Ignite Creation Date:
2025-12-24 @ 7:13 PM
Ignite Modification Date:
2025-12-30 @ 12:13 PM
Study NCT ID:
NCT03011203
Status:
COMPLETED
Last Update Posted:
2018-05-01
First Post:
2016-12-22
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Acute Nutritional Ketosis in GSD IIIa
Sponsor:
University Medical Center Groningen
Organization:
Study Overview
Official Title:
Acute Nutritional Ketosis and Exercise in Glycogen Storage Disease Type IIIa
Status:
COMPLETED
Status Verified Date:
2018-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The investigators will study if acute nutritional ketosis in adult GSD IIIa patients can boost muscle mitochondrial function in vivo.
Detailed Description:
Glycogen Storage Disease type IIIa (GSD IIIa) is an inborn error of carbohydrate metabolism caused by impaired glycogen debranching enzyme (GDE) activity. The ageing GSD IIIa cohort shows that muscle involvement -despite dietary management- is a common disabling phenotype in adulthood. Currently, no specific therapy has been established for muscle problems in adult GSD IIIa patients.
However, it could be hypothesized that nutritional ketosis (NK) will be highly beneficial to patients. Amongst others, ketone bodies could take on the role of primary energy source in exercising muscle. Recently, acute NK in exercise was studied in humans. NK was found to enhance muscle mitochondrial function in athletes.
The investigators will study if acute NK in adult GSD IIIa patients can boost muscle mitochondrial function in vivo.
However, it could be hypothesized that nutritional ketosis (NK) will be highly beneficial to patients. Amongst others, ketone bodies could take on the role of primary energy source in exercising muscle. Recently, acute NK in exercise was studied in humans. NK was found to enhance muscle mitochondrial function in athletes.
The investigators will study if acute NK in adult GSD IIIa patients can boost muscle mitochondrial function in vivo.
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: