Hippotherapy in Children With Spinal Muscular Atrophy

NCT ID: NCT05341453

Last Updated: 2024-08-20

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: NA
• Total Enrollment: 16 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-04-22
• Study Completion Date: 2025-10-22

Brief Summary

The randomized controlled trial is aimed to discover the physiotherapy and hippotherapy effect and efficacy on children with SMA. The concept is to utilized two types of physiotherapy - the first concept is classic physiotherapy and the second one is hippotherapy. The hippotherapy concept will be in intervals of 15 minutes twice a day, the physiotherapy will be in intervals of 30 minutes once a day. In-patient therapy will be for 6 days. The efficacy will be assessed by biomedical measures - Qualisys Motion Capture Systems 2020.3., by molecular biological markers (lncRNA) in blood and by surface electromyography (EMG). The primary goal of this study is to compare two physiotherapeutic approaches - the recommended form of classical physiotherapy and the method on a neurophysiological basis - hippotherapy. The secondary intention of the research will be the appropriate intensity of therapy so that unwanted muscle fatigue does not occur.

Detailed Description

Spinal muscular atrophy is rare neuromuscular disease, it is caused by gene mutation survival motor neuron 1 (SMN1). This gene encodes survival motor neuron(SMN) protein. This protein is most important to survive alfa-motoric neurons in spinal cord. When this protein doesn´t work, no signals go from brain to skeletal muscles and fazic muscles of body. In the end weakening breathing muscles and internal organs muscles. The first treatment is pharmacological treatment. There is a lot of research about medicament treatment, the famous treatment in Czech Republic is medicine Spinraze and from year 2020 medicine Zongelsma. This, however, more often puts more difficulty on complex rehabilitation. An empirical observation has found out that complex rehabilitation has positive effect on progress of disease and on self-sufficiency of people and children with SMA. Physiotherapy is one of the recommended rehabilitation types.

The use of rehabilitation procedures is common in clinical practice. In a 2016 study, 105 adults with SMA were asked how many of them used physiotherapy. A total of 86% of respondents received these services, yet the authors of the article conclude that further research is needed to understand the impact of physiotherapy in SMA.

Nevertheless, there is a lot of discussion if physiotherapy is really good for people and children with SMA. Some research shows us, that physiotherapy doesn´t worsen this disease. Researchers showed that strength training 3 times a week does not show side effects in children under 10 years of age, in some cases trends in improving muscle strength and motor functions have been observed.

Because in physiotherapy therapists work primarily with muscles, and a lot of activity can cause fatigue of skeletal muscles and worsen their function. But from clinical work scientists know that the effect of physiotherapy is extensive. There is a research on mice which shows the effect of physiotherapy on the strength activities and the aerobic activities.

Research to better elucidate the intensity of physiotherapy has not yet been published. Motes et al. monitored a total of 16 patients with SMA aged 10 - 55 years, who regularly underwent home autotherapy for 16 months - cycling and strengthening. The authors did not find any side effects of exercise on strength, function or fatigue during the study. However, the oxidation capacity has been reduced and according to the authors, further research is needed in this area.

In the United States, the "Standard of Care" (SMA-SOC) has been developed for SMA, which regulates the rehabilitation care of children and adults with SMA according to the degree of mobility. In the framework of the said directive, the stretching of contractures, swimming, water therapy, light strength exercises and a verticalization stand are recommended within the framework of physiotherapy, and horse riding is also mentioned - but without more detailed information. Vibration techniques and positioning are recommended to improve breathing. Recommended exercises for seated persons also include concentric and eccentric exercises and aerobic and general fitness exercises with and without resistance. According to the directive, the duration of therapy should be adapted to the individual patient. For walkers, the exercise program may include some forms of balance exercises, dynamic and static.

In a study from 2021, physiotherapists were asked about their knowledge of SMA-SOC and its use in practice. Most of them stated that after individuals with SMA started disease-modifying pharmacotherapy, they would recommend increasing the frequency and duration of therapeutic interventions. The authors agree that in practice there are differences in the care of people with SMA, especially in the frequency and duration of specific interventions. At the same time, they state that these findings can identify future research needs and thus extend SMA-SOC to rehabilitation management best practices.

Our goal is to compare two physiotherapeutic approaches - the recommended form of classical physiotherapy and the method on a neurophysiological basis - hippotherapy. Due to the nature of the disease, investigators expect a greater effect in the method of Hippotherapy in cerebral palsy (CP), where several muscle chains work simultaneously and thus succeeds in activating weakened muscles within the chain, which eliminates possible fatigue and worsening of the condition. However, investigators also expect a positive effect in the recommended form of classical physiotherapy. Here, however, due to the lower intensity of the sensory environment and the possible complexity of movement, investigators assume less effectiveness than hippotherapy.

The secondary intention of the research will be the appropriate intensity of therapy so that unwanted muscle fatigue does not occur. Investigators will therefore investigate the neurophysiological mechanisms of rehabilitation that investigators will monitor using long non-coding RNAs (lncRNAs) involved in important cellular regulation, including regulation of genomic imprinting, epigenetic chromatin modification, transcriptional interference, and nuclear export. The lncRNA, SMN-antisense 1 (SMN-AS1), has been shown to repress the expression of the duplicate survival motor neuron 2 (SMN2) gene by binding Polycomb repressive complex 2 (PRC2) to its locus. Increasing the transcriptional activity of an almost identical SMN2 gene can functionally compensate for the loss of SMN1, increase the amount of SMN protein and thus improve the prognosis of the disease. Chemically modified oligonucleotides have been proposed that disrupt the interaction between SMN-AS1 and PRC2, which inhibits the action of PRC2 and thus leads to increased SMN2 expression. Investigation of lncRNAs involved in the processes of gene regulation of these genes may help to understand the neurophysiological effects of rehabilitation of children with SMA.

