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sepofarsen · 1 trial · 10 indications
Change in Best-corrected visual acuity (BCVA) relative to baseline after 12 months of treatment versus sham-procedure
| Arm | Type | Description |
|---|---|---|
| Group 1: Dose 1 sepofarsen (QR-110) | EXPERIMENTAL | Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated |
| Group 2: Dose 2 sepofarsen (QR-110) | ACTIVE_COMPARATOR | Initial loading dose, followed by maintenance doses at month 3 and every 6 months there after, administered by intravitreal injection (24 months duration of treatment). After 12 months treatment of the contralateral eye may be initiated |
| Group 3: Sham | SHAM_COMPARATOR | Sham procedure (no experimental drug administered), Day 1, month 3 and every six months there after. After 12 months cross over to active study drug may be initiated |
| Name | Type | Description |
|---|---|---|
| sepofarsen | DRUG | RNA antisense oligonucleotide for intravitreal injection |
| Sham | OTHER | Sham-Procedure (no experimental drug administered) |
Main Inclusion Criteria Relating to Study Initiation: * Male or female, ≥ 8 years of age at Screening with a clinical diagnosis of LCA10 and a molecular diagnosis of homozygosity or compound heterozygosity for the c.2991+1655A\>G mutation, based on genotyping analysis at Screening. A historic genot...
Sepofarsen is an investigational small molecule being developed for Leber Congenital Amaurosis 10 (LCA10), a rare inherited retinal disease that causes severe vision loss and blindness. It is designed to address the underlying genetic cause of the condition. Sepofarsen is currently in Phase 2 clinical development.
Sepofarsen is an antisense oligonucleotide (ASO), a class of small molecules that target RNA to modulate gene expression. It is designed to correct the splicing defect associated with Leber Congenital Amaurosis 10, potentially restoring functional protein production. This mechanism aims to address the root cause of the disease.
Sepofarsen is being developed by ProQR Therapeutics N.V., a biopharmaceutical company focused on RNA-based therapies. ProQR Therapeutics is publicly traded under the ticker symbol PRQR. The company is advancing sepofarsen through clinical trials for Leber Congenital Amaurosis 10.
Sepofarsen is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory authorities and is still being studied for safety and efficacy. The ongoing Phase 2 trial is evaluating the drug in patients with Leber Congenital Amaurosis 10.
Sepofarsen is being studied in a Phase 2 clinical trial with the identifier NCT03913143, titled "A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE)." The trial is active but not recruiting, with an enrollment of 36 participants across multiple countries.
Yes, sepofarsen is also known as QR-110. The clinical trial NCT03913143 refers to sepofarsen as QR-110 in its title. Both names refer to the same investigational drug being developed by ProQR Therapeutics for Leber Congenital Amaurosis 10.