Vascular MalformationsPhase 2NCT07477548
A Study to Evaluate the Efficacy and Safety of Everolimus in Patients With Teratment-refractory Vascular Anomalies
A Phase 2 study testing Everolimus in people with Vascular Malformations and Arteriovenous Malformations, run by Yonsei University.
Trial summary
Background and Objectives Vascular anomalies are a heterogeneous group of disorders classified into vascular tumors and vascular malformations according to the ISSVA classification. Read more about this study
- Who is it for?
- Ages 1 year and older
- Conditions the study is for
- Vascular Malformations, Arteriovenous Malformations, Venous Malformation
- What is being tested?
- Everolimus
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 67 participants
- Where
- No sites listed yet
- Study dates
- 1 Apr 2026to 30 Nov 2030
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
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 1 year and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- ① Diagnosis per the 2014 ISSVA classification: Group 1 (hemangioendothelioma, tufted angioma): histologically confirmed tumor, OR Kasabach-Merritt Syndrome (histologically confirmed or histologic diagnosis not feasible) Group 2 (vascular tumors not in Group 1, or vascular malformations): histologically confirmed, OR radiologically diagnosed when biopsy is not feasible
- Age ≥1 year ③ Failure of at least one prior therapy (e.g., vincristine, corticosteroids, interferon), stratified as: Cohort 1: sirolimus-naïve Cohort 2: prior sirolimus failure
- At least one measurable target lesion ≥1 cm in longest diameter per RECIST 1.1 on CT or MRI
- ECOG Performance Score 0, 1, or 2 ⑥ WOCBP must have a negative pregnancy test prior to enrollment; adequate contraception required during the study and for 8 weeks after completion ⑦ Written informed consent obtained
Who cannot join
- Pregnancy or breastfeeding (WOCBP must use adequate contraception)
- Documented allergy or hypersensitivity to everolimus
- ③ Inadequate organ function: Bone marrow: ANC <1,000/µL or platelets <75,000/µL Renal: serum creatinine >1.5×ULN; if >1.5×ULN, 24-hour creatinine clearance <60 mL/min Hepatic: total bilirubin >1.5×ULN or ALT >3.0×ULN
- KMP associated with vascular tumors or malformations is not an exclusion criterion, including: thrombocytopenia (<100,000/µL), hypofibrinogenemia, anemia (Hb <8 g/dL), consumptive coagulopathy, or overlying skin changes (edema, warmth, erythema, purplish/dark discoloration)
- Uncontrolled hyperlipidemia (fasting cholesterol >300 mg/dL or triglycerides >2.5×ULN)
- Uncontrolled diabetes (fasting glucose >1.5×ULN)
Show all 13Show fewer
- Active uncontrolled infection
- Hepatitis B (HBsAg positive) or hepatitis C (anti-HCV positive) ⑨ Known HIV infection (positive serology)
- Clinically significant symptomatic pulmonary dysfunction; PFTs and room air SpO₂ performed if clinically indicated; exclusion if FEV₁ ≤70% or DLCO ≤70% of predicted (assessed in patients ≥8 years)
- ⑪ Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart)
- ⑫ Concomitant investigational agents (e.g., mTOR inhibitors: sirolimus, temsirolimus)
- ⑬ Concurrent chemotherapy (e.g., mTOR inhibitors: sirolimus, temsirolimus)
- ⑭ Concurrent other malignancy not meeting eligibility criteria
Trial locations and contacts
No sites are listed yet. Contact the study team or a site near you to ask about taking part.
About this study
Official title: Non-randomized, Phase II, Open-label Study for Efficacy and Safety of Everolimus in Relapsed or Refractory Hemangioendothelioma and Other ISSVA Group I or II Vascular Malformation and Neoplasms
Background and Objectives Vascular anomalies are a heterogeneous group of disorders classified into vascular tumors and vascular malformations according to the ISSVA classification. Although most follow a benign course, a subset causes serious complications including organ dysfunction, chronic pain, thrombocytopenia, and hemorrhage. Kaposiform hemangioendothelioma (KHE) complicated by Kasabach-Merritt Phenomenon (KMP) carries a mortality rate of 14-24%. Surgical resection is the primary treatment when organ damage is not anticipated; however, when surgery is not feasible, pharmacologic therapy is considered. Agents such as interferon, corticosteroids, vincristine, cyclophosphamide, and propranolol have been used with variable efficacy, and no established therapy exists for patients refractory to these treatments.
The PI3K-Akt-mTOR and RAS-MEK-ERK pathways have been identified as key molecular mechanisms underlying vascular anomalies. Targeted therapies against these pathways are emerging, including anti-VEGF antibodies, PI3K/Akt inhibitors (e.g., alpelisib, miransertib), and mTOR inhibitors. Sirolimus has demonstrated clinical benefit in 50-80% of patients with vascular anomalies, with a 96% symptom response rate in KMP-associated vascular tumors. Everolimus, another mTOR inhibitor, is already approved and established for tuberous sclerosis-associated angiomyolipoma and SEGA in pediatric patients, with a well-characterized safety profile. Given its shared mechanism with sirolimus and emerging case reports supporting efficacy in KHE with KMP, this phase 2 study aims to evaluate the efficacy and safety of everolimus in patients with treatment-refractory vascular anomalies.
Study Design This is a single-center, open-label, uncontrolled phase 2 clinical trial enrolling 67 patients over 60 months from IRB approval, stratified into two cohorts: Cohort 1 (sirolimus-naïve, n=39) and Cohort 2 (prior sirolimus failure, n=28). Everolimus is administered orally at age- and CYP3A4/P-gp inducer-adjusted doses, with maintenance dosing titrated to a target trough level of 5-15 ng/mL. The primary endpoint is overall response rate (ORR) at 6 months. Secondary endpoints include toxicity per NCI CTCAE v4.0, ORR at 12 months, platelet recovery rate at 4 weeks (KMP patients), 1-year overall survival, and 3-year progression-free survival.
Read the detailed description
- Study Design This is a single-center, open-label, uncontrolled phase 2 clinical trial. The study period is 60 months from IRB approval; patients may not be enrolled within 1 year of the planned study end date. A total of 67 patients will be enrolled and stratified into two cohorts based on prior sirolimus exposure: Cohort 1 (sirolimus-naïve, n=39) and Cohort 2 (prior sirolimus failure, n=28).
- Eligibility Criteria
- Inclusion Criteria: ① Diagnosis per the 2014 ISSVA classification: Group 1 (hemangioendothelioma, tufted angioma): histologically confirmed tumor, OR Kasabach-Merritt Syndrome (histologically confirmed or histologic diagnosis not feasible) Group 2 (vascular tumors not in Group 1, or vascular malformations): histologically confirmed, OR radiologically diagnosed when biopsy is not feasible
- Age ≥1 year ③ Failure of at least one prior therapy (e.g., vincristine, corticosteroids, interferon), stratified as: Cohort 1: sirolimus-naïve Cohort 2: prior sirolimus failure ④ At least one measurable target lesion ≥1 cm in longest diameter per RECIST 1.1 on CT or MRI
- ECOG Performance Score 0, 1, or 2
