BIIB111 for Choroideremia & BIIB112 for X-Linked Retinitis Pigmentosa

(SOLSTICE Trial)

Enrolling by invitation at 49 trial locations

Age: 18+

Sex: Male

Trial Phase: Phase 3

Sponsor: NightstaRx Ltd, a Biogen Company

Disqualifiers: Others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial examines the long-term safety and effectiveness of two treatments, BIIB111 and BIIB112, for eye conditions that can lead to vision loss. BIIB111 targets Choroideremia (CHM), a condition causing gradual blindness, while BIIB112 addresses X-linked retinitis pigmentosa (XLRP), another disorder affecting vision. Participants should have received these injections in earlier studies. This follow-up study assesses the treatments' effectiveness over time. As a Phase 3 trial, it represents the final step before FDA approval, allowing participants to contribute to the potential availability of these treatments.

Will I have to stop taking my current medications?

The trial information does not specify whether you need to stop taking your current medications. Please consult with the study team for guidance.

Is there any evidence suggesting that this trial's treatments are likely to be safe?

In a previous study, researchers found that BIIB111 was generally safe for people with choroideremia. The treatment involved an injection directly into the eye. Overall, the safety was considered acceptable, with no major safety concerns noted. Some participants experienced changes in health or unusual lab results, but these were expected.

For BIIB112, studies have shown that it was well-tolerated in people with X-linked retinitis pigmentosa. Like BIIB111, this treatment is administered as an injection into the eye. Participants generally did well, and the treatment showed early and lasting improvements in some areas of vision over 12 months.

Both BIIB111 and BIIB112 are in later stages of clinical trials, indicating sufficient safety in earlier studies to continue testing. While no treatment is without risk, the available data suggest that these gene therapies are relatively safe for most people. ^{1 2 3 4 5}

Why are researchers excited about this trial's treatments?

Researchers are excited about BIIB111 and BIIB112 because they offer innovative approaches to treating eye conditions that currently have limited options. Unlike most treatments for choroideremia and X-linked retinitis pigmentosa, which focus on managing symptoms, these therapies use gene therapy to address the underlying genetic causes. BIIB111 and BIIB112 are administered through sub-retinal injections, directly delivering the treatment to the affected area in the eye. This targeted delivery method aims to restore or preserve vision by correcting genetic defects, a significant step forward compared to existing treatments that do not tackle the root cause of these conditions.

What evidence suggests that this trial's treatments could be effective for Choroideremia and X-Linked Retinitis Pigmentosa?

This trial will compare BIIB111 and BIIB112 as separate treatments. Research has shown that BIIB111 may help treat choroideremia. In one study, some patients experienced noticeable vision improvement for up to 24 months after treatment. However, a later study did not achieve its main goals, indicating mixed results overall.

For BIIB112, another treatment option in this trial, early research suggests it might improve vision in people with X-linked retinitis pigmentosa. Some participants experienced better vision and retinal sensitivity after treatment. But like BIIB111, BIIB112 also faced difficulties in meeting its main goals in recent studies. Overall, both treatments have shown potential but have had inconsistent results. ^{4 5 6 7 8}

Who Is on the Research Team?

Medical Director

Principal Investigator

Biogen

Are You a Good Fit for This Trial?

This trial is for individuals with Choroideremia (CHM) or X-linked Retinitis Pigmentosa (XLRP) who have previously received BIIB111 or BIIB112 treatments in earlier studies. Participants must have completed the prior research to be eligible.

Inclusion Criteria

You have previously taken part in a study where a medicine called BIIB111 was injected into your eye for a condition called CHM, and you have completed that study.

I have had a BIIB112 injection for XLRP and completed a prior study.

The study is not open to people with X-linked retinitis pigmentosa (XLRP).

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Follow-up

Long-term follow-up to evaluate the safety and efficacy of the treatment

Up to 5 years

Regular visits as per study protocol

Assessment

Participants undergo various assessments including BCVA, fundus photography, and microperimetry

Up to 5 years

Assessments at each visit

What Are the Treatments Tested in This Trial?

Interventions

• BIIB111
• BIIB112

Trial Overview The study aims to assess the long-term safety and effectiveness of sub-retinal injections of BIIB111 for CHM and BIIB112 for XLRP by comparing previously treated participants with untreated controls from a related study.

How Is the Trial Designed?

Group I: BIIB112 Experimental Treatment 1 Intervention

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.

Group II: BIIB111 Experimental Treatment 1 Intervention

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.

Group III: Untreated Active Control 1 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 Is Running the Clinical Trial?

NightstaRx Ltd, a Biogen Company

Lead Sponsor

Trials

6

Recruited

1,100+

Citations

1. clinicaltrials.gov clinicaltrials.gov/study/NCT03496012

NCT03496012 | Efficacy and Safety of BIIB111 for the ... The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of BIIB111 in participants with choroideremia (CHM).

2. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/29940166/

Two-Year Results After AAV2-Mediated Gene Therapy for ... Secondary outcome measures were the change from baseline in best-corrected visual acuity (BCVA) in the treated eye compared to the untreated eye ...

3. nature.com nature.com/articles/s41591-023-02520-3

Subretinal timrepigene emparvovec in adult men with ... Three patients (9%) achieved and maintained a clinically significant gain of ≥15 ETDRS letters at 24 months. Here we report the results from the ...

4. investors.biogen.com investors.biogen.com/news-releases/news-release-details/biogen-announces-topline-results-phase-3-gene-therapy-study

Biogen Announces Topline Results from Phase 3 Gene ... The Phase 3 STAR study did not meet primary or key secondary endpoints; Choroideremia is a rare inherited retinal disease that results in ...

5. biogentrialtransparency.com biogentrialtransparency.com/content/dam/global-development/general/biogen-trial-link/educational/en-us/pdf/csr/NCT03507686-Synopsis.pdf

NCT03507686 Synopsis - Transparency in clinical trials AAV2-REP1 (BIIB111). Study Indication: Choroideremia. The percent change from baseline of the total area of preserved AF and the square root of total area of ...

6. clinicaltrials.gov clinicaltrials.gov/study/NCT03507686

NCT03507686 | A Safety Study of Retinal Gene Therapy ... 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 ...

7. clinicaltrials.gov clinicaltrials.gov/study/NCT03496012?limit=100&cond=choroideremia&intr=aav&rank=4

Efficacy and Safety of BIIB111 for the Treatment ... An unfavorable change in the health of a participant, including abnormal laboratory findings, that happens during a clinical study or within a certain amount of ...

8. biogentrialtransparency.com biogentrialtransparency.com/content/dam/global-development/general/biogen-trial-link/educational/en-us/pdf/lls/english-master/NCT03507686-LLS.pdf

A Study to Learn About the Safety of the Gene Therapy ... ... safety of BIIB111 and the potential to help people with choroideremia. Overall, the researchers in this study found the safety of BIIB111 was acceptable.