BIIB111 for Choroideremia & BIIB112 for X-Linked Retinitis Pigmentosa

(SOLSTICE Trial)

Recruiting at 47 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

330 Participants Needed

Trial Summary

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.

What data supports the effectiveness of this treatment for Choroideremia and X-Linked Retinitis Pigmentosa?

Research on similar gene therapies for Choroideremia shows that some patients experienced improvements in vision, with a few gaining more than one line of vision. However, results have been variable, and the primary goal of significant vision improvement was not consistently met across studies. ^{1 2 3 4 5}

What is known about the safety of BIIB111 and BIIB112 treatments?

In a clinical trial for choroideremia, the gene therapy timrepigene emparvovec (related to BIIB111) was generally safe, with most side effects being mild or moderate. The study involved subretinal injections in adult men, and while it didn't meet its primary goal for improving vision, the safety profile was acceptable. ^{2 6 7 8 9}

How does the treatment BIIB111 differ from other treatments for choroideremia?

BIIB111 is a gene therapy that uses an adeno-associated viral (AAV) vector to deliver a functional copy of the CHM gene, which is unique compared to other treatments as it targets the genetic cause of choroideremia directly. ^{2 3 5 8 10}

What is the purpose of this trial?

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.

Research Team

Medical Director

Principal Investigator

Biogen

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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+

References

1. Switzerland pubmed.ncbi.nlm.nih.gov

Update on Gene Therapy Clinical Trials for Choroideremia and Potential Experimental Therapies. [2023]

2. United States pubmed.ncbi.nlm.nih.gov

Subretinal timrepigene emparvovec in adult men with choroideremia: a randomized phase 3 trial. [2023]

3. England pubmed.ncbi.nlm.nih.gov

Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial. [2023]

4. United States pubmed.ncbi.nlm.nih.gov

Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia. [2023]

5. England pubmed.ncbi.nlm.nih.gov

CHM/REP1 cDNA delivery by lentiviral vectors provides functional expression of the transgene in the retinal pigment epithelium of choroideremia mice. [2021]

6. United States pubmed.ncbi.nlm.nih.gov

The Spectrum of CHM Gene Mutations in Choroideremia and Their Relationship to Clinical Phenotype. [2022]

7. United States pubmed.ncbi.nlm.nih.gov

Clinical features of Japanese families with a 402delT or a 555-556delAG mutation in choroideremia gene. [2019]

8. United States pubmed.ncbi.nlm.nih.gov

Choroideremia: variability of clinical and electrophysiological characteristics and first report of a negative electroretinogram. [2006]

9. Korea (South) pubmed.ncbi.nlm.nih.gov

Identification of Pathogenic Variants in the CHM Gene in Two Korean Patients With Choroideremia. [2018]

10. England pubmed.ncbi.nlm.nih.gov

Is subretinal AAV gene replacement still the only viable treatment option for choroideremia? [2023]