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NCT07903376

SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)

StatusNot Yet Recruiting
PhaseEarly Phase 1
Primary completion (estimated)
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Record History

All 1 version: 1 with changes below.

  1. Study First Submitted

Endpoints

Primary

Occurrence and Severity of Adverse Events and Serious Adverse Events
Timeframe: Through Month 12
Change in Alanine Aminotransferase (ALT) Levels From Baseline
Timeframe: Baseline through Month 12
Change in Aspartate Aminotransferase (AST) Levels From Baseline
Timeframe: Baseline through Month 12
Change in Platelet Count From Baseline
Timeframe: Baseline through Month 12
Change in Serum Creatinine Levels From Baseline
Timeframe: Baseline through Month 12
Change in Cardiac Troponin I Levels From Baseline
Timeframe: Baseline through Month 12
Change in Lactate Dehydrogenase (LDH) Levels From Baseline
Timeframe: Baseline through Month 12
Change in Hemoglobin Levels From Baseline
Timeframe: Baseline through Month 12
Change in Total Bilirubin Levels From Baseline
Timeframe: Baseline through Month 12
Occurrence of Thrombotic Microangiopathy (TMA) Following SPX-01 Administration
Timeframe: Through Month 12

Secondary (11)

Overall Survival Status
Event-Free Survival Status
Change in Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Total Score From Baseline
Age at First Achievement of Full Head Control as Assessed by HINE-2
Age at First Achievement of Independent Rolling as Assessed by HINE-2
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Age at First Achievement of Independent Sitting as Assessed by HINE-2
Age at First Achievement of Standing With Support as Assessed by HINE-2
Change in Compound Muscle Action Potential (CMAP) Amplitude From Baseline
Ventilatory Support Status
Swallowing Function Status
Feeding Support Status

Arms and interventions

Experimental: SPX-01Experimental

One infant with genetically confirmed spinal muscular atrophy type 1 will receive a single intravenous administration of SPX-01 at a target dose of 1.1 × 10\^14 vector genomes/kg.

Genetic: SPX-01

Sites

1 site in 1 country

  • Children's Medical CenterTehran, Tehran Province, Iran