Does Botulinum Toxin A Make Walking Easier in Children With Cerebral Palsy?

NCT02546999 | Completed Phase 4 DMC

• 2 other identifiers: 2013/11952014-002539-32
• Study Type: interventional
• Target: 61
• Locations: 3 countries
• Sites: 7

Brief Summary

In Norway, about 60% of all children with cerebral palsy (CP) are being treated with botulinum toxin A (BoNT-A) at 6 years of age, mainly in the legs. Despite this widespread use of the drug, the evidence for a positive effect on walking is insufficient. Moreover, large variation in effect is seen by clinicians. The main objective of the present study is to investigate whether injections with BoNT-A in the calf muscles make walking easier in children with spastic CP within 6 months, reflected by reduced energy cost during walking.

Conditions

Cerebral Palsy; Muscle Spasticity

Keywords

Botulinum Toxins, Type A; Walking

Outcome Measures

Primary Outcomes (1)

• Energy cost during walking

  Will be measured by a 5 minutes walk test (overground walking at comfortable speed) with simultaneous gas exchange.

  6 months

Secondary Outcomes (4)

• Activity

  6 months

• Perceived improved performance and satisfaction

  6 months

• Recurrent musculoskeletal pain

  6 months

• Walking capacity

  6 months

Other Outcomes (4)

• Gait pattern

  6 months

• Ankle strength

  6 months

• Spasticity

  6 months

Study Arms (2)

botox

EXPERIMENTAL

Botox® (onabotulinumtoxin A),injections in the calf muscles. The total maximum body dose of Botox® in this study will be 420 Units. Maximum dose per injection site will be 50 Units. The gastrocnemius muscle will receive 5-6 Units Botox® per kg, but maximum 180 Units in each leg. The soleus muscle will receive 2 Units Botox® per kg with maximum dose 60 Units in each leg. Dilution: 100 Units Botox® in 1 ml 0.9% sodium chloride, and the maximum volume per injection site will be 0,5 ml in both study groups. The route of administration is intramuscular injection.

Drug: botox

placebo

PLACEBO COMPARATOR

Sterile 0,9% Sodium Chloride injection The placebo dose will be the same dose in ml as the reconstituted Botox

Drug: placebo

Interventions

botox DRUG

The agent will be given only once at point zero in the time scheme for the project.

Also known as: botulinum toxin A

botox

placebo DRUG

The agent will be given only once at point zero in the time scheme for the project.

Also known as: sodium chloride

placebo

Eligibility Criteria

• Age: 4 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Diagnosed with unilateral or bilateral CP
• GMFCS level I and II
• Signed informed consent
• expected cooperation of the patients for the treatment and follow up.

You may not qualify if:

• BoNT-A injections in the lower legs in the last 6 months before intervention
• history of adverse reactions to BoNT-A
• Known hypersensitivity to BoNT-A or to any of the excipients
• Orthopedic surgery in the legs in the last 2 years
• Major cognitive impairments (must be able to take verbal instructions and conduct the test procedure)
• infection at the proposed injection site(s)
• Subclinical or clinical evidence of defective neuromuscular transmission e.g. myasthenia gravis or Lambert-Eaton Syndrome in patients with peripheral motor neuropathic diseases (e.g. amyotrophic lateral sclerosis or motor neuropathy)
• other underlying neurological disorders that may be affected by BoNT-A injections
• Use of aminoglycoside antibiotics or spectinomycin, or other medicinal products that interfere with neuromuscular transmission (e.g. neuromuscular blocking agents)
• Pregnant or breast-feeding
• Childbearing potential not using contraception
• any reason why, in the opinion of the investigator, the patient should not participate
• Children needing deep sedation under treatment

Sponsors & Collaborators

• St. Olavs Hospital: lead
• Norwegian University of Science and Technology: collaborator
• The Hospital of Vestfold: collaborator
• University Hospital of North Norway: collaborator
• Oslo University Hospital: collaborator
• Haukeland University Hospital: collaborator
• Fondation Lenval: collaborator
• Mazowieckie Centrum Neuropsychiatrii, Warszawa: collaborator

Study Sites (7)

Lenval Foundation Children's Hospital

Nice, France

Location

Haukeland University Hospital

Bergen, 5000, Norway

Location

Oslo University Hospital

Oslo, Norway

Location

University Hospital of North-Norway

Tromsø, Norway

Location

Department of Orthopaedic Surgery, St. Olavs University Hospital

Trondheim, Norway

Location

Vestfold Hospital trust

Tønsberg, Norway

Location

Mazowieckie Centrum Neuropsychiatrii, Zagorze

Warsaw, Poland

Location

Related Publications (2)

• Braendvik SM, Roeleveld K, Andersen GL, Raftemo AE, Ramstad K, Majkic-Tajsic J, Lamvik T, Lund B, Follestad T, Vik T. The WE-Study: does botulinum toxin A make walking easier in children with cerebral palsy?: Study protocol for a randomized controlled trial. Trials. 2017 Feb 6;18(1):58. doi: 10.1186/s13063-016-1772-8.

  PMID: 28166806 BACKGROUND

• Braendvik SM, Ross Raftemo AE, Roeleveld K, Andersen GL, Ramstad K, Follestad T, Aarli A, Bonikowski M, Vik T; Walking Easier. Does botulinum neurotoxin A make walking easier in children with cerebral palsy? A randomized clinical trial. Dev Med Child Neurol. 2025 Feb;67(2):263-271. doi: 10.1111/dmcn.16038. Epub 2024 Jul 26.

  PMID: 39058740 DERIVED

MeSH Terms

Conditions

Cerebral Palsy; Muscle Spasticity

Interventions

Botulinum Toxins, Type A; Sodium Chloride

Condition Hierarchy (Ancestors)

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

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; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Petter Aadahl, md prof

  St. Olavs Hospital

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 9, 2015
• First Posted: September 11, 2015
• Study Start: September 1, 2015
• Primary Completion: October 15, 2021
• Study Completion: October 15, 2021
• Last Updated: January 11, 2022

Record last verified: 2022-01

Locations

PL (1); FR (1); NO (5)