BOTOX® Treatment in Pediatric Upper Limb Spasticity

NCT01603602 | Completed Phase 3 Results DMC

• 2 other identifiers: 191622-1012012-000062-38
• Study Type: interventional
• Target: 235
• Locations: 9 countries
• Sites: 45

Brief Summary

This study will evaluate the safety and efficacy of BOTOX® (botulinum toxin Type A) in pediatric patients with upper limb spasticity.

Conditions

Pediatrics; Muscle Spasticity; Cerebral Palsy; Stroke

Outcome Measures

Primary Outcomes (2)

• Average Change From Baseline in Modified Ashworth Scale-Bohannon (MAS-B) Score of the Principal Muscle Group at Weeks 4 and 6

  The MAS-B was used to evaluate spasticity based on grading the resistance encountered in the principal muscle group (elbow and wrist) by means of passively moving a limb through its range of motion at a study specified velocity. The resistance encountered to passive stretch was graded using a 6-point scale where: 0=no increase in muscle tone (best) to 4=affected part(s) rigid in flexion or extension (worst). For analysis purposes, the MAS-B was recoded as follows: 0=1, 1=1, 1+=2, 2=3, 3=4, 4=5. The scores at Weeks 4 and 6 were averaged. A Mixed Model Repeated Measures (MMRM) model was used for analysis. A negative change from Baseline indicates improvement.

  Baseline (Day 1) to Weeks 4 and 6

• Average Clinical Global Impression (CGI) of Overall Change by Physician at Weeks 4 and 6

  The CGI of overall change (improvement or worsening) was assessed by the physician considering the participant's clinical condition and severity of side effects using a 9-point scale where: -4=very marked worsening to +4=very marked improvement. The scores at Weeks 4 and 6 were averaged. A MMRM model was used for analysis.

  Weeks 4 and 6

Secondary Outcomes (3)

• Average Change From Baseline in MAS-B Score of the Finger Flexor Muscle Group at Weeks 4 and 6

  Baseline (Day 1) to Weeks 4 and 6

• Goal Attainment Score (GAS) as Assessed by Physician Using a 6-Point Scale

  Week 8 and 12

• Change From Baseline in Severity of Spasticity of the Principal Muscle Group (R2-R1) Calculated Using the Modified Tardieu Scale (MTS)

  Baseline (Day 1) to Week 6

Study Arms (3)

BOTOX® 3 U/kg

EXPERIMENTAL

Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 3 units (U) per kilogram (kg) of body weight (3 U/kg) into specified muscles of the upper limb on Day 1. Participants received weekly occupational therapy (OT).

Biological: botulinum toxin Type A

BOTOX® 6 U/kg

EXPERIMENTAL

Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 6 U per kg of body weight (6 U/kg) into specified muscles of the upper limb on Day 1. Participants received weekly OT.

Biological: botulinum toxin Type A

Placebo

PLACEBO COMPARATOR

Participants received intramuscular injections of normal saline (placebo) into specified muscles of the upper limb on Day 1. Participants received weekly OT.

Drug: Normal Saline (Placebo)

Interventions

botulinum toxin Type A BIOLOGICAL

Intramuscular injections of botulinum toxin Type A into specified muscles of the upper limb on Day 1.

Also known as: BOTOX®, onabotulinumtoxinA

BOTOX® 3 U/kg; BOTOX® 6 U/kg

Normal Saline (Placebo) DRUG

Intramuscular injections of normal saline (placebo) into specified muscles of the upper limb on Day 1.

Placebo

Eligibility Criteria

• Age: 2 Years - 16 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Minimum weight of 10 kg/22 lb
• Upper limb spasticity due to cerebral palsy or stroke

You may not qualify if:

• Muscular dystrophy, myasthenia gravis, Eaton-Lambert syndrome, amyotrophic lateral sclerosis, or mitochondrial disease
• Uncontrolled epilepsy
• Botulinum Toxin therapy of any serotype for any condition within the last 6 months
• Previous surgical treatment of the study limb (except tendon lengthening), or planned surgery of the study limb during the study
• Previous casting of the study limb for spasticity within 6 months or with a dynamic splint within 3 months, or planned casting or dynamic splinting for spasticity of the study limb or affected lower limb during the study

Sponsors & Collaborators

• Allergan: lead

Study Sites (45)

AMS Neurology

Pasadena, California, 91106, United States

Location

Harrison Clinical Management

Pomona, California, 91766, United States

Location

Children's Hospital Colorado Dept. of PM&R

Aurora, Colorado, 80045, United States

Location

Associated Neurologists of Southern CT, P.C.

