Dysport® Pediatric Lower Limb Spasticity Study

Study Type:	Interventional
Study Design:	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Conditions:	Cerebral Palsy; Muscle Spasticity; Children
Interventions:	Drug: Botulinum type A toxin (Dysport®); Drug: Placebo

  Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
Each centre that participated in the study was expected to randomise between five and 25 subjects. It was planned to have at least two participating centres per country.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Screening details: A total of 241 subjects were randomised to the study. The safety population included 239 subjects who were treated. Out of the 239 treated subjects, 4 subjects were excluded from the Intent-to-Treat (ITT) population because no MAS score was obtained at the baseline visit and/or at Week 4.

Reporting Groups

	Description
Dysport 10 U/Kg	10 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. Botulinum type A toxin (Dysport®): I.M. (in the muscle) injection on day 1 of a single treatment cycle.
Dysport 15 U/Kg	15 U/Kg per lower limb. Either one or both lower limbs can be treated. Total volume injected, 2ml per leg. Botulinum type A toxin (Dysport®): I.M. (in the muscle) injection on day 1 of a single treatment cycle.
Placebo	Total volume to be injected per lower limb - 2ml. Either one or both lower limbs can be treated. Placebo: I.M. injection on day 1 of a single treatment cycle.

Participant Flow for 2 periods

Period 1:   Randomised Population

	Dysport 10 U/Kg	Dysport 15 U/Kg	Placebo
STARTED	80	80	81
Treated [1]	80	80	79
COMPLETED	79	79	77
NOT COMPLETED	1	1	4
Study treatment not received	0	0	2
No MAS score at baseline and/or Week 4	1	1	2

[1]	Constitutes the safety population.

Period 2:   Treatment Phase - ITT Population

	Dysport 10 U/Kg	Dysport 15 U/Kg	Placebo
STARTED	79	79	77
COMPLETED	78	75	73
NOT COMPLETED	1	4	4
Not specified	0	0	1
Adverse Event	0	0	1
Withdrawal by Subject	1	3	1
Lost to Follow-up	0	1	1

  Baseline Characteristics

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Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
Baseline characteristic values were based on the ITT population, defined as all randomised subjects who received at least one injection of study treatment and who had a MAS score in the GSC assessed both at baseline and at Week 4.

Reporting Groups

	Description
Dysport 10 U/Kg	Dysport 10 U/Kg intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment.
Dysport 15 U/Kg	Dysport 15 U/Kg intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment.
Placebo	Placebo intramuscular injection on either one or both lower limbs (2 ml per leg), single treatment.
Total	Total of all reporting groups

Baseline Measures

	Dysport 10 U/Kg	Dysport 15 U/Kg	Placebo	Total
Overall Participants Analyzed; [Units: Participants]	79	79	77	235
Age; [Units: Years]; Mean (Standard Deviation)	6 (3.3)	5.7 (3.2)	5.9 (3.5)	5.9 (3.3)
Age, Customized; [Units: Participants]
Children (2-9 years)	67	67	65	199
Children (10-17 years)	12	12	12	36
Gender; [Units: Participants]; Count of Participants
Female	45 57.0%	48 60.8%	48 62.3%	141 60.0%
Male	34 43.0%	31 39.2%	29 37.7%	94 40.0%
Ethnicity (NIH/OMB); [Units: Participants]; Count of Participants
Hispanic or Latino	21 26.6%	21 26.6%	20 26.0%	62 26.4%
Not Hispanic or Latino	58 73.4%	58 73.4%	57 74.0%	173 73.6%
Unknown or Not Reported	0 0.0%	0 0.0%	0 0.0%	0 0.0%
Race/Ethnicity, Customized; [Units: Participants]
Black/African American	2	0	5	7
Caucasian/White	57	60	55	172
American Indian/Alaskan Native	1	0	0	1
Multiple	19	19	17	55
Botulinum toxin Status; [Units: Participants]
Naive	40	41	41	122
Non-naive	39	38	36	113
Number of legs being treated; [Units: Participants]
1 Leg injected	42	50	47	139
2 Legs injected	37	29	30	96
Gross Motor Function Classification System (GMFCS) [1]; [Units: Participants]
GMFCS level 1	46	45	40	131
GMFCS level 2	24	24	30	78
GMFCS level 3	9	10	7	26
[1] Participants were staged according to the following GMFCS criteria: level 1 (walks without limitations (best outcome)), level 2 (walks with limitations) and level 3 (walks using a hand held mobility device (worst outcome)). Only participants classified as GMFCS levels 1 to 3 were eligible for entry into the study.
Height; [Units: Cm]; Mean (Standard Deviation)	117.1 (20.7)	111.6 (18.5)	114.6 (19.7)	114.4 (19.7)
Weight; [Units: Kg]; Mean (Standard Deviation)	23.1 (13.4)	21.1 (10.7)	22.6 (11.9)	22.3 (12.0)
Body Mass Index (BMI); [Units: Kg/m2]; Mean (Standard Deviation)	15.8 (2.9)	16.1 (2.7)	16.2 (2.7)	16.0 (2.8)
Modified Ashworth Scale (MAS) score [1]; [Units: Units on a scale]; Mean (Standard Deviation)	3.1 (0.3)	3.1 (0.3)	3.2 (0.4)	3.1 (0.4)
[1] The MAS is a 6-point scale which measures the intensity of muscle tone by measuring the resistance of the muscle to passive lengthening or stretching. The Investigator graded muscle tone in the gastrocnemius-soleus complex (GSC) from 0 (no increase in tone) to 4 (affected parts rigid in flexion or extension).

  Outcome Measures

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1. Primary:

Change in MAS Score in the Gastrocnemius-soleus Complex (GSC) at the Ankle Joint of the (Most) Affected Lower Limb [ Time Frame: Change from baseline to Week 4 ]

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2. Secondary:

Physician’s Global Assessment (PGA) of the Treatment Response. [ Time Frame: Week 4 ]

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3. Secondary:

Goal Attainment Scale (GAS) Score [ Time Frame: Week 4 ]

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  Serious Adverse Events

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  Other Adverse Events

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  Limitations and Caveats

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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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  More Information

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Certain Agreements:  

Principal Investigators are NOT employed by the organization sponsoring the study.

There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact:  

Name/Title: Medical Director, Neurology
Organization: Ipsen
phone: clinical.trials@ipsen.com
e-mail: clinical.trials@ipsen.com

Responsible Party:	Ipsen
ClinicalTrials.gov Identifier:	NCT01249417 History of Changes
Other Study ID Numbers:	Y-55-52120-141; 2009-017709-12 ( EudraCT Number )
First Submitted:	November 25, 2010
First Posted:	November 29, 2010
Results First Submitted:	August 30, 2016
Results First Posted:	February 3, 2017
Last Update Posted:	February 3, 2017