- WOCBP must have a negative pregnancy test prior to enrollment; adequate contraception required during the study and for 8 weeks after completion ⑦ Written informed consent obtained - Exclusion Criteria: ① Pregnancy or breastfeeding (WOCBP must use adequate contraception)
- Documented allergy or hypersensitivity to everolimus ③ Inadequate organ function: Bone marrow: ANC <1,000/µL or platelets <75,000/µL Renal: serum creatinine >1.5×ULN; if >1.5×ULN, 24-hour creatinine clearance <60 mL/min Hepatic: total bilirubin >1.5×ULN or ALT >3.0×ULN
- KMP associated with vascular tumors or malformations is not an exclusion criterion, including: thrombocytopenia (<100,000/µL), hypofibrinogenemia, anemia (Hb <8 g/dL), consumptive coagulopathy, or overlying skin changes (edema, warmth, erythema, purplish/dark discoloration)
- Uncontrolled hyperlipidemia (fasting cholesterol >300 mg/dL or triglycerides >2.5×ULN)
- Uncontrolled diabetes (fasting glucose >1.5×ULN)
- Active uncontrolled infection
- Hepatitis B (HBsAg positive) or hepatitis C (anti-HCV positive)
- Known HIV infection (positive serology)
- Clinically significant symptomatic pulmonary dysfunction; PFTs and room air SpO₂ performed if clinically indicated; exclusion if FEV₁ ≤70% or DLCO ≤70% of predicted (assessed in patients ≥8 years) ⑪ Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart) ⑫ Concomitant investigational agents (e.g., mTOR inhibitors: sirolimus, temsirolimus) ⑬ Concurrent chemotherapy (e.g., mTOR inhibitors: sirolimus, temsirolimus)
- Concurrent other malignancy not meeting eligibility criteria
- Treatment Everolimus is administered orally with doses adjusted by age and concomitant CYP3A4/P-gp inducer use: -Starting dose: Age <10 years: 6.0 mg/m²/day (9.0 mg/m²/day with CYP3A4/P-gp inducers) Age 10 to <18 years: 5.0 mg/m²/day (8.0 mg/m²/day with CYP3A4/P-gp inducers) Age ≥18 years: 3.0 mg/m²/day (5.0 mg/m²/day with CYP3A4/P-gp inducers) -Maintenance dose: Adjusted to achieve a target trough level of 5-15 ng/mL
- Endpoints
- Primary endpoint: Overall response rate (ORR) at 6 months
- Secondary endpoints: Toxicity assessment per NCI CTCAE v4.0 ORR at 12 months Rate of platelet recovery at 4 weeks (KMP patients)
1-year overall survival (OS) 3-year progression-free survival (PFS)
- Conditions
- Vascular Malformations, Arteriovenous Malformations, Venous Malformation, Lymphangioma, Nevus, Port-Wine, Hemangioendothelioma
- Being tested
- Everolimus
- Medical subject headings
- Vascular Malformations, Arteriovenous Malformations, Lymphangioma, Nevus, Hemangioendothelioma, Hemangioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EverolimusDrug
Everolimus (dose varies by age and concomitant use of CYP3A4/P-gp inducers) -Starting dose: Age <10 years: 6.0 mg/m²/day (9.0 mg/m²/day with CYP3A4/P-gp inducers) Age 10 to <18 years: 5.0 mg/m²/day (8.0 mg/m²/day with CYP3A4/P-gp inducers) Age ≥18 years: 3.0 mg/m²/day (5.0 mg/m²/day with CYP3A4/P-gp inducers)
-Maintenance dose: Adjusted to achieve a target trough level of 5-15 ng/mL
| Study group | Role | What they receive |
|---|---|---|
| Everolimus | Experimental | Everolimus administered orally, dose adjusted by age and concomitant CYP3A4/P-gp inducer use. Target trough level: 5-15 ng/mL. Receives: Drug: Everolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall Response Rate (ORR)
ORR is defined as the proportion of patients achieving complete response (CR) or partial response (PR) per RECIST 1.1 criteria on CT or MRI at 6 months from treatment initiation.
Measured: 6 months from treatment initiation
Secondary outcomes (5)
Overall Response Rate (ORR)
ORR is defined as the proportion of patients achieving complete response (CR) or partial response (PR) per RECIST 1.1 criteria on CT or MRI at 6 months from treatment initiation.
ORR is defined as the proportion of patients achieving complete response (CR) or partial response (PR) per RECIST 1.1 criteria on CT or MRI at 6 months from treatment initiation.