Conditions

Spinal Muscular Atrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: SUPPORTIVE_CARE
• Blinding Strategy: SINGLE

Study Groups

Individual physiotherapy (SMA-SOC)

Individual physiotherapy will be performed according to the recommended SMA-SOC guidelines.

Group Type: ACTIVE_COMPARATOR

Individual physiotherapy (SMA-SOC)

Intervention Type: PROCEDURE

Therapeutic procedures according to the recommended standard (SMA-SOC) will be used in the therapy. Elements of breathing gymnastics, vibration techniques, stretching, eccentric and concentric exercise procedures will be combined. Individual physiotherapy will be conducted on the basis of set goals from the kinesiological examination, it will be adapted to the individuality of the child - his cooperation, fatigue and sleep time.

Hippotherapy by the children with cerebral palsy

Hippotherapy by the children with cerebral palsy is an accredited form of hippotherapy, whose methodology is based on the clinical picture of cerebral palsy, but its procedures are applicable to a wider group of children with disabilities. Therefore, investigators assume its effect for children with SMA.

Group Type: EXPERIMENTAL

Hippotherapy

Intervention Type: PROCEDURE

HT by CP is an accredited form of hippotherapy, whose methodology is based on the clinical picture of cerebral palsy, but its procedures are applicable to a wider group of children with disabilities. Therefore, investigators anticipate it´s effect for children with SMA.

Based on a special type of examination, this method precisely determines the "type / breed" of the horse according to the biomechanics of the movement of its back in the step, the position of the child and the neurophysiological function of the back - differentiation or sensorimotor skills. Furthermore, according to the course of therapy, the physiotherapist determines the pace of the horse's step, its length, selects special manual contacts according to the current situation and adjusts the duration of therapy to the client's fatigue.

Therapy will be performed by therapists with professional competence to perform hippotherapy by children with CP.

Therapeutic grooming

In order to influence the psychomotor development in a comprehensive way, the psychosocial activity of therapeutic grooming will also be included in the study. Its goal is to support children's communication, their interaction with the environment, the ability to establish contact with the horse and the overall emotional support of children with SMA.

Group Type: OTHER

Therapeutic grooming

Intervention Type: PROCEDURE

In order to influence the psychomotor development in a comprehensive way, the psychosocial activity of therapeutic grooming will also be included in the study. Its goal is to support children's communication, their interaction with the environment, the ability to establish contact with the horse and the overall emotional support of children with SMA. The purpose of this activity is to clean the horse, its possible guide, stroking and contact with the horse, work from the ground.

Interventions

Individual physiotherapy (SMA-SOC)

Therapeutic procedures according to the recommended standard (SMA-SOC) will be used in the therapy. Elements of breathing gymnastics, vibration techniques, stretching, eccentric and concentric exercise procedures will be combined. Individual physiotherapy will be conducted on the basis of set goals from the kinesiological examination, it will be adapted to the individuality of the child - his cooperation, fatigue and sleep time.

Intervention Type: PROCEDURE

Hippotherapy

HT by CP is an accredited form of hippotherapy, whose methodology is based on the clinical picture of cerebral palsy, but its procedures are applicable to a wider group of children with disabilities. Therefore, investigators anticipate it´s effect for children with SMA.

Based on a special type of examination, this method precisely determines the "type / breed" of the horse according to the biomechanics of the movement of its back in the step, the position of the child and the neurophysiological function of the back - differentiation or sensorimotor skills. Furthermore, according to the course of therapy, the physiotherapist determines the pace of the horse's step, its length, selects special manual contacts according to the current situation and adjusts the duration of therapy to the client's fatigue.

Therapy will be performed by therapists with professional competence to perform hippotherapy by children with CP.

Intervention Type: PROCEDURE

Therapeutic grooming

In order to influence the psychomotor development in a comprehensive way, the psychosocial activity of therapeutic grooming will also be included in the study. Its goal is to support children's communication, their interaction with the environment, the ability to establish contact with the horse and the overall emotional support of children with SMA. The purpose of this activity is to clean the horse, its possible guide, stroking and contact with the horse, work from the ground.

Intervention Type: PROCEDURE

Other Intervention Names

Hippotherapy by the children with cerebral palsy (HT by CP)

Eligibility Criteria

Inclusion Criteria

• clinic diagnosis SMA (I, II, and III types)
• no changing medicine 6 months at least
• without another grave diseases

Exclusion Criteria

• hip subluxation
• allergies to horses and the environment of horse stables
• insurmountable fear of the horse

• Minimum Eligible Age: 1 Year
• Maximum Eligible Age: 10 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Charles University, Czech Republic

OTHER

Sponsor Role: lead

Responsible Party

Kamila Řasová

assoc. prof. PhDr. Kamila Řasová, Ph.D.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Kamila Rasova, as.prof.Dr.

Role: STUDY_DIRECTOR

Clinic of rheumatology and rehabilitation, Third medical faculty CU and Faculty Thomayer Hospital

Locations

Kamila Řasová

Prague, , Czechia

Site Status: RECRUITING

Countries

Czechia

Central Contacts

Kamila Rasova, as.prof.Dr.

Role: CONTACT

kamila.rasova@lf3.cuni.cz

+420604511416

Kateřina Marikova, Mgr.

Role: CONTACT

katerina.marikova@chmirakl.cz

+420607616104

Facility Contacts

Kamila Řasová, Ph.D.

Role: primary

kamila.rasova@gmail.com

+420604511416

Natalia Hrušková

Role: backup

natalia0087@gmail.com

+420702836959

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Study Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Document Type: Informed Consent Form

View Document

Other Identifiers

18032022

Identifier Type: -

Identifier Source: org_study_id