The Effect of Arginine on Classic Galactosemia (ARGALT)
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|ClinicalTrials.gov Identifier: NCT03580122|
Recruitment Status : Completed
First Posted : July 9, 2018
Last Update Posted : September 27, 2018
Rationale: Classic galactosemia is a rare inherited metabolic disease that presents in neonatal patients with a life-threatening multi-organ toxic syndrome. Although the current standard of care - a galactose-restricted diet - quickly relieves the severe neonatal clinical picture, it fails to prevent brain and gonadal sequelae. There is a need for new therapeutic strategies.
As arginine is an amino acid that is therapeutically widely used with no side effects described, we propose to use it in a pilot-clinical study. We aim to evaluate the effects of arginine in classic galactosemia patients, in order to determine its potential therapeutic role in this disease.
Objective: To evaluate the possible effect of arginine on the whole body galactose oxidative capacity in classic galactosemia patients.
Study design: Interventional pilot-clinical study with pre-post single arm design.
Study population: We aim to include 5 classic galactosemia adult patients homozygous for the p.Q188R mutation.
Intervention: All participants will receive arginine in the form of Asparten ® (arginine aspartate) during 1 month, by oral administration.
The main study parameter is whole body galactose galactose oxidative capacity.
|Condition or disease||Intervention/treatment||Phase|
|Classic Galactosemia||Drug: Arginine Aspartate||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||4 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Does Arginine Enhance Galactose Oxidative Capacity in Classic Galactosemia: A Pilot Study|
|Actual Study Start Date :||December 5, 2017|
|Actual Primary Completion Date :||January 12, 2018|
|Actual Study Completion Date :||September 5, 2018|
Asparten (arginine aspartate) 5000mg/10mL 3x/day
Drug: Arginine Aspartate
- whole body galactose oxidative capacity [ Time Frame: 6 hours ]Whole body galactose oxidative capacity is breathing test that quantifies [1-13C]-galactose conversion into 13CO2, thus allowing to delineate the exact extent of impaired galactose metabolism, providing clear information on a patient's ability to oxidize galactose.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03580122
|Academisch Ziekenhuis Maastricht|
|Maastricht, Limburg, Netherlands, 6202 AZ|