NCT01194141

Brief Summary

Barth syndrome (BTHS) is a genetic disease that results in heart failure, muscle weakness and exercise intolerance. Several studies in non-BTHS heart failure suggest that endurance exercise training is beneficial in improving exercise intolerance, heart function and quality of life in young men with BTHS. This study will examine the effects of Endurance (i.e. aerobic) exercise training on exercise tolerance, heart function, and quality of life in adolescents and young adults with BTHS. We hypothesize that 3 months of endurance training will improve exercise tolerance, heart function and quality of life in adolescents and young men with BTHS.

Trial Health

100
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
4

participants targeted

Target at below P25 for not_applicable

Timeline
Completed

Started Jul 2010

Longer than P75 for not_applicable

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

July 1, 2010

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

August 30, 2010

Completed
3 days until next milestone

First Posted

Study publicly available on registry

September 2, 2010

Completed
3.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2013

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2013

Completed
Last Updated

January 10, 2017

Status Verified

January 1, 2017

Enrollment Period

3.4 years

First QC Date

August 30, 2010

Last Update Submit

January 6, 2017

Conditions

Keywords

Barth syndromecardiomyopathymitochondriacardiolipin

Outcome Measures

Primary Outcomes (1)

  • Peak oxygen consumption

    peak oxygen consumption measured by indirect calorimetry

    Enrollment and 3 months

Secondary Outcomes (2)

  • Cardiac output

    Enrollment and 3 months

  • muscle oxygen extraction

    Enrollment and 3 months

Study Arms (1)

Exercise training

EXPERIMENTAL

Aerobic exercise training 45-60 min/3x/week/12 weeks

Behavioral: Exercise training

Interventions

aerobic exercise training, 45-60 minutes, 3x/week, 12 weeks (3-months)

Exercise training

Eligibility Criteria

Age15 Years - 30 Years
Sexmale
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Age 15-30 years
  • Sedentary (exercises less than 2x/wk)
  • Motivated to exercise
  • Stable on medications for ≥ 3 months
  • Lives in North America.
  • Planning on attending the Barth Syndrome International Conference in July 2010.

You may not qualify if:

  • Unstable heart disease
  • Any concurrent disease that may contraindicate exercise testing and training.
  • Cardiac transplantation

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Related Publications (1)

  • Cade WT, Reeds DN, Peterson LR, Bohnert KL, Tinius RA, Benni PB, Byrne BJ, Taylor CL. Endurance Exercise Training in Young Adults with Barth Syndrome: A Pilot Study. JIMD Rep. 2017;32:15-24. doi: 10.1007/8904_2016_553. Epub 2016 Jun 11.

Related Links

MeSH Terms

Conditions

Barth SyndromeCardiomyopathies

Interventions

Exercise

Condition Hierarchy (Ancestors)

Heart Defects, CongenitalCardiovascular AbnormalitiesCardiovascular DiseasesHeart DiseasesAbnormalities, MultipleCongenital AbnormalitiesCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesGenetic Diseases, X-LinkedGenetic Diseases, InbornLipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Motor ActivityMovementMusculoskeletal Physiological PhenomenaMusculoskeletal and Neural Physiological Phenomena

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
PRINCIPAL INVESTIGATOR
PI Title
Assisant Professor of Physical Therapy and Medicine

Study Record Dates

First Submitted

August 30, 2010

First Posted

September 2, 2010

Study Start

July 1, 2010

Primary Completion

December 1, 2013

Study Completion

December 1, 2013

Last Updated

January 10, 2017

Record last verified: 2017-01