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)

Part of paid clinical trials in Aurora, Colorado.

Sponsor
Splice Bio
Study ID
NCT06942572
Phase
PHASE1/PHASE2
Status
Recruiting
Apply to this trial

Conditions

  • Stargardt Disease
  • Stargardt Macular Degeneration
  • Stargardt Macular Dystrophy

Eligibility Criteria

Sex
ALL
Age
12 Years - 65 Years
Healthy Volunteers
Not accepted

Interventions

  • SB-007 — GENETIC
    Subretinal Administration of SB-007

Study Details

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.

Key Dates

Start date
Feb 11, 2025
Status verified
Apr 2025
Primary completion
Oct 31, 2028
Completion
Dec 31, 2028

Study Design

Enrollment
57 participants (estimated)
Allocation
RANDOMIZED
Intervention model
SEQUENTIAL
Primary purpose
TREATMENT

Arms

  • Experimental: Phase 1 Dose Escalation - Low Dose
    Low Dose: Subjects will receive subretinal injection of SB-007 in the low dose group
  • Experimental: Phase 1 Dose Escalation - Medium Dose
    Medium Dose: Subjects will receive subretinal injection of SB-007 in the medium dose group
  • Experimental: Phase 1 Dose Escalation - High Dose
    High Dose: Subjects will receive subretinal injection of SB-007 in the high dose group
  • Experimental: Phase 2 Dose Expansion: Dose 1 from Phase 1 Randomised Arm
    Subjects will receive a subretinal injection of SB-007 with Maximum tolerated dose (MTD) from Phase 1
  • Experimental: Phase 2 Dose Expansion: Dose 2 from Phase 1 Randomised Arm
    Subjects will receive a subretinal injection of SB-007 with lower dose than Maximum tolerated dose (MTD) from Phase 1
  • No Intervention: Phase 2 No Intervention - Randomised Control Arm
    No Intervention Control Arm: Subject will not receive any active study intervention

Primary Outcome Measure

Safety Measures [ Time Frame: 96 weeks ]

Central Contacts

Locations (5)

FacilityCityStateZIPSite coordinators
UCHealth Sue Anschutz-Rodgers Eye Center,AuroraColorado80045
Site 109 (PRINCIPAL_INVESTIGATOR)
Bascom Palmer Eye InstituteMiamiFlorida33136
Site 104 (PRINCIPAL_INVESTIGATOR)
Massachusetts Eye and Ear InfirmaryBostonMassachusetts02114
Center for Clinical Research
Site 105 (PRINCIPAL_INVESTIGATOR)
Oregon Health & Science UniversityPortlandOregon97239
Site 103 (PRINCIPAL_INVESTIGATOR)
Retina Foundation of the SouthwestDallasTexas75261
Site 102 (PRINCIPAL_INVESTIGATOR)

Find similar trials in Aurora, CO

By research site
UCHealth Sue Anschutz-Rodgers Eye Center· Aurora, COBascom Palmer Eye Institute· Miami, FLMassachusetts Eye and Ear Infirmary· Boston, MAOregon Health & Science University· Portland, ORRetina Foundation of the Southwest· Dallas, TX

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