ORR is defined as the proportion of patients achieving complete response (CR) or partial response (PR) per RECIST 1.1 criteria on CT or MRI at 6 months from treatment initiation.
Measured: 12 months from treatment initiation
Overall Survival (OS)
Proportion of patients alive at 1 year from treatment initiation
Measured: 12 months from treatment initiation
Overall Response Rate (ORR)
ORR is defined as the proportion of patients achieving complete response (CR) or partial response (PR) per RECIST 1.1 criteria on CT or MRI at 6 months from treatment initiation.
Measured: 12 months from treatment initiation
Toxicity
Incidence and severity of adverse events assessed per NCI CTCAE v4.0
Measured: Throughout treament period (every 4 weeks)
Platelet Recovery Rate at 4 weeks (KMP patients)
4 weeks from treatment initiation
Measured: Rate of platelet recovery to ≥100,000/µL at 4 weeks from treatment initiation, assessed in patients with Kasabach-Merritt Phenomenon at enrollment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Mar 2026
- Study start1 Apr 2026 (estimated)
- Primary completion31 Oct 2030 (estimated)
- Study completion30 Nov 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Vascular MalformationsStudies of the same condition, open and under way.
- More trials run by Yonsei 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 67 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Yonsei University
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07477548
- Sponsor's protocol ID
- 4-2025-1169
- Status last verified
- Mar 2026
- First submitted
- 12 Mar 2026
- Last updated
- 17 Mar 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Non-randomized, Phase II, Open-label Study for Efficacy and Safety of Everolimus in Relapsed or Refractory Hemangioendothelioma and Other ISSVA Group I or II Vascular Malformation and Neoplasms
- ClinicalTrials.gov ID
- NCT07477548
- Acronym / sponsor ID
- 4-2025-1169
- Conditions
- Vascular Malformations, Arteriovenous Malformations, Venous Malformation, Lymphangioma, Nevus, Port-Wine, Hemangioendothelioma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 67 participants (estimated)
- Lead sponsor
- Yonsei UniversityUniversity, hospital or other
- Study start
- 1 Apr 2026 (estimated)
- Primary completion
- 31 Oct 2030 (estimated)
- Study completion
- 30 Nov 2030 (estimated)
- First posted
- 17 Mar 2026 (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.
Background and Objectives Vascular anomalies are a heterogeneous group of disorders classified into vascular tumors and vascular malformations according to the ISSVA classification. Although most follow a benign course, a subset causes serious complications including organ dysfunction, chronic pain, thrombocytopenia, and hemorrhage. Kaposiform hemangioendothelioma (KHE) complicated by Kasabach-Merritt Phenomenon (KMP) carries a mortality rate of 14-24%. Surgical resection is the primary treatment when organ damage is not anticipated; however, when surgery is not feasible, pharmacologic therapy is considered. Agents such as interferon, corticosteroids, vincristine, cyclophosphamide, and propranolol have been used with variable efficacy, and no established therapy exists for patients refractory to these treatments.
The PI3K-Akt-mTOR and RAS-MEK-ERK pathways have been identified as key molecular mechanisms underlying vascular anomalies. Targeted therapies against these pathways are emerging, including anti-VEGF antibodies, PI3K/Akt inhibitors (e.g., alpelisib, miransertib), and mTOR inhibitors. Sirolimus has demonstrated clinical benefit in 50-80% of patients with vascular anomalies, with a 96% symptom response rate in KMP-associated vascular tumors. Everolimus, another mTOR inhibitor, is already approved and established for tuberous sclerosis-associated angiomyolipoma and SEGA in pediatric patients, with a well-characterized safety profile. Given its shared mechanism with sirolimus and emerging case reports supporting efficacy in KHE with KMP, this phase 2 study aims to evaluate the efficacy and safety of everolimus in patients with treatment-refractory vascular anomalies.
Study Design This is a single-center, open-label, uncontrolled phase 2 clinical trial enrolling 67 patients over 60 months from IRB approval, stratified into two cohorts: Cohort 1 (sirolimus-naïve, n=39) and Cohort 2 (prior sirolimus failure, n=28). Everolimus is administered orally at age- and CYP3A4/P-gp inducer-adjusted doses, with maintenance dosing titrated to a target trough level of 5-15 ng/mL. The primary endpoint is overall response rate (ORR) at 6 months. Secondary endpoints include toxicity per NCI CTCAE v4.0, ORR at 12 months, platelet recovery rate at 4 weeks (KMP patients), 1-year overall survival, and 3-year progression-free survival.
Detailed description
- Study Design This is a single-center, open-label, uncontrolled phase 2 clinical trial. The study period is 60 months from IRB approval; patients may not be enrolled within 1 year of the planned study end date. A total of 67 patients will be enrolled and stratified into two cohorts based on prior sirolimus exposure: Cohort 1 (sirolimus-naïve, n=39) and Cohort 2 (prior sirolimus failure, n=28).
- Eligibility Criteria
- Inclusion Criteria: ① Diagnosis per the 2014 ISSVA classification: Group 1 (hemangioendothelioma, tufted angioma): histologically confirmed tumor, OR Kasabach-Merritt Syndrome (histologically confirmed or histologic diagnosis not feasible) Group 2 (vascular tumors not in Group 1, or vascular malformations): histologically confirmed, OR radiologically diagnosed when biopsy is not feasible
- Age ≥1 year ③ Failure of at least one prior therapy (e.g., vincristine, corticosteroids, interferon), stratified as: Cohort 1: sirolimus-naïve Cohort 2: prior sirolimus failure ④ At least one measurable target lesion ≥1 cm in longest diameter per RECIST 1.1 on CT or MRI
- ECOG Performance Score 0, 1, or 2
- WOCBP must have a negative pregnancy test prior to enrollment; adequate contraception required during the study and for 8 weeks after completion ⑦ Written informed consent obtained - Exclusion Criteria: ① Pregnancy or breastfeeding (WOCBP must use adequate contraception)
- Documented allergy or hypersensitivity to everolimus ③ Inadequate organ function: Bone marrow: ANC <1,000/µL or platelets <75,000/µL Renal: serum creatinine >1.5×ULN; if >1.5×ULN, 24-hour creatinine clearance <60 mL/min Hepatic: total bilirubin >1.5×ULN or ALT >3.0×ULN
- KMP associated with vascular tumors or malformations is not an exclusion criterion, including: thrombocytopenia (<100,000/µL), hypofibrinogenemia, anemia (Hb <8 g/dL), consumptive coagulopathy, or overlying skin changes (edema, warmth, erythema, purplish/dark discoloration)
- Uncontrolled hyperlipidemia (fasting cholesterol >300 mg/dL or triglycerides >2.5×ULN)
- Uncontrolled diabetes (fasting glucose >1.5×ULN)
- Active uncontrolled infection
- Hepatitis B (HBsAg positive) or hepatitis C (anti-HCV positive)
- Known HIV infection (positive serology)
- Clinically significant symptomatic pulmonary dysfunction; PFTs and room air SpO₂ performed if clinically indicated; exclusion if FEV₁ ≤70% or DLCO ≤70% of predicted (assessed in patients ≥8 years) ⑪ Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart) ⑫ Concomitant investigational agents (e.g., mTOR inhibitors: sirolimus, temsirolimus) ⑬ Concurrent chemotherapy (e.g., mTOR inhibitors: sirolimus, temsirolimus)
- Concurrent other malignancy not meeting eligibility criteria
- Treatment Everolimus is administered orally with doses adjusted by age and concomitant CYP3A4/P-gp inducer use: -Starting dose: Age <10 years: 6.0 mg/m²/day (9.0 mg/m²/day with CYP3A4/P-gp inducers) Age 10 to <18 years: 5.0 mg/m²/day (8.0 mg/m²/day with CYP3A4/P-gp inducers) Age ≥18 years: 3.0 mg/m²/day (5.0 mg/m²/day with CYP3A4/P-gp inducers) -Maintenance dose: Adjusted to achieve a target trough level of 5-15 ng/mL
