New drug shows promise for rare overgrowth conditions
NCT ID NCT06975618
First seen Jun 25, 2026 · Last updated Jun 30, 2026 · Updated 3 times
Summary
This study tests a new drug called CYH33 in people with rare conditions that cause abnormal tissue growth and blood vessel malformations (PROS and PRVM). The trial has two phases: phase I finds the safest dose, and phase II checks if the drug shrinks growths. About 141 adults and adolescents will take part.
What this could mean
Our plain-language read of the trial. This is informational only — not medical advice or a prediction.
- Active substance
- CYH33 (a PI3Kα inhibitor)
- What this could lead to
- If successful, this could lead to a new treatment option that shrinks abnormal growths and improves symptoms for people with PROS and PRVM.
- What could go wrong
- This is an early-phase trial (phase I/II) with a small number of participants, so safety and effectiveness are not yet proven. The drug may cause side effects or fail to show benefit.
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
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Capital Center for Children's Health, Capital Medical University
RECRUITINGBeijing, Beijing Municipality, 100000, China
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Fujian Medical University Union Hospital
RECRUITINGFuzhou, Fujian, 350001, China
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Gifu University Hospital
RECRUITINGGifu, 501-1194, Japan
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Guangzhou Women and Children's Medical Center
NOT_YET_RECRUITINGGuangzhou, Guangdong, 510000, China
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Henan Provincial People's Hospital
RECRUITINGZhengzhou, Henan, 450003, China
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Kyorin University Hospital
RECRUITINGMitaka, Tokyo, 181-8611, Japan
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National Hospital Organization Kobe Medical Center
RECRUITINGKobe, Hyōgo, 654-0155, Japan
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Plastic Surgery Hospital, Chinese Academy of Medical Sciences
RECRUITINGBeijing, Beijing Municipality, 100144, China
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Shanghai Ninth People Hospital, Shanghai Jiaotong University School of Medicine
RECRUITINGShanghai, Shanghai Municipality, 200011, China
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Shinshu University Hospital
RECRUITINGMatsumoto, Nagano, 390-8621, Japan
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The Second Xiangya Hospital of Central South University
RECRUITINGChangsha, Hunan, 410011, China
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Tohoku University Hospital
RECRUITINGSendai, Miyagi, 980-8574, Japan
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Tonan Hospital
RECRUITINGSapporo, Hokkaido, 060-0004, Japan
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West China Hospital of Sichuan University
RECRUITINGChengdu, Sichuan, 610041, China
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Yokohama City University Hospital
RECRUITINGYokohama, Kanagawa, 236-0004, Japan
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Alpelisib's Long-Term safety tracked in PIK3CA patients
- Could a pill shrink abnormal growths in PROS? new trial hopes to find out.
- Last-Resort drug alpelisib made available for patients with no other options
- New drug targets root cause of rare overgrowth syndromes
- New hope for rare overgrowth disorder: daily pill could tame symptoms
- Drug shows promise for rare overgrowth disorder in long-term study