Status:
UNKNOWN
Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation
Lead Sponsor:
Peking Union Medical College Hospital
Collaborating Sponsors:
Air Force General Hospital of the PLA
Chinese Academy of Medical Sciences
Conditions:
Blue Rubber Bleb Nevus Syndrome
Venous Malformation
Eligibility:
All Genders
Phase:
PHASE4
Brief Summary
A prospective, nonrandomized, open-label, single-arm clinical trial to study efficacy of rapamycin (sirolimus) in the treatment of Blue Rubber Bleb Nevus Syndrome, hereditary or sporadic venous malfor...
Detailed Description
Blue rubber bleb nevus syndrome (BRBNS) and venous malformation are mainly caused by somatic mutation of TEK and PIK3CA, which activates the PI3K/AKT signaling pathway. As an important protein kinase ...
Eligibility Criteria
Inclusion
- Patients diagnosed with BRBNS, VMCM, sporadic multiple VM, or large single VM;
- Age and gender are not limited;
- Physical status ECOG 0\~3;
- Organ function is good, biochemical examination meets the following conditions: AST ≤ 2.5 × upper limit of normal value (ULN), ALT ≤ 2.5 × upper limit of normal value (ULN), serum total bilirubin ≤ 1.5 × upper limit of normal value (ULN), creatinine ≤ 1.5 × upper limit of normal (ULN);
- Patients volunteer to participate in the trial and sign the informed consent form by the participant or his/her legal guardian.
Exclusion
- Patients need emergency surgery due to intestinal obstruction, intussusception, or gastrointestinal bleeding;
- History of surgery within 1 month;
- allergic to rapamycin;
- Any disease or condition that may affect the study implementation or result interpretation, including: known hemoglobinopathy, suffering from gastrointestinal infections at the same time, severe heart, liver, kidney and other serious concomitant diseases that may endanger lives
- Pregnant or lactating women;
- Alcohol or drugs (eg, laxatives) abusers;
- Participating in another clinical trial that may affect this study within one month;
- Being believed not suitable to be enrolled by the investigator for other reasons.
- Exit Criteria:
- An allergic reaction to rapamycin occurs.
- The patient requests withdrawal: at his own discretion or at the request of his legal representative. Subjects may refuse to participate in further studies at any time without reasons. Subjects will not be affected because of such decision.
- Subjects are required to withdraw from the study in certain special circumstances (eg, there is significant issues of compliance, safety, or surgical intervention for the disease)
- Other situations in which the study must be terminated. For example, the investigators believe that continuing the study may be harmful to the health of subjects.
- Rejection Criteria:
- Patients who violate the requirements of the test protocol
- Patients with poor recording (with incomplete, or inaccurate data)
Key Trial Info
Start Date :
July 31 2018
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
July 1 2022
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT03767660
Start Date
July 31 2018
End Date
July 1 2022
Last Update
December 19 2018
Active Locations (1)
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1
Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
Beijing, Beijing Municipality, China, 100000