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Efficacy And Safety Of Dysport In The Treatment Of Upper Limb Spasticity In Children (PUL)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02106351
Recruitment Status : Completed
First Posted : April 8, 2014
Results First Posted : December 24, 2019
Last Update Posted : December 24, 2019
Sponsor:
Information provided by (Responsible Party):
Ipsen

Brief Summary:
The purpose of this study is to assess the efficacy and safety of multiple doses of Dysport used in the treatment of upper limb spasticity (altered skeletal muscle performance) in children with cerebral palsy (CP).

Condition or disease Intervention/treatment Phase
Upper Limb Spasticity (Altered Skeletal Muscle Performance) in Children Biological: Botulinum toxin type A Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 212 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Phase III, Multicentre, Double Blind, Prospective, Randomised, Controlled, Multiple Treatment Study Assessing Efficacy And Safety Of DYSPORT® Used In The Treatment Of Upper Limb Spasticity In Children
Actual Study Start Date : April 2014
Actual Primary Completion Date : September 21, 2017
Actual Study Completion Date : September 4, 2018

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Botox

Arm Intervention/treatment
Experimental: Group A
Group A - Treatments 1, 2, 3 and 4: Dysport 16 Units (U)/kg in one upper extremity (the study limb).
Biological: Botulinum toxin type A
Subjects randomised to receive Dysport 2 U/kg, 8 U/kg or 16 U/kg administered intramuscularly in the study limb.
Other Name: AbobotulinumtoxinA (Dysport®)

Experimental: Group B
Group B - Treatments 1, 2, 3 and 4: Dysport 8 U/kg in one upper extremity (the study limb).
Biological: Botulinum toxin type A
Subjects randomised to receive Dysport 2 U/kg, 8 U/kg or 16 U/kg administered intramuscularly in the study limb.
Other Name: AbobotulinumtoxinA (Dysport®)

Experimental: Group C

Group C - Treatment 1: Dysport 2 U/kg in one upper extremity (the study limb).

Group C - Treatments 2, 3 and 4: Dysport 8 or 16 U/kg in one upper extremity (the study limb).

Biological: Botulinum toxin type A
Subjects randomised to receive Dysport 2 U/kg, 8 U/kg or 16 U/kg administered intramuscularly in the study limb.
Other Name: AbobotulinumtoxinA (Dysport®)




Primary Outcome Measures :
  1. Mean Change From Baseline to TC 1, Week 6 in MAS Score in the TC 1 PTMG [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Week 6. ]
    The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.


Secondary Outcome Measures :
  1. Mean Physician's Global Assessment (PGA) Score at TC 1, Week 6 [ Time Frame: TC 1, Week 6. ]
    The PGA of treatment response was assessed by asking the investigator the following question: 'How would you rate the response to treatment in the subject's upper limb since the start of the study?'. Answers were on a 9-point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved and +4: markedly improved). The mean scores for each treatment group at TC 1, Week 6 are presented.

  2. Mean Goal Attainment Scale (GAS) Total Score at TC 1, Week 6 [ Time Frame: TC 1, Week 6. ]
    The GAS is a functional 5-point scale used to measure progress towards individual therapy goals. At start of each TC, 1 to 3 individual goals were defined for each subject by investigator and child's parents/guardians/caregivers prior to treatment. Outcome to reach each goal was rated on a 5-point scale ranging from -2 to +2 (-2: much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, +1: somewhat more than expected outcome, +2: much more than expected outcome). Higher score indicates a better outcome. A GAS T-score was calculated as: 50+(10∑_(i=1)^n wi xi)/√(0.7∑_(i=1)^n wi^2 +0.3(∑_(i=1)^n wi)^2) where, xi = rating of ith goal post-baseline; wi = weight of ith goal, calculated as importance * difficulty as defined at baseline; n = number of goals assessed at baseline and post-baseline. A GAS T-score of 50 indicates goals achieved as expected. Scores below 50 reflect under attainment of goals and scores above 50 reflect over attainment of goals.


Other Outcome Measures:
  1. Mean Change From Baseline to TC 1 Week 16 in MAS Score in the TC 1 PTMG [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Week 16. ]
    The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone.

  2. Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Elbow Flexors of the Study Limb [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16. ]
    The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the elbow flexors.

  3. Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Wrist Flexors of the Study Limb [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16. ]
    The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the wrist flexors.

  4. Mean Change From Baseline to TC 1 Weeks 6 and 16 in MAS Score in the Finger Flexors of the Study Limb [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Weeks 6 and 16. ]
    The MAS was used to assess muscle tone in the upper limb PTMG and consists of 6 grades: 0 (no increase in muscle tone), 1 (slight increase in muscle tone, manifested by a catch and release or by minimal resistance at the end of the ROM) when the affected part is moved in flexion or extension, 1+ (slight increase in muscle tone, manifested by a catch, followed by minimal resistance throughout the remainder (less than half) of the ROM), 2 (more marked increase in muscle tone), 3 (considerable increase in muscle tone) or 4 (affected part(s) rigid in flexion or extension). The original score '+1' was given a derived numeric score of '2' and the higher numeric scores were incremented by 1 so that the MAS score range was from 0 to 5 with higher scores indicating greater muscle tone. A negative change from baseline indicates a decrease in muscle tone. Data is presented for subjects injected in the finger flexors.

