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Efficacy and Safety of Rapamycin to Complex Vascular Anomalies in Pediatric Patients

NCT04598204 · Status: UNKNOWN · Phase: PHASE2/PHASE3 · Type: INTERVENTIONAL · Enrollment: 30

Last updated 2021-11-10

No results posted yet for this study

Summary

KHE and TA are rare tumors and some of the cases may lead to life-threatening complications including Kasabach-Merritt Phenomenon. Typically treated with steroids and vincristine, a majority of the cases do not have good prognosis. Complex vascular malformations are always managed by surgery,sclerotherapy and embolization therapy. While many of the cases still lead to complications such as disfigurement, chronic pain, recurrent infections, coagulopathies. Different medical centers are exploring new therapy for these tough problems. This study is plotted to determine the efficacy and safety of rapamycin monotherapy in KHE/TA and complex vascular malformations in pediatric patients.

Conditions

  • Vascular Anomalies

Interventions

DRUG

Treatment with oral rapmycin

To treat the enrolled patients with oral rapamycin with an initial dosage of 0.8mg/m2, once daily for children under 3 years old and twice daily (every 12 hours) for those above 3 years, and adjust the dosage to target a trough concentration of rapamycin in plasma as 10-15ng/ml （OR 15-20ng/ml if the efficacy of treatment is not satisfactory）. One course lasts for 12weeks and no more than 4 course is given.

Sponsors & Collaborators

  • Zhujiang Hospital

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Month
Max Age
14 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2017-12-01
Primary Completion
2022-12-01
Completion
2022-12-01

Countries

  • China

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04598204 on ClinicalTrials.gov