Modified Ketogenic Diet in Patients With McArdle Disease Part A

NCT03843606 | Completed DMC

• 1 other identifier: H-18013022
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief Summary

McArdle disease, glycogen storage disease type V, is a rare metabolic disease. Affected individuals are unable to utilize sugar stored as glycogen in muscle. We hypothesize that a modified ketogenic diet could be a potential treatment option, by providing ketones as alternative fuel substrates for working muscle. In this open interventional pilot study we wish to investigate 3 different modified ketogenic diet regimes, to find an optimal composition of a modified ketogenic diet that ensures adequate degree of ketosis and at the same time is well tolerated for patients with McArdle disease.

Conditions

McArdle Disease

Outcome Measures

Primary Outcomes (1)

• Heart rate

  heart rate during constant load cycling exercise

  2 times

Secondary Outcomes (6)

• Compliance

  3 weeks

• indirect calorimetry

  2 times

• level of ketosis

  daily

• Safety parameters

  2 times

• Perceived exertion

  2 times

Study Arms (3)

diet 1

EXPERIMENTAL

60% fat, 15% protein, 25% carbohydrates

Other: Modified ketogenic diets

diet 2

EXPERIMENTAL

70% fat, 15% protein, 15% carbohydrates

Other: Modified ketogenic diets

diet 3

EXPERIMENTAL

80% fat, 15% protein, 5% carbohydrates

Other: Modified ketogenic diets

Interventions

Modified ketogenic diets OTHER

Different diet composition

diet 1; diet 2; diet 3

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Genetically confirmed GSDV
• Patient is willing and able to provide written informed consent prior to participation.
• Patient is ambulatory.
• Women in fertile age must be willing to practice the following medically acceptable methods of birth control

You may not qualify if:

• Patient has any prior or current medical conditions that, in the judgment of the Investigator, would prevent the patient from safely participating in and/or completing all study requirements.
• Pregnancy or breastfeeding
• Patient does not have the cognitive capacity to understand/comprehend and complete all study assessments
• Patients with porphyria or disorders of fat metabolism (primary carnitine deficiency, carnitine palmitoyltransferase I or II, β-oxidation defects etc.).

Sponsors & Collaborators

• Rigshospitalet, Denmark: lead

Study Sites (1)

Copenhagen Neuromuscular Center, Rigshospitalet

Copenhagen, Denmark

Location

MeSH Terms

Conditions

Glycogen Storage Disease Type V

Condition Hierarchy (Ancestors)

Glycogen Storage Disease; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: MD, PhD-student

Model Details: Open interventional study to investigate the effects of 3 different ketogenic diet regimes

Study Record Dates

• First Submitted: December 12, 2018
• First Posted: February 18, 2019
• Study Start: February 16, 2019
• Primary Completion: May 1, 2019
• Study Completion: May 8, 2019
• Last Updated: May 10, 2019

Record last verified: 2019-05

Data Sharing

• IPD Sharing: Will not share

Locations

DK (1)