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NCT03349242

Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR)

Completed Last updated 20 June 2024
What this trial tests

trial in Retinitis Pigmentosa in 140 participants. Completed in 19 April 2024.

Timeline
19 December 2017
Primary endpoint
19 April 2024
19 April 2024

Quick facts

Lead sponsorMeiraGTx UK II Ltd
StatusCompleted
Study typeOBSERVATIONAL
Enrollment140
Start date19 December 2017
Primary completion19 April 2024
Estimated completion19 April 2024
Sites8 locations across United Kingdom, Canada, United States

Conditions studied

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):

  1. 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
  2. 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
  3. X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects.
    Mansouri V. · · 2023 · cited 18× · PMID 36346573 · DOI 10.1007/s40123-022-00602-y
  4. 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
  5. 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
  6. 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
  7. 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

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Other recruiting trials for Retinitis Pigmentosa

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Data sources for this page

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.

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