SCMC Trial on KHE With KMP (V.2020)

NCT ID: NCT04409691

Last Updated: 2020-06-09

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

UNKNOWN

Clinical Phase

PHASE1

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2020-07-01

Study Completion Date

2023-06-30

Brief Summary

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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)

Detailed Description

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Conditions

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

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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prednison group

Group Type ACTIVE_COMPARATOR

prednison

Intervention Type 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+sirolimus group

Group Type EXPERIMENTAL

prednison and Sirolimus

Intervention Type 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

Interventions

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prednison and Sirolimus

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

Intervention Type DRUG

prednison

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.

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Kaposiform Hemangioendotheliomas with Kasabach-Merritt Phenomenon
* 0 - 12 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

Exclusion Criteria

* 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
Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shanghai Children's Medical Center

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Locations

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

Shanghai, Shanghai Municipality, China

Site Status

Countries

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China

Central Contacts

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Song Gu

Role: CONTACT

18930830716

Facility Contacts

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Song Gu, Doctor

Role: primary

18930830716

Other Identifiers

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SCMC2020

Identifier Type: -

Identifier Source: org_study_id

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