Kaposiform HemangioendotheliomaPhase 4NCT04921722
Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies
A Phase 4 study testing Percutaneous sirolimus and Oral sirolimus in people with Kaposiform Hemangioendothelioma and Tufted Angioma, run by Children's Hospital of Fudan University at 1 site in China.
Trial summary
In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies. Read more about this study
- Who is it for?
- Ages 0 to 18 years
- Conditions the study is for
- Kaposiform Hemangioendothelioma, Tufted Angioma, Superficial Vascular Anomalies
- What is being tested?
- Percutaneous sirolimus and Oral sirolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 75 participants
- Where
- 1 siteChina
- Study dates
- 22 Oct 2021to 1 Nov 2025
- Phase
- Phase 4
- 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 0 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components.
- The case is initial, with a relatively limited superficial lesion.
- The participant has residual surface lesions after oral medication.
- Participant with no use of other medication or surgical treatment
- Participant with detailed medical records of the disease at the time of screening
- Participant with signed and dated informed consent from the guardian(s)
Who cannot join
- Participants with Kasabach-Merritt Phenomenon, with platelets <50×10 9 /L.
- Participants with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
- Participants with other hematological diseases or solid tumor.
- Participants allergic to sirolimus or dressing.
Trial locations and contacts
1 site in 1 country.
China1 site, 1 recruiting
- Recruiting
Children's Hospital of Fudan University
Shanghai, 210012
Kai Li, MD, PhD, +86 02164931114, likai2727@163.com
Kai Li, MD, PhD
About this study
Official title: Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies: a Randomized Controlled Trial
In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.
Read the detailed description
Vascular anomaly is a kind of rare disease. According to histology, biological behavior and clinical manifestations, it can be divided into two categories: vascular tumor and vascular malformation.
mTOR inhibitors are proved with the properties of anti-proliferation and anti-angiogenesis. Therefore, they have been used in the treatment of vascular anomalies. Sirolimus, by its ability to prevent downstream protein synthesis and subsequent cell proliferation and angiogenesis, has become a novel and effective treatment. However, after the children reach complete response, there may still be skin manifestations that affect the appearance and cause psychological shadows. Therefore, intervention is required.
Studies have reported that topical sirolimus is effective in treating Kaposiform Hemangioendothelioma (KHE). It is absorbed through the skin, avoiding the first pass elimination effect of the liver. Fewer adverse reactions have been observed. In this study, we investigate the efficacy and safety of percutaneous administration of sirolimus in the treatment of superficial complicated vascular anomalies.
- Conditions
- Kaposiform Hemangioendothelioma, Tufted Angioma, Superficial Vascular Anomalies, Superficial Lymphatic Malformations
- Being tested
- Percutaneous sirolimus, Oral sirolimus
- Medical subject headings
- Kaposiform Hemangioendothelioma, Tufted angioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Percutaneous sirolimusDrug
Also called Rapamycin
We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In experimental group, we administrate percutaneous sirolimus. Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months.
Oral sirolimusDrug
Also called Rapamycin
We compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In active comparator group, we administrate oral sirolimus. Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml.
| Study group | Role | What they receive |
|---|---|---|
| Topical use of sirolimus | Experimental | Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months. Receives: Drug: Percutaneous sirolimus |
| Oral use of sirolimus | Active comparator | Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml. Receives: Drug: Oral sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Effective rate
Achauer BM et al. made the four-level standard as follows. Grade I: tumor size and skin lesion color regression ≤ 25%; grade II: tumor size and skin lesion color regression 25%-50%; grade III: tumor size and skin lesion color regression 50-75%; grade IV: tumor size and skin lesion color regression ≥75%. After 6 months of treatment, the pzrticipant will be evaluated. Grade I will be viewed as invalid. Grade II and grade III will be viewed as effective, and grade IV will be viewed as very effective. Those in grade Ⅱ, Ⅲ or Ⅳ will be calculated in effectiveness rate.
Measured: From admission to follow-up six months
Secondary outcomes (4)
Changes of resistance coefficient
Measured by ultrosonic doppler flowmetery at follow-up
Measured: From admission to follow-up six months
Incidence of adverse events
Adverse events will be reported according to Common Terminology Criteria for Adverse Events, version 4.0 (CTCAE v4.0). Incidence of complications such as oral ulcers, abnormal liver enzymes, infections will be recorded. It is defined as occurring if individual subject has any of the above complications during the 6-month intervention
Measured: From admission to follow-up six months
Platelet count
Platelet count is one of the major indicators of response to treatment. It is supposed to be greater than 100×10^9/L.
Measured: From admission to follow-up six months
Changes of peak blood flow
Measured by ultrosonic doppler flowmetery at follow-up
Measured: From admission to follow-up six months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jun 2021
- Study start22 Oct 2021
- Primary completion1 Nov 2025 (estimated)
- Study completion1 Nov 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Kaposiform HemangioendotheliomaStudies of the same condition, open and under way.
