ChoroideremiaPhase 2REGENERATENCT02407678
REP1 Gene Replacement Therapy for Choroideremia
A Phase 2 study testing AAV-mediated REP1 gene replacement in people with Choroideremia, run by University of Oxford at 2 sites in United Kingdom.
Trial summary
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… Read more about this study
- Who is it for?
- Ages 18 to 90 years, men only
- Conditions the study is for
- Choroideremia
- What is being tested?
- AAV-mediated REP1 gene replacement
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 30 participants
- Where
- 2 sitesUnited Kingdom
- Study dates
- 16 Aug 2016to 23 Jul 2021
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 90 years
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Candidate is willing and able to give informed consent for participation in the study.
- Male aged 18 years or above.
- Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency).
- Active disease visible clinically within the macula region.
- Best corrected visual acuity better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.
Who cannot join
- Any female, or a male aged below 18 years.
- An additional cause for sight loss (e.g. amblyopia) in the eye to be treated.
- Any other significant ocular and non-ocular disease or disorder which, in the opinion of the investigator, may put the participants at risk because of participation in the study.
- Inability to take systemic prednisolone for a period of 45 days.
- Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.
- Participation in another research study involving an investigational product in the preceding 12 weeks.
Trial locations and contacts
2 sites in 1 country.
United Kingdom2 sites
Moorfields Eye Hospital NHS Foundation Trust
London, EC1V 2PD
Oxford University Hospitals NHS Foundation Trust
Oxford, OX3 9DU
About this study
Official title: An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
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.
- Conditions
- Choroideremia
- Being tested
- AAV-mediated REP1 gene replacement
- Medical subject headings
- Choroideremia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV-mediated REP1 gene replacementGenetic
AAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection
| Study group | Role | What they receive |
|---|---|---|
| Treatment | Experimental | Treated eye undergoes AAV-mediated REP1 gene replacement. AAV vector is delivered by subretinal injection. Receives: Genetic: AAV-mediated REP1 gene replacement |
| Control | No intervention | Untreated eye |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline in best corrected visual acuity in the treated eye
Measured: 2 years
Secondary outcomes (2)
Change from baseline in the central visual field in the treated eye as determined by microperimetry
Measured: 2 years
Change from baseline in the area of surviving retinal pigment epithelium in the treated eye as measured by fundus autofluorescence, compared to the untreated fellow eye (control eye) after randomisation of treatment to one eye or the other
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Apr 2015 (estimated)
- Study start16 Aug 2016
- Primary completion23 Jul 2021
- Study completion23 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ChoroideremiaStudies of the same condition, open and under way.
- More trials run by University of OxfordThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- The decision about which eye to treat will be made on clinical grounds and will generally be the worse eye affected in cases where BCVA differs between the two eyes by 2 lines or more of ETDRS letters. The eye to be treated will be randomised in cases where the degeneration is relatively symmetrical between the two eyes, defined as: * a difference in BCVA of no more than 1 line of ETDRS letters, and * no more than 25% difference in the area of surviving RPE as measured by fundus autofluorescence. Prospective participants having non-symmetrical retinal degeneration will be allocated to the non-randomised arm. The treated eye will generally be the worse eye. Prospective participants having relatively symmetrical retinal degeneration will be allocated to the randomised arm.
- Masking
- None (open label)
- Enrolment
- 30 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Oxford
- Sponsor type
- University, hospital or other
- Collaborators
- Moorfields Eye Hospital NHS Foundation Trust, University College, London
- Responsible party
- Sponsor
- Investigators
- Robert E MacLaren, MB ChB DPhil, Principal investigator, University of Oxford
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02407678
- Sponsor's protocol ID
- REGEN2015
- Acronym
- REGENERATE
- Status last verified
- Jul 2021
- First submitted
- 10 Mar 2015
- Last updated
- 5 Aug 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Phase 2 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
- ClinicalTrials.gov ID
- NCT02407678
- Acronym / sponsor ID
- REGENERATE / REGEN2015
- Conditions
- Choroideremia
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 30 participants (estimated)
- Lead sponsor
- University of OxfordUniversity, hospital or other
- Collaborators
- Moorfields Eye Hospital NHS Foundation Trust, University College, London
- Study start
- 16 Aug 2016 (actual)
- Primary completion
- 23 Jul 2021 (actual)
- Study completion
- 23 Jul 2021 (actual)
- First posted
- 3 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- The decision about which eye to treat will be made on clinical grounds and will generally be the worse eye affected in cases where BCVA differs between the two eyes by 2 lines or more of ETDRS letters. The eye to be treated will be randomised in cases where the degeneration is relatively symmetrical between the two eyes, defined as: * a difference in BCVA of no more than 1 line of ETDRS letters, and * no more than 25% difference in the area of surviving RPE as measured by fundus autofluorescence. Prospective participants having non-symmetrical retinal degeneration will be allocated to the non-randomised arm. The treated eye will generally be the worse eye. Prospective participants having relatively symmetrical retinal degeneration will be allocated to the randomised arm.
| Arm | Type | Receives |
|---|---|---|
| Treatment | Experimental | Genetic: AAV-mediated REP1 gene replacementTreated eye undergoes AAV-mediated REP1 gene replacement. AAV vector is delivered by subretinal injection. |
| Control | No intervention | –Untreated eye |
Who may qualify
- Ages
- 18 to 90 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Candidate is willing and able to give informed consent for participation in the study.
- Male aged 18 years or above.
- Genetic or molecular confirmed diagnosis of choroideremia (REP1 protein deficiency).
- Active disease visible clinically within the macula region.
- Best corrected visual acuity better than or equal to 6/60 (20/200; Decimal 0.1; LogMAR 1.0) in the study eye.
Key exclusion criteria (6 listed)
- Any female, or a male aged below 18 years.
- An additional cause for sight loss (e.g. amblyopia) in the eye to be treated.
- Any other significant ocular and non-ocular disease or disorder which, in the opinion of the investigator, may put the participants at risk because of participation in the study.
- Inability to take systemic prednisolone for a period of 45 days.
- Unwillingness to use barrier contraception methods for a period of three months following gene therapy surgery.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV-mediated REP1 gene replacementAAV vector carrying human REP1 gene is delivered into the treated eye by subretinal injection | Genetic | Treatment |
Primary outcome
Change from baseline in best corrected visual acuity in the treated eye
Time frame: 2 years
2 secondary outcomes
Change from baseline in the central visual field in the treated eye as determined by microperimetry
Time frame: 2 years
Change from baseline in the area of surviving retinal pigment epithelium in the treated eye as measured by fundus autofluorescence, compared to the untreated fellow eye (control eye) after randomisation of treatment to one eye or the other
Time frame: 2 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2021.
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Choroideremia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Robert E MacLaren, MB ChB DPhil principal investigator
University of Oxford
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing AAV-mediated REP1 gene replacement in people with Choroideremia, run by University of Oxford at 2 sites in United Kingdom.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 6 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United Kingdom. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline in best corrected visual acuity in the treated eye (time frame: 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2021.