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A Safety Study of Retinal Gene Therapy for Choroideremia (GEMINI)

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ClinicalTrials.gov Identifier: NCT03507686
Recruitment Status : Recruiting
First Posted : April 25, 2018
Last Update Posted : April 25, 2018
Sponsor:
Information provided by (Responsible Party):
Nightstar Therapeutics

Brief Summary:
This is a multi-centre, open-label, prospective, two-period, interventional safety study of bilateral use of AAV2-REP1 in adult male subjects with genetically confirmed CHM.

Condition or disease Intervention/treatment Phase
Choroideremia Drug: AAV2-REP1 Phase 2

Detailed Description:

This is a phase 2 clinical trial of a gene therapy vector made from adenoassociated virus (AAV) called AAV2-REP1 for the treatment of Choroideremia. Participating subjects will be required to attend a screening visit during which their suitability for the study will be assessed, and eligible subjects will undergo a surgical procedure under general anaesthesia consisting of vitrectomy, retinal detachment and sub-retinal administration of AAV2-REP1.

Subjects will be treated bilaterally, with the interval between eyes determined by discussion between the investigator and Nightstar. Participating subjects will be required to attend follow up visits for 12 months following the second eye surgery.


Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)
Actual Study Start Date : November 6, 2017
Estimated Primary Completion Date : March 31, 2020
Estimated Study Completion Date : March 31, 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: AAV2-REP1
Bilateral sub-retinal administration of AAV2-REP1 at a range of intervals.
Drug: AAV2-REP1
Sub-retinal injection of AAV2-REP1 after vitrectomy.
Other Name: Gene Therapy




Primary Outcome Measures :
  1. Adverse events as a measure of safety and tolerability [ Time Frame: up to 2 years ]
    Incidence of treatment emergent adverse events (%)


Secondary Outcome Measures :
  1. Best Corrected Visual Acuity [ Time Frame: up to 2 years ]
    ETDRS visual acuity chart

  2. Fundus autofluorescence (AF) [ Time Frame: up to 2 years ]
    Change in AF (mm2)

  3. Optical coherence tomography [ Time Frame: up to 2 years ]
    Ellipsoid Zone

  4. Microperimetry [ Time Frame: up to 2 years ]
    Change in Sensitivity (dB)



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Are willing and able to give informed consent for participation in the study to have both eyes treated.
  2. Are male and ≥18 years of age.
  3. Have a genetically-confirmed diagnosis of CHM.
  4. Have active disease clinically visible within the macular region of both eyes.
  5. Have a BCVA within certain visual acuity criteria in both eyes

Exclusion Criteria:

  1. Have a history of amblyopia or inflammatory disorder in either eye.
  2. Are unwilling to use barrier contraception methods, for a period of 3 months following treatment with AAV2-REP1 in either eye.
  3. Have had previous intraocular surgery performed within 3 months of the Screening Visit in either eye
  4. Have any other significant ocular or non-ocular disease/disorder which, in the opinion of the investigator, may either put the subjects at risk because of participation in the study, or may influence the results of the study, or the subject's ability to participate in the study.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03507686


Contacts
Contact: Nightstar Therapeutics +44 207 611 2077 researchenquiries@nightstartx.com

Locations
United States, Florida
Bascom Palmer Eye Institute Recruiting
Miami, Florida, United States, 33136
Sponsors and Collaborators
Nightstar Therapeutics

Responsible Party: Nightstar Therapeutics
ClinicalTrials.gov Identifier: NCT03507686     History of Changes
Other Study ID Numbers: NSR-REP-02
2017-002395-75 ( EudraCT Number )
First Posted: April 25, 2018    Key Record Dates
Last Update Posted: April 25, 2018
Last Verified: April 2018
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Nightstar Therapeutics:
NightstaRx
NSR-REP1
Choroideremia
CHM
Gene Therapy
AAV
REP1

Additional relevant MeSH terms:
Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked