The effect of Triheptanoin on excercise in adults and adolescence with glycogenoses

Phase 1

• Conditions: Cori Forbe's Disease Also called: glycogen storage disease Type III or debrancher deficiency.Tarui's diseaseAlso called: glycogen storage disease Type VII or phosphofructokinase deficiency.Glycogenin-1 deficiency or glycogen storage disease Type XV.; MedDRA version: 20.0Level: LLTClassification code 10016983Term: Forbes' diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 20.1Level: PTClassification code 10053241Term: Glycogen storage disease type VIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; MedDRA version: 20.1Level: PTClassification code 10053250Term: Glycogen storage disease type IIISystem Organ Class: 10010331 - Congenital, familial and genetic disorders; Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]; MedDRA version: 20.0Level: LLTClassification code 10053255Term: Tarui diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders

• Registration Number: EUCTR2017-004153-17-DK
• Lead Sponsor: Copenhagen Neuromuscular Center

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-03-26
• Last Update Posted: 21 September 2020

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 20

Inclusion Criteria

- Male/Female Age > 15 years
- Genetically and/or biochemically verified diagnosis of GSD III, GSD VII or GSD XV
- Capacity to consent
- For women in fertile age on contraceptive treatment with: Birth control
pills, coil, ring, transdermal hormone patch injection of gestagen or
subdermal implant.
Are the trial subjects under 18? yes
Number of subjects for this age range: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 14
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 2

Exclusion Criteria

- Significant cardiac or pulmonary disease
- Pregnancy (confirmed by urine stix or plasma-HCG) or breastfeeding.
- Inability to perform cycling exercise
- Any other significant disorder that may confound the interpretation of
the findings

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified