HIPOSMA: Hippotherapy in Children With Spinal Muscular Atrophy

Sponsor

Charles University, Czech Republic (Other)

Overall Status

Not yet recruiting

CT.gov ID

NCT05341453

Collaborator

(none)

Enrollment

Location

Arms

Anticipated Duration (Months)

0.5

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Spinal Muscular Atrophy	Procedure: Individual physiotherapy (SMA-SOC) Procedure: Hippotherapy Procedure: Therapeutic grooming	N/A

Detailed Description

Spinal muscular atrophy is rare neuromuscular disease, it is caused by gene mutation SMN1. This gene encodes 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 rehabilition 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. (16)

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. Lewelt et al. in their research they 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 we work primarily with muscles, and a lot of activity can cause fatigue of skeletal muscles and worsen their function. But from clinical work we 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 acitivities.

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, we expect a greater effect in the method of Hippotherapy in DMO, 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, we 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, we 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. We will therefore investigate the neurophysiological mechanisms of rehabilitation that we 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 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. (Woo C J et al.) 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.

Study Design

Study Type:

Interventional

Anticipated Enrollment :

16 participants

Allocation:

Randomized

Intervention Model:

Parallel Assignment

Intervention Model Description:

A randomized controlled trial. 16 children will be randomized into two interventional groups, on classic physiotherapy, the second hippotherapy. Participants both groups will undergo 12 therapies, twice a day, 15 minutes long. Before and in 6 day all children will be examined by a blinded independent clinician.A randomized controlled trial. 16 children will be randomized into two interventional groups, on classic physiotherapy, the second hippotherapy. Participants both groups will undergo 12 therapies, twice a day, 15 minutes long. Before and in 6 day all children will be examined by a blinded independent clinician.

Masking:

Single (Outcomes Assessor)

Masking Description:

All participants of the study will undergo a clinical examination including a questionnaire survey, led by a blinded independent examiner, who will not have access to intervention documentation. The examiner will see each patient twice, before the first intervention and 6 days later in the end last therapy. The examiner will be prohibited to talk about the therapy attended. The parents of patients will be informed about the character of the examination ant the importance of the most objectivity possible. All therapists can apply both interventions, therefore masking is more efficient.

Primary Purpose:

Supportive Care

Official Title:

The Effect of Intensive Hippotherapy on the Psychomotoric Development of Children With Spinal Muscular Atrophy

Anticipated Study Start Date :

Apr 22, 2022

Anticipated Primary Completion Date :

Oct 22, 2023

Anticipated Study Completion Date :

Oct 22, 2024

Arms and Interventions

Arm	Intervention/Treatment
Active Comparator: Individual physiotherapy (SMA-SOC) Individual physiotherapy will be performed according to the recommended SMA-SOC guidelines.	Procedure: 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.
Experimental: Hippotherapy by the children with cerebral palsy Hippotherapy by the children with cerebral palsy is an accredited form of hipotherapy, whose methodology is based on the clinical picture of cereberal palsy, but its procedures are applicable to a wider group of children with disabilities. Therefore, we assume its effect for children with SMA.	Procedure: Hippotherapy HT by CP is an accredited form of hippotherapy, whose methodology is based on the clinical picture of cereberal palsy, but its procedures are applicable to a wider group of children with disabilities. Therefore, we 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. Other Names: Hipotherapy by the chidlren with cereberal palsy (HT by CP)
Other: 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.	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.

Arm

Intervention/Treatment

Active Comparator: Individual physiotherapy (SMA-SOC)

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

Procedure: 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.

Experimental: Hippotherapy by the children with cerebral palsy

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

Procedure: Hippotherapy

HT by CP is an accredited form of hippotherapy, whose methodology is based on the clinical picture of cereberal palsy, but its procedures are applicable to a wider group of children with disabilities. Therefore, we 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.

Other Names:

Hipotherapy by the chidlren with cereberal palsy (HT by CP)

Other: 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.

Procedure: Therapeutic grooming

Outcome Measures

Primary Outcome Measures

The ability of the thorax to change shape [15 minutes]
The examination will take place lying on your back. 3 position markers will be placed on each side of the thorax, in the area of the distal end of the sternum axes, at the level of the 3rd rib and at the lowest arch of the 10th rib. Their position during exhalation and inspiration will be measured, and the change in thorax circumference will be evaluated. The aim of this test is to objectify the change in respiratory stereotype. After therapy, we expect a greater ability of the thorax to change direction.
Sitting symmetry [5 minues]
Observations will be made by sitting on a therapy cylinder. There will be special markers on the body (on clothes) of children: lower angles of the scapulae axes, ridge of the iliac axes, sacroiliac joint (SI) on both sides and in the area of the L4 vertebra and navel. We will measure the symmetry of these marks in each other's position and the movement in which the child will walk back and forth with his hands. Markers will be analyzed using Qualisys Motion Capture Systems 2020.3. Acceleration and change of position will be evaluated in the second time.
Work with the center of gravity [10 minutes]
Observation will be performed by sitting on therapeutic cylinder. 3 position markers will be used on each side of the body, at the root of the palm, in the area of the SI joint and at the level of the temporomandibular joint. The change of markers will be measured from the starting position until the child is stretched to the side (torso tilt with lug) and forward (torso flexion with forearm). The monitoring child will be motivated to reach for the toy. The goal is to evaluate the stability of children. We assume, that after therapy we can see better work with the center of gravity.
Muscle fatigue [10 minutes]
We will measure muscle fatigue after physical activity using surface EMG. The child will lie on his back, therapist will provide a toy in the area of his opposite knee. The child will stretch for a toy. This act will be 30 s. We will evaluate activity m.obliquus externus abdominis at the beginning and end of this specific activity. Goal is to evaluate muscle fatigue. We assume, that after intensive therapeutic program will be muscle fatigue lower.
Hammersmith function scale for children with spinal muscular atrophy [40 minutes]
The examination will follow a standardized procedure.
Deep neck flexor endurance test [5 minutes]
The examination will follow a standardized procedure.
Neck extensor endurance test [5 minutes]
The examination will follow a standardized procedure.
Spirometric measure [2 minutes]
The spirometer will be used to determine the forced expiratory volume in 1 second FEV1 value, i.e the volume of air exhaled after the maximum inspiration with the greatest effort within 1 second. The change in value at the beginning and end of the rehabilitation stay will be evaluated.

Secondary Outcome Measures

Quality of life assessment [120 minutes]
Parents of child with SMA will fill special questionnaire at the beginning and 4-5 weeks after end of the rehabilitation. The ICF score set, more precisely the ICF-based Documentation Form, the category Children with cereberal palsy Brief (below 6 years old),supplemented by some items from the basic rehabilitation set, will be used to evaluate the quality of life.
Monitoring of molecular biological indicators of rehabilitation [10 minutes]
Samples treated in this way can be safely stored at room temperature for longer periods of time (up to three days or more). Blood samples stored in RNAlater® Solution achieve RNA quality comparable to the quality of samples processed immediately according to commercial websites. The expected average total RNA yields will be about 2-4 /g / 0.5 ml all blood. Total RNA will be transcribed into cDNA by reverse transcriptase. To avoid error, the expression of human lncRNA and internal endogenous gene (e.g., GAPDH) will be quantified using RNA obtained from blinded samples (i.e., concealment of sample origin allocation).