Experimental drug aims to tame rare mast cell disease
NCT ID NCT00449748
First seen Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time
Summary
This phase 2 trial tests whether the drug RAD001 (everolimus) can help control systemic mastocytosis, a rare condition where too many mast cells build up in organs like the bone marrow. Ten adults with the disease take a daily pill for 30 days. Researchers measure success by changes in blood tryptase levels or bone marrow mast cell counts, and they also watch for side effects.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- RAD001 (everolimus)
- What this could lead to
- If it works, this could point toward a treatment that helps control systemic mastocytosis, reducing symptoms and disease activity.
- What could go wrong
- This is a small, early-phase trial with only 10 participants, so results may not apply to everyone. The drug may not work or could cause side effects like liver problems or bleeding.
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
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Locations
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The University of Texas M.D. Anderson Cancer Center
Houston, Texas, 77030, United States
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