Double-blind Comparative Randomized Multicenter

Innopharm LLC80 enrolled

Overview

The name of the study: is a double-blind comparative randomized trial to evaluate the safety and efficacy of the drug Miotox® with the participation of children with cerebral palsy The code/number of the Protocol No. MTK-III-D-cerebral palsy-04/2019, version 5.0 dated 09/01/2021. Investigational drug Trade Name: Miotox®. Description: Transparent or slightly opalescent liquid from colorless to light yellow. Manufacturer: FSBI "M.P. Chumakov FNCRIP RAS", Russia Comparison drug: Trade Name: Botox® Description: dried white substance in the form of a barely noticeable film located at the bottom of the bottle. Manufacturer: Allergan Pharmaceuticals Ireland, Ireland. The studied indication for use is Focal spasticity associated with dynamic foot deformity of the "horse foot" type due to spasticity in patients 2 years and older with cerebral palsy who are on outpatient treatment Study design Double-blind comparative randomized multicenter The sponsor of the study is Innopharm LLC, Russia Clinical Development: Phase III The start date of the study is 10/15/2021. The date of completion of the study is 06/29/2022.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

SUPPORTIVE_CARE

Masking

TRIPLE

Enrollment

Conditions

Cerebral Palsy (CP)

Interventions

During the study, patients were injected with the investigational drug Miotox® . For the treatment of spasticity and equine varus deformity of the foot in children with cerebral palsy, drugs were injected into two points of each head of the calf muscle (medial and lateral). In case of damage to one limb, the total dose was 4 units / kg of body weight per affected limb. In diplegia, the total dose was 6 units / kg of body weight for both affected limbs. The total total dose should not exceed 200 units. This course of treatment is based on the current instructions for the medical use of Botox®.

BOTOX®DRUG

During the study, patients were injected with the investigational comparison drug Botox®. For the treatment of spasticity and equine varus deformity of the foot in children with cerebral palsy, drugs were injected into two points of each head of the calf muscle (medial and lateral). In case of damage to one limb, the total dose was 4 units / kg of body weight per affected limb. In diplegia, the total dose was 6 units / kg of body weight for both affected limbs. The total total dose should not exceed 200 units. This course of treatment is based on the current instructions for the medical use of Botox®.

Eligibility

Sex: ALLMin age: 2 YearsMax age: 12 Years

Medical Language ↔ Plain English

Inclusion Criteria: All patients were checked for compliance with the following inclusion criteria: * Written and dated Informed consent of the child's parent to participate in the clinical trial; The age of the child is from 2 to 12 years (2 years 0 months 0 days - 11 years 11 months 29 days); * Focal spasticity associated with dynamic foot deformity of the "horse foot" type due to spasticity in children with cerebral palsy undergoing outpatient treatment; * The degree of spasticity on the modified Ashworth scale (MAS) 2 or more; * Grade level on the scale of basic motor functions for cerebral palsy, extended and refined version (GMFCS - E\&R) I - III; * Children and their parents who are able to fulfill the requirements of the Protocol (i.e., fill out a self-observation diary, come to control visits). Exclusion Criteria: During the study, patients were excluded if any of the following criteria were present: * Withdrawal of Informed consent by the patient's parent; * Serious adverse events or adverse events that do not meet the criteria of severity, in the development of which, in the opinion of the researcher, further participation in the study may be detrimental to the health or well-being of the patient; * The need for procedures and/or drug treatment that are not permitted by the Protocol of this study; * The patient was included in violation of the criteria for inclusion/non-inclusion of the Protocol; * The appearance of non-inclusion criteria during the study; * The need for surgical intervention. * The patient's incompetence with the examination procedures. * Any condition of the patient that requires, in the reasonable opinion of the research physician, the withdrawal of the patient from the study; * The patient refuses to cooperate or is undisciplined (for example, the patient does not show up for a scheduled visit without warning the researcher and/or loss of communication with the patient); * The patient is out of observation; * Non-compliance by the patient with the established Protocol requirements, rules of stay at the clinical base; For administrative reasons (termination of the study by the Sponsor or regulatory authorities), as well as in case of gross violations of the Protocol that may affect the results of the study.

Locations (3)

SPb GBUZ "DGP No. 45 of the Nevsky district

Saint Petersburg, Russia

GBUZ SODKB named after N.N. Ivanova, Candidate of Medical Sciences;

Samara, Russia

Limited Liability Company "Santerra" (LLC "Santerra")

Stavropol, Russia

Outcomes

Primary Outcomes

Safety assessment

The frequency of adverse events during the study: adverse events of an immediate type (allergic reactions) that occur within 2 hours after administration of the drug under study and identified both by the researcher and according to information provided to the researcher by the patient's parent; adverse events (local and systemic reactions) that occur within 7 days after administration of the test drug and are identified according to information provided to the researcher by the patient's parent; other adverse events that occur 7 days after administration of the test drug (from 8 to 84 days after administration of the test drug, without taking into account the permissible interval of visits) and noted by the patient's parent in the Self-observation Diary.

Time frame: 14 weeks

Efficasy assessment

The change in the level of spasticity (decrease in score) on the Modified Ashworth Scale (MAS) scale at week 4 compared to the baseline level from 1 to 4 points.

Time frame: 14 weeks

Secondary Outcomes

Safety assessment

The frequency of serious adverse events during the study.

Time frame: 14 weeks

Safety assessment

The frequency of adverse events and serious adverse events, at least probably related to the study drug during the study.

Time frame: 14 weeks

Safety assessment

Cases of early termination of patients' participation in the study due to the development of NYA/NYA associated with the use of research drugs.

Time frame: 14 weeks

Efficasy assessment

Change in the level of spasticity on the Modified Ashworth Scale (MAS) scale (decrease in score) at 1, 8 and 12 weeks compared to the baseline level.

Time frame: 14 weeks

Efficasy assessment

Severity of pain on a 10-point Visual Analogue Scale (VAS) at 1, 4, 8 and 12 weeks compared to the baseline level (decrease in pain severity).

Time frame: 14 weeks

Efficasy assessment

The state of motor functions according to the scale of basic motor functions for cerebral palsy (GMFCS - E\&R) at 1, 4, 8 and 12 weeks compared with the baseline level.

Time frame: 14 weeks

Efficasy assessment

Measurement of spasticity on the Mental Toughness Scale (MTS )(decrease in score) at 1, 4, 8 and 12 weeks compared to the baseline level.

Time frame: 14 weeks