BOTOX® Treatment in Pediatric Lower Limb Spasticity

NCT01603628 | Completed Phase 3 Results DMC

• 2 other identifiers: 191622-1112012-000042-35
• Study Type: interventional
• Target: 384
• Locations: 9 countries
• Sites: 53

Brief Summary

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

Conditions

Pediatrics; Muscle Spasticity; Cerebral Palsy

Outcome Measures

Primary Outcomes (2)

• Average Change From Baseline in Modified Ashworth Scale-Bohannon (MAS-B) Ankle Score With Knee Extended 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 Mixed Model Repeated Measures (MMRM) model was used for analysis.

  Weeks 4 and 6

Secondary Outcomes (2)

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

  Weeks 8 and 12

• Change From Baseline in Severity of Spasticity of the Ankle With Knee Extended and Knee Flexed (R2-R1) Calculated Using the Modified Tardieu Scale (MTS)

  Baseline (Day 1) to Weeks 2, 4, 6, 8 and 12

Study Arms (3)

BOTOX® 4 U/kg

EXPERIMENTAL

Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 4 U per kg of body weight (4 U/kg) into specified muscles of the lower limb on Day 1. Participants received weekly physical therapy (PT).

Biological: botulinum toxin Type A

BOTOX® 8 U/kg

EXPERIMENTAL

Participants received intramuscular injections of BOTOX® (botulinum toxin Type A) 8 U per kg of body weight (8 U/kg) into specified muscles of the lower limb on Day 1. Participants received weekly PT.

Biological: botulinum toxin Type A

Normal Saline (Placebo)

PLACEBO COMPARATOR

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

Drug: Normal Saline (Placebo)

Interventions

botulinum toxin Type A BIOLOGICAL

Participants received intramuscular injections of botulinum toxin Type A into specified muscles of the lower limb on Day 1.

Also known as: BOTOX®

BOTOX® 4 U/kg; BOTOX® 8 U/kg

Normal Saline (Placebo) DRUG

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

Normal Saline (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
• Cerebral palsy with dynamic muscle contracture /spasticity of the ankle

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
• History of surgical intervention of the lower study leg or planned surgery of any 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 upper limb during the study

Sponsors & Collaborators

• Allergan: lead

Study Sites (53)

AMS Neurology

Pasadena, California, 91106, United States

Location

Harrison Clinical Management

Pomona, California, 91767, 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

New England Center for Clinical Research

Stamford, Connecticut, 06905, 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, 32819, United States

Location

Children's Healthcare of Atlanta Children's Rehabilitation Associates

Atlanta, Georgia, 30342, United States

Location

Rehab Institute of Chicago

Chicago, Illinois, 60611, United States

Location

The Children's Mercy Hospital & Clinics

Kansas City, Missouri, 64108, 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 of Charlotte, LLC

Charlotte, North Carolina, 28203, United States

Location

Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

University of Texas Southwestern Medical Center

Dallas, Texas, 75235, 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

Semmelweis Egyetem- Ortopédiai Klinika

Budapest, 1113, Hungary

Location

Debrecen University Clinical Center, Orthopedic Clinic

Debrecen, 4032, Hungary

Location

Istituto IRCCS G. Gaslini

Genoa, 16148, Italy

Location

Philippine Orthopedic Center

Quezon City, 1100, Philippines

Location

St. Luke's Medical Center-Quezon City

Quezon City, 1102, Philippines

Location

Philippine Children's Medical Center

Quezon City, 1104, Philippines

Location

Uniwersytecki Dzieciecy Szpita

Bialystok, 15-274, Poland

Location

Uni Centrum Kliniczne

Gdansk, 80-219, Poland

Location

Centrum Rehabilitacji Krok po Kroku

Gdansk, 80-389, Poland

Location

Specjal. Gabinet Neurologiczny

Krakow, 30-359, Poland

Location

INTERMED, Lublin

Lublin, 20-058, Poland

Location

CRH ŻAGIEL MED, Lublin

Lublin, 20-601, Poland

Location

Szpital Wojewódzki Nr 2

Rzeszów, 35-301, 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

Gyeonggi-do, 10444, South Korea

Location

Seoul National University Hospital

Seoul, 03080, South Korea

Location

Asan Medical Center

Seoul, 05505, South Korea

Location

Samsung Medical Center

Seoul, 06351, South Korea

Location

Severance Hospital, Yonsei University Health System

Seoul, 120-752, South Korea

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

Selçuk Üniversitesi

Konya, 42250, 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

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

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

clinicaltrials@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: September 11, 2012
• Primary Completion: June 28, 2017
• Study Completion: June 28, 2017
• Last Updated: August 14, 2018
• Results First Posted: August 14, 2018

Record last verified: 2018-07

Locations

TH (2); KR (6); PL (9); PH (3); HU (2); US (24); IT (1); RU (3); TU (3)