NCT02853240: TOXIMUS_CP

Effect of Botulinum Toxin on Muscles of Children With Cerebral Palsy

Completed NA Last updated 18 January 2020

What this trial tests

NA trial testing Muscle biopsy in Cerebral Palsy in 20 participants. Completed in 10 January 2020.

Timeline

24 October 2016

Primary endpoint
10 January 2020

10 January 2020

Quick facts

Lead sponsor	Hospices Civils de Lyon
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	basic science
Enrollment	20
Start date	24 October 2016
Primary completion	10 January 2020
Estimated completion	10 January 2020
Sites	1 location across France

Drugs / interventions tested

• Muscle biopsy

Conditions studied

• Cerebral Palsy — all drugs for Cerebral Palsy →

Sponsor

Hospices Civils de Lyon — full company profile →

Who can join

Adults 8 to 17, any sex, with Cerebral Palsy. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Cerebral palsy (CP) is a group of non-progressive motor dysfunction but often changing, secondary to injury or brain abnormalities that occur in early stages of development. In children with CP, the brain injury lead to a delayed motor development in the first weeks, associated with muscular spasticity. Drug treatments include oral treatments (baclofen and tizanidine) and injectable treatments like Botox (intramuscular injection) and neurolysis with alcohol or phenol (local injection into the nerve).

Regarding botulinum toxin, there is no study questioning its effectiveness. However, no publication on the pathophysiology of human muscle of the CP child after toxin injection was found. The action of the toxin on the neuromuscular junction (NMJ) and muscle structure is unknown in children with CP.

The primary objective of this study is to describe structural abnormalities of the CP child's muscle following multiple toxin injections in terms of NMJ fragmentation and axonal sprouting.

Secondary objectives:

To evaluate the relationship between:

* The severity of the motor impairment and muscle structural abnormalities.
* The clinical measure of spasticity and muscle structural abnormalities.
* To compare the structure spastic muscles with toxin injections and spastic muscle without toxin injections

For muscles with multiple toxin injections, assessing the relationship between :

* The number of toxin injections and muscle structural abnormalities.
* The date of the first injection and muscle structural abnormalities.
* The total dose of injected toxin in the muscle and its structural abnormalities.
* The nature of the product injected in the muscle and its structural abnormalities.

This innovative study will improve the knowledge on the effects of long-term botulinum toxin injections on the muscle (and therefore its safety in usual care), on the spastic muscle NMJ of CP children, on the pathophysiology of the CP child's muscle.

All the visits all acts will be performed according to usual patient follow-up. Only a biopsy will be performed in addition, taken from an injected muscle during a planned operation. A biopsy may also be performed on a muscle without toxin injection if the act is made possible by the planned surgery. No biopsy will be made on a muscle that would not require surgery.

Publications & conference data

No peer-reviewed publications indexed yet for this trial. Completed trials usually publish results within 12-18 months.