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

NCT00374075 | Completed Phase 1

• 1 other identifier: 11893
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Spinal Muscular Atrophy

Keywords

Spinal Muscular Atrophy (SMA); Valproic Acid

Outcome Measures

Primary Outcomes (1)

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

Secondary Outcomes (5)

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

• Measures of gross motor function

• Electrophysiologic measures of denervation

• DEXA estimates of body composition, bone mineral density and content

• Measures of pulmonary function

Interventions

Valproic Acid DRUG

Eligibility Criteria

• Age: 2 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• 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

Sponsors & Collaborators

• University of Utah: lead
• Families of Spinal Muscular Atrophy: collaborator
• Leadiant Biosciences, Inc.: collaborator
• Abbott: collaborator

Study Sites (1)

University of Utah/Primary Children's Medical Center

Salt Lake City, Utah, 84132, United States

Location

Related Publications (43)

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MeSH Terms

Conditions

Muscular Atrophy, Spinal

Interventions

Valproic Acid

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; Neuromuscular Diseases

Intervention Hierarchy (Ancestors)

Pentanoic Acids; Valerates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids, Volatile; Fatty Acids; Lipids

Study Officials

• Kathryn J Swoboda, M.D

  University of Utah/Primary Children's Medical Center

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 6, 2006
• First Posted: September 8, 2006
• Study Start: September 1, 2003
• Study Completion: February 1, 2006
• Last Updated: May 10, 2023

Record last verified: 2016-08

Locations

US (1)