Kaposiform Hemangioendothelioma (KHE)Phase 2NCT02110069
A Study to Compare Vincristine to Sirolimus for Treatment of High Risk Vascular Tumors
A Phase 2 study testing Vincristine and Sirolimus in people with Kaposiform Hemangioendothelioma (KHE) and Kasabach-Merritt Syndrome, run by Boston Children's Hospital at 7 sites in United States.
Trial summary
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular tumors such as Tufted Angioma (TA). Read more about this study
- Who is it for?
- Ages Up to 31 years
- Conditions the study is for
- Kaposiform Hemangioendothelioma (KHE), Kasabach-Merritt Syndrome, Tufted Angioma
- What is being tested?
- Vincristine and Sirolimus
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 4 participants
- Where
- 7 sitesUnited States
- Study dates
- 14 Jun 2017to 27 Oct 2020
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 31 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Diagnosis: All patients must have one of the following vascular anomalies as determined by clinical, radiologic and histologic criteria (when possible). Biopsy strongly recommended (but not required) with suggested immunostains: CD34, PROX-1 or D240, Glut-1 and MIB-1.
- Kaposiform Hemangioendotheliomas
- Tufted angioma
- High Risk Stratification: In addition to the above diagnosis, all of the following criteria need to be met:
- Kasabach Merritt Syndrome defined at a platelet counts less than 50,000 K/µl and/or fibrinogen level < 100 mg/dl at the time of diagnosis.
- Age: Patients must be 0 - 31 years of age at the time of study entry. Enrollment includes patients of both genders and all ethnic groups.
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- Organ function requirements:
- Adequate liver function defined as:
- Total bilirubin ≤ 1.5 x ULN for age, and
- SGPT (ALT) ≤ 5 x ULN for age, and
- Serum albumin >/= 2 g/dL.
- Fasting LDL cholesterol of <160 mg/dL
- Fasting triglyceride <400 mg/dl
- Adequate Bone Marrow Function defined as:
- Peripheral absolute neutrophil count (ANC) >/= 1000/uL
- Hemoglobin >/= 8.0 g/dL (may receive RBC transfusions)
- No Platelet requirement
- Adequate Renal Function Defined as:
- A serum creatinine based on age as follows:
- Age (Years) Maximum Serum Creatinine (mg/dL)
- 5 0.8 6 to ≤10 1.0 11 to ≤15 1.2 >15 1.5
- Urine protein to creatinine ratio (UPC) < 0.3 g/l
- Performance Status: Karnofsky >/= 50 (≥16 years of age) and Lansky >/= 50 for patients <16 years of age.
- Prior therapy
- Patients who have undergone surgical resection or interventional radiology procedures for disease control are eligible if they meet all inclusion criteria after surgery/procedure
- Surgery: At least 2 weeks since undergoing any major surgery
- Radiation: > 6 months from involved field radiation
- Prior vincristine therapy is permitted. Patients may also have received up to 2 doses of vincristine prior to randomization.
Who cannot join
- Concurrent severe and/or uncontrolled medical disease that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration).
- Patients who require medications that are strong inhibitors/inducers CYP3A4 enzyme activity, including anticonvulsants, (Appendix II) to control concurrent medical conditions are not eligible. Patients who discontinue use of prohibited medications with a one week washout prior to start of study treatment are eligible.
- Known history of HIV seropositivity or known immunodeficiency. Testing is not required unless a condition is suspected.
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of sirolimus (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection). A gastric tube or nasogastric tube is allowed.
- Females who are pregnant or breast feeding.
- Males or females of reproductive potential may not participate unless they have agreed to use an effective contraceptive method during the period they are receiving the study drug and for 3 months thereafter. Abstinence is an acceptable method of birth control. Females of childbearing potential will be given a pregnancy test within 7 days prior to administration of study treatment and must have a negative urine or serum pregnancy test.
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- Patients who have received prior treatment with an mTOR inhibitor.
- Patients unwilling or unable to comply with the protocol or who in the opinion of the investigator may not be able to comply with the safety monitoring requirements of the study.
- Patients who currently have an uncontrolled infection, defined as receiving intravenous antibiotics.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Emory Children's Healthcare of Atlanta
Atlanta, Georgia, 30322
Johns Hopkins
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02115
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Texas Children's Hospital
Houston, Texas, 77094
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226
Lucille Packard Children's Hospital Stanford
Palo Alto, California, 94304
About this study
Official title: A Randomized Phase 2 Study of Vincristine Versus Sirolimus to Treat High Risk Kaposiform Hemangioendothelioma (KHE).
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular tumors such as Tufted Angioma (TA). In these tumors, the blood cells that help your blood clot called platelets become trapped in the tumor causing swelling, pain, and bruising. Vascular tumors can be life threatening. There are few medical treatments that will work to shrink the vascular tumor. Some doctors will use steroids and vincristine to try and shrink vascular tumors.
In this research study, the study doctor will compare two different drugs to see which one will work better to help shrink your vascular tumor. One of the drugs is vincristine. Vincristine is approved by the Food and Drug Administration (FDA) to treat people with cancer. Vincristine is used to stop the abnormal cells from growing such as cells that make up blood vessels.
