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OpenTrials
Completed

NCT Number: NCT00374075

Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atrophy

This is an open label phase I/II clinical trial to assess safety, tolerability and potential effect on SMN mRNA and protein in vivo of a compound in which preliminary evidence supports a potential effect on SMN levels in vitro.

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Key information

Conditions

Spinal Muscular Atrophy Central Nervous System Diseases Motor Neuron Disease Muscular Atrophy, Spinal Nervous System Diseases Neurodegenerative Diseases Neuromuscular Diseases Spinal Cord Diseases

Age range

2 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

University of Utah/Primary Children's Medical Center

Salt Lake City, Utah, 84132, United States

About this study

This is an open label phase I/II trial of valproic acid in 40 SMA subjects > 2 years of age with severe, intermediate, and mild phenotypes. Primary outcome measures includes laboratory and physical examination assessments to monitor effects on liver, hematologic, metabolic and nutritional status. Secondary outcomes includes measures of gross motor function; electrophysiologic measures of denervation; DEXA estimates of body composition, bone mineral density and content; measures of pulmonary function; and quantitative SMN mRNA and protein levels in blood cells. Subjects will need 2-3 baseline visits over a 3 -6 month period prior to enrollment. Follow-up visits will be scheduled at 3, 6 and 12 months on treatment.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

Inclusion criteria

  • Patients must have a diagnosis of SMA, confirmed by genetic testing
  • Only patients 2 years of age and older at enrollment will be eligible

Exclusion criteria

  • Patients taking any medications with known hepatotoxicity, congenital metabolic disorders or on multiple anticonvulsant medications
  • Patients taking medications which may interact with VPA
  • Patients on ventilatory support for more than 16 hours per day
  • Patients currently enrolled in other treatment trials

Treatment and study plan

Valproic acid

Drug

Primary outcomes

  1. To assess safety and tolerability of VPA in SMA patients greater than 2 years of age

Secondary outcomes

  1. To look for a potential in vivo effect of VPA on SMN mRNA in patient blood cells at routinely used clinical doses

  2. Measures of gross motor function

  3. Electrophysiologic measures of denervation

  4. DEXA estimates of body composition, bone mineral density and content

  5. Measures of pulmonary function

Sponsors and collaborators

Lead sponsor

University of Utah

Other

Collaborators

  • Abbott
  • Families of Spinal Muscular Atrophy
  • Leadiant Biosciences, Inc.

Registry information

Official study title

In Vivo Study of Safety, Tolerability and Dosing Effect on SMN mRNA and Protein Levels of Valproic Acid in Patients With Spinal Muscular Atrophy

Important dates

Study start
2003
Study completion
2006
First posted
Sep 8, 2006
Registry last updated
May 10, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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