Safety and Tolerability of Dalfampridine in Subjects With Cerebral Palsy

Study Overview
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Brief Summary
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A double-blind, placebo-controlled, crossover study in subjects with cerebral palsy (CP) to evaluate the safety and tolerability and the effect of dalfampridine extended release (ER) tablets on sensorimotor function

Official Title
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A Double-Blind, Placebo-Controlled, Crossover Study in Subjects With Cerebral Palsy to Evaluate the Safety and Tolerability and the Effect on Sensorimotor Function of Dalfampridine-ER

Conditions
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Cerebral Palsy (CP)

Intervention / Treatment
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* Drug: dalfampridine-ER 10mg
* Other: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: A diagnosis of CP No previous use of any dalfampridine formulation Ability to perform all the required study procedures. Subjects should be capable of fully extending and flexing both hands Exclusion Criteria: Presence of any progressive neurological disease Severe CP defined as the requirement to use a wheelchair at all times and a care taker for constant assistance in daily activities. This definition includes spastic quadriplegia Pregnant or breastfeeding

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 70 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Crossover Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Placebo Comparator: (PART A) AB: dalfampridine-ER 10mg then placebo<br>Each subject randomized to the AB arm will receive a single witnessed dose of (A) dalfampridine-ER 10 mg, and a single witnessed dose of (B) placebo, two days apart | Drug: dalfampridine-ER 10mg<br> <br> Other: Placebo<br> <br> |
| Placebo Comparator: (PART A) BA: placebo then dalfampridine-ER 10mg<br>Each subject randomized to the BA arm will receive a single witnessed dose of (B) placebo, and a single witnessed dose of (A) dalfampridine-ER 10 mg, two days apart | Drug: dalfampridine-ER 10mg<br> <br> Other: Placebo<br> <br> |
| Placebo Comparator: (PART B) AB: dalfampridine-ER 10mg then placebo<br>Each subject randomized to the AB arm will receive multiple doses of (A) dalfampridine-ER 10mg and multiple doses of (B) placebo | Drug: dalfampridine-ER 10mg<br> <br> Other: Placebo<br> <br> |
| Placebo Comparator: (PART B) BA: Placebo then dalfampridine-ER 10mg<br>Each subject randomized to the BA arm will receive multiple doses of (B) placebo, and multiple doses of (A) dalfampridine-ER 10mg | Drug: dalfampridine-ER 10mg<br> <br> Other: Placebo<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and Tolerability of Dalfampridine-ER 10mg in Subjects With Cerebral Palsy (CP) | Safety and tolerability will be assessed primarily by monitoring Treatment Emergent Adverse Events (TEAEs) TEAEs are defined as Adverse Events (AEs) with date of onset (or worsening) on or after the start-date of double-blind treatment and no more than 5 days after the last dose of double-blind treatment for Part A of the study and no more than 9 days for Part B of the study. The severity categories of mild, moderate or severe, are defined below: Mild is defined as causing no limitation of usual activities Moderate is defined as causing some limitation of usual activities Severe is defined as causing inability to carry out usual activities | up to 31 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Measure the Effects of Both Single and Multiple Doses of Dalfampridine-ER 10 mg on Sensorimotor Function | Hand strength as measured by a composite Z-score derived from the grip test, and key, tip and palmar pinch tests Manual dexterity as measured by the Box and Block Test Walking speed as measured by the Timed 25 Foot Walk (T25FW) Gait as measured by gait analysis equipment (to be performed by sites that have the capability to perform it) For Part B only, subjective impressions of treatment as measured by: Subject Global Impression (SGI) Clinician Global Impression (CGI) | up to 31 days |