One patient gets experimental enzyme to fight rare kidney disease
NCT ID NCT04737720
First seen Jun 24, 2026 · Last updated Jul 17, 2026 · Updated 6 times
Summary
This study tested an artificial version of the LCAT enzyme (ACP-501) in one person with familial LCAT deficiency, a rare genetic condition that leads to low 'good' cholesterol and kidney failure. The participant received several infusions of the drug over weeks, with regular blood tests to see if it raised HDL cholesterol and improved kidney function. The goal was to see if replacing the missing enzyme could reverse or prevent the disease's worst effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- ACP-501 (recombinant human LCAT enzyme)
- What this could lead to
- If it works, this could point toward a treatment for familial LCAT deficiency, potentially preventing or reversing kidney problems.
- What could go wrong
- This is a single-person study with no control group, so results may not apply to others. The treatment is still experimental and may not improve kidney function or have unknown side effects.
This is an AI summary of the original study and may miss details. Read our disclaimer.
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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
Locations
-
National Institutes of Health Clinical Center
Bethesda, Maryland, 20892, United States