- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02553135
Choroideremia Gene Therapy Clinical Trial
July 25, 2019 updated by: Byron Lam
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)
Phase II gene therapy study, involving a total of 6 male patients with choroideremia.
The study will be conducted at the Bascom Palmer Eye Institute, University of Miami.
Patients will be required to attend a total of 11 study visits over a 24 month period with an additional 3 year follow-up.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
This is a Phase II, open label study involving patients with a clinical phenotype of choroideremia and a confirmed CHM genotype.
Following consent, patients will be required to attend an initial screening visit (Visit 1).
Within 2 weeks of the screening visit patients will undergo a surgical procedure (Visit 2) under general anesthesia which will include a standard vitrectomy, retinal detachment and administration of a subretinal injection of AAV2-REP1 (1x1011 genome particles).
Patients will be required to attend a further 9 study visits (Visits 3-11) over a 24 month period for functional, and anatomical assessments as well as monitoring of adverse events.
The primary endpoint is the change from baseline in visual acuity in the study eye, compared to control eye.
Secondary study endpoints are, change from baseline in autofluorescence evaluation, microperimetry readings and other anatomic and functional outcomes (all in the study eye compared to control eye).
Secondary endpoints also include safety assessments to be conducted throughout the study.
The fellow eyes of these patients will be utilized as controls in this study and will receive no study treatment.
Study Type
Interventional
Enrollment (Actual)
6
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
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Florida
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Miami, Florida, United States, 33136
- Bascom Palmer Eye Institute, University of Miami
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
Male
Description
Inclusion Criteria:
- 18 Years and older
- Male
- Able to give informed consent
- Genetically confirmed diagnosis of choroideremia
- Active disease visible clinically within the macula region
- Best-corrected visual acuity equal to or worse than 20/32 but better than or equal to 20/200 in the study eye.
Exclusion Criteria:
- Female
- Under the age of 18
- History of amblyopia in the study eye
- Men unwilling to use barrier contraception methods
- Relevant grossly asymmetrical disease or other ocular morbidity which might confound use of the fellow eye as a long-term control
- Any other significant ocular and non-ocular disease/disorder or retinal surgery
- Contraindication to use of medications or contrast agents
- Participated in research study involving an investigational product in the past 12 weeks
- Having had gene or cellular therapy at any time prior to this study.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Injection of AAV2-REP1
Injection of AAV-REP1, 1.00x10e11 vg, subretinal injection of total volume of 100 μL.
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Single Group: single arm study
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Best Corrected Visual Acuity From Baseline
Time Frame: Baseline, 24 Months
|
Patients will have an assessment of visual acuity using the Early Treatment of Diabetic Retinopathy Study (ETDRS) vision charts in both eyes.
Low Luminance BCVA was measured by placing a 2.0 log unit neutral density filter over the best correction for that eye and having the participant read the normally illuminated ETDRS chart and was reported as the number of letters read correctly (ranging from 0 to 100 letters) in the study eye.
The lower the number of letters read correctly on the eye chart, the worse the vision (or visual acuity).
The study eye was defined as the eye that met inclusion/exclusion criteria with the worst standard BCVA.
If the BCVA in both eyes was similar the Patient determines the worse eye be selected as the study eye.
A positive change from baseline indicates an improvement and a negative change from baseline indicates a worsening.
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Baseline, 24 Months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change in Retinal Macular Autofluorescence From Baseline
Time Frame: 12 and 24 months
|
Measured in mm^2 by Fundus Autofluorescence and shows changes in the integrity and metabolism of retinal cells.
A negative change from baseline indicates a decrease in size of the retained retina (worsening; disease progression).
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12 and 24 months
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Changes in Microperimetry From Baseline
Time Frame: Baseline to 24 months
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Microperimetry assessments.
A negative change from baseline indicates disease worsening.
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Baseline to 24 months
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Number of Participants Who Experience an Adverse Event
Time Frame: 24 months
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Adverse events during treatment and follow-up period
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24 months
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: BYRON LAM, MD, University of Miami, Bascom Palmer Eye Institute
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
September 1, 2015
Primary Completion (Actual)
February 1, 2018
Study Completion (Actual)
February 1, 2018
Study Registration Dates
First Submitted
June 17, 2015
First Submitted That Met QC Criteria
September 16, 2015
First Posted (Estimate)
September 17, 2015
Study Record Updates
Last Update Posted (Actual)
July 29, 2019
Last Update Submitted That Met QC Criteria
July 25, 2019
Last Verified
July 1, 2019
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 20150371
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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