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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Biogen Idec Research Limited

Participant type

Patients

Age range

18-64 years

Gender

Male

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Trial duration

7 Oct 2024 → ongoing

Decision date (initial)

2024-10-01

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

No

Funding sources

NightstaRx Ltd (A Biogen Company)

External identifiers

EU CT number

2023-507994-16-00

EudraCT number

2017-003104-42

ClinicalTrials.gov

NCT03584165

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Therapy, Efficacy

To evaluate the long-term safety and efficacy of a sub retinal injection
of:
• AAV2-REP1 in participants with CHM who have been previously treated
with AAV2-REP1 and who have exited an antecedent study; these
treated participants will be compared with untreated control participants
who have exited the STAR study.
• AAV8-RPGR in participants with XLRP who have been previously
treated with AAV8-RPGR and who have exited an antecedent study.

Secondary objectives 1

1. Not applicable

Conditions and MedDRA coding

Choroideremia (CHM) X-Linked Retinitis Pigmentosa (XLRP)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. CHM Participants: a. Are willing and able to give informed consent for participation in the study, and
2. CHM Participants: b. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of AAV2- REP1 for CHM
3. XLRP Participants: a. Are willing and able to give informed consent for participation in the study
4. XLRP Participants: b. Have received a sub-retinal injection of AAV8-RPGR for XLRP and have exited an antecedent study

Exclusion criteria 1

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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Safety assessments will be performed as per protocol

Secondary endpoints 13

1. Change from Baseline in best-corrected visual acuity (BCVA) as measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) chart
2. Proportion of participants with no decrease from Baseline in BCVA or a decrease from Baseline in BCVA of <5 ETDRS letters (in CHM subjects only)
3. Proportion of participants with an increase from Baseline in BCVA of ≥ 10 ETDRS letters (in CHM subjects only)
4. Proportion of participants with an increase from Baseline in BCVA of ≥ 15 ETDRS letters (in CHM subjects only)
5. Available assessments of fundus autofluorescence at each visit
6. Available assessments of fundus photography at each visit
7. Available assessments of spectral-domain optical coherence tomography (SD-OCT) at each visit
8. Available assessments of microperimetry at each visit
9. Change from Baseline in the 25-Item Visual Function Questionnaire (VFQ-25)
10. Change from Baseline in visual field (in AAV8-RPGR-treated participants only)
11. Proportion of participants with an increase from Baseline in low luminance visual acuity (LLVA) of ≥10 ETDRS letters (in AAV8-RPGRtreated participants only)
12. Proportion of participants with an increase from Baseline in LLVA of ≥ 15 ETDRS letters (in AAV8-RPGR-treated participants only)
13. Datasets from the 2 disease populations will be analyzed separately.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Biogen Idec Research Limited

Sponsor organisation

Biogen Idec Research Limited

Address

Innovation House, 70 Norden Road 70 Norden Road

City

Maidenhead

Postcode

SL6 4AY

Country

United Kingdom

Scientific contact point

Organisation

Biogen Idec Research Limited

Contact name

CTA Lead Department

Public contact point

Organisation

Biogen Idec Research Limited

Contact name

Clinical Operations Department

Third parties 4

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 13 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications