A clinical study to Evaluate the Safety, Tolerability and Preliminary Efficacy of SB-007 in Subjects with Stargardt Disease.

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Splicebio S.L.

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Eye Diseases [C11]

Decision date (initial)

2026-05-06

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

SpliceBio, S.L.

External identifiers

EU CT number

2024-519535-42-00

WHO UTN

U1111-1317-4444

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

The objective of this study is to evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt disease type 1 (STGD1) confirmed genotypically to have bi-allelic ABCA4 gene mutations.

Conditions and MedDRA coding

Stargardt Disease

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 8

1. 1. Provide written consent.
2. 2. Are male or female subjects ≥18 to ≤65 years (inclusive).
3. 3. Are able to understand and comply with the study procedures.
4. 4. Have a diagnosis of Stargardt disease type 1 (STGD1).
5. 5. Clinical evidence consistent with Stargardt Disease type 1.
6. 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.
7. 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.
8. 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 10

1. 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.
2. 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.
3. 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.
4. 4. Have a history of amblyopia in the study eye.
5. 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.
6. 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.
7. 7. Have received any investigational therapy within 90 days of the Screening Visit or 5 half-lives, whichever is longer.
8. 8. Have known serious allergies to the fluorescein dye.
9. 9. Have any significant ocular or non-ocular disease/disorder.
10. 10. Are an immediate family member (e.g., child, sibling) of the Sponsor or study site personnel.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. 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).

Secondary endpoints 8

1. 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.
2. 2. Change from baseline in lesion size growth (as measured by DDAF) on FAF imaging.
3. 3.Change from baseline in total lesion size growth (as measured by DDAF and well demarcated questionably decreased autofluorescence (QDAF)) on FAF imaging.
4. 4. Change from baseline in ellipsoid zone area as measured by spectral-domain optical coherence tomography (SD-OCT).
5. 5. Change from baseline in retinal sensitivity based on microperimetry.
6. 6. Change from baseline in best-corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS).
7. 7. Change from baseline in low luminance visual acuity (LLVA) using ETDRS.
8. 8. Change from baseline in contrast sensitivity scores.

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Splicebio S.L.

Sponsor organisation

Splicebio S.L.

Address

Carrer De Baldiri Reixac 10-12, Poligono Industrial Parc Cientific De Barcelona Poligono Industrial Parc Cientific De Barcelona

City

Barcelona

Postcode

08028

Country

Spain

Scientific contact point

Organisation

Splicebio S.L.

Contact name

Leigh Shaw

Public contact point

Organisation

Splicebio S.L.

Contact name

Leigh Shaw

Third parties 7

Locations

2 EU/EEA countries · 3 investigational sites

By country

Investigational sites

Results and documents

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

Documents 23 files

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

Application history

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