Double-blind Comparative Randomized Multicenter

A Double-blind Comparative Randomized Trial to Evaluate the Safety and Efficacy of the Drug Miotox® in Children with Cerebral Palsy

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 Overview

• Status: Completed
• Conditions: Cerebral Palsy (CP)
• Intervention / Treatment: Drug: Miotox (Botulinum toxin type A is a hemagglutinin complex); Drug: BOTOX®

• Study Type: Interventional
• Enrollment (Actual): 80
• Phase: Phase 3

Contacts and Locations

Study Locations

• Russian Federation
  • Saint Petersburg, Russian Federation
    • SPb GBUZ "DGP No. 45 of the Nevsky district
  • Samara, Russian Federation
    • GBUZ SODKB named after N.N. Ivanova, Candidate of Medical Sciences;
  • Stavropol, Russian Federation
    • Limited Liability Company "Santerra" (LLC "Santerra")

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Supportive Care
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Miotox®(Botulinum toxin type A is a hemagglutinin complex); Children of the younger age group from 2 to 12 years (2 years 0 months 0 days - 11 years 11 months 29 days) Group 1 - 40 children who were injected with the drug Miotox®;	Drug: Miotox (Botulinum toxin type A is a hemagglutinin complex); 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®.
Active Comparator: Botox (Botulinum toxin type A - hemagglutinin complex - 100 units); Children of the younger age group from 2 to 12 years (2 years 0 months 0 days - 11 years 11 months 29 days) Group 2 - 40 children who were injected with Botox®.	Drug: BOTOX®; 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®.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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.	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.	14 weeks

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Safety assessment	The frequency of serious adverse events during the study.	14 weeks
Safety assessment	The frequency of adverse events and serious adverse events, at least probably related to the study drug during the study.	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.	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.	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).	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.	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.	14 weeks

Collaborators and Investigators

• Sponsor: Innopharm LLC

Study record dates

Study Major Dates

• Study Start (Actual): October 15, 2021
• Primary Completion (Actual): May 15, 2022
• Study Completion (Actual): June 29, 2022

Study Registration Dates

• First Submitted: January 27, 2025
• First Submitted That Met QC Criteria: February 17, 2025
• First Posted (Actual): March 25, 2025

Study Record Updates

• Last Update Posted (Actual): March 25, 2025
• Last Update Submitted That Met QC Criteria: February 17, 2025
• Last Verified: February 1, 2025

More Information

Terms related to this study

• Keywords: botulinum toxin
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Brain Damage, Chronic; Cerebral Palsy; Immunologic Factors; Physiological Effects of Drugs; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Coagulants; Neurotransmitter Agents; Membrane Transport Modulators; Cholinergic Agents; Neuromuscular Agents; Acetylcholine Release Inhibitors; Agglutinins; Botulinum Toxins, Type A; abobotulinumtoxinA; Botulinum Toxins; Hemagglutinins
• Other Study ID Numbers: № MTK-III-D-DCP-04/2019

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No