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NCT05085964 · ClinicalTrials.gov registry record · Phase 2

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

A Phase 2 study, sponsored by Laboratoires Thea.

Terminated
Registry status
Phase 2
Development phase
21
Enrollment target

NCT05085964: Clinical Trial Phase 2 study, sponsored by Laboratoires Thea.

NCT05085964 is a Phase 2 study that was terminated before completion, run by Laboratoires Thea. The registered enrollment target is 21 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (84% lower). According to ClinicalTrials.gov, the official US trial registry.

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The verdict

NCT05085964, a Phase 2 study, was terminated before completion, sponsored by Laboratoires Thea.

TERMINATED
Registry status
Phase 2
Development phase
21 participants
Enrollment target

Study 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.

Primary Outcome

Number of subjects with ocular treatment emergent adverse events (TEAEs) in the contralateral eye (CE) is presented. Frequency of individual TEAEs by system organ class and preferred term is presented in the safety section and clinical trial summary report. Time frame of reporting is the maximum followup period from first subject first visit to last end of study visit.

Interventions

  • DRUG RNA antisense oligonucleotide for intravitreal injection

Trial Details

FieldValue
Enrollment Target 21 participants
Start Date 2021-09-16
Est. Completion 2022-10-18
Phase Phase 2
Laboratoires Thea

11 total trials

Why NCT05085964 stopped before completion

NCT05085964 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 21 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (84% lower).

The record links to 0 conditions, and to 1 intervention - of which RNA antisense oligonucleotide for intravitreal injection is the first listed.

NCT05085964 does not publish any study locations in the registry export this page uses.

Frequently Asked Questions

What is clinical trial NCT05085964 about?

NCT05085964 is a clinical study titled "An Open-Label Extension Study to Evaluate Safety & Tolerability of QR-421a in Subjects With Retinitis Pigmentosa". 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 p...

What is the current status of trial NCT05085964?

This trial is currently terminated. It is a Phase 2 study. The enrollment target is 21 participants. The study started on 2021-09-16. Estimated completion is 2022-10-18.

What interventions are being tested in trial NCT05085964?

The interventions under investigation include: RNA antisense oligonucleotide for intravitreal injection (DRUG).

Who is sponsoring clinical trial NCT05085964?

This trial is sponsored by Laboratoires Thea, which has 11 total clinical trials registered on ClinicalTrials.gov.

How this trial's enrollment target compares

Where NCT05085964's enrollment target sits among peer trials

21 1794th of 2000 higher than 196 of 2,000 other Phase 2 trials

participants (enrollment target), bucketed by value

Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.

Source ClinicalTrials.gov registry export · 2026-08-08

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Source: ClinicalTrials.gov NCT05085964, the US trial registry maintained by the National Library of Medicine. NCT05085964 (small enrollment · none site footprint · terminated) retrieved and formatted by PlainTrial, see methodology.