Trial Summary
What is the purpose of this trial?
This is a prospective interventional study of patients with Friedreich's Ataxia that receive respiratory strength training for a period of 12 weeks with two research visits at the beginning and at the end of the study period. Visits include swallowing evaluation with fiberoptic endoscopic evaluation of swallowing, pulmonary function testing, surface electromyography and patient surveys.
Do I need to stop my current medications for this trial?
The trial protocol does not specify if you need to stop your current medications. However, you cannot have taken antibiotics, systemic corticosteroids, or neuromuscular blocking agents within 15 days before the baseline screening.
What data supports the idea that Respiratory Training for Friedreich's Ataxia is an effective treatment?
The available research shows that Respiratory Training, also known as Respiratory Muscle Training, can improve the strength of breathing muscles in people with neurological disorders. For example, in patients with late-onset Pompe disease, a similar condition, this training increased the strength of muscles used for breathing by up to 74%. Although the research does not specifically mention Friedreich's Ataxia, these findings suggest that similar improvements might be possible for people with this condition. However, the effectiveness of this treatment for Friedreich's Ataxia specifically is not clearly established in the available research.12345
What safety data exists for respiratory muscle training?
The available research on respiratory muscle training (RMT) primarily focuses on its effectiveness rather than safety. Studies have shown improvements in respiratory muscle strength and pulmonary function in various conditions, such as spinal cord injury and neurological disorders. However, specific safety data is limited, and the effects on inspiratory muscle strength, respiratory muscle endurance, quality of life, exercise performance, and respiratory complications remain inconclusive. No significant adverse effects have been reported in the studies reviewed, but comprehensive safety evaluations are lacking.16789
Is Respiratory Strength Training a promising treatment for Friedreich's Ataxia?
Yes, Respiratory Strength Training is promising for Friedreich's Ataxia because it can help improve breathing and heart function, which are often affected by the disease. This type of training can enhance the body's ability to use oxygen and increase endurance, potentially leading to better overall health and quality of life for patients.1011121314
Eligibility Criteria
This trial is for individuals who can perform pulmonary function tests, have genetically confirmed Friedreich's Ataxia, and show a certain level of swallowing difficulty. It's not specified who cannot join the trial.Inclusion Criteria
My genetic test confirmed I have Friedreich's Ataxia.
My swallowing test score is 1 or higher.
I can do lung function tests.
Treatment Details
Interventions
- Respiratory Strength Training (Behavioural Intervention)
Trial OverviewThe study examines the effects of Respiratory Strength Training (RST) on patients with Friedreich's Ataxia over 12 weeks. Participants will undergo evaluations including swallowing assessments, lung function tests, muscle activity measurements, and surveys before and after training.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: Respiratory Strength Training (RST)Experimental Treatment1 Intervention
Participants will receive a respiratory strength training device to take home and use up to 5 times a week for 12 weeks.
Find a Clinic Near You
Research Locations NearbySelect from list below to view details:
Clinical Research CenterGainesville, FL
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Who Is Running the Clinical Trial?
University of FloridaLead Sponsor
References
Effects of respiratory muscle training on cough function in neurological disorders: A systematic review with meta-analysis. [2021]Patients with neurological disorders can present the weakness of respiratory muscle and impaired cough function. Previous studies have shown that respiratory muscle strength training (RMT) is an effective method of improving the strength of respiratory muscle. The effects of RMT on cough function remain controversial.
Respiratory muscle training for respiratory deficits in neurodegenerative disorders: a systematic review. [2018]Studies of the impact of respiratory muscle training (RMT) on central neurodegenerative pathologies have been aimed at improving pulmonary function. However, there is no certainty about the effectiveness of RMT in patients affected by these groups of disorders. The purpose of this review was to assess the evidence regarding the efficacy of inspiratory muscle training (IMT) and expiratory muscle training (EMT) on respiratory function in patients with neurodegenerative disorders of the CNS.
