NCT06942572: ASTRA

A Phase 1/2, First-in-Human Dose Escalation/Expansion Study to Evaluate the Safety, Tolerability and Preliminary Efficacy of a Subretinal Injection of SB-007 in Subjects With Stargardt Disease (STGD1)

Recruiting now Phase 1, PHASE2 Last updated 29 April 2025

What this trial tests

Phase 1, PHASE2 trial testing SB-007 in Stargardt Disease in 57 participants. Currently enrolling.

Timeline

11 February 2025

Primary endpoint
31 October 2028

31 December 2028

Quick facts

Lead sponsor	Splice Bio
Phase	Phase 1, PHASE2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	randomized
Design	sequential
Masking	quadruple
Primary purpose	treatment
Enrollment	57
Start date	11 February 2025
Primary completion	31 October 2028
Estimated completion	31 December 2028
Sites	5 locations across United States

Drugs / interventions tested

• SB-007

Conditions studied

• Stargardt Disease — all drugs for Stargardt Disease →
• Stargardt Macular Degeneration — all drugs for Stargardt Macular Degeneration →
• Stargardt Macular Dystrophy — all drugs for Stargardt Macular Dystrophy →

Sponsor

Splice Bio

Who can join

Adults 12 to 65, any sex, with Stargardt Disease or Stargardt Macular Degeneration. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This Phase 1/2 study will evaluate the safety, tolerability, and preliminary efficacy of subretinal SB-007 administration to determine dose selection in subjects with Stargardt's Type 1 (STGD1).

This is a multicenter study which will enroll approximately 57 subjects, followed up over a 96 week period post treatment after a single administration of SB-007.

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Modalities of vision restoration in optic neuropathies and retinal disease.
   Rubin B, Lavinsky F, Cain R, Kuriyan AE, et al · · 2026 · PMID 42107518 · DOI 10.1016/j.preteyeres.2026.101474