Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)Phase 1SCMC-KK2020NCT04409691
SCMC Trial on KHE With KMP (V.2020)
A Phase 1 study testing prednison and Sirolimus and prednison in people with Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP), run by Shanghai Children's Medical Center at 1 site in China.
Trial 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) Read more about this study
- Who is it for?
- Ages Up to 12 years
- Conditions the study is for
- Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
- What is being tested?
- prednison and Sirolimus and prednison
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 20 participants
- Where
- 1 siteChina
- Study dates
- 1 Jul 2020to 30 Jun 2023
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
China1 site
Shanghai Children's Medical Center Shanghai Jiaotong University School of Medicine
Shanghai, Shanghai Municipality, 200127
Song Gu, Doctor, 18930830716, gusong@shsmu.edu.cn
About this study
Official title: A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance
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)
- Being tested
- prednison and Sirolimus, prednison
- Medical subject headings
- Kaposiform Hemangioendothelioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
prednison and SirolimusDrug
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
prednisonDrug
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.
| Study group | Role | What they receive |
|---|---|---|
| prednison group | Active comparator | Not described Receives: Drug: prednison |
| prednison+sirolimus group | Experimental | Not described Receives: Drug: prednison and Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 6 months after taking the drug
Secondary outcomes (1)
side effect rate
Number of participants with treatment-related adverse events as assessed by CTCAE v5.0 Monitoring patient's clinical biochemical indicators and symptoms
Measured: 6 months after taking the drug
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Jun 2020
- Study start1 Jul 2020 (estimated)
- Primary completion31 Jan 2023 (estimated)
- Study completion30 Jun 2023 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 20 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Children's Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04409691
- Sponsor's protocol ID
- SCMC2020
- Acronym
- SCMC-KK2020
- Status last verified
- Jun 2020
- First submitted
- 27 May 2020
- Last updated
- 9 Jun 2020
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 9 Jun 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Clinical Study on the Treatment of Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP) by Hormone Shock and Sirolimus Maintenance
- ClinicalTrials.gov ID
- NCT04409691
- Acronym / sponsor ID
- SCMC-KK2020 / SCMC2020
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Shanghai Children's Medical CenterUniversity, hospital or other
- Study start
- 1 Jul 2020 (estimated)
- Primary completion
- 31 Jan 2023 (estimated)
- Study completion
- 30 Jun 2023 (estimated)
- First posted
- 1 Jun 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| prednison group | Active comparator | Drug: prednison |
| prednison+sirolimus group | Experimental | Drug: prednison and Sirolimus |
Who may qualify
- Ages
- Up to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (5 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| prednison and SirolimusIf 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 | Drug | prednison+sirolimus group |
| prednisonPrednison 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. | Drug | prednison group |
Primary outcome
response to treatment
Time frame: 6 months after taking the drug
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.
1 secondary outcome
side effect rate
Time frame: 6 months after taking the drug
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Jun 2020.
Related trials for Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP)
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Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing prednison and Sirolimus and prednison in people with Kaposiform Hemangioendothelioma (KHE) With Kasabach-Merritt Phenomenon (KMP), run by Shanghai Children's Medical Center at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 9 Jun 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Song Gu (18930830716, gusong@shsmu.edu.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
response to treatment (time frame: 6 months after taking the drug). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 9 Jun 2020.