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Clinical trial

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

ID
Name
11893
Description
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.
Trial arms
Trial start
2003-09-01
Trial end
2006-02-01
Status
Completed
Phase
Early phase I
Treatment
Valproic Acid
Size
42
Primary endpoint
To assess safety and tolerability of VPA in SMA patients greater than 2 years of age
Eligibility criteria
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
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'SINGLE_GROUP', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 42}}
Updated at
2023-05-10

1 organization

1 product

1 indication

Organization
University of Utah
Product
Valproic Acid
Indication
Spinal Muscular Atrophy