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NCT03349242
Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)
trial in Retinitis Pigmentosa in 140 participants. Completed in 19 April 2024.
19 April 2024
Quick facts
| Lead sponsor | MeiraGTx UK II Ltd |
|---|---|
| Status | Completed |
| Study type | OBSERVATIONAL |
| Enrollment | 140 |
| Start date | 19 December 2017 |
| Primary completion | 19 April 2024 |
| Estimated completion | 19 April 2024 |
| Sites | 8 locations across United Kingdom, Canada, United States |
Conditions studied
- Retinitis Pigmentosa — all drugs for Retinitis Pigmentosa →
Sponsor
MeiraGTx UK II Ltd — full company profile →
Who can join
5 and older, any sex, with Retinitis Pigmentosa. Patients with the condition only — healthy volunteers not accepted.
Sponsor's own description
The rod-cone dystrophies (often referred to as retinitis pigmentosa (RP)) are a clinically and genetically heterogeneous group of disorders in which there is progressive loss of rod and later cone photoreceptor function leading to severe visual impairment. RP usually occurs as an isolated retinal disorder, but it may also be seen in association with systemic abnormalities.
Publications & conference data
7 peer-reviewed publications reference this trial (live from Europe PMC):
-
Adeno-Associated Viral Gene Therapy for Inherited Retinal Disease.
Ong T, Pennesi ME, Birch DG, Lam BL, et al · · 2019 · cited 46× · PMID 30617669 · DOI 10.1007/s11095-018-2564-5 -
Molecular Strategies for RPGR Gene Therapy.
Cehajic Kapetanovic J, McClements ME, Martinez-Fernandez de la Camara C, MacLaren RE. · · 2019 · cited 33× · PMID 31487940 · DOI 10.3390/genes10090674 -
X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects.
Mansouri V. · · 2023 · cited 18× · PMID 36346573 · DOI 10.1007/s40123-022-00602-y -
Impact of Reference Center Choice on Adaptive Optics Imaging Cone Mosaic Analysis.
Roshandel D, Sampson DM, Mackey DA, Chen FK. · · 2022 · cited 8× · PMID 35446344 · DOI 10.1167/iovs.63.4.12 -
Pearls and Pitfalls of Adaptive Optics Ophthalmoscopy in Inherited Retinal Diseases.
Ashourizadeh H, Fakhri M, Hassanpour K, Masoudi A, et al · · 2023 · cited 4× · PMID 37510157 · DOI 10.3390/diagnostics13142413 -
Health state utilities associated with X-linked retinitis pigmentosa (XLRP).
Matza LS, Li N, Stewart KD, Hashim M, et al · · 2025 · cited 2× · PMID 40095340 · DOI 10.1007/s10198-025-01761-y -
Advances in RPGR gene therapy for X‑linked retinitis pigmentosa: From preclinical insights to clinical application (Review).
Long Y, Qi J, Zhang W, Qin H, et al · · 2026 · PMID 41480687 · DOI 10.3892/ijmm.2025.5723
Verify or expand the search:
- PubMed search for NCT03349242
- 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 MeiraGTx UK II Ltd trials
Trials by the same sponsor.
- NCT03758404 — Gene Therapy for Achromatopsia (CNGA3) · Phase 1, PHASE2 · completed
- NCT04043104 — A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects · Phase 1 · completed
- NCT03252847 — Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) · Phase 1, PHASE2 · completed
- NCT03278873 — Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 · terminated
- NCT03001310 — Gene Therapy for Achromatopsia (CNGB3) · Phase 1, PHASE2 · 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 NCT03349242 (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 MeiraGTx UK II Ltd
- Last refreshed: 20 June 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/NCT03349242.
Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing