ChoroideremiaPhase 3SOLSTICENCT03584165
Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
A Phase 3 study testing BIIB111 and BIIB112 in people with Choroideremia and X-Linked Retinitis Pigmentosa, run by NightstaRx Ltd, a Biogen Company at 25 sites in 9 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Choroideremia, X-Linked Retinitis Pigmentosa
- What is being tested?
- BIIB111 and BIIB112
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 277 participants
- Where
- 25 sitesin 9 countries
- Study dates
- 4 Jun 2018to 22 Jun 2026
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- CHM Participants
- 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
- Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.
Who cannot join
- Participants are not eligible for study participation if they meet the following exclusion criterion.
- 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.
Trial locations and contacts
25 sites in 9 countries.
25 sites in 9 countries
United States10 sites
Johns Hopkins Hospital
Baltimore, Maryland, 21287-0005
MEEI Massachusets Eye and Ear Infirmary
Boston, Massachusetts, 02114-3002
Cincinnati Eye Institute - Blue Ash
Cincinnati, Ohio, 45242-5664
Retina Foundation of the Southwest
Dallas, Texas, 75231-5080
Vitreo Retinal Associates PA - The Millennium Center
Gainesville, Florida, 32607
UCLA - Jules Stein Eye Institute
Los Angeles, California, 90095-7065
University of Wisconsin School of Medicine
Madison, Wisconsin, 53705-3644
University of Miami
Miami, Florida, 33136
Columbia University Medical Center
New York, New York, 10032
OHSU - Casey Eye Institute
Portland, Oregon, 97239
United Kingdom4 sites
Moorfields Eye Hospital
London, EC1V 2PD
Manchester Royal Eye Hopsital
Manchester, M13 9WL
John Radcliffe Hospital
Oxford, OX3 9DU
Southampton General Hospital
Southampton, SO16 6YD
Canada3 sites
The Northern Alberta Clinical Trials and Research Centre
Edmonton, Alberta, T5H 3V9
McGill University Health Centre
Montreal, Quebec, H4A 3JI
The University of British Columbia - Eye Care Centre
Vancouver, British Columbia, V5Z 39N
France2 sites
CHU Montpellier - Saint Eloi
Montpellier, 34295
Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
Paris, 75012
Germany2 sites
Universitäts-Augenklinik Bonn
Bonn, 53127
Universitats Klinikum Tubingen - Institute for Ophthalmic Research
Tübingen, 72076
Denmark1 site
Rigshospitalet-Glostrup, Oejenafdelingen
Glostrup Municipality, 2600
About this study
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
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.
Read the detailed description
This study was previously posted by NightstaRx Ltd. In October 2020, sponsorship of the trial was transferred to Biogen.
- Conditions
- Choroideremia, X-Linked Retinitis Pigmentosa
- Being tested
- BIIB111, BIIB112
- Keywords
- NightstaRx, Biogen, NSR-REP1, CHM, Gene Therapy, AAV, REP1, AAV8, RPGR
- Medical subject headings
- Choroideremia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BIIB111Genetic
Also called AAV2-REP1, rAAV2-REP1
Administered as specified in the treatment arm.
BIIB112Genetic
Also called AAV8-RPGR
Administered as specified in the treatment arm.
| Study group | Role | What they receive |
|---|---|---|
| BIIB111 | Experimental | 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. Receives: Genetic: BIIB111 |
| BIIB112 | Experimental | 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. Receives: Genetic: BIIB112 |
| Untreated | No intervention | 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants with Adverse Events (AEs)
Measured: Up to 5 years
Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
Measured: Up to 5 years
Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
Measured: Up to 5 years
Ophthalmic Examination Assessment: Lens Opacity Grading
Measured: Up to 5 years
Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
Measured: Up to 5 years
Ophthalmic Examination Assessment: Indirect Ophthalmoscopy
Measured: Up to 5 years
Secondary outcomes (12)
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.
Measured: Up to 5 years
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.
Measured: Up to 5 years
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.
Measured: Up to 5 years
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.
Measured: Up to 5 years
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).
Measured: Up to 5 years
Assessment of Fundus Photography at Each Visit
Fundus photography will be performed on both eyes following the dilation of the participant's pupils.
Measured: Up to 5 years
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).
