SCMC Trial on KHE With KMP (V.2020)

NCT04409691 | Unknown Phase 1

• 1 other identifier: SCMC2020
• Study Type: interventional
• Target: 20
• Locations: 1 country
• Sites: 1

Brief Summary

a phase I trial focusing on safety and efficacy of prednison shock plus sirolimus maintenance in treating Kaposiform hemangioendothelioma (KHE) with Kasabach-Merritt phenomenon (KMP)

Conditions

Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)

Outcome Measures

Primary Outcomes (1)

• response to treatment

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

  6 months after taking the drug

Secondary Outcomes (1)

• side effect rate

  6 months after taking the drug

Study Arms (2)

prednison group

ACTIVE COMPARATOR

Drug: prednison

prednison+sirolimus group

EXPERIMENTAL

Drug: prednison and Sirolimus

Interventions

prednison and Sirolimus DRUG

If the intravenous prednisolone 4 mg / kg / d (one time in the morning and one time in the evening, 2mg / kg body weight each time) is effective, it will be gradually reduced to 2mg / kg / D (one time in the morning and one time in the evening, 1mg / kg body weight each time), gradually converted to oral prednisone of equal dose, and the hormone will be removed within 4-6 weeks. At the same time, the dosage of sirolimus oral liquid is 0.8 mg / m2 twice a day, with an interval of 12 hours, maintaining the blood concentration of 8-15 ng / ml. if there is no intolerable side effect, the treatment will last for 6 months

prednison+sirolimus group

prednison DRUG

Prednison is taken at a dose of 4mg/kg/d. If the hormone treatment is effective, it will be gradually reduced to 2mg / kg / d (one time in the morning and one time in the evening, 1 mg / kg body weight each time), and repeated after 2 months of continuous treatment and 1 month of drug withdrawal.

prednison 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 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

• Shanghai Children's Medical Center: lead

Study Sites (1)

Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine

Shanghai, Shanghai Municipality, 200127, China

Location

MeSH Terms

Conditions

Kaposiform Hemangioendothelioma

Interventions

Sirolimus; Prednisone

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals; Pregnadienediols; Pregnadienes; Pregnanes; Steroids; Fused-Ring Compounds; Polycyclic Compounds

Central Study Contacts

Song Gu

CONTACT

18930830716; gusong@shsmu.edu.cn

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 27, 2020
• First Posted: June 1, 2020
• Study Start: July 1, 2020
• Primary Completion: January 31, 2023
• Study Completion: June 30, 2023
• Last Updated: June 9, 2020

Record last verified: 2020-06

Locations

CN (1)