as of Oct 22, 2026

Find a Clinical Trial Near Me

See recruiting studies for sepofarsen and check eligibility with the study team.

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Treatment type

"Added to GLP-1" means the investigational treatment is being studied on top of an existing GLP-1-based therapy.

Your ZIP code is only used to calculate distance to trial sites — it isn't stored.

Trial details
  • Recruiting NCT NCT06891443

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

    Not yet reported Laboratoires Thea 32 participants Est. completion 2028-10 18 sites
    Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Sensation Disorders, Vision Disorder, Neurological Manifestations, Eye Diseases, Hereditary, Eye Diseases, Eye Disorders Congenital, Retinal Disease
    View official trial listing →
  • Completed NCT NCT03140969

    Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene

    Not yet reported, Not yet reported Laboratoires Thea 11 participants Est. completion 2019-10-02 3 sites Stand-alone
    Other conditions: Leber's Congenital Amaurosis
    View official trial listing →
  • Terminated NCT NCT03913130

    Extension Study to Study PQ-110-001 (NCT03140969)

    Not yet reported, Not yet reported Laboratoires Thea 9 participants Est. completion 2022-10-03 3 sites Stand-alone
    Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
    View official trial listing →
  • Active, not recruiting NCT NCT03913143

    A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)

    Not yet reported, Not yet reported ProQR Therapeutics 36 participants Est. completion 2023-03 14 sites Stand-alone
    Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Diseases, Hereditary, Eye Disorders Congenital, Retinal Disease
    View official trial listing →
  • Status unknown NCT NCT04855045

    An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene.

    Not yet reported, Not yet reported ProQR Therapeutics 15 participants Est. completion 2023-12 9 sites Stand-alone
    Other conditions: Leber Congenital Amaurosis 10, Blindness, Leber Congenital Amaurosis, Vision Disorders, Sensation Disorders, Neurologic Manifestations, Eye Diseases, Eye Disorders Congenital, Retinal Disease, Retinal Degeneration, Retinal Dystrophies
    View official trial listing →
Efficacy history

Not yet reported.

Safety

Not yet reported.

Sources

Also known as: sepofarsen, QR-110

Related On The Pen coverage

No related coverage yet.

This drug may be investigational and not yet approved for any use. Whether someone can join a clinical trial is determined only by that trial's own study team — On The Pen does not determine eligibility. Information here can change; always confirm current details on the official ClinicalTrials.gov listing or with the sponsor.