Measured: Up to 5 years
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).
Measured: Up to 5 years
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.
Measured: Up to 5 years
Change from Baseline in Visual Field
The outcome measure will be assessed in BIIB112-treated participants.
Measured: Up to 5 years
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.
Measured: 18 Months to 60 Months, Post-Day 0 Visits
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.
Measured: 18 Months to 60 Months, Post-Day 0 Visits
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Jul 2018
- Study start4 Jun 2018
- Primary completion22 Jun 2026
- Study completion22 Jun 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for ChoroideremiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 277 participants (actual)
Sponsor and investigators
- Lead sponsor
- NightstaRx Ltd, a Biogen Company
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Medical Director, Study director, Biogen
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03584165
- Sponsor's protocol ID
- 273CH201
- Other IDs
- 2017-003104-42, 2017
- Acronym
- SOLSTICE
- Status last verified
- Jul 2026
- First submitted
- 29 Jun 2018
- Last updated
- 6 Jul 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03584165
- Acronym / sponsor ID
- SOLSTICE / 273CH201
- Conditions
- Choroideremia, X-Linked Retinitis Pigmentosa
- Keywords
- NightstaRx, Biogen, NSR-REP1, CHM, Gene Therapy, AAV, REP1, AAV8
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 277 participants (actual)
- Lead sponsor
- NightstaRx Ltd, a Biogen CompanyUniversity, hospital or other
- Study start
- 4 Jun 2018 (actual)
- Primary completion
- 22 Jun 2026 (actual)
- Study completion
- 22 Jun 2026 (actual)
- First posted
- 12 Jul 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Other
- Allocation
- Non-randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| BIIB111 | Experimental | Genetic: BIIB111Participants 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. |
| BIIB112 | Experimental | Genetic: BIIB112Participants 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 | No intervention | –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. |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- CHM Participants
- 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
- Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.
Key exclusion criteria (3 listed)
- Participants are not eligible for study participation if they meet the following exclusion criterion.
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BIIB111Also: AAV2-REP1, rAAV2-REP1Administered as specified in the treatment arm. | Genetic | BIIB111 |
| BIIB112Also: AAV8-RPGRAdministered as specified in the treatment arm. | Genetic | BIIB112 |
Primary outcome
Percentage of Participants with Adverse Events (AEs)
Time frame: Up to 5 years
Ophthalmic Examination Assessment: Intraocular Pressure (IOP)
Time frame: Up to 5 years
Ophthalmic Examination Assessment: Abnormal Slit Lamp Examination
Time frame: Up to 5 years
Ophthalmic Examination Assessment: Lens Opacity Grading
Time frame: Up to 5 years
Ophthalmic Examination Assessment: Anterior Chamber and Vitreous Inflammation
Time frame: Up to 5 years
Ophthalmic Examination Assessment: Indirect Ophthalmoscopy
Time frame: Up to 5 years
12 secondary outcomes
Change from Baseline in Best-Corrected Visual Acuity (BCVA)
Time frame: Up to 5 years
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
Percentage of Participants with an Increase from Baseline in BCVA of ≥10 ETDRS Letters in CHM Participants
Time frame: Up to 5 years
Percentage of Participants with an Increase from Baseline in BCVA of ≥15 ETDRS Letters in CHM Participants
Time frame: Up to 5 years
Assessment of Fundus Autofluorescence (AF) at Each Visit
Time frame: Up to 5 years
Assessment of Fundus Photography at Each Visit
Time frame: Up to 5 years
Assessment of Spectral Domain Optical Coherence Tomography (SD-OCT) at Each Visit
Time frame: Up to 5 years
Assessment of Microperimetry at Each Visit
Time frame: Up to 5 years
Change from Baseline in 25-Item Visual Function Questionnaire (VFQ-25)
Time frame: Up to 5 years
Change from Baseline in Visual Field
Time frame: Up to 5 years
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
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medical Director study director
Biogen
25 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BIIB111 and BIIB112 in people with Choroideremia and X-Linked Retinitis Pigmentosa, run by NightstaRx Ltd, a Biogen Company at 25 sites in 9 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 25 sites in United States, United Kingdom, Canada, France, Germany and 4 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants with Adverse Events (AEs) (time frame: Up to 5 years). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Jul 2026.