NCT04312672 is a clinical trial of AAV5-hRKp.RPGR in X-Linked Retinitis Pigmentosa, sponsored by Janssen Research & Development, LLC.

Who is sponsoring NCT04312672?

NCT04312672 is sponsored by Janssen Research & Development, LLC.

What drugs are being tested in NCT04312672?

Interventions in NCT04312672: AAV5-hRKp.RPGR.

What condition does NCT04312672 study?

NCT04312672 studies X-Linked Retinitis Pigmentosa.

Is NCT04312672 still recruiting?

NCT04312672 is currently completed. Last updated 21 January 2026.

Last reviewed 2026-01-21 · How we verify

NCT04312672

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

Long-term Follow-up Gene Therapy Study for RPGR- XLRP

Completed Last updated 21 January 2026

What this trial tests

trial testing AAV5-hRKp.RPGR in X-Linked Retinitis Pigmentosa in 42 participants. Completed in 17 September 2025.

Timeline

31 July 2017

Primary endpoint
17 September 2025

17 September 2025

Quick facts

Lead sponsor	Janssen Research & Development, LLC
Status	Completed
Study type	OBSERVATIONAL
Enrollment	42
Start date	31 July 2017
Primary completion	17 September 2025
Estimated completion	17 September 2025
Sites	5 locations across United Kingdom, United States

Drugs / interventions tested

AAV5-hRKp.RPGR — full drug profile →

Conditions studied

X-Linked Retinitis Pigmentosa — all drugs for X-Linked Retinitis Pigmentosa →

Sponsor

Janssen Research & Development, LLC — full company profile →

Who can join

5 and older, male only, with X-Linked Retinitis Pigmentosa. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This is a long-term follow-up study assessing safety of patients for up to 60 months following advanced therapy investigational medicinal product (ATIMP) AAV5-hRKp.RPGR vector in participants with XLRP caused by mutations in RPGR.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Assessing Photoreceptor Status in Retinal Dystrophies: From High-Resolution Imaging to Functional Vision.
Sahel JA, Grieve K, Pagot C, Authié C, et al · · 2021 · cited 23× · PMID 34000280 · DOI 10.1016/j.ajo.2021.04.013
X-Linked Retinitis Pigmentosa Gene Therapy: Preclinical Aspects.
Mansouri V. · · 2023 · cited 18× · PMID 36346573 · DOI 10.1007/s40123-022-00602-y
AAV2 and AAV9 tropism and transgene expression in the mouse eye and major tissues after intravitreal and subretinal delivery.
Koponen S, Kokki E, Tamminen T, Ylä-Herttuala S. · · 2023 · cited 11× · PMID 40838057 · DOI 10.3389/fddev.2023.1148795
Adeno-Associated Viral Vectors as Versatile Tools for Neurological Disorders: Focus on Delivery Routes and Therapeutic Perspectives.
Fajardo-Serrano A, Rico AJ, Roda E, Honrubia A, et al · · 2022 · cited 6× · PMID 35453499 · DOI 10.3390/biomedicines10040746
Pre-Clinical and Clinical Advances in Gene Therapy of X-Linked Retinitis Pigmentosa: Hope on the Horizon.
Pechnikova NA, Poimenidou M, Iliadis I, Zafeiriou-Chatziefraimidou M, et al · · 2025 · cited 4× · PMID 39941570 · DOI 10.3390/jcm14030898
Advances in Precision Therapeutics and Gene Therapy Applications for Retinal Diseases: Impact and Future Directions.
AlEissa MM, Alhawsawi AA, Alonazi R, Magharbil E, et al · · 2025 · cited 2× · PMID 40725503 · DOI 10.3390/genes16070847
Genetic engineering and the eye.
Murphy R, Martin KR. · · 2025 · cited 2× · PMID 39516652 · DOI 10.1038/s41433-024-03441-2
Systematic Identification of Candidate Genes for Inherited Retinal Disease Gene Therapy Integrating Worldwide IRD Cohort and Single-Cell Analysis.
Wang CY, Chen L, Lin TY, Huang SP. · · 2025 · cited 1× · PMID 40547876 · DOI 10.1155/joph/7014745

Verify or expand the search:

Other trials of AAV5-hRKp.RPGR

Trials testing the same drug.

NCT06646289 — A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis · Phase 2 · active not recruiting
NCT05926583 — A Study of AAV5-hRKp.RPGR for the Treatment of Japanese Participants With X-linked Retinitis Pigmentosa · Phase 3 · active not recruiting

Other recruiting trials for X-Linked Retinitis Pigmentosa

Currently open trials in the same condition.

NCT06646289 — A Follow-on Study for Second-Eye Treatment for Participants Previously Treated With Gene Therapy for X-Linked Retinitis · Phase 2 · active not recruiting
NCT04850118 — A Clinical Trial Evaluating the Safety and Efficacy of a Single Subretinal Injection of AGTC-501 in Participants With XL · Phase 2, PHASE3 · active not recruiting
NCT05874310 — Gene Therapy for Subjects With RPGR Mutation-associated X-linked Retinitis Pigmentosa · EARLY_PHASE1 · recruiting
NCT04794101 — Follow-up Gene Therapy Trial for the Treatment of X-linked Retinitis Pigmentosa Associated With Variants in the RPGR Gen · Phase 3 · active not recruiting

Other Janssen Research & Development, LLC trials

Trials by the same sponsor.

NCT07518186 — A Study Comparing JNJ-79635322 and Teclistamab in Participants With Relapsed or Refractory Multiple Myeloma · Phase 3 · not yet recruiting
NCT07309445 — A Study to Assess Real-World Use and Outcomes of TAR-200 for Participants With Non-Muscle Invasive Bladder Cancer (NMIBC · recruiting
NCT07499232 — A Study of Guselkumab Versus Risankizumab in Participants With Moderately to Severely Active Crohn's Disease · Phase 3 · not yet recruiting
NCT07438496 — A Study of Nipocalimab in Adults With Moderate to Severe Systemic Lupus Erythematosus · Phase 3 · recruiting
NCT07227025 — A Study of Amivantamab and Olomorasib Combination Therapy in Participants With Metastatic Non-Small Cell Lung Cancer · Phase 1, PHASE2 · recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT04312672 (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 Janssen Research & Development, LLC
Last refreshed: 21 January 2026

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/NCT04312672.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing