Baclofen Efficacy and Safety Trials- Pediatric Pharmacokinetic and Pharmacodynamic Study of Oral Baclofen in the Treatment of Spasticity Associated with Cerebral Palsy (BEST-PK/PD)

This is an open-label, multiple-dose, ascending dose study in approximately 85 subjects in 3 age groups (ages 2 to 6 years, 6 to 12 years and 12 to 16 years), to be conducted at approximately eleven investigative sites with access to general clinical research centers (GCRCs) with pediatric experience.

Each of these sites will enroll approximately 12 subjects (for a total of approximately 110 subjects) to allow for discontinuation of subjects prior to study completion and titration of a sufficient number of subjects to each dose level.

Primary Objectives 

1. Determine pharmacokinetic parameters of oral baclofen in children with spasticity associated with cerebral palsy (CP). 
2. Describe the relationship between plasma concentrations of oral baclofen and clinical measures of spasticity. 
3. Determine optimal dosing range and interval for administration of oral baclofen for use in a randomized clinical trial of safety and efficacy.

Secondary Objectives 

1. Describe the relationship between plasma concentrations of oral baclofen and measures of strength, function, ease of care, pain/comfort and health related quality of life. 
2. Describe the safety and tolerability of oral baclofen in children with spasticity associated with CP.
3. Investigate preliminarily whether oral baclofen improves dystonia.