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GITOPWTSMANCT06772402

GCB-001 in Treatment of Patients With Type II (SMA) Spinal Muscular Atrophy

StatusEnrolling by Invitation
PhaseNot applicable
Primary completion (estimated)

At risk: Primary completion moved at least 11 months later.

See at-risk trials from Genecombio Ltd.

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Record History

All 3 versions: 3 with changes below.

  1. Primary completion pushed from Dec 2026 to Dec 2027v3
  2. Trial status: Not Yet Recruiting to Enrolling by Invitation
    • Study sites: 0 to 1
    • Start date changed from estimate of Feb 2025 to Jan 15, 2025
    v2
  3. Study First Submitted
  • Notable: the plan changed

Endpoints

Primary

The rate of adverse events from baseline to 12 months after administration Assessed by CTCAE v4.0.
Timeframe: 0-12 months
AAV viral load after a single administration.
Timeframe: 0-12 months
AAV viral immunogenicity after a single administration.
Timeframe: 0-12 months
AAV viral shedding after a single administration.
Timeframe: 0-12 months
The change in total HFMSE (Hammersmith Functional Motor Scale Expanded) score from baseline at 12 months after a single administration
Timeframe: 0-12 months
The change in total RULM (Revised Upper Limb Module) score from baseline at 12 months after a single administration
Timeframe: 0-12 months

Arms and interventions

Experimental : Low doseExperimental

Low dose is the first cohort of the study with a low dose level.

Experimental : High doseExperimental

High dose is the first cohort of the study with a high dose level.

Genetic: GCB-001

Sites

1 site in 1 country

  • Children's Hospital ZheJiang Univisity School Of MedicineHangzhou, Zhejiang, China