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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)

Primary Purpose

Choroideremia, X-Linked Retinitis Pigmentosa

Status
Enrolling by invitation
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
BIIB111
BIIB112
Sponsored by
NightstaRx Ltd, a Biogen Company
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional other trial for Choroideremia focused on measuring NightstaRx, Biogen, NSR-REP1, CHM, Gene Therapy, AAV, REP1, AAV8, RPGR

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)MaleDoes not accept healthy volunteers

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.

Sites / Locations

  • UCLA - Jules Stein Eye Institute
  • Vitreo Retinal Associates PA - The Millennium Center
  • University of Miami
  • Johns Hopkins Hospital
  • MEEI Massachusets Eye and Ear Infirmary
  • Columbia University Medical Center
  • Cincinnati Eye Institute - Blue Ash
  • OHSU - Casey Eye Institute
  • Retina Foundation of the Southwest
  • University of Wisconsin School of Medicine
  • Instituto Genetica Ocular
  • The Northern Alberta Clinical Trials and Research Centre
  • The University of British Columbia - Eye Care Centre
  • McGill University Health Centre
  • Rigshospitalet-Glostrup, Oejenafdelingen
  • Helsinki University Central Hospital (HUCH)
  • CHU Montpellier - Saint Eloi
  • Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
  • Universitäts-Augenklinik Bonn
  • Universitats Klinikum Tubingen - Institute for Ophthalmic Research
  • Radboudumc
  • Moorfields Eye Hospital
  • Manchester Royal Eye Hopsital
  • John Radcliffe Hospital
  • Southampton General Hospital

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm Type

Experimental

Experimental

No Intervention

Arm Label

BIIB111

BIIB112

Untreated

Arm Description

Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.

Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.

Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.

Outcomes

Primary Outcome Measures

Percentage of Participants with Adverse Events (AEs)
Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
Ophthalmic Examination Assessment: Lens Opacity Grading
Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
Ophthalmic Examination Assessment: Indirect Ophthalmoscopy

Secondary Outcome Measures

Change from Baseline in Best-Corrected Visual Acuity (BCVA)
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.
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
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.
Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants
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.
Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants
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.
Assessment of Fundus Autofluorescence (AF) at Each Visit
Fundus Autofluoroscence will be performed on both eyes to assess the change in the area of viable retinal tissue. It will be reported in square millimetres (mm^2).
Assessment of Fundus Photography at Each Visit
Fundus photography will be performed on both eyes following the dilation of the participant's pupils.
Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit
SD-OCT will be performed on both eyes to assess the foveal changes and other potential anatomic changes. The measurements were taken after dilation of the participant's pupil and would be reported in micrometres (µm).
Assessment of Microperimetry at Each Visit
Microperimetry will be performed on both eyes to assess changes in retinal sensitivity within the macula. It will be reported in decibels (dB).
Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25)
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.
Change from Baseline in Visual Field
The outcome measure will be assessed in BIIB112-treated participants.
Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants
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.
Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants
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.

Full Information

First Posted
June 29, 2018
Last Updated
May 5, 2023
Sponsor
NightstaRx Ltd, a Biogen Company
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1. Study Identification

Unique Protocol Identification Number
NCT03584165
Brief Title
Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
Acronym
SOLSTICE
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
Study Type
Interventional

2. Study Status

Record Verification Date
May 2023
Overall Recruitment Status
Enrolling by invitation
Study Start Date
June 4, 2018 (Actual)
Primary Completion Date
June 4, 2026 (Anticipated)
Study Completion Date
June 4, 2026 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
NightstaRx Ltd, a Biogen Company

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. Study Description

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

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Choroideremia, X-Linked Retinitis Pigmentosa
Keywords
NightstaRx, Biogen, NSR-REP1, CHM, Gene Therapy, AAV, REP1, AAV8, RPGR

