Phase I/II Trial of Valproic Acid and Carnitine in Infants with Spinal Muscular Atrophy Type I (CARNI-VAL Type I) - CARNI-VAL Type I

• Conditions: Spinal Muscular Atrophy Type I in infants; MedDRA version: 9.1Level: LLTClassification code 10051203Term: Spinal muscular atrophy congenital

• Registration Number: EUCTR2008-003915-11-DE
• Lead Sponsor: niversity of Utah

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2009-06-25
• Last Update Posted: 17 December 2012

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 36

Inclusion Criteria

• Laboratory documentation of SMN mutation or deletion consistent with genetic diagnosis of SMA
• Clinical diagnosis of SMA type I
• Age between 2 weeks and 12 months
• Written informed consent of parent/guardian
Are the trial subjects under 18? yes
Number of subjects for this age range:
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

• Any clinical or laboratory evidence of hepatic or pancreatic insufficiency.
• Laboratory results drawn within 14 days prior to start of study drug demonstrating:
- Liver transaminases (AST, ALT), lipase, amylase: >1.5 x ULN,
- White Blood Cell Count: < 3 x 10^9/l
- Neutropenia: <1 x 10^9/l
- Platelet: <100 x 10^9/l
- Hematocrit: <30 %, persisting over a 30-day period
• Serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study entry.
• Use of medications or supplements within 30 days of study enrollment that
- interfere with VPA or carnitine metabolism
- that increase the potential risks of VPA or carnitine
- that are hypothesized to have a beneficial effect in SMA animal models or human neuromuscular disorders, including riluzole, valproic acid, hydroxyurea, oral use of albuterol, sodiumphenylbutyrate, butyrate derivatives, creatinine, growth hormone, anabolic steroids, probenecid, oral or parenteral use of corticosteroids at entry, or
agents anticipated to increase or decrease muscle strength or agents with presumed histone deacetylase (HDAC) inhibition
• Infants who have participated in a treatment trial for SMA within 30 days of study entry or who will become enrollees in any other treatment trial during the course
of this study.
• Unwillingness to travel for study assessments.
• Coexisting medical conditions that contradict use of VPA/carnitine or travel to and from study site.
• Requirement of ventilator support for more than 12 hours per day.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified