Clinical Trial: Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Energy Supplements to Improve Exercise Tolerance in Metabolic Myopathies

Brief Summary: Patients suffering from the metabolic myopathy Glycogen Storage Disease type IIIa (GSDIIIa) have a problem releasing sugar stored in cells that is needed for energy production. This causes several systemic impairments, but only recently have the exercise-related symptoms in the muscles been examined. A previous study showed signs that intravenous infusion of glucose relieves some of these symptoms. The purpose of this study is to investigate in a randomized and placebo-controlled fashion whether oral ingestion of sugar can alleviate muscular symptoms in patients with GSDIIIa.

Detailed Summary: It has recently been documented how patients with GSDIIIa have a moderate to severely reduced exercise capacity, and that exercise induces muscle pain and cramps. These symptoms are caused by the inability to mobilize skeletal muscle glycogen and are most likely the consequence of a severe energy deficiency within muscles. The study changed the phenotype of GSDIIIa, to include exercise-induced symptoms, which is a typical presentation in other metabolic myopathies. It also documented that exercise capacity was significantly improved while exercise-induced muscular symptoms were relieved by an intravenous glucose infusion. Based on these findings, this study wishes to investigate if oral ingestion of sucrose has the same effects on work capacity on a larger number of patients, in a randomized, placebo-controlled, cross-over setup. Ingestion of sucrose has the potential to be an effective, cheap and easily accessible dietary treatment of muscular symptoms in GSDIIIa.
Sponsor: Rigshospitalet, Denmark

Current Primary Outcome: maximal work capacity [ Time Frame: After up to 1 hour of bicycling on the 2nd and 4th day. ]

Area Under the Curve (AUC)


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Peak oxygen consumption [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (VO2peak)
  • Peak workload [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (Wpeak)
  • Peak respiratory exchange ratio [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (RER)
  • p-lactate [ Time Frame: measured at rest and max on day 1, and before first dose of soft drink, before exercise and every 10 minutes during exercise at day 2 and 4. ]
  • Heart rate [ Time Frame: Continously during the cycle test (max. 1 hour) on the 2nd and 4th day ]
  • Borg score [ Time Frame: Measured periodically during the cycle test (max. 1 hour) on the 2nd and 4th day ]


Original Secondary Outcome:

  • Peak oxygen consumption [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (VO2peak)
  • Peak workload [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (Wpeak)
  • Peak respiratory exchange ratio [ Time Frame: After up to 1 hour of cycling on the 2nd and 4th day. ]
    (RER)
  • Peak lactate [ Time Frame: measured every 10 minutes during exercise test at day 2 and 4 and once on day 3 and 5. ]
  • Heart rate [ Time Frame: Continously during the cycle test (max. 1 hour) on the 2nd and 4th day ]
  • Borg score [ Time Frame: Measured periodically during the cycle test (max. 1 hour) on the 2nd and 4th day ]


Information By: Rigshospitalet, Denmark

Dates:
Date Received: May 15, 2015
Date Started: January 2015
Date Completion: February 2018
Last Updated: January 19, 2017
Last Verified: January 2017