Safety and Efficacy of Low-dose Sirolimus to Kaposiform Hemangioendothelioma

NCT04077515 | Completed Phase 4

• 1 other identifier: 2019-161
• Study Type: interventional
• Target: 92
• Locations: 1 country
• Sites: 1

Brief Summary

to evaluate the safety and efficacy of Low-dose sirolimus in Kaposiform Hemangioendothelioma in Chinese children by a prospective, randomized open trial.

Conditions

Hemangioma; Kaposiform Hemangioendothelioma

Keywords

Kaposiform Hemangioendothelioma; Kasabach-Merritt Syndrome; Sirolimus; Rapamycin

Outcome Measures

Primary Outcomes (1)

• response to treatment

  it is a variable outcome, including complete responses, partial response and no response. The definitions are : Complete Response： 1. platelets counts is greater than 100×10\^9/L. 2. significant volume reduction is greater than 80%. 3. Fibrinogen levels at 2-4g/L. 4. The surface skin of the tumor is lighter or the tumor is softer significantly. Partial Response: 1. platelets counts is greater than 40×10\^9/L. 2. significant volume reduction is greater than 50%. 3. Fibrinogen levels at less than 50% reduction from baseline. 4. The surface skin of the tumor and palpation of the tumor have no change or less change. No Response: 1. platelets counts is less than 40×10\^9/L. 2. significant volume reduction is less than 50% or the tumor is bigger. 3. Fibrinogen levels at grater then 50% reduction from baseline. 4. The surface skin of the tumor is darker or the tumor is harder.

  1 year after taking the drug

Secondary Outcomes (1)

• Number of participants with treatment-related adverse events as assessed by CTCAE v4.0

  1 year after taking the drug

Study Arms (2)

high blood concentration group

ACTIVE COMPARATOR

The blood concentration is maintained at 10-15ng/ml (not including 10ng/ml).

Drug: Sirolimus(0.8mg/m2)

low blood concentration group

EXPERIMENTAL

The blood concentration is maintained at 7-10ng/ml (including 10ng/ml).

Drug: Sirolimus(0.7mg/m2)

Interventions

Sirolimus(0.8mg/m2) DRUG

The initial use of sirolimus is 0.8mg/m2 administered twice daily.After two weeks of taking the drug, blood concentrations are measured and adjusted appropriately to maintain the targeted blood concentration.

Also known as: Rapamycin

high blood concentration group

Sirolimus(0.7mg/m2) DRUG

The initial use of sirolimus is 0.7mg/m2 administered twice daily.After two weeks of taking the drug, blood concentrations are measured and adjusted appropriately to maintain the targeted blood concentration.

Also known as: Rapamycin

low blood concentration group

Eligibility Criteria

• Age: Up to 12 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Kaposiform Hemangioendotheliomas with or without Kasabach-Merritt Phenomenon.
• years of age at the time of study entry.
• Male or female.
• Consent of parents (or the person having parental authority in families): Signed and dated written informed consent.

You may not qualify if:

• with hematological diseases.
• with other solid tumors.
• with hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
• with tuberculosis,cytomegalovirus and Epstein-Barr virus infection before the treatment.

Sponsors & Collaborators

• Children's Hospital of Fudan University: lead

Study Sites (1)

Children's Hospital of Fudan University

Shanghai, 210012, China

Location

Related Publications (4)

• Drolet BA, Trenor CC 3rd, Brandao LR, Chiu YE, Chun RH, Dasgupta R, Garzon MC, Hammill AM, Johnson CM, Tlougan B, Blei F, David M, Elluru R, Frieden IJ, Friedlander SF, Iacobas I, Jensen JN, King DM, Lee MT, Nelson S, Patel M, Pope E, Powell J, Seefeldt M, Siegel DH, Kelly M, Adams DM. Consensus-derived practice standards plan for complicated Kaposiform hemangioendothelioma. J Pediatr. 2013 Jul;163(1):285-91. doi: 10.1016/j.jpeds.2013.03.080. No abstract available.

  PMID: 23796341 BACKGROUND

• Yoon HY, Hwang JJ, Kim DS, Song JW. Efficacy and safety of low-dose Sirolimus in Lymphangioleiomyomatosis. Orphanet J Rare Dis. 2018 Nov 14;13(1):204. doi: 10.1186/s13023-018-0946-8.

  PMID: 30428897 BACKGROUND

• Kazmierczak D, Maliszewska A. [Disturbances of tooth and maxillary development in Down's syndrome and their treatment]. Czas Stomatol. 1986 Nov;39(11):755-9. No abstract available. Polish.

  PMID: 2958244 BACKGROUND

• Ozgonenel B, Martin A. Low-dose sirolimus controls recurrent iron deficiency in a patient with blue rubber bleb nevus syndrome. Pediatr Blood Cancer. 2015 Nov;62(11):2054-5. doi: 10.1002/pbc.25590. Epub 2015 Jul 1. No abstract available.

  PMID: 26132912 BACKGROUND

MeSH Terms

Conditions

Hemangioma; Kaposiform Hemangioendothelioma; Kasabach-Merritt Syndrome

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Neoplasms, Vascular Tissue; Neoplasms by Histologic Type; Neoplasms; Thrombocytopenia; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Kai Li, MD, PhD

  Children's Hospital of Fudan University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2019
• First Posted: September 4, 2019
• Study Start: May 1, 2019
• Primary Completion: December 10, 2022
• Study Completion: December 31, 2022
• Last Updated: January 30, 2023

Record last verified: 2023-01

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)