Fairfield, Connecticut, 06824, United States

Location

NW FL Clinical Research Group, LLC

Gulf Breeze, Florida, 32561, United States

Location

Axcess Medical Research, LLC

Loxahatchee Groves, Florida, 33470, United States

Location

Pediatric Neurology, PA

Orlando, Florida, 32891, United States

Location

Children's Healthcare of Atlanta Children's Rehabilitation Associates

Atlanta, Georgia, 30342, United States

Location

Gillette Children's Specialty Healthcare

Saint Paul, Minnesota, 55101, United States

Location

Washington University School of Medicine

St Louis, Missouri, 63110, United States

Location

Clinical Research Center of New Jersey

Voorhees Township, New Jersey, 08043, United States

Location

NYU Hospital for Joint Diseases

New York, New York, 10003, United States

Location

Columbia University Medical Center Dept. of Rehab. & Regenerative Medicine

New York, New York, 10032, United States

Location

OnSite Clinical Solutions, LLC

Charlotte, North Carolina, 28203, United States

Location

PMG Research if Charlotte, LLC

Charlotte, North Carolina, 28203, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

Baylor College of Medicine Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Road Runner Research

San Antonio, Texas, 78258, United States

Location

Seattle Children's Hospital

Seattle, Washington, 98105, United States

Location

Marshfield Clinic

Marshfield, Wisconsin, 54449, United States

Location

Holland Bloorview Kids Rehab

Toronto, Ontario, M4G 1R8, Canada

Location

Debrecen University Clinical Center, Orthopedic Clinic

Debrecen, 4032, Hungary

Location

De La Salle University Medical Center

Dasmariñas, Cavite, 4114, Philippines

Location

Philippine Children's Medical Center

Bagong Pag-asa, Quezon City, 1104, Philippines

Location

Uni Centrum Kliniczne

Gdansk, 80-219, Poland

Location

Specjal. Gabinet Neurologiczny

Krakow, 30-359, Poland

Location

Centrum Medyczne "POMOC"

Lodz, 93-271, Poland

Location

INTERMED, Lublin

Lublin, 20-058, Poland

Location

CRH ŻAGIEL MED, Lublin

Lublin, 20-601, Poland

Location

Neuro - Dzieci I Mlodziezy Aga

Warsaw, 02-315, Poland

Location

NZOZ Mazowieckie Centrum

Warsaw, 05-462, Poland

Location

Childrens Republic Hospital

Kazan', 420138, Russia

Location

Smolensk Regional Hospital- Regional Budget State Healthcare institution

Smolensk, 214018, Russia

Location

Tyumen Regional Hospital #2 - State Budget Healthcare Institution of Tyumen region

Tyumen, 625039, Russia

Location

Daegu Fatima Hospital

Daegu, 41199, South Korea

Location

National Health Insurance Service Ilsan Hospital

Goyang-si, 10444, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, 03722, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Siriraj Hospital, Mahidol University

Bangkok, 10700, Thailand

Location

Maharaj Nakorn Chiang Mai Hospital, Chiang Mai University

Chiang Mai, 50200, Thailand

Location

Srinagarind Hospital, Khon Kaen University

Khon Kaen, 40002, Thailand

Location

Ankara Diskapi Yildrim Beyazit

Ankara, 6110, Turkey (Türkiye)

Location

Kocaeli Üniversitesi

Kocaeli, 41380, Turkey (Türkiye)

Location

Related Links

• Additional information on study locations near you may be found at AllerganClinicalTrials.com. For any study not on AllerganClinicalTrials.com, please contact IR-CTRegistration@Allergan.com for assistance.

MeSH Terms

Conditions

Muscle Spasticity; Cerebral Palsy; Stroke

Interventions

Botulinum Toxins, Type A; Saline Solution

Condition Hierarchy (Ancestors)

Muscular Diseases; Musculoskeletal Diseases; Muscle Hypertonia; Neuromuscular Manifestations; Neurologic Manifestations; Nervous System Diseases; Signs and Symptoms; Pathological Conditions, Signs and Symptoms; Brain Damage, Chronic; Brain Diseases; Central Nervous System Diseases; Cerebrovascular Disorders; Vascular Diseases; Cardiovascular Diseases

Intervention Hierarchy (Ancestors)

Botulinum Toxins; Metalloendopeptidases; Endopeptidases; Peptide Hydrolases; Hydrolases; Enzymes; Enzymes and Coenzymes; Metalloproteases; Bacterial Proteins; Proteins; Amino Acids, Peptides, and Proteins; Bacterial Toxins; Toxins, Biological; Biological Factors; Crystalloid Solutions; Isotonic Solutions; Solutions; Pharmaceutical Preparations

Results Point of Contact

• Title: Therapeutic Area Head
• Organization: Allergan

IR-CTRegistration@Allergan.com

714-246-4500

Study Officials

• Rozalina Dimitrova

  Allergan

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 21, 2012
• First Posted: May 22, 2012
• Study Start: July 12, 2012
• Primary Completion: June 29, 2017
• Study Completion: July 6, 2017
• Last Updated: August 14, 2018
• Results First Posted: August 14, 2018

Record last verified: 2018-07

Locations

TH (3); KR (6); HU (1); PH (2); RU (3); US (20); PL (7); TU (2); CA (1)