Phase 2/3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Single-Treatment Efficacy and Safety Study of MYOBLOC in Treatment of Adult Upper Limb Spasticity and Open-Label Extension, Multiple-Treatment Safety Study of MYOBLOC
Overview
- Phase
- Phase 2
- Intervention
- Phase 2; Low Dose MYOBLOC
- Conditions
- Spasticity
- Sponsor
- Supernus Pharmaceuticals, Inc.
- Enrollment
- 272
- Locations
- 9
- Primary Endpoint
- Effect of MYOBLOC on Clinical Global Impression of Change (CGI-C) Scale [Phase 2 and Phase 3]
- Status
- Active, not recruiting
- Last Updated
- 3 years ago
Overview
Brief Summary
Phase 2/3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial assessing the efficacy and safety of MYOBLOC for the treatment of upper limb spasticity in adults followed by an open-label extension safety trial.
Detailed Description
Phase 2, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial will compare the efficacy and safety of two doses of MYOBLOC versus volume-matched placebo in the treatment of upper limb spasticity in adults. An interim analysis will evaluate all available safety and efficacy data from the Phase 2 double-blind trial in order to recommend which dose will be evaluated in subsequent Phase 3 trial. The Phase 3, randomized, double-blind, placebo-controlled, single-treatment, multicenter trial will compare the efficacy and safety of MYOBLOC versus placebo in the treatment of upper limb spasticity in adults. Subjects who complete either the Phase 2 or Phase 3 trial will continue into an open-label extension where each will receive 5 separate MYOBLOC treatments (\~13 week apart) for upper limb spasticity.
Investigators
Eligibility Criteria
Inclusion Criteria
- Not provided
Exclusion Criteria
- Not provided
Arms & Interventions
Phase 2; Low Dose MYOBLOC
Low Dose MYOBLOC is a single treatment and will be compared to volume-matched placebo
Intervention: Phase 2; Low Dose MYOBLOC
Phase 2; High Dose MYOBLOC
High Dose MYOBLOC is a single treatment and will be compared to volume-matched placebo
Intervention: Phase 2; High Dose MYOBLOC
Phase 2; Placebo
Volume-matched placebo is a single treatment
Intervention: Phase 2; Placebo
Phase 3; MYOBLOC
MYOBLOC is a single treatment and will be compared to volume-matched placebo
Intervention: Phase 3; MYOBLOC
Phase 3; Placebo
Volume-matched placebo is a single treatment
Intervention: Phase 3; Placebo
Outcomes
Primary Outcomes
Effect of MYOBLOC on Clinical Global Impression of Change (CGI-C) Scale [Phase 2 and Phase 3]
Time Frame: Week 4
The second co-primary endpoint is the Clinical Global Impression of Change (CGI-C) score in functional ability at Week 4 post-injection. The CGI-C scale is a single item clinician assessment of how much the patient's functional ability has improved, worsened or has not changed relative to the patient's baseline state prior to treatment (injection). The CGI-C is rated on a 7-point Likert scale from 1 to 7, where 1 = "Very much improved", 2 = "Much improved", 3 = "Minimally improved", 4 = "No change", 5 = "Minimally worse", 6 = "Much worse", and 7 = "Very much worse". Successful therapy is indicated by a lower score (\<4) in subsequent testing.
Effect of MYOBLOC on the Modified Ashworth Scale (MAS) for tone of the Primary Target Muscle Group (PTMG) [Phase 2 and Phase 3]
Time Frame: Baseline and Week 4
The first co-primary endpoint is the change from baseline in Modified Ashworth Scale (MAS) score for tone of the Primary Target Muscle Group (PTMG) selected for treatment at Week 4 post-injection. The MAS is an internationally accepted and validated instrument used to measure resistance during passive soft-tissue stretching. Resistance will be measured and recorded using a 6-point scale ranging from 0 (no increase in muscle tone) to 4 (affected part\[s\] rigid in flexion or extension). A lower change from baseline MAS score (\<0) represents a better outcome.
Secondary Outcomes
- Effect of MYOBLOC on the Patient Global Impression of Change (PGI-C) [Phase 2 and Phase 3](Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Modified Ashworth Scale (MAS) for tone in each muscle group selected for treatment [Phase 2 and Phase 3](Baseline and Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Modified Barthel Index (MBI) [Phase 2 and Phase 3](Baseline and Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Modified Ashworth Scale (MAS) Responder Rate for the Primary Target Muscle Group (PTMG) [Phase 2 and Phase 3](Baseline and Week 4)
- Effect of MYOBLOC on the Modified Ashworth Scale (MAS) for tone in the Primary Target Muscle Group (PTMG) [Phase 2 and Phase 3](Baseline and Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on Clinical Global Impression of Change (CGI-C) Scale [Phase 2 and Phase 3](Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Caregiver Global Impression of Change (GGI-C) [Phase 2 and Phase 3](Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Pain Numeric Rating Scale (Pain-NRS) [Phase 2 and Phase 3](Baseline and Weeks 2, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Clinical Global Impression of Severity (CGI-S) [Phase 2 and Phase 3](Baseline and Weeks 2, 4, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Patient Global Impression of Severity (PGI-S) [Phase 2 and Phase 3](Baseline and Weeks 2, 4, 8, and 13 (and, if applicable, at re-evaluation visit))
- Effect of MYOBLOC on the Caregiver Global Impression of Severity (GGI-S) [Phase 2 and Phase 3](Baseline and Weeks 2, 4, 8, and 13 (and, if applicable, at re-evaluation visit))