Last reviewed · How we verify
NCT05158296
Study to Evaluate the Efficacy Safety and Tolerability of Ultevursen in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene (Sirius)
Phase 2, PHASE3 trial testing Ultevursen in Retinitis Pigmentosa in 7 participants. Terminated before completion.
12 October 2022
Quick facts
| Lead sponsor | Laboratoires Thea |
|---|---|
| Phase | Phase 2, PHASE3 |
| Status | Terminated |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 7 |
| Start date | 8 December 2021 |
| Primary completion | 12 October 2022 |
| Estimated completion | 12 October 2022 |
| Sites | 15 locations across United Kingdom, Netherlands, United States, Germany |
Drugs / interventions tested
- Ultevursen — full drug profile →
- Sham-procedure
Conditions studied
- Retinitis Pigmentosa — all drugs for Retinitis Pigmentosa →
- Usher Syndrome Type 2 — all drugs for Usher Syndrome Type 2 →
- Deaf Blind — all drugs for Deaf Blind →
- Retinal Disease — all drugs for Retinal Disease →
Sponsor
Laboratoires Thea — full company profile →
Who can join
12 and older, any sex, with Retinitis Pigmentosa or Usher Syndrome Type 2. Patients with the condition only — healthy volunteers not accepted.
Adverse events — posted to ClinicalTrials.gov
Time frame: up to 10 months for each participant. Study was terminated prematurely, therefore follow-up maximum range is first patient first visit to last patient last visit.. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Other adverse events (14 terms — click to expand)
| Reaction | System | Ultevursen 60/60 µg | Ultevursen 180/60 µg | Sham-procedure |
|---|---|---|---|---|
| Conjunctival haemorrhage | Eye disorders | — | — | — |
| Eye pain | Eye disorders | — | — | — |
| Visual impairment | Eye disorders | — | — | — |
| Vision blurred | Eye disorders | — | — | — |
| Photopsia | Eye disorders | — | — | — |
| Urinary tract infection | Infections and infestations | — | — | — |
| Tonsillitis | Infections and infestations | — | — | — |
| Sinusitis | Infections and infestations | — | — | — |
| Iron deficiency | Metabolism and nutrition disorders | — | — | — |
| Tooth injury | Injury, poisoning and procedural complications | — | — | — |
| Vertigo labyrinthine | Ear and labyrinth disorders | — | — | — |
| Arthralgia | Musculoskeletal and connective tissue disorders | — | — | — |
| Inguinal hernia | Gastrointestinal disorders | — | — | — |
| Hydrocele | Congenital, familial and genetic disorders | — | — | — |
Data from ClinicalTrials.gov NCT05158296 adverse events section.
Sponsor's own description
The purpose of this study is to evaluate the efficacy safety and tolerability of ultevursen administered via intravitreal injection (IVT) in subjects with Retinitis Pigmentosa (RP) due to mutations in exon 13 of the USH2A gene.
Publications & conference data
8 peer-reviewed publications reference this trial (live from Europe PMC):
-
A perspective on oligonucleotide therapy: Approaches to patient customization.
Thakur S, Sinhari A, Jain P, Jadhav HR. · · 2022 · cited 74× · PMID 36339619 · DOI 10.3389/fphar.2022.1006304 -
Nucleic acid drugs: recent progress and future perspectives.
Sun X, Setrerrahmane S, Li C, Hu J, et al · · 2024 · cited 72× · PMID 39609384 · DOI 10.1038/s41392-024-02035-4 -
Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8 -
Antisense Oligonucleotide Therapy for the Nervous System: From Bench to Bedside with Emphasis on Pediatric Neurology.
Amanat M, Nemeth CL, Fine AS, Leung DG, et al · · 2022 · cited 47× · PMID 36365206 · DOI 10.3390/pharmaceutics14112389 -
RNA-based therapies in inherited retinal diseases.
Girach A, Audo I, Birch DG, Huckfeldt RM, et al · · 2022 · cited 28× · PMID 36388727 · DOI 10.1177/25158414221134602 -
Modeling complex age-related eye disease.
Becker S, L'Ecuyer Z, Jones BW, Zouache MA, et al · · 2024 · cited 20× · PMID 38365085 · DOI 10.1016/j.preteyeres.2024.101247 -
Whole genome sequencing for <i>USH2A</i>-associated disease reveals several pathogenic deep-intronic variants that are amenable to splice correction.
Reurink J, Weisschuh N, Garanto A, Dockery A, et al · · 2023 · cited 20× · PMID 36785559 · DOI 10.1016/j.xhgg.2023.100181 -
Update on Clinical Trial Endpoints in Gene Therapy Trials for Inherited Retinal Diseases.
Igoe JM, Lam BL, Gregori NZ. · · 2024 · cited 18× · PMID 39336999 · DOI 10.3390/jcm13185512
Verify or expand the search:
- PubMed search for NCT05158296
- Europe PMC full search
- ASCO Meeting Library
- ESMO Meeting Library
- bioRxiv preprints
- medRxiv preprints
- Google Scholar
Related trials
Other recruiting trials for Retinitis Pigmentosa
Currently open trials in the same condition.
- NCT06891885 — A Study to Investigate the Safety of DSP-3077 After a Unilateral Eye Injection in Male and Female Participants 18 Years · Phase 1, PHASE2 · recruiting
- NCT07408232 — A Phase 1/2 Study in Healthy Volunteers and Participants With Autosomal Dominant Retinitis Pigmentosa (RHO-adRP) · Phase 1, PHASE2 · recruiting
- NCT07228793 — Natural History Study of Patients With EYS-Associated RP · recruiting
- NCT06319872 — The Effects of Disulfiram (Antabuse®) on Visual Acuity in Patients With Retinal Degeneration · Phase 1 · recruiting
- NCT06936787 — An Open-label, Dose-ascending Study of IGT001 for Retinitis Pigmentosa · Phase 1 · recruiting
Other Laboratoires Thea trials
Trials by the same sponsor.
- NCT07169695 — A Study to Compare the Effectiveness and Safety of T1695 Versus Ciclosporin in Participants With Moderate to Severe Vern · Phase 2 · recruiting
- NCT07234318 — A Non-interventional Study to Evaluate the Anti-inflammatory Effects and the Clinical Efficacy of Topical Water Free Cyc · active not recruiting
- NCT07234292 — Evaluation of Patient Satisfaction With Blephaderm as a Complementary Care in Post-eyelid Surgery. · not yet recruiting
- NCT07007702 — Anti-inflammatory Effects and Clinical Efficacy of Topical Thealoz Total Eye Drops in Patients With Chronic Dry Eye Dise · completed
- NCT06375499 — Performance and Safety of T2769 Versus Hylo-Forte® in the Treatment of Moderate to Severe Dry Eye Syndrome. · NA · completed
Verify against primary sources
- ClinicalTrials.gov — authoritative US registry record
- WHO ICTRP — international registry index
- EU Clinical Trials Register
- Sponsor press releases (Google)
- Trial protocol + status: ClinicalTrials.gov NCT05158296 (US National Library of Medicine, public domain)
- Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
- Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
- Sponsor: as reported to ClinicalTrials.gov by Laboratoires Thea
- Last refreshed: 12 July 2024
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT05158296.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing