Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03767660
• Date of registration: 24/11/2018
• Prospective Registration: No
• Primary sponsor: Peking Union Medical College Hospital
• Public title: Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation
• Scientific title: Efficacy of Rapamycin (Sirolimus) in the Treatment of Blue Rubber Bleb Nevus Syndrome, Hereditary or Sporadic Venous Malformation
• Date of first enrolment: July 31, 2018
• Target sample size: 20
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/show/NCT03767660
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 4

Countries of recruitment

China

Contacts

• Name: Jiaolin Zhou, MD

• Name: Jiaolin Zhou, MD

Key inclusion & exclusion criteria

Inclusion Criteria:

- 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 Criteria:

- 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)



Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Blue Rubber Bleb Nevus Syndrome

Venous Malformation

Intervention(s)

Drug: Rapamycin

Primary Outcome(s)

Total venous malformation lesion load [Time Frame: The time from start of therapy to 1 year]

Secondary Outcome(s)

Amount of daily oral iron supplements [Time Frame: The time from start of therapy to 1 year]

Frequency of blood transfusion [Time Frame: The time from start of therapy to 1 year]

Concentration of hemoglobin in blood [Time Frame: The time from start of therapy to 1 year]

Concentration of D-dimer in blood [Time Frame: The time from start of therapy to 1 year]

Secondary ID(s)

HS-1606

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Chinese Academy of Medical Sciences

Air Force General Hospital of the PLA