Increased inspiratory and expiratory muscle strength following respiratory muscle strength training (RMST) in two patients with late-onset Pompe disease. [2011]Respiratory muscle strength training (RMST) is an exercise-based intervention which targets respiratory muscle weakness. We implemented RMST in two patients with late-onset Pompe disease (LOPD), both who had received long-term enzyme replacement therapy and had severe respiratory weakness. Over 16-32 weeks, inspiratory muscle strength increased by 73-74%. Expiratory muscle strength increased 31-48% over 12-22 weeks. These findings suggest that RMST may increase respiratory muscle strength, even in the setting of LOPD and severe baseline weakness.
Respiratory strength training: concept and intervention outcomes. [2011]Respiratory muscle strength training (RMST) focuses on increasing the force-generating capacity of the inspiratory and expiratory muscles. The choice of respiratory muscles that are targeted using RMST depends on the outcome desired. For example, if an individual has reduced inspiratory muscle strength due to a neurogenic injury and is unable to ventilate the lungs, then inspiratory muscle strength training may be the chosen rehabilitation target. On the other hand, if a professional voice user is complaining of difficulty generating adequate vocal loudness during song production and is suffering from laryngeal dysfunction, then an expiratory muscle strength training paradigm may be the chosen rehabilitation target. Our most recent work with RMST has focused on increasing expiratory muscle force generation for those with Parkinson's disease who have difficulty with breathing, swallowing, and cough production. This difficulty typically worsens as the disease progresses. Highlights of these outcomes are summarized in this article.
Should respiratory muscle training be part of the treatment of Parkinson's disease? A systematic review of randomized controlled trials. [2022]To determine the effectiveness of respiratory muscle training in persons with Parkinson's disease.
Respiratory muscle training in persons with spinal cord injury: a systematic review. [2022]The purpose of this paper was to review the effectiveness of respiratory muscle training (RMT) on respiratory muscle strength and endurance, pulmonary function, quality of life, respiratory complications and exercise performance in persons with spinal cord injury. A MEDLINE (National Library of Medicine, Bethesda, MD, USA) database was used for selection of the literature (from 1980 to November 2004), and relevant references from peer-reviewed articles were retrieved as well. Studies investigating the effects of RMT (i.e. resistive breathing weight lifting or normocapnic hyperpnea) in persons with spinal cord injury were selected. Two independent reviewers investigated controlled studies for methodological quality by using a modification of the framework for methodological quality. Methodological quality ranged between 15 and 29 (maximal feasible score=40). Twenty-three papers were retrieved and six controlled trials were kept for further analysis. A meta-analysis and calculation of effect-size of each individual study and weighted summary effect-size was intended. However, unreported data and heterogeneity in outcome variables did not allow performing a meta-analysis. From the systematic review it is concluded that RMT tended to improve expiratory muscle strength, vital capacity and residual volume. Insufficient data was available to make conclusions concerning the effects on inspiratory muscle strength, respiratory muscle endurance, quality of life, exercise performance and respiratory complications.
Comprehensive Respiratory Muscle Training Improves Pulmonary Function and Respiratory Muscle Strength in Acute Stroke Patients. [2021]To evaluate the effects of respiratory muscle training (RMT) on pulmonary function, respiratory complications, and stroke-related disabilities in patients with acute stroke.
Respiratory muscle training in athletes with spinal cord injury. [2009]The effect of respiratory muscle endurance training (RMET) on RM function, dyspnoea and exercise performance was evaluated in SCI athletes. Nine endurance athletes (7 paraplegics T4-L1, 2 post-polio syndromes) were evaluated on three occasions (T1-T3), with a 1-month interval between evaluations. Participants performed between T1 and T2 their standard individual exercise training program (control), and between T2 and T3 the same program with 5 additional RMET sessions per week. Each evaluation included: lung function tests, RM strength and endurance tests, a maximal incremental arm cranking test and a field test (simulated competition). Ventilation and dyspnoea were evaluated during each exercise test. Lung function variables and maximal inspiratory strength were not modified (p>0.05) while maximal expiratory strength (+23+/-36 cm H2O; p
Training of respiratory muscles in patients with multiple sclerosis: a systematic review. [2018]The aim of this systematic review was to summarize the level of evidence and grades of recommendation regarding therapeutic respiratory muscle training interventions in patients with multiple sclerosis (MS).
