Phase 3, blinded, multicentre study assessing efficacy and safety of Dysport for treatment of upper limb spasticity (altered skeletal muscle performance) in childre

Phase 1

• Conditions: pper limb spasticity in children; MedDRA version: 20.0Level: LLTClassification code 10048970Term: Arm spasticitySystem Organ Class: 100000014461; Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

• Registration Number: EUCTR2010-021817-22-BE
• Lead Sponsor: IPSEN INNOVATIO

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2015-10-28
• Last Update Posted: 26 November 2018

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 210

Inclusion Criteria

(1) Signed informed consent obtained from the child’s parent(s)/guardian(s) and, if applicable, a signed assent from the child.
(2) Be from 2 to 17 years of age, inclusive.
(3) Body weight of 10 kg or more at the baseline visit.
(4) Have a diagnosis of CP, as defined by Rosenbaum.
(5) Have increased muscle tone/spasticity in at least one upper limb.
(6) Have a MAS score =2 in the upper limb PTMG (elbow flexors or wrist flexors) of the study limb (the limb to be injected in the first treatment) at the baseline visit.
(7) Be classified as Gross Motor Function Classification System Level 1 to 4.
(8) If undergoing therapy, such as physiotherapy, occupational therapy, or use of splints and/or orthoses, therapy must have been initiated at least 30 days prior to the baseline visit and agreed to continue the therapy throughout the study and at the minimum, up to Week 16 following the first treatment administration.

Are the trial subjects under 18? yes
Number of subjects for this age range: 210
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

(1) Fixed myocontracture in the PTMG (elbow flexors or wrist flexors) of the study limb defined as having an available range of motion angle of <40°, regardless of the starting and finishing angles, measured at the Tardieu Scale (TS) slow (XV1) speed at the baseline visit. (Available range for wrist flexors should be measured without holding the fingers and allowing free finger flexion).
(2) Subjects likely to be treated with BTX in the lower limb(s) and/or the non-study upper limb before Treatment 2 of the study.
(3) Inadequate washout from previous BTX injection of any serotype for any condition:
• Within 6 months prior to the baseline visit in the study limb,
or
• Within 3 months prior to the baseline visit in any other part of the body.
(4) Subjects who require BTX treatment in a single muscle group in the study limb in Treatment 1.
(5) Severe athetoid or dystonic movements in the study limb.
(6) Previous or planned surgery for spasticity in the PTMG(s) of the study limb.
(7) Previous injection of alcohol and/or phenol within 1 year prior to the baseline visit affecting the study limb wrist flexors and/or elbow flexors selected for injection in Treatment 1.
(8) Treatment with any drug that interferes either directly or indirectly with neuromuscular function (e.g. aminoglycoside antibiotics) or neuroblocking agents used during surgery (e.g. curare) within the last 30 days prior to study treatment.
(9) Are pregnant and/or lactating.
(10) Female subjects not willing to use contraceptive measures throughout the course of the study if postpubertal and sexually active.
(11) Inability or unwillingness to comply with the protocol.
(12) Subjects with any clinical (or sub-clinical) evidence of marked defective neuromuscular transmission (e.g. Lambert-Eaton syndrome or myasthenia gravis) or persistent clinically significant neuromuscular disorders.
(13) Known sensitivity to BTX or to any of the components in the formulation or allergy to cow’s milk protein.
(14) An infection at the injection site(s).
(15) Previous rhizotomy less than 6 months prior to the baseline visit or rhizotomy planned/anticipated during the course of the study.
(16) Subjects treated or likely to be treated with intrathecal baclofen within 30 days prior to the baseline visit or during the course of the study.
(17) Treatment with a new investigational drug within 30 days prior to the baseline visit or scheduled to receive such a drug during the course of the study.
(18) Subjects with a history of aspiration or conditions which put them at risk of aspiration, such as severe dysphagia.
(19) Concurrent or history of frequent lower respiratory tract infections, aspiration pneumonia, or, as judged by the Investigator, has compromised respiratory function.
(20) Any known medical condition, laboratory or diagnostic procedure finding, which might compromise compliance with the objectives and procedures of this protocol or preclude administration of botulinum toxin type A (BTX-A), as judged by the Investigator.
(21) Any uncontrolled clinically significant medical condition other than CP.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified