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An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa

NCT05085964 · Status: TERMINATED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 21

Last updated 2024-08-07

Study results available
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Summary

PQ-421a-002 (Helia) is an open-label, extension study to evaluate the safety, tolerability and efficacy of QR 421a (ultevursen) administered via intravitreal (IVT) injection in one or both eyes, in subjects ≥ 12 years of age with RP due to mutations in exon 13 of the USH2A gene, for an anticipated period of 24 months, or until provision of continued treatment by other means is available, provided the subject's benefit-risk determination remains positive.

Conditions

Interventions

DRUG

RNA antisense oligonucleotide for intravitreal injection

QR-421a will be first administered to the fellow eye (as defined in the preceding study), and will be repeated every 6 months. Treatment of the study eye (as defined in the preceding study) can commence 3 months (9 months for subjects from study PQ-421a-001) after the treatment of the fellow eye has been initiated and will be repeated every 6 months as well. Continued subject treatment in this study will be pursued provided that the benefit-risk balance is positive for the individual subject.

Sponsors & Collaborators

  • Sepul Bio

    collaborator INDUSTRY

  • Laboratoires Thea

    lead INDUSTRY

Principal Investigators

  • Sepul Bio Medical Monitor · Sepul Bio

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
12 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2021-09-16
Primary Completion
2022-10-18
Completion
2022-10-18
FDA Drug
Yes

Countries

  • United States
  • Canada
  • France

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05085964 on ClinicalTrials.gov