Sugar supplement shows promise for rare disease in new trial
NCT ID NCT05402332
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This phase 2b trial tests AVTX-801, a D-galactose supplement, in 8 adults with PGM1-CDG, a rare genetic disorder affecting sugar metabolism. Participants currently on D-galactose will receive either the study drug or a placebo to see if it reduces disease-related events like low blood sugar or liver problems. The study aims to find a better way to manage this condition.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
Active substance
D-galactose (AVTX-801)
What this could lead to
If successful, this could provide a better treatment option for people with PGM1-CDG, helping manage symptoms like low blood sugar and liver issues.
What could go wrong
This is a small, early-phase trial with only 8 participants, so results may not apply to everyone. The treatment is already used, so benefits may be limited.
Disclaimer
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This is a summary of
the original study
.
Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
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Study contacts
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Contact
Phone: •••-•••-•••• Email: •••••@•••••
Locations
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Mayo Clinic Rochester
Rochester, Minnesota, 55905, United States