Resistance Exercise in Barth Syndrome

NCT01629459 | Completed DMC

• 1 other identifier: 201205024
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

Barth syndrome (BTHS) is a disorder that is characterized by heart failure, exercise intolerance and skeletal muscle weakness. Preliminary evidence demonstrates that endurance exercise training does not significantly improve exercise tolerance in BTHS. Because endurance exercise training targets a metabolic pathway that is adversely affected by BTHS, the investigators hypothesized that resistance training may improve exercise tolerance in BTHS because this type of training targets a different metabolic pathway than does endurance exercise. Therefore, the overall objective of the pilot/feasibility/proof-of-concept proposal is to collect preliminary data on the following hypothesis: Supervised resistance exercise training (3x/wk, 45min, 12 wks) will improve exercise tolerance, heart function, muscle strength and quality of life, and will be found safe in adolescents and young adults with BTHS.

Conditions

Barth Syndrome

Keywords

exercise; strength; training; barth syndrome; mitochondria; amino acid; protein

Outcome Measures

Primary Outcomes (1)

• Change in exercise tolerance

  This study will examine the effect of resistance exercise training on exercise tolerance: peak oxygen consumption, exercise time and exercise work during graded exercise test on cycle ergometer

  Enrollment, 3 months

Secondary Outcomes (3)

• Change in muscle strength

  Enrollment, 3 months

• Change in quality of life

  Enrollment, 3 months

• Change in left ventricular systolic strain

  Enrollment, 3 months

Study Arms (1)

Resistance exercise training

EXPERIMENTAL

Participants will undergo resistance exercise training 3x/wk for 12 weeks at a physical therapy or cardiac rehabilitation facility near the participant's home.

Behavioral: Resistance exercise and protein supplementation

Interventions

Resistance exercise and protein supplementation BEHAVIORAL

Resistance exercise training will occur 3x/wk for 12 wks at a physical therapy or cardiac rehabilitation facility near the participant's home. Participant will take 42 grams of supplementation protein daily.

Resistance exercise training

Eligibility Criteria

• Age: 15 Years - 35 Years
• Sex: male
• Healthy Volunteers: Yes
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Age 15-35 years
• Sedentary (exercises less than 2x/wk)
• Motivated to exercise (BTHS only)
• Stable on medications for ≥ 3 months (BTHS only)
• Lives in North America
• Lives in the St. Louis area (Controls only)

You may not qualify if:

• Unstable heart disease
• Cardiac transplantation

Sponsors & Collaborators

• Washington University School of Medicine: lead

Study Sites (1)

Washington University

St Louis, Missouri, 63110, United States

Location

MeSH Terms

Conditions

Barth Syndrome; Motor Activity

Condition Hierarchy (Ancestors)

Heart Defects, Congenital; Cardiovascular Abnormalities; Cardiovascular Diseases; Heart Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Lipid Metabolism, Inborn Errors; Metabolism, Inborn Errors; Lipid Metabolism Disorders; Metabolic Diseases; Nutritional and Metabolic Diseases; Behavior

Study Officials

• William T Cade, PT, PhD

  Washington University School of Medicine

  PRINCIPAL INVESTIGATOR

Study Design

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

Model Details: Resistance exercise training

Study Record Dates

• First Submitted: June 20, 2012
• First Posted: June 27, 2012
• Study Start: June 1, 2012
• Primary Completion: June 30, 2019
• Study Completion: June 30, 2019
• Last Updated: May 13, 2020

Record last verified: 2020-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)