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Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa (SOLSTICE)

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ClinicalTrials.gov Identifier: NCT03584165
Recruitment Status : Enrolling by invitation
First Posted : July 12, 2018
Last Update Posted : February 8, 2021
Sponsor:
Information provided by (Responsible Party):
Biogen ( NightstaRx Ltd, a Biogen Company )

Brief Summary:
The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.

Condition or disease Intervention/treatment
Choroideremia X-Linked Retinitis Pigmentosa Genetic: BIIB111 Genetic: BIIB112

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 : Observational
Estimated Enrollment : 440 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent Study
Actual Study Start Date : June 4, 2018
Estimated Primary Completion Date : March 23, 2027
Estimated Study Completion Date : March 23, 2027


Group/Cohort Intervention/treatment
BIIB111
Participants previously treated with sub-retinal injection of BIIB111 in the antecedent study 273CH301 (NCT03496012) will be enrolled.
Genetic: BIIB111
Administered as specified in the treatment arm.
Other Name: AAV2-REP1

BIIB112
Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled.
Genetic: BIIB112
Administered as specified in the treatment arm.
Other Name: AAV8-RPGR

Untreated
Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled.



Primary Outcome Measures :
  1. Percentage of Participants with Adverse Events (AEs) [ Time Frame: Up to 5 years ]
  2. Ophthalmic Examination Assessment: Intraocular Pressure (IOP) [ Time Frame: Up to 5 years ]
  3. Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination [ Time Frame: Up to 5 years ]
  4. Ophthalmic Examination Assessment: Lens Opacity Grading [ Time Frame: Up to 5 years ]
  5. Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation [ Time Frame: Up to 5 years ]
  6. Ophthalmic Examination Assessment: Indirect Ophthalmoscopy [ Time Frame: Up to 5 years ]
  7. Immunogenicity: Percentage of Participants with Antibodies Against Viral Vector BIIB111 and BIIB112 [ Time Frame: Up to 5 years ]
  8. Immunogenicity: Percentage of Participants with Cell Based Responses Against the Viral Vector BIIB111 and BIIB112 [ Time Frame: Up to 5 years ]

Secondary Outcome Measures :
  1. Change from Baseline in Best-Corrected Visual Acuity (BCVA) [ Time Frame: Up to 5 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. Percentage of Participants with no Decrease from Baseline in BCVA or a Decrease from Baseline in BCVA of <5 ETDRS Letters in Choroideremia (CHM) Participants [ Time Frame: Up to 5 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.

  3. Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants [ Time Frame: Up to 5 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.

  4. Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants [ Time Frame: Up to 5 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.

  5. Change from Baseline in Fundus Autofluorescence (AF) [ Time Frame: Up to 5 years ]
  6. Change from Baseline in Fundus Photography [ Time Frame: Up to 5 years ]
  7. Change from Baseline in Spectral Domain Optical Coherence Tomography (SD-OCT) [ Time Frame: Up to 5 years ]
  8. Change from Baseline in Microperimetry [ Time Frame: Up to 5 years ]
  9. Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25) [ Time Frame: Up to 5 years ]
    VFQ-25 questionnaire measures dimensions of self-reported vision-targeted health status that are most important to persons with eye disease. Total score ranges from 0-100, where a score of 0 represents the worst outcome and 100 represents the best outcome.

  10. Change from Baseline in Visual Field [ Time Frame: Up to 5 years ]
    The outcome measure will be assessed in BIIB112-treated participants.

  11. Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants [ Time Frame: 18 Months to 60 Months, Post-Day 0 Visits ]
    The LLVA 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. The assessment was performed prior to dilating the eyes. Initially, letters are read at a distance of 4 metres from the chart. If <20 letters are read at 4 metres, testing at 1 metre should be performed. BCVA is to be reported as number of letters read correctly by the participant.

  12. Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants [ Time Frame: 18 Months to 60 Months, Post-Day 0 Visits ]
    The LLVA 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. The assessment was performed prior to dilating the eyes. Initially, letters are read at a distance of 4 metres from the chart. If <20 letters are read at 4 metres, testing at 1 metre should be performed. BCVA is to be reported as number of letters read correctly by the participant.



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Ages Eligible for Study:   10 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Participants previously treated with BIIB111 and BIIB112 and participants with CHM previously untreated in an antecedent study.
Criteria

Key Inclusion Criteria:

CHM Participants

a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM.

XLRP Participants

a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.

Key Exclusion Criteria:

Participants are not eligible for study participation if they meet the following exclusion criterion.

a. In the opinion of the investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.

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): NCT03584165


Locations
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United States, Florida
Research Site
Gainesville, Florida, United States, 32607
Research Site
Miami, Florida, United States, 33136
United States, Ohio
Research Site
Cincinnati, Ohio, United States, 45242-5664
United States, Oregon
Research Site
Portland, Oregon, United States, 97239
United States, Texas
Research Site
Dallas, Texas, United States, 75231-5080
United States, Wisconsin
Research Site
Madison, Wisconsin, United States, 53705-3644
Canada, Alberta
Research Site
Edmonton, Alberta, Canada, T5H 3V9
Finland
Research Site
Helsinki, Finland
Germany
Research Site
Bonn, Germany
Research Site
Tübingen, Germany
United Kingdom
Research Site
Oxford, United Kingdom
Sponsors and Collaborators
NightstaRx Ltd, a Biogen Company
Investigators
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Study Director: Medical Director Biogen
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Responsible Party: NightstaRx Ltd, a Biogen Company
ClinicalTrials.gov Identifier: NCT03584165    
Other Study ID Numbers: 273CH201 (NSR-CHM-OS2)
2017-003104-42 ( EudraCT Number )
First Posted: July 12, 2018    Key Record Dates
Last Update Posted: February 8, 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: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biogen ( NightstaRx Ltd, a Biogen Company ):
NightstaRx
Biogen
NSR-REP1
CHM
Gene Therapy
AAV
REP1
AAV8
RPGR
Additional relevant MeSH terms:
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Retinitis
Retinitis Pigmentosa
Choroideremia
Retinal Diseases
Eye Diseases
Eye Diseases, Hereditary
Retinal Dystrophies
Retinal Degeneration
Genetic Diseases, Inborn
Choroid Diseases
Uveal Diseases
Genetic Diseases, X-Linked