  5. Mean PGA Score at TC 1 Week 16 [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Week 16. ]
    The PGA of treatment response was assessed by asking the investigator the following question: 'How would you rate the response to treatment in the subject's upper limb since the start of the study?'. Answers were on a 9-point rating scale (-4: markedly worse, -3: much worse, -2: worse, -1: slightly worse, 0: no change, +1: slightly improved, +2: improved, +3: much improved and +4: markedly improved). The mean scores for each treatment group at TC 1 Week 16 are presented.

  6. Mean GAS Total Score at TC 1, Week 16 [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Week 16. ]
    The GAS is a functional 5-point scale used to measure progress towards individual therapy goals. At start of each TC, 1 to 3 individual goals were defined for each subject by investigator and child's parents/guardians/caregivers prior to treatment. Outcome to reach each goal was rated on a 5-point scale ranging from -2 to +2 (-2: much less than expected outcome, -1: somewhat less than expected outcome, 0: expected outcome, +1: somewhat more than expected outcome, +2: much more than expected outcome). Higher score indicates a better outcome. A GAS T-score was calculated as: 50+(10∑_(i=1)^n wi xi)/√(0.7∑_(i=1)^n wi^2 +0.3(∑_(i=1)^n wi)^2) where, xi = rating of ith goal post-baseline; wi = weight of ith goal, calculated as importance * difficulty as defined at baseline; n = number of goals assessed at baseline and post-baseline. A GAS T-score of 50 indicates goals achieved as expected. Scores below 50 reflect under attainment of goals and scores above 50 reflect over attainment of goals.

  7. Mean Change From Baseline to TC 1, Week 16 in the Paediatric Quality of Life (PedsQL) Scores [ Time Frame: Baseline (TC 1, Day 1) and TC 1, Week 16. ]
    Parents/guardians completed questionnaires on their child's quality of life. The PedsQL parent inventory measured healthcare concepts for children/adolescents aged 2-18 years. The Generic Core Scales include physical, emotional, social and school aspects. The CP module was also completed. Scores were transformed on a scale from 0 to 100 with higher scores indicating a better quality of life. Mean changes from baseline to TC 1, Week 16 are presented for the General Core Scale and for the CP module. A positive change from baseline indicates an improvement in quality of life.



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Ages Eligible for Study:   2 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Upper limb spasticity due to cerebral palsy
  • Body weight 10 kg or over
  • MAS score of 2 or more in affected elbow or wrist flexors

Exclusion Criteria:

  • Fixed myocontracture
  • Previous phenol or alcohol injection within 1 year
  • Severe athetoid or dystonic movements
  • Previous or planned surgery for spasticity in elbow or wrist flexors
  • Neuromuscular disorders
  • Previous Rhizotomy within 6 months
  • Intrathecal baclofen within 30 days

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02106351


Locations
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United States, Colorado
Aurora, Colorado, United States
United States, District of Columbia
Washington, District of Columbia, United States
United States, Louisiana
New Orleans, Louisiana, United States
United States, Michigan
Royal Oak, Michigan, United States
United States, Minnesota
Saint Paul, Minnesota, United States
United States, Nebraska
Omaha, Nebraska, United States
United States, New Mexico
Albuquerque, New Mexico, United States
United States, New York
New York, New York, United States
United States, Ohio
Columbus, Ohio, United States
United States, Oregon
Portland, Oregon, United States
United States, Texas
Austin, Texas, United States
Dallas, Texas, United States
Belgium
Brussels, Belgium
Czechia
Brno, Czechia
Prague, Czechia
Israel
Be'er Sheva, Israel
Jerusalem, Israel
Petaẖ Tiqwa, Israel
Tel Aviv, Israel
Tel Hashomer, Israel
Mexico
La Paz, Baja California Sur, Mexico
Celaya, Mexico
Monterrey, Mexico
Poland
Gdansk, Poland
Poznan, Poland
Wiazowna, Poland
Spain
Barcelona, Spain
Terrassa, Spain
Turkey
Istanbul, Turkey
Izmir, Turkey
Kocaeli, Turkey
Sponsors and Collaborators
Ipsen
Investigators
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Study Director: Ipsen Medical Director Ipsen
  Study Documents (Full-Text)

Documents provided by Ipsen:
Study Protocol  [PDF] July 24, 2017
Statistical Analysis Plan  [PDF] October 19, 2018


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Ipsen
ClinicalTrials.gov Identifier: NCT02106351    
Other Study ID Numbers: Y-52-52120-153
2010-021817-22 ( EudraCT Number )
First Posted: April 8, 2014    Key Record Dates
Results First Posted: December 24, 2019
Last Update Posted: December 24, 2019
Last Verified: December 2019

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Muscle Spasticity
Muscular Diseases
Musculoskeletal Diseases
Muscle Hypertonia
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Signs and Symptoms
Botulinum Toxins
Botulinum Toxins, Type A
abobotulinumtoxinA
Acetylcholine Release Inhibitors
Membrane Transport Modulators
Molecular Mechanisms of Pharmacological Action
Cholinergic Agents
Neurotransmitter Agents
Physiological Effects of Drugs
Neuromuscular Agents
Peripheral Nervous System Agents