Adeno-associated virus vector serotype 8 encoding the ABCA4 protein (N-region and C-region) for Stargardt disease

Inclusion criteria

• {"criterion_text":"- 1. Provide written consent.\n- 2. Are male or female subjects ≥18 to ≤65 years (inclusive).\n- 3. Are able to understand and comply with the study procedures.\n- 4. Have a diagnosis of Stargardt disease type 1 (STGD1).\n- 5. Clinical evidence consistent with Stargardt Disease type 1.\n- 6. For women of child-bearing potential (WOCBP), have a negative pregnancy test at Screening and, if due to receive active treatment, at Day 0.\n- 7. For both WOCBP and male subjects (or their female partners who are of child-bearing potential), agree to either strict abstinence or, if sexually active, use an acceptable contraception measure for 4 months from Day 0.\n- 8. Must have clear ocular media and adequate pupillary dilation in the study eye, including no allergy to dilating eyedrops, to permit good quality retinal imaging."}

Exclusion criteria

• {"criterion_text":"- 1. Have had any intraocular surgery (including cataract surgery) or thermal laser within 90 days of the Screening Visit or planned intraocular surgery (including cataract surgery) or thermal laser during the period of the study, in the study eye.\n- 10. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.\n- 2. Have had any major surgical procedure within 30 days of the Screening Visit or planned or anticipated major surgery during the period of the study.\n- 3. Have two pathogenic or likely pathogenic variants in inherited retinal dystrophy (IRD) genes (other than ABCA4) or a single pathogenic or likely pathogenic variant in autosomal dominant or X-linked IRD genes.\n- 4. Have a history of amblyopia in the study eye.\n- 5. Are unwilling to stop taking the following products at Screening and throughout the study: a. Supplements containing vitamin A or beta-carotene, liver-based products. b. Prescription oral retinoids. Topical products containing vitamin A or retinoids are not exclusionary.\n- 6. Have any ophthalmic history of gene therapy, stem cell therapy, surgical implantation of prosthetic retinal chips, or intravitreal or sub-retinal or supra-choroidal injections.\n- 7. Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer.\n- 8. Have known serious allergies to the fluorescein dye.\n- 9. Have any significant ocular or non-ocular disease/disorder."}

Primary endpoints

• {"endpoint_text":"- The primary endpoint is safety and tolerability through Week 96 assessed by incidence and/or clinically significant changes in ocular and non-ocular adverse events (AEs).","definition_or_measurement_approach":"Assessed by incidence and/or clinically significant changes in ocular and non-ocular adverse events (AEs) through Week 96."}

Secondary endpoints

• {"endpoint_text":"- 1. Change from baseline in lesion size growth (as measured by definitely decreased autofluorescence [DDAF]) on fundus autofluorescence (FAF) imaging compared between SB-007 and untreated control.","definition_or_measurement_approach":"Change from baseline in lesion size growth measured by DDAF on FAF imaging; comparison between SB-007 and untreated control."}
• {"endpoint_text":"- 2. Change from baseline in lesion size growth (as measured by DDAF) on FAF imaging.","definition_or_measurement_approach":"Change from baseline in lesion size growth measured by DDAF on FAF imaging."}
• {"endpoint_text":"- 3.Change from baseline in total lesion size growth (as measured by DDAF and well demarcated questionably decreased autofluorescence (QDAF)) on FAF imaging.","definition_or_measurement_approach":"Change from baseline in total lesion size growth measured by DDAF and QDAF on FAF imaging."}
• {"endpoint_text":"- 4. Change from baseline in ellipsoid zone area as measured by spectral-domain optical coherence tomography (SD-OCT).","definition_or_measurement_approach":"Change from baseline in ellipsoid zone area measured by SD-OCT."}
• {"endpoint_text":"- 5. Change from baseline in retinal sensitivity based on microperimetry.","definition_or_measurement_approach":"Change from baseline in retinal sensitivity measured by microperimetry."}
• {"endpoint_text":"- 6. Change from baseline in best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS).","definition_or_measurement_approach":"Change from baseline in BCVA measured by ETDRS charts."}
• {"endpoint_text":"- 7. Change from baseline in low luminance visual acuity (LLVA) using ETDRS.","definition_or_measurement_approach":"Change from baseline in LLVA measured using ETDRS under low luminance conditions."}
• {"endpoint_text":"- 8. Change from baseline in contrast sensitivity scores.","definition_or_measurement_approach":"Change from baseline in contrast sensitivity measured by specified contrast sensitivity testing (method as per protocol)."}

Germany

Earliest CTIS Part Ii Submission Date

23-06-2025

Latest Decision Or Authorization Date

07-05-2026

Processing Time Days

318

Number Of Sites

2

Number Of Participants

10

Belgium

Earliest CTIS Part Ii Submission Date

23-04-2026

Latest Decision Or Authorization Date

06-05-2026

Processing Time Days

13

Number Of Sites

1

Number Of Participants

3

Primary sponsor

Full Name

Splicebio S.L.

Organisation Type

Pharmaceutical company

Country Of Registered Address

Spain

Contract research organisations

Name

Medpace Finland Oy

Responsibilities

Sponsor duties codes: 1,10,11,12,13,2,3,5,6,7,8,9 (multiple trial management and operational responsibilities listed)

Name

Almac Pharma Services Limited

Responsibilities

Sponsor duties code: 14

Name

Precision For Medicine Inc.

Responsibilities

Sponsor duties code: 4 (laboratory services)

Name

Precision Vision

Responsibilities

Provides vision charts

Name

Global Eye Trials Limited

Responsibilities

BCVA Lane Certification

Name

Doheny Eye Institute

Responsibilities

Central imaging reader

Third parties

• {"country":"United Kingdom","full_name":"Global Eye Trials Limited","duties_or_roles":"BCVA Lane Certification","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Precision Vision","duties_or_roles":"Provides vision charts","organisation_type":"Industry"}
• {"country":"Finland","full_name":"Blueprint Genetics Oy","duties_or_roles":"Sponsor duties code: 4","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"Finland","full_name":"Medpace Finland Oy","duties_or_roles":"Sponsor duties codes: 1,10,11,12,13,2,3,5,6,7,8,9","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom (Northern Ireland)","full_name":"Almac Pharma Services Limited","duties_or_roles":"Sponsor duties code: 14","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Precision For Medicine Inc.","duties_or_roles":"Sponsor duties code: 4","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Doheny Eye Institute","duties_or_roles":"Central imaging reader (sponsor duties code: 15)","organisation_type":"Educational Institution"}