7. Study Design

Primary Purpose
Other
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
Outcomes Assessor
Allocation
Non-Randomized
Enrollment
330 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
BIIB111
Arm Type
Experimental
Arm Description
Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.
Arm Title
BIIB112
Arm Type
Experimental
Arm Description
Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.
Arm Title
Untreated
Arm Type
No Intervention
Arm Description
Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.
Intervention Type
Genetic
Intervention Name(s)
BIIB111
Other Intervention Name(s)
AAV2-REP1, rAAV2-REP1
Intervention Description
Administered as specified in the treatment arm.
Intervention Type
Genetic
Intervention Name(s)
BIIB112
Other Intervention Name(s)
AAV8-RPGR
Intervention Description
Administered as specified in the treatment arm.
Primary Outcome Measure Information:
Title
Percentage of Participants with Adverse Events (AEs)
Time Frame
Up to 5 years
Title
Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
Time Frame
Up to 5 years
Title
Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
Time Frame
Up to 5 years
Title
Ophthalmic Examination Assessment: Lens Opacity Grading
Time Frame
Up to 5 years
Title
Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
Time Frame
Up to 5 years
Title
Ophthalmic Examination Assessment: Indirect Ophthalmoscopy
Time Frame
Up to 5 years
Secondary Outcome Measure Information:
Title
Change from Baseline in Best-Corrected Visual Acuity (BCVA)
Description
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.
Time Frame
Up to 5 years
Title
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
Description
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.
Time Frame
Up to 5 years
Title
Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants
Description
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.
Time Frame
Up to 5 years
Title
Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants
Description
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.
Time Frame
Up to 5 years
Title
Assessment of Fundus Autofluorescence (AF) at Each Visit
Description
Fundus Autofluoroscence will be performed on both eyes to assess the change in the area of viable retinal tissue. It will be reported in square millimetres (mm^2).
Time Frame
Up to 5 years
Title
Assessment of Fundus Photography at Each Visit
Description
Fundus photography will be performed on both eyes following the dilation of the participant's pupils.
Time Frame
Up to 5 years
Title
Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit
Description
SD-OCT will be performed on both eyes to assess the foveal changes and other potential anatomic changes. The measurements were taken after dilation of the participant's pupil and would be reported in micrometres (µm).
Time Frame
Up to 5 years
Title
Assessment of Microperimetry at Each Visit
Description
Microperimetry will be performed on both eyes to assess changes in retinal sensitivity within the macula. It will be reported in decibels (dB).
Time Frame
Up to 5 years
Title
Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25)
Description
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.
Time Frame
Up to 5 years
Title
Change from Baseline in Visual Field
Description
The outcome measure will be assessed in BIIB112-treated participants.
Time Frame
Up to 5 years
Title
Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥10 ETDRS Letters in BIIB112-treated Participants
Description
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.
Time Frame
18 Months to 60 Months, Post-Day 0 Visits
Title
Percentage of Participants with an Increase from Baseline in Luminance Visual Acuity (LLVA) of ≥15 ETDRS Letters in BIIB112-treated Participants
Description
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.
Time Frame
18 Months to 60 Months, Post-Day 0 Visits

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility 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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medical Director
Organizational Affiliation
Biogen
Official's Role
Study Director
Facility Information:
Facility Name
UCLA - Jules Stein Eye Institute
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095-7065
Country
United States
Facility Name
Vitreo Retinal Associates PA - The Millennium Center
City
Gainesville
State/Province
Florida
ZIP/Postal Code
32607
Country
United States
Facility Name
University of Miami
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287-0005
Country
United States
Facility Name
MEEI Massachusets Eye and Ear Infirmary
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114-3002
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Cincinnati Eye Institute - Blue Ash
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45242-5664
Country
United States
Facility Name
OHSU - Casey Eye Institute
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Retina Foundation of the Southwest
City
Dallas
State/Province
Texas
ZIP/Postal Code
75231-5080
Country
United States
Facility Name
University of Wisconsin School of Medicine
City
Madison
State/Province
Wisconsin
ZIP/Postal Code
53705-3644
Country
United States
Facility Name
Instituto Genetica Ocular
City
Sao Paulo
ZIP/Postal Code
04039
Country
Brazil
Facility Name
The Northern Alberta Clinical Trials and Research Centre
City
Edmonton
State/Province
Alberta
ZIP/Postal Code
T5H 3V9
Country
Canada
Facility Name
The University of British Columbia - Eye Care Centre
City
Vancouver
State/Province
British Columbia
ZIP/Postal Code
V5Z 39N
Country
Canada
Facility Name
McGill University Health Centre
City
Montreal
State/Province
Quebec
ZIP/Postal Code
H4A 3JI
Country
Canada
Facility Name
Rigshospitalet-Glostrup, Oejenafdelingen
City
Glostrup
ZIP/Postal Code
2600
Country
Denmark
Facility Name
Helsinki University Central Hospital (HUCH)
City
Helsinki
ZIP/Postal Code
00290 HUS
Country
Finland
Facility Name
CHU Montpellier - Saint Eloi
City
Montpellier
ZIP/Postal Code
34295
Country
France
Facility Name
Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
City
Paris
ZIP/Postal Code
75012
Country
France
Facility Name
Universitäts-Augenklinik Bonn
City
Bonn
ZIP/Postal Code
53127
Country
Germany
Facility Name
Universitats Klinikum Tubingen - Institute for Ophthalmic Research
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Radboudumc
City
Nijmegen
ZIP/Postal Code
6525 GA
Country
Netherlands
Facility Name
Moorfields Eye Hospital
City
London
ZIP/Postal Code
EC1V 2PD
Country
United Kingdom
Facility Name
Manchester Royal Eye Hopsital
City
Manchester
ZIP/Postal Code
M13 9WL
Country
United Kingdom
Facility Name
John Radcliffe Hospital
City
Oxford
ZIP/Postal Code
OX3 9DU
Country
United Kingdom
Facility Name
Southampton General Hospital
City
Southampton
ZIP/Postal Code
SO16 6YD
Country
United Kingdom

12. IPD Sharing Statement

Plan to Share IPD
No

Learn more about this trial

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

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