- More trials run by Children's Hospital of Fudan UniversityThe sponsor's other registered studies.
- 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 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 75 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Hospital of Fudan University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04921722
- Sponsor's protocol ID
- LK210106
- Status last verified
- Mar 2024
- First submitted
- 6 Jun 2021
- Last updated
- 15 Mar 2024
- 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 15 Mar 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies: a Randomized Controlled Trial
- ClinicalTrials.gov ID
- NCT04921722
- Acronym / sponsor ID
- LK210106
- Conditions
- Kaposiform Hemangioendothelioma, Tufted Angioma, Superficial Vascular Anomalies, Superficial Lymphatic Malformations
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 75 participants (estimated)
- Lead sponsor
- Children's Hospital of Fudan UniversityUniversity, hospital or other
- Study start
- 22 Oct 2021 (actual)
- Primary completion
- 1 Nov 2025 (estimated)
- Study completion
- 1 Nov 2025 (estimated)
- First posted
- 10 Jun 2021 (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.
In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.
Detailed description
Vascular anomaly is a kind of rare disease. According to histology, biological behavior and clinical manifestations, it can be divided into two categories: vascular tumor and vascular malformation.
mTOR inhibitors are proved with the properties of anti-proliferation and anti-angiogenesis. Therefore, they have been used in the treatment of vascular anomalies. Sirolimus, by its ability to prevent downstream protein synthesis and subsequent cell proliferation and angiogenesis, has become a novel and effective treatment. However, after the children reach complete response, there may still be skin manifestations that affect the appearance and cause psychological shadows. Therefore, intervention is required.
Studies have reported that topical sirolimus is effective in treating Kaposiform Hemangioendothelioma (KHE). It is absorbed through the skin, avoiding the first pass elimination effect of the liver. Fewer adverse reactions have been observed. In this study, we investigate the efficacy and safety of percutaneous administration of sirolimus in the treatment of superficial complicated vascular anomalies.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Topical use of sirolimus | Experimental | Drug: Percutaneous sirolimusDrop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months. |
| Oral use of sirolimus | Active comparator | Drug: Oral sirolimusOral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml. |
Who may qualify
- Ages
- 0 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (6 listed)
- Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components.
- Participant with no use of other medication or surgical treatment
- Participant with detailed medical records of the disease at the time of screening
- Participant with signed and dated informed consent from the guardian(s)
Key exclusion criteria (4 listed)
- Participants with Kasabach-Merritt Phenomenon, with platelets <50×10 9 /L.
- Participants with general disease such as hypertension, diabetes, adrenal insufficiency, neurological diseases, liver and kidney dysfunction, and cardiopulmonary insufficiency.
- Participants with other hematological diseases or solid tumor.
- Participants allergic to sirolimus or dressing.
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 |
|---|---|---|
| Percutaneous sirolimusAlso: RapamycinWe compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In experimental group, we administrate percutaneous sirolimus. Drop 5 ml of sirolimus oral solution and 5 g of dressing into the mixed bottle. Apply mixed gel of topical sirolimus to affected area. Use it twice a day for 6 months. | Drug | Topical use of sirolimus |
| Oral sirolimusAlso: RapamycinWe compare topical and oral use of sirolimus in the treatment of superficial complicted vascular anomalies. In active comparator group, we administrate oral sirolimus. Oral dose of sirolimus is calculated according to body surface area. Take it twice a day for 6 months. Maintain the blood concentration of sirolimus at 5-15ng/ml. | Drug | Oral use of sirolimus |
Primary outcome
Effective rate
Time frame: From admission to follow-up six months
Achauer BM et al. made the four-level standard as follows. Grade I: tumor size and skin lesion color regression ≤ 25%; grade II: tumor size and skin lesion color regression 25%-50%; grade III: tumor size and skin lesion color regression 50-75%; grade IV: tumor size and skin lesion color regression ≥75%. After 6 months of treatment, the pzrticipant will be evaluated. Grade I will be viewed as invalid. Grade II and grade III will be viewed as effective, and grade IV will be viewed as very effective. Those in grade Ⅱ, Ⅲ or Ⅳ will be calculated in effectiveness rate.
4 secondary outcomes
Changes of resistance coefficient
Time frame: From admission to follow-up six months
Incidence of adverse events
Time frame: From admission to follow-up six months
Platelet count
Time frame: From admission to follow-up six months
Changes of peak blood flow
Time frame: From admission to follow-up six months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Mar 2024.
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RecruitingPhase 2/3
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 4 study testing Percutaneous sirolimus and Oral sirolimus in people with Kaposiform Hemangioendothelioma and Tufted Angioma, run by Children's Hospital of Fudan University 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 6 inclusion and 4 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 15 Mar 2024. 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 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
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?
Each site lists its own contact: choose the nearest one under Trial locations. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Effective rate (time frame: From admission to follow-up six months). 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 15 Mar 2024.