"DOES MYOBLOC™ IMPROVE FUNCTIONAL HAND USE IN YOUNG CHILDREN WITH A HYPERTONIC UPPER EXTREMITY?"

NCT00238641 | Completed Phase 1

• 1 other identifier: Myobloc2002
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

Brief Summary

In children with cerebral palsy, stiffness of the arm can develop early and delay or prevent the acquisition of normal hand skills. Improvement in functional use of the hand may therefore be dependent upon early treatment of upper extremity hypertonia. We propose to test a series of injections of Myobloc™ in a non-randomized one-way crossover pilot clinical trial and dose-finding study, with clinical assessments and blinded video evaluations. Ten children age 2-17 years with increased tone at the elbow or wrist will be expected to complete the study. A 1-month baseline evaluation period will be followed by an injection of low-dose Myobloc™ to affected muscle(s) of the arm according to standardized per-kilogram dosing with a maximum of 25U/kg in each affected arm. Three months later, a second injection of up to 50U/kg will be performed in each affected arm. Three months after the second dose, a third dose of up to 100U/kg will be performed in each affected arm. Neurological assessments will be performed at study entry, prior to each injection, and at 1 and 3 months following each injection. Routine physical therapy and non-study medications will be continued during the study.

Conditions

Cerebral Palsy

Outcome Measures

Primary Outcomes (1)

• The primary outcome measure is the time it takes to complete maximum arm extension during voluntary reaching, measured from the coraco-acromial joint to the midpoint of the dorsum of the wrist.

Secondary Outcomes (1)

• The rater will compare improvement or worsening in global arm function through the Unified Dystonia Rating Scale (UDRS), the motor subscale of the Unified Parkinson's Disease rating scale (UPDRS), and the Burke-Fahn-Marsden dystonia scale (BFM). Compar

Interventions

Botulinum Toxin type B DRUG

Eligibility Criteria

• Age: 2 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• \. Age 2- 17 years 2. Hypertonia affecting one or both upper extremities 3. Hypertonia caused by static encephalopathy due to prenatal or perinatal injury 4. Cognitive and motor function sufficient so that the child will reliably reach toward a target or a small toy 5. Concurrent enrollment in "standard of care" physical therapy or occupational therapy services

You may not qualify if:

• \. Injection of neuromuscular blocking agents to any extremity within six months prior to enrollment 2. Dose changes of trihexyphenidyl, dantrolene, baclofen, dopaminergic agents or benzodiazepines during the course of the study.
• \. Any use of aminoglycoside antibiotics during the study. 4. Prior surgery to either upper extremity 5. Initiation or change in physical or occupational therapy regimen within 3 months of study entry 6. Progressive or neurodegenerative disease, or suspicion of an inborn error of metabolism 7. Neuropathy, myopathy, or neuromuscular junction disease 8. Congenital deformities of either upper extremity 9. Respiratory or airway compromise, or use of respiratory medications 10. Cardiomyopathy 11. Any other medical condition that would place the child at risk for participation

Sponsors & Collaborators

• Stanford University: lead
• Elan Pharmaceuticals: collaborator

Study Sites (1)

Stanford University

Stanford, California, 94305-5235, United States

Location

MeSH Terms

Conditions

Cerebral Palsy

Interventions

rimabotulinumtoxinB

Condition Hierarchy (Ancestors)

Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases

Study Officials

• Terence Sanger, MD, PhD

  Stanford University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER

Study Record Dates

• First Submitted: October 11, 2005
• First Posted: October 13, 2005
• Study Start: January 1, 2002
• Study Completion: May 1, 2005
• Last Updated: October 13, 2005

Record last verified: 2005-10

Locations

US (1)