Effect of Dysport Injections on Energy Expenditure and Walking Efficiency in Children With Cerebral Palsy

NCT03469999 | Completed Phase 3 Results FDA Drug

• 1 other identifier: AAAR1322
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to evaluate the efficacy of single event multi level chemoneurolysis with the abobotulinumtoxin A drug, Dysport®, on walking energy expenditure and gait in children with spastic diplegia cerebral palsy.

Conditions

Cerebral Palsy

Keywords

Botulinum toxin A; Walking efficiency; Spasticity; Dysport; Pediatric

Outcome Measures

Primary Outcomes (1)

• Oxygen Consumption (ml/kg/Min)

  Energy expenditure (oxygen consumption (VO2)) will be measured during the 6 minute walk test using a portable metabolic cart. The participants will use a 10 meter walkway to walk back and forth during the 6 minutes to coverage as much distance as possible. During this time they will be wearing a portable spirometric device that collects the oxygen consumption (VO2) per minute.

  Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Secondary Outcomes (4)

• Gait Velocity (cm/s)

  Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

• Muscle Spasticity Using MAS

  Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

• Muscle Spasticity Using MTS

  Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

• Score on the Cerebral Palsy Quality of Life (CP QOL) Questionnaire

  Baseline (1 week prior to injection), 4 weeks post injection, 12 weeks post injection

Study Arms (1)

Dysport Injectable Product

EXPERIMENTAL

All participants will participate in baseline data collection of energy expenditure, gait analysis, and lower limb spasticity assessment. All participants will receive single event multi level chemoneurolysis with Dysport and will have repeat data collection at 4 weeks and 12 weeks post injection.

Drug: Dysport Injectable Product

Interventions

Dysport Injectable Product DRUG

Selected dose of medication will be determined by affected muscle(s), severity of spasticity, and the patient's body weight. We will follow the recommended total Dysport dose of 10-15 units/kg per limb, not to exceed 15 units/kg for unilateral lower limb, 30 units/kg for bilateral lower limb, or a total of 1000 units, whichever is lower in a given session.

Also known as: Botulinum toxin A

Dysport Injectable Product

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Age: 5-17 years. Must be \<18 prior to injection.
• \>10 kilograms at screening and injection visits
• Diagnosis of spastic diplegia or mild- to moderate spastic quadriplegia Cerebral Palsy
• Gross Motor Function Classification System level: I, II, III
• Ability to ambulate independently without aid, equinus gait
• Absent of joint or bone deformities
• Eligible to receive single-event multi-level chemoneurolysis (SEMLC)
• Cooperative and tolerant to testing procedures during clinic screening
• Presence of spasticity in one or both legs
• Be on a stable dose and regimen if on any prescribed medication/s
• Parent must have signed written informed consent and the Patient Authorization for Use and Release of Health and Research Study Information

You may not qualify if:

• Ankle contractures no more than -10 degrees with the knee extended
• Hemiplegia
• Wheelchair dependent
• Received Botulinum toxin within previous 4 months
• Uncontrolled epilepsy or certain types of seizures
• Fracture in the study limb within previous 12 months
• Infection or skin disorder at planned injection site
• Shortness of breath or other respiratory issues
• Uncontrolled clinically significant medical condition
• Received phenol or alcohol block in the study limb within previous 6 months
• Surgery in the study limb within previous 12 months
• Serial casting within previous 12 months
• New physiotherapy and/or orthotic regimen \<1 month before study start. (physiotherapy and/or orthotic regimen will be permitted if it began \>1 month before study start and maintained throughout study)

Sponsors & Collaborators

• Columbia University: lead

Study Sites (1)

Columbia University Irving Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

Cerebral Palsy; Muscle Spasticity

Interventions

Botulinum Toxins, Type A

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

Results Point of Contact

• Title: Dr. Heakyung Kim
• Organization: Columbia University Irving Medical Center

hk2641@cumc.columbia.edu

212-305-9416

Study Officials

• Heakyung Kim, MD

  Columbia University Medical Center- Department of Rehabilitation

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 24, 2018
• First Posted: March 19, 2018
• Study Start: December 12, 2018
• Primary Completion: May 26, 2021
• Study Completion: May 26, 2021
• Last Updated: June 1, 2022
• Results First Posted: June 1, 2022

Record last verified: 2022-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)