Respiratory Training in Friedreich's Ataxia

NCT06539598 | Completed

• 1 other identifier: IRB202401099
• Study Type: interventional
• Target: 1
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Friedreich Ataxia

Outcome Measures

Primary Outcomes (7)

• Force Vital Capacity

  Forced Vital Capicity will be measured before and after the 12 week intervention

  Through study completion (4 months)

• Maximal Inspiratory Pressure (MIP)

  Maximal Inspiratory Pressure (MIP) will be measured before and after the 12 week intervention

  Through study completion (4 months)

• Maximal Expiratory Pressure (MEP)

  Maximal Expiratory Pressure (MEP) will be measured before and after the 12 week intervention

  Through study completion (4 months)

• Sniff Nasal Inspiratory Pressure (SNIP)

  Sniff Nasal Inspiratory Pressure (SNIP) will be measured before and after the 12 week intervention

  Through study completion (4 months)

• Swallowing

  Fiberoptic Endoscopic Evaluation of Swallowing (FEES) will be measured before and after the 12 week intervention.

  Through study completion (4 months)

• Surface Electromyography (sEMG)

  sEMG will be placed on the skin to measure muscle activity involved in breathing and swallowing

  Through study completion (4 months)

• Diaphragm Ultrasound

  Diaphragm movement will be measured through an ultrasound probe positioned on the skin before and after the 12 week intervention.

  Through study completion (4 months)

Study Arms (1)

Respiratory Strength Training (RST)

EXPERIMENTAL

Participants will receive a respiratory strength training device to take home and use up to 5 times a week for 12 weeks.

Other: Respiratory Strength Training (RST)

Interventions

Respiratory Strength Training (RST) OTHER

Respiratory Strength training involves inhaling/exhaling against a fix resistant to potentially increase respiratory strength

Respiratory Strength Training (RST)

Eligibility Criteria

• Age: 18 Years - 65 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• confirmed genetic testing of Friedreich's Ataxia
• have an overall DIGEST score of 1 or higher based on FEES screening
• are able to perform pulmonary function testing

You may not qualify if:

• have been on antibiotics within 15 days prior to baseline screening.
• have been prescribed systemic corticosteroids or neuromuscular blocking agents within 15 days of the baseline screening
• have an allergy or contraindication to topical lidocaine or oxymetazoline
• have any other concurrent medical condition which, in the opinion of the investigators, would make the subject inappropriate to participate in testing or respiratory exercise

Sponsors & Collaborators

• University of Florida: lead

Study Sites (1)

Clinical Research Center

Gainesville, Florida, 32610, United States

Location

MeSH Terms

Conditions

Friedreich Ataxia

Condition Hierarchy (Ancestors)

Spinocerebellar Degenerations; Cerebellar Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Spinal Cord Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Carmen Leon Astudillo, MD

  University of Florida

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 30, 2024
• First Posted: August 6, 2024
• Study Start: August 29, 2024
• Primary Completion: August 7, 2025
• Study Completion: August 7, 2025
• Last Updated: November 3, 2025

Record last verified: 2024-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)