- Endpoints
- Primary endpoint: Overall response rate (ORR) at 6 months
- Secondary endpoints: Toxicity assessment per NCI CTCAE v4.0 ORR at 12 months Rate of platelet recovery at 4 weeks (KMP patients)
1-year overall survival (OS) 3-year progression-free survival (PFS)
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Everolimus | Experimental | Drug: EverolimusEverolimus administered orally, dose adjusted by age and concomitant CYP3A4/P-gp inducer use. Target trough level: 5-15 ng/mL. |
Who may qualify
- Ages
- 1 year and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- ① Diagnosis per the 2014 ISSVA classification: Group 1 (hemangioendothelioma, tufted angioma): histologically confirmed tumor, OR Kasabach-Merritt Syndrome (histologically confirmed or histologic diagnosis not feasible) Group 2 (vascular tumors not in Group 1, or vascular malformations): histologically confirmed, OR radiologically diagnosed when biopsy is not feasible
- Age ≥1 year ③ Failure of at least one prior therapy (e.g., vincristine, corticosteroids, interferon), stratified as: Cohort 1: sirolimus-naïve Cohort 2: prior sirolimus failure
Key exclusion criteria (13 listed)
- Pregnancy or breastfeeding (WOCBP must use adequate contraception)
- ③ Inadequate organ function: Bone marrow: ANC <1,000/µL or platelets <75,000/µL Renal: serum creatinine >1.5×ULN; if >1.5×ULN, 24-hour creatinine clearance <60 mL/min Hepatic: total bilirubin >1.5×ULN or ALT >3.0×ULN
- ⑪ Prior solid organ or hematopoietic stem cell transplantation (bone marrow, liver, kidney, lung, or heart)
- ⑫ Concomitant investigational agents (e.g., mTOR inhibitors: sirolimus, temsirolimus)
- ⑬ Concurrent chemotherapy (e.g., mTOR inhibitors: sirolimus, temsirolimus)
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 |
|---|---|---|
| EverolimusEverolimus (dose varies by age and concomitant use of CYP3A4/P-gp inducers) -Starting dose: Age <10 years: 6.0 mg/m²/day (9.0 mg/m²/day with CYP3A4/P-gp inducers) Age 10 to <18 years: 5.0 mg/m²/day (8.0 mg/m²/day with CYP3A4/P-gp inducers) Age ≥18 years: 3.0 mg/m²/day (5.0 mg/m²/day with CYP3A4/P-gp inducers) -Maintenance dose: Adjusted to achieve a target trough level of 5-15 ng/mL | Drug | Everolimus |
Primary outcome
Overall Response Rate (ORR)
Time frame: 6 months from treatment initiation
ORR is defined as the proportion of patients achieving complete response (CR) or partial response (PR) per RECIST 1.1 criteria on CT or MRI at 6 months from treatment initiation.
5 secondary outcomes
Overall Response Rate (ORR)
Time frame: 12 months from treatment initiation
Overall Survival (OS)
Time frame: 12 months from treatment initiation
Overall Response Rate (ORR)
Time frame: 12 months from treatment initiation
Toxicity
Time frame: Throughout treament period (every 4 weeks)
Platelet Recovery Rate at 4 weeks (KMP patients)
Time frame: Rate of platelet recovery to ≥100,000/µL at 4 weeks from treatment initiation, assessed in patients with Kasabach-Merritt Phenomenon at enrollment
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Everolimus in people with Vascular Malformations and Arteriovenous Malformations, run by Yonsei University.
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 4 inclusion and 13 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: opening soon, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Jung Woo Han, Professor (+82-2-2228-2050, JWHAN@yuhs.ac), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall Response Rate (ORR) (time frame: 6 months from treatment initiation). 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 17 Mar 2026.