Read more
The other drug to be used in this study is sirolimus. Sirolimus is currently approved by the Food and Drug Administration (FDA) to prevent transplanted organ rejection. Sirolimus is not approved by the FDA for treatment of vascular abnormalities and is considered experimental. Sirolimus belongs to a class of drugs call 'mTOR inhibitors'. mTOR (mammilian target of rapamycin) helps cells to grow and may also help blood vessels to grow in a more normal fashion. Sirolimus is currently being tested in patients with vascular tumors and cancer. In vascular tumors, we hope sirolimus will stop the blood vessel growth.
Funding Source: FDA - OOPD (Office of Orphan Products Development)
Read the detailed description
Kaposiform hemangioendotheliomas (KHE) are extremely rare life threatening tumors which can be associated with Kasabach-Merritt Phenomenon consisting of profound thrombocytopenia and hypofibrinogenemia causing a significant risk of bleeding and an associated mortality rate as high as 20% to 30%. Despite the severity of potential complications, we lack uniform guidelines for the treatment and response to treatment of children and young adults with these tumors. KHE patients have been treated with a multitude of aggressive drug regimens without prospective evaluation of response or safety. Presently, vincristine is considered the standard of practice. We have treated a subset of these patients on study SIR-DA-0901 (FDA Grant# 5RO1FD003712-01). This study is a phase II trial assessing the efficacy and safety of sirolimus for the treatment of complicated vascular anomalies. Although the numbers are small, the response has been extremely promising with excellent tolerability. There is pre-clinical and clinical data supporting the essential regulatory function of the PI3 kinase/AKT/mTOR pathway in vascular growth and organization which suggests a therapeutic target for patients with complicated vascular anomalies. The overall goal of this trial is to objectively assess the efficacy of sirolimus compared to vincristine for the treatment of patients with high risk KHE.
Hypothesis: Sirolimus treatment for children and young adults with Kaposiform hemangioendotheliomas will be more effective than vincristine, assessed by time to response in an induction period and provide equivalent safety parameters.
Study Rationale We propose a multi-center, phase II trial with participation from 8 sites. The study will consist of two phases. The first of these is an initial induction phase in which vincristine and steroids will be compared to sirolimus and steroids. Response in the induction phase will be assessed as time to hematologic response. At the end of induction phase, cross over can occur if there is failure to respond. Part 2 is a maintenance phase which will be 1 year in length. Continued safety and efficacy data will be collected during maintenance and there will be cross over at any time for patients who lose their response following induction. Failure will be defined as worsening of hematological parameters on two separate laboratory evaluations at any time during maintenance or if they meet the definition of progressive disease following response assessments. Formal response in maintenance will be evaluated by imaging studies, functional assessment, and quality of life as per study SIR-DA-0901. Present therapies are very limited and new therapies are desperately needed for this devastating disease. Based on our preliminary data, there is a very good rationale for sirolimus therapy in KHE patients and so a phase II trial is urgently needed to determine if this therapy is to become the new standard of care for KHE patients.
Our secondary aims will be addressing biomarker analysis. There are limited studies describing the biology of these tumors. Per study SIR-DA-0901 there is some preliminary data indicating the importance of VEGF-C and other upregulated markers in the mTor pathway. This needs to be further investigated especially in KHE patients. Furthermore there are no clear objective measurements to determine response data.
- Being tested
- Vincristine, Sirolimus
- Keywords
- KHE, Vascular anomaly, Kasabach-Merritt Syndrome, Tufted angioma, hemangioendothelioma
- Medical subject headings
- Kaposiform Hemangioendothelioma, Kasabach-Merritt Syndrome, Tufted angioma, Vascular Malformations, Hemangioendothelioma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
VincristineDrug
Also called Oncovin, Vincasar® PFS, Vincrex
Vincristine dose dependent upon weight. Weekly for 2 months (Induction); Weekly 2 months; every 2 weeks for next 5 months; every 3 weeks for 5 months (Maintenance)
SirolimusDrug
Also called Rapamune
Continuous dosing to maintain trough level of 10-15ng/ml.
| Study group | Role | What they receive |
|---|---|---|
| Vincristine | Active comparator | Induction phase: participants assigned to vincristine will receive vincristine weekly at a dose of 0.05mg/kg/dose IV (for participants less than 10kg) or a dose of 1.5 mg/m2/dose (for participants greater than 10kg) for 2 months.
Maintenance: if participants continue to receive vincristine weekly for 2 months, every 2 weeks for 5 months and every 3 weeks for 5 months. Receives: Drug: Vincristine |
| Sirolimus | Experimental | Sirolimus will be administered at a dose of 0.8mg/m2/dose twice a day on a continuous dosing schedule throughout the trial for participants randomized to Sirolimus or for participants who fail vincristine may cross-over to the sirolimus arm.
Sirolimus trough levels will be maintained between 10-15 ng/ml. Receives: Drug: Sirolimus |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Hematologic Parameters
Hematologic parameters are defined as a platelet count greater than 100,000/uL (or a 2 times increase in platelet count compared to baseline) and a fibrinogen level greater than 150mg/dl.
Measured: 2 months
Number of Serious and Non-Serious Adverse Events
Serious and non-serious adverse events will be recorded for all participants and graded using CTCAE v4.0 (Common Toxicity Criteria for Adverse Effects). Adverse event rates will be calculated for both sirolimus and vincristine.
Measured: 2 months; 12 months
Secondary outcomes (4)
Evaluation of Disease Response - Maintenance
Disease evaluation will be measured using a combination of quality of life assessments, clinical parameters, and radiologic images.
Measured: 6 months; 12 months
Number of Serious and Non Serious Adverse Events - Maintenance
Serious adverse events and adverse events