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A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111 (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 18, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen ( NightstaRx Ltd, a Biogen Company )

Brief Summary:
The objective of the study is to evaluate the safety of bilateral, sequential sub-retinal administration of a single dose of BIIB111 in adult male participants with Choroideremia (CHM).

Condition or disease Intervention/treatment Phase
Choroideremia Drug: BIIB111 Phase 2

Detailed Description:
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.

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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 2, 2018
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: BIIB111
Participants will receive a single dose of sub-retinal injection of BIIB111 in each eye at Day 0 separated by an interval of <6 months, 6-12 months, or >12 months.
Drug: BIIB111
Administered as specified in the treatment arm.
Other Names:
  • Gene Therapy
  • AAV2-REP1




Primary Outcome Measures :
  1. Best-Corrected Visual Acuity (BCVA) [ Time Frame: Up to 2 years ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (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). An increase in the number of letters read correctly means that vision has improved.

  2. Ophthalmic Examination Assessment: Intraocular Pressure (IOP) [ Time Frame: Up to 2 years ]
  3. Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination [ Time Frame: Up to 2 years ]
  4. Ophthalmic Examination Assessment: Dilated Ophthalmoscopy [ Time Frame: Up to 2 years ]
  5. Ophthalmic Examination Assessment: Lens Opacity Grading [ Time Frame: Up to 2 years ]
  6. Spectral Domain Optical Coherence Tomography (SD-OCT) [ Time Frame: Up to 2 years ]
  7. Fundus Autofluorescence (AF) [ Time Frame: Up to 2 years ]
  8. Fundus Photography [ Time Frame: Up to 2 years ]
  9. Microperimetry [ Time Frame: Up to 2 years ]
  10. Percentage of Participants with Adverse Events (AEs) [ Time Frame: Up to 2 years ]
  11. Vector Shedding Post-treatment [ Time Frame: Up to 2 years ]
  12. Number of Participants with Cell-Based and Antibody-Based Immune Responses Against BIIB111 [ Time Frame: Up to 2 years ]
  13. Number of Participants with Significant Change in Vital Signs Post-Treatment [ Time Frame: Up to 2 years ]

Secondary Outcome Measures :
  1. Change from Baseline in BCVA [ Time Frame: Up to 2 years ]
    BCVA will be assessed for both eyes using the Early Treatment of Diabetic Retinopathy Study (ETDRS) visual acuity (VA) chart. BCVA test should be performed prior to pupil dilation, and distance refraction should be carried out before BCVA is measured. Initially, letters are read at a distance of 4 meters from the chart. If <20 letters are read at 4 meters, testing at 1 meter should be performed. BCVA is to be reported as number of letters read correctly by the participant using the ETDRS Scale (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). An increase in the number of letters read correctly means that vision has improved.

  2. Change from Baseline in AF [ Time Frame: Up to 2 years ]
  3. Change from Baseline in SD-OCT [ Time Frame: Up to 2 years ]
  4. Change from Baseline in Microperimetry [ Time Frame: Up to 2 years ]


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
Accepts Healthy Volunteers:   No
Criteria

Key Inclusion Criteria:

  1. Are willing and able to give informed consent for participation in the study to have both eyes treated.
  2. Have documentation of a genetically-confirmed diagnosis of CHM.
  3. Have active disease clinically visible within the macular region of both eyes.
  4. 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 BIIB111*

    *If previously treated with BIIB111 in an antecedent study, participants may be eligible for participation following Sponsor approval.

  5. For participants who received treatment with BIIB111 in an antecedent study, have biological samples available to complete an adequate immunology profile.

Key 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 BIIB111 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 participants at risk because of participation in the study, or may influence the results of the study or the participant's ability to participate in the study. This includes but is not limited to a potential participants:

    • 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 BIIB111.

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply


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
Research Site
Miami, Florida, United States, 33136
United States, Massachusetts
Research Site
Boston, Massachusetts, United States, 02114
United States, Ohio
Research Site
Cincinnati, Ohio, United States, 45242
United States, Oregon
Research Site
Portland, Oregon, United States, 97232
France
Research Site
Paris, France, 75571
Germany
Research Site
Tübingen, Germany
Sponsors and Collaborators
NightstaRx Ltd, a Biogen Company
Investigators
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Study Director: Medical Director Biogen
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: 273CH203 (NSR-REP-02)
2017-002395-75 ( EudraCT Number )
First Posted: April 25, 2018    Key Record Dates
Last Update Posted: February 18, 2021
Last Verified: February 2021
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 Biogen ( NightstaRx Ltd, a Biogen Company ):
NightstaRx
NSR-REP1
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