BOTOX treatment in pediatric upper limb spasticity: Double-blind study. Protocol: 191622-101
This study is being done to see if the investigational drug, botulinum neurotoxin type A (BoNT) also known as BOTOX[RegisteredTM], can treat upper limb spasticity. This study will consist of 8 clinic visits over an approximate 16 -week period. On the 3rd visit, the participant will receive the BOTOX injections into the affected region of the upper limb. Additionally, the participant will begin the first of 13 weekly hourly sessions of occupational therapy two weeks prior to treatment day. Both the visits to the Study Doctor and the follow-up visits at the OT will take approximately 1 hour. The whole study will last about 4 months.
The visits will occur as follows:
* Visit 1 Screening (up to 28 days before treatment visit)
* Visit 2 (approximately 14 days before treatment visit)
* Visit 3 (treatment visit)
* Follow-up visits at Weeks 2, 4, 6, and 8
* Exit visit 12 weeks after treatment
There are 3 different groups in this study so that different amounts of the medicine can be tested to see which one is best. Everyone that joins the study, will be randomly assigned into one of these 3 groups:
1. One group will get a small dose of the medicine, - at 3 units per kilogram of body weight (1 in 3 chance).
2. Another group will get a higher dose of medicine, - at 6 units per kilogram of body weight (1 in 3 chance).
3. And another group will get a placebo with no medicine in it placebo (1 in 3 chance).
In other words, if the participant is a good candiate for the study, they will be assigned randomly like drawing straws to receive either the small amount of BOTOX, the large amount of BOTOX, or the placebo.
* 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
* Upper limb monoplegic, hemiplegic, or triplegic spasticity (spasticity confirmed by Hypertonia Assessment Tool [HAT]) with single-arm sparing (only 1 arm requiring botulinum toxin treatment for spasticity during the study) resulting from cerebral palsy, or post-stroke with the stroke onset prior to age 2 and at least 12 months prior to the day 1 visit
* Patient[Single Quote]s spasticity meets at least one of the conditions below in the study limb at the screening and day 1 visits:
o Elbow flexor tone of 2 or greater as measured by the MAS-B AND elbow flexor muscle contracture of no more than 30 degrees, and/or
o Wrist flexor tone of 2 or greater with finger flexor tone of 1+ or greater as measured by the MAS-B
AND at least neutral position for wrist with fingers at maximum extension
* Has demonstrable movement and attempted utilization of the study limb
* Gross Motor Function Classification System Expanded and Revised (GMFCS-E&R) level I to IV at the screening visit
* Manual Ability Classification System (MACS) level I to IV at the screening visit
* Stable medical condition in the investigator[Single Quote]s opinion
* Acceptable clinical laboratory results 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
* Patients who are on an intrathecal baclofen pump must be on a continuous infusion at a stable dose and regimen (ie, not on bolus infusions) 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)