Endurance exercise training in Friedreich ataxia. [2006]The purpose of this case study was to determine whether a patient with Friedreich ataxia (FA) would experience a clinically significant physiologic adaptation to aerobic endurance training. A 38-year-old man with FA underwent graded exercise testing with collection of expired gases on a bicycle ergometer before and after training, to determine maximum work capacity and oxygen consumption. Training consisted of 27 electrocardiographically monitored exercise sessions on the ergometer, each for 20 to 25 minutes at a workload adjusted to achieve an exercising heart rate equal to 70% to 85% of his pretest maximum, preceded and followed by a stretching routine. Large increases in cardiorespiratory and work measures demonstrated clinically important physiologic adaptations to aerobic conditioning in this patient. Peak VO2 increased 27% and peak ventilation increased 21%. Total exercise time increased five minutes, reflecting a 50-watt increase in maximum workload. In addition, the patient experienced a 4.75-kg weight loss. A medically supervised endurance training program can increase aerobic work capacity and promote weight loss in patients with FA who can pedal a bicycle at training level intensities.
Mitochondrial capacity, muscle endurance, and low energy in friedreich ataxia. [2022]In this study we noninvasively evaluated skeletal muscle mitochondrial capacity, muscle-specific endurance, and energy/fatigue feelings in persons with Friedreich ataxia (FRDA) and able-bodied controls (AB).
Regulation of respiration in Friedreich's ataxia. [2019]Friedreich's Ataxia (F.A.) is a degenerative disease which commonly leads to premature death of cardiorespiratory origin. To explain the early death of these patients, previous investigations have established the existence of 1) a cardiomyopathy in nearly 100% of cases, 2) a restrictive pulmonary syndrome of scoliotic origin and 3) a mild hypoxemia associated with slight respiratory alkalosis and a normal oxyhemoglobin dissociation curve. To further assess the cause of early death in patients with such neuromyopathy, we evaluated, in eleven F.A. patients, the sensitivity of the respiratory centers to hypercapnia, hypoxia, and hyperoxia. Ventilatory (VE, VT, F, VT/Ti) and occlusion pressure (P0.1) responses were taken as indices of the respiratory centers output during progressive hypercapnia (Read's method) and isocarbic hypoxia (Weil's method). We studied 11 Friedreich's Ataxia patients and 11 age, sex, and armspan matched controls. The responses of patients to hypercapnia were significantly greater than controls but their responses to hypoxia were similar to controls. Our study establishes that the respiratory centers are functioning adequately in early Friedreich's Ataxia and do not contribute to cardio-respiratory insufficiency in such neuromyopathy.
Safety and feasibility of upper limb cardiopulmonary exercise test in Friedreich ataxia. [2022]To explore the feasibility of upper limbs cardiopulmonary exercise test (CPET) in Friedreich ataxia (FRDA) patients and to compare the results with sex, age, and body mass index (BMI) matched cohort of healthy controls (HC).
In vivo assessment of OXPHOS capacity using 3 T CrCEST MRI in Friedreich's ataxia. [2022]Friedreich's ataxia (FRDA) is a neurodegenerative disease caused by decreased expression of frataxin, a protein involved in many cellular metabolic processes, including mitochondrial oxidative phosphorylation (OXPHOS). Our objective was to assess skeletal muscle oxidative metabolism in vivo in adults with FRDA as compared to adults without FRDA using chemical exchange saturation transfer (CrCEST) MRI, which measures free creatine (Cr) over time following an in-magnet plantar flexion exercise.