Efficacy and Safety of Sirolimus to Vascular Anomalies

NCT03583307 | Completed

• 1 other identifier: 2018-618
• Study Type: interventional
• Target: 126
• Locations: 1 country
• Sites: 1

Brief Summary

To evaluate the safety and efficacy of Sirolimus in complicated vascular anomalies in Chinese children

Conditions

Vascular Anomaly

Keywords

Sirolimus; Vascular anomalies

Outcome Measures

Primary Outcomes (2)

• Volumetric changes in complicated vascular anomalies to sirolimus

  Response to sirolimus treatment was measured by volumetric magnetic resonance imaging (MRI) analyses, which were performed at baseline and 6 and 12 months after treatment and were independently assessed by 2 radiologists. Changes in size of vascular anomalies were classified as further growth (increase of ≥10%), no change (\<10% increase and \<10% decrease), partial involution (decrease of ≥10% and \<75%), nearly complete involution (decrease of ≥75% and \<100%), or complete involution (100%). Photographs of the complicated vascular anomalies were taken at months 0, 3, 6 and 12 by a medical photographer. Complete/nearly complete resolution of the vascular anomalies at month 12 compared to baseline based on the intra-patient blinded centralized independent qualitative assessments of month 12 MRI.

  Baseline, 6, and 12 months

• The changes in the patient's symptoms and/or complications.

  Baseline, 3, 6, and 12 months

Secondary Outcomes (4)

• Quality of Life in patients by the Pediatric Quality of Life Inventory TM (PedsQLTM) 4.0 Generic Core Scales.

  Baseline, 6, 12 months

• Measuring the impact of vascular anomalies on family functioning by PedsQLTM 4.0 Family Impact Module (FIM).

  Baseline, 6, 12 months

• Frequency of adverse events as assessed by CTCAE v4.0

  Baseline, 3, 6, 12 months

• Changes in plasma levels fibrinogen and/ or D-dimers

  Baseline, 3, 6, 12 months

Study Arms (1)

Sirolimus

EXPERIMENTAL

Drug: Sirolimus

Interventions

Sirolimus DRUG

Sirolimus was initiated at a dosage of 0.8 mg/m2 administered twice daily. Subsequently, the sirolimus dosage was adjusted monthly to achieve trough levels between 10 and 15 ng/mL.

Also known as: Rapamycin

Sirolimus

Eligibility Criteria

• Age: 0 Years - 18 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• All patients included in the present research must be diagnosed with one of the following vascular anomalies:
• Kaposiform Hemangioendotheliomas without Kasabach-Merritt Phenomenon
• Tufted Angioma without Kasabach-Merritt Phenomenon
• Capillary Malformations
• Lymphatic Malformations
• Venous Malformations
• Capillary-Venous Malformation (CVM)
• Capillary-Lymphatic Malformation (CLM)
• Lymphatic-Venous Malformation (LVM)
• Capillary-Lymphatic-Venous Malformation (CLVM)
• Multifocal Lymphangiomatosis and Thrombocytopenia (MLT)
• Patients must be 0 - 18 years of age at the time of study entry.
• Without functional impairment requiring treatment of corticosteroid.
• Organ function requirements:
• Adequate liver function Total bilirubin less than or equal to 1.5 x upper limit of normal (ULN)for age, and alanine transaminase (ALT) and aspartate aminotransferase (AST) less than or equal to 2.5 x upper limit normal (ULN) for age.

You may not qualify if:

• Allergy to sirolimus or other rapamycin analogues.
• Allergy to sirolimus or other rapamycin analogues.
• Any known evidence of significant local or systemic uncontrolled infection, defined as receiving intravenous antibiotics at the time of randomization.
• Patients must not be known to be Human Immunodeficiency Virus positive or known immunodeficiency. Testing is not required unless a condition is suspected.
• Other concurrent severe and/or uncontrolled medical disease which could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe malnutrition, chronic liver or renal disease, active upper gastrointestinal tract ulceration).
• Impairment of gastrointestinal function or chronic gastrointestinal disease that may significantly alter the absorption of sirolimus.
• Patients who have a history of malignancy.
• Patients with an inability to participate or to follow the study treatment and assessment plan.
• Patients who have a history of treatment with sirolimus or other mTOR inhibitor.

Sponsors & Collaborators

• West China Hospital: lead

Study Sites (1)

West China Hospital of Sichuan University

Chengdu, Sichuan, 61004·, China

Location

Related Publications (1)

• Ji Y, Chen S, Yang K, Zhou J, Zhang X, Jiang X, Xu X, Lu G, Qiu L, Kong F, Zhang Y. A prospective multicenter study of sirolimus for complicated vascular anomalies. J Vasc Surg. 2021 Nov;74(5):1673-1681.e3. doi: 10.1016/j.jvs.2021.04.071. Epub 2021 May 31.

  PMID: 34082006 DERIVED

MeSH Terms

Conditions

Vascular Malformations

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Cardiovascular Abnormalities; Cardiovascular Diseases; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Yi Ji

  West China Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: June 17, 2018
• First Posted: July 11, 2018
• Study Start: June 1, 2018
• Primary Completion: February 19, 2021
• Study Completion: February 19, 2021
• Last Updated: March 15, 2022

Record last verified: 2022-03

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)