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Trial record 2 of 3 for:    NSR-REP1

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03507686
Recruitment Status : Active, not recruiting
First Posted : April 25, 2018
Last Update Posted : February 24, 2020
Sponsor:
Information provided by (Responsible Party):
NightstaRx Ltd, a Biogen Company

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 adeno-associated 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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: N/A
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 : February 28, 2022
Estimated Study Completion Date : February 28, 2022

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: AAV2-REP1
Bilateral sub-retinal administration of AAV2-REP1.
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 ]
    Evaluation of safety as measured by adverse events.


Secondary Outcome Measures :
  1. Best Corrected Visual Acuity (BCVA) [ 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 (OCT) [ Time Frame: up to 2 years ]
    Ellipsoid Zone

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



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Gender Eligibility Description:   Male subjects only.
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 documentation of a genetically-confirmed diagnosis of CHM.
  4. Have active disease clinically visible within the macular region of both eyes.
  5. Have a BCVA of ≥34 ETDRS letters (20/200 or better Snellen acuity) in both eyes, or in the untreated eye, if the other eye was previously treated with AAV2-REP1*

    *If previously treated with AAV2-REP1 in an antecedent study, subjects may be eligible for participation following Sponsor approval.

  6. For subjects who received treatment with AAV2-REP1 in an antecedent study, have biological samples available to complete an adequate immunology profile.

Exclusion Criteria:

  1. Have a history of amblyopia or inflammatory disorder in either eye.
  2. Are unwilling to use barrier contraception methods or abstain from sexual intercourse 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. This includes but is not limited to a potential subject:

    • with a contraindication to oral corticosteroid (e.g., prednisolone/prednisone)
    • with clinically significant cataract in either eye
    • who, in the clinical opinion of the Investigator, is not an appropriate candidate for sub-retinal surgery.
  5. Have participated in another research study involving an investigational product in the past 12 weeks or received a gene/cell-based therapy at any time previously, except if treated within an antecedent study with AAV2-REP1.

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


Locations
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United States, Florida
Study Site
Miami, Florida, United States, 33136
United States, Massachusetts
Study Site
Boston, Massachusetts, United States, 02114
United States, Ohio
Study Site
Cincinnati, Ohio, United States, 45242
United States, Oregon
Study Site
Portland, Oregon, United States, 97232
Germany
Study Site
Tübingen, Germany
Sponsors and Collaborators
NightstaRx Ltd, a Biogen Company
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: NightstaRx Ltd, a Biogen Company
ClinicalTrials.gov Identifier: NCT03507686    
Other Study ID Numbers: NSR-REP-02
2017-002395-75 ( EudraCT Number )
First Posted: April 25, 2018    Key Record Dates
Last Update Posted: February 24, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by NightstaRx Ltd, a Biogen Company:
NightstaRx
NSR-REP1
Choroideremia
CHM
Gene Therapy
AAV
REP1
Timrepigene Emparvovec
Additional relevant MeSH terms:
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Choroideremia
Eye Diseases, Hereditary
Eye Diseases
Choroid Diseases
Uveal Diseases
Genetic Diseases, Inborn
Genetic Diseases, X-Linked