Trials Sponsors Conditions Drugs Pricing

Conditions

McArdle Disease

Outcome Measures

Primary Outcomes (1)

Change in mean heart rate
Change in mean heart rate (bpm) during constant load cycling exercise (30 minute submaximal cycle test). Heart rate will be measured every minute during the cycle test at all visits.
At Visit 1 (baseline), Visit 2 (4 weeks), Visit 3 (8 weeks), and Visit 4 (12 weeks)

Secondary Outcomes (15)

Compliance
up 12 weeks
Change in Indirect calorimetry
At Visit 1 (baseline), Visit 2 (4 weeks), Visit 3 (8 weeks), and Visit 4 (12 weeks)
Change in self-rated daily function scores
At Visit 1 (baseline), Visit 2 (4 weeks), Visit 3 (8 weeks), and Visit 4 (12 weeks)
Change in self-rated fatigue
At Visit 1 (baseline), Visit 2 (4 weeks), Visit 3 (8 weeks), and Visit 4 (12 weeks)
Change in blood ketones
At Visit 1 (baseline), Visit 2 (4 weeks), Visit 3 (8 weeks), and Visit 4 (12 weeks)
+10 more secondary outcomes

Study Arms (2)

Interventional diet

ACTIVE COMPARATOR

A modified ketogenic diet (with the composition found in the pilot study, 75%fat, 15% protein, 10% carbohydrates)

Dietary Supplement: Ketocal 4:1 liquid Nutricia (intervention)

Placebo diet

PLACEBO COMPARATOR

A placebo diet (over 100 g of carbohydrates per day)

Dietary Supplement: Fortini multifibre Nutrica (placebo)

Interventions

Ketocal 4:1 liquid Nutricia (intervention)Dietary supplement

Modified ketogenic diet

Interventional diet

Fortini multifibre Nutrica (placebo)Dietary supplement

Placebo diet

Registry-reported names are shown in full. Names without a link have not yet been matched to a Syfrah profile.

Eligibility Criteria

Age18 Years - 80 Years

Sexall

Healthy VolunteersNo

Age GroupsAdult (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.

Sponsors & Collaborators

University College, London
Othercollaborator
Copenhagen University Hospital, Rigshospitalet
Otherlead

Study Sites (2)

Copenhagen Neuromuscular Center, Rigshospitalet

Copenhagen, Denmark

National Hospital for Neurology and neurosurgery

London, United Kingdom

Related Publications (1)

Lokken N, Nielsen MR, Stemmerik MG, Ellerton C, Revsbech KL, Macrae M, Slipsager A, Krett B, Beha GH, Emanuelsson F, van Hall G, Quinlivan R, Vissing J. Can a modified ketogenic diet be a nutritional strategy for patients with McArdle disease? Results from a randomized, single-blind, placebo-controlled, cross-over study. Clin Nutr. 2023 Nov;42(11):2124-2137. doi: 10.1016/j.clnu.2023.09.006. Epub 2023 Sep 20.
PMID: 37769369DERIVED

MeSH Terms

Conditions

Glycogen Storage Disease Type V

Interventions

Methods

Condition Hierarchy (Ancestors)

Glycogen Storage DiseaseCarbohydrate Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesMetabolic DiseasesNutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Investigative Techniques

Enrollment

20 participants (actual enrollment reported by the registry).

Study Design

Study Type: interventional
Phase: not applicable
Allocation: RANDOMIZED
Masking: DOUBLE
Who Masked: PARTICIPANT, INVESTIGATOR
Masking Details: Disguise products (Oral supplements: Nutricia 'ketocal' (intervention) or Nutricia 'fortini' (placebo)
Purpose: TREATMENT
Intervention Model: CROSSOVER
Sponsor Type: OTHER
Responsible Party: PRINCIPAL INVESTIGATOR
PI Title: MD, Research fellow

Study Record Dates

First Submitted

December 12, 2018

First Posted

August 5, 2019

Study Start

Modified Ketogenic Diet in Patients With McArdle Disease Part B

Brief Summary

Registry Overview

Trial Relationships

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (15)

Study Arms (2)

Interventional diet

Placebo diet

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

Related Publications (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations

Brief Summary

Registry Overview

Trial Relationships

Related Scientific Literature

Study Timeline

First Submitted

Study Start

First Posted

Primary Completion

Study Completion

Conditions

Outcome Measures

Primary Outcomes (1)

Secondary Outcomes (15)

Study Arms (2)

Interventional diet

Placebo diet

Interventions

Eligibility Criteria

You may qualify if:

Sponsors & Collaborators

Study Sites (2)

Related Publications (1)

MeSH Terms

Conditions

Interventions

Condition Hierarchy (Ancestors)

Intervention Hierarchy (Ancestors)

Enrollment

Study Design

Study Record Dates

You may not qualify if:

Data Sharing

Source record

Locations