BOTOX(RegisteredTM) Treatment in Pediatric Lower Limb Spasticity: Double-blind Study. Protocol: 191622-111
This is a multicenter, randomized, double-blind, placebo-controlled, parallel-group, 12-week study evaluating 2 doses of BoToX, 4 and 8 u/kg, and placebo with standardized PT for the treatment of spasticity involving the ankle plantar flexors in monoplegic and hemiplegic children. Patients who successfully complete this study without major protocol deviations (eg, noncompliance to protocol-required procedures) will be given the option of enrolling in the open-label extension Study 191622-112 if they meet eligibility criteria.
The maximum study duration per patient will be approximately 16 weeks (including up to 4 weeks in the screening period). Patients will complete 8 study visits as follows: screening (week -4 to week -2), initiation of standardized PT (week -2), randomization and treatment visit (day 1), and post-injection follow-up visits at weeks 2, 4, 6, 8, and 12.
There will be 1 treatment cycle in the study. only 1 affected lower limb (referred to as the study limb) will be treated. Patients will be randomized in a 1:1:1 ratio to one of the following treatment groups: 4 u/kg (not to exceed 150 u) + PT, 8 u/kg (not to exceed 300 u) + PT, or placebo + PT.
The following are requirements for entry into the study:
* Male or female, 2 to 16 years and 11 months of age (prior to 17th birthday) at the day 1 visit
* Minimum weight of 10 kg at the screening and day 1 visits
* Monoplegic or hemiplegic patients with cerebral palsy who have dynamic muscle contracture
(spasticity confirmed by Hypertonia Assessment Tool [HAT]) of the ankle plantar flexors.
Equinovarus or equinovalgus deformities are acceptable.
* MAS-B score >= 2 for the ankle plantar flexors and minimum range of dorsiflexion of 0 degrees of the
ankle with knee fully extended in the study limb at screening and day 1 visits
* Patients for whom study treatment in the 3 ankle plantar flexors (namely gastrocnemius, soleus, and
tibialis posterior) of the study leg is appropriate
* Gross Motor Function Classification System x Expanded and Revised (GMFCS-E&R) level I to IV at
the screening visit
* Patients who are on anti-spastic medications or muscle relaxants (eg, oral baclofen, tizanidine,
dantrolene, scopolamine [oral or patch], vigabatrin, or benzodiazepine therapy) must be on a stable
dose and regimen for at least 30 days prior to the day 1 visit
* Patients who are on anti-epileptic medications must be on a stable dose and regimen for at least
30 days prior to the day 1 visit
* Written informed consent has been obtained from parent/legally authorized representative
* Written minor assent has been obtained in accordance with local laws and institutional review board (IRB)/independent ethics committee (IEC) requirements
* Written documentation has been obtained in accordance with the relevant country and local privacy
requirements, where applicable (eg, Written Authorization for Use and Release of Health and Research
Study Information for United States [US] sites and written Data Protection consent for European
Union [EU] sites)
* Negative urine pregnancy test at the screening and day 1 visits (for females of childbearing potential, defined as females post menarche)