NCT02407678 (REGENERATE) is a Phase 2 clinical trial of AAV-mediated REP1 gene replacement in Choroideremia, sponsored by University of Oxford.

Who is sponsoring NCT02407678?

NCT02407678 is sponsored by University of Oxford.

What drugs are being tested in NCT02407678?

Interventions in NCT02407678: AAV-mediated REP1 gene replacement.

What condition does NCT02407678 study?

NCT02407678 studies Choroideremia.

Is NCT02407678 still recruiting?

NCT02407678 is currently completed. Last updated 5 August 2021.

Last reviewed 2021-08-05 · How we verify

NCT02407678: REGENERATE

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

REP1 Gene Replacement Therapy for Choroideremia

Completed Phase 2 Last updated 5 August 2021

What this trial tests

Phase 2 trial testing AAV-mediated REP1 gene replacement in Choroideremia in 30 participants. Completed in 23 July 2021.

Timeline

16 August 2016

Primary endpoint
23 July 2021

23 July 2021

Quick facts

Lead sponsor	University of Oxford
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	30
Start date	16 August 2016
Primary completion	23 July 2021
Estimated completion	23 July 2021
Sites	2 locations across United Kingdom

Drugs / interventions tested

AAV-mediated REP1 gene replacement

Conditions studied

Choroideremia — all drugs for Choroideremia →

Sponsor

University of Oxford

Who can join

Adults 18 to 90, male only, with Choroideremia. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

The assessment of the efficacy (with respect to preservation of visual function and retinal structure) and safety of a single subretinal injection of AAV2.REP1 in participants with a confirmed diagnosis of choroideremia, as evaluated by various functional and anatomical outcomes measured over a number of time points up to 24 months post-treatment.

Publications & conference data

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

Viral vector platforms within the gene therapy landscape.
Bulcha JT, Wang Y, Ma H, Tai PWL, et al · · 2021 · cited 899× · PMID 33558455 · DOI 10.1038/s41392-021-00487-6
Progress in treating inherited retinal diseases: Early subretinal gene therapy clinical trials and candidates for future initiatives.
Garafalo AV, Cideciyan AV, Héon E, Sheplock R, et al · · 2020 · cited 136× · PMID 31899291 · DOI 10.1016/j.preteyeres.2019.100827
Technique of retinal gene therapy: delivery of viral vector into the subretinal space.
Xue K, Groppe M, Salvetti AP, MacLaren RE. · · 2017 · cited 130× · PMID 28820183 · DOI 10.1038/eye.2017.158
Inner Ear Gene Therapies Take Off: Current Promises and Future Challenges.
Delmaghani S, El-Amraoui A. · · 2020 · cited 92× · PMID 32708116 · DOI 10.3390/jcm9072309
Let There Be Light: Gene and Cell Therapy for Blindness.
Dalkara D, Goureau O, Marazova K, Sahel JA. · · 2016 · cited 89× · PMID 26751519 · DOI 10.1089/hum.2015.147
Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.
Bennett J. · · 2017 · cited 84× · PMID 28391961 · DOI 10.1016/j.ymthe.2017.03.008
Advancing Clinical Trials for Inherited Retinal Diseases: Recommendations from the Second Monaciano Symposium.
Thompson DA, Iannaccone A, Ali RR, Arshavsky VY, et al · · 2020 · cited 82× · PMID 32832209 · DOI 10.1167/tvst.9.7.2
Characterizing the Natural History of Visual Function in Choroideremia Using Microperimetry and Multimodal Retinal Imaging.
Jolly JK, Xue K, Edwards TL, Groppe M, et al · · 2017 · cited 76× · PMID 29084330 · DOI 10.1167/iovs.17-22486

Verify or expand the search:

Other recruiting trials for Choroideremia

Currently open trials in the same condition.

NCT06375239 — Observational Study to Assess Endpoint Operational Feasibility & Measurement Properties in Patients with Retinal Degener · recruiting
NCT04483440 — Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia · Phase 1 · active not recruiting

Other University of Oxford trials

Trials by the same sponsor.

NCT05380388 — A Safety, Immunogenicity and Efficacy Study of PvRII/Matrix-M in Healthy Thai Adults Living in Thailand ( MIST3 ) · Phase 2 · not yet recruiting
NCT07470424 — A Clinical Study of Piperaquine, Pyronaridine, and Artesunate Administered in Combination in Healthy Adults · Phase 1 · not yet recruiting
NCT07345910 — Environment, Pathogens, and Host Interactions in Melioidosis · not yet recruiting
NCT07434973 — Stratification and Treatment in Early Psychosis Study - PROMOTE · Phase 3 · not yet recruiting
NCT07460401 — 'Do Patient Characteristics Associate With Poor Outcome With Femoral Acetabular Impingement Syndrome (FAIS) Following Ph · not yet 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 NCT02407678 (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 University of Oxford
Last refreshed: 5 August 2021

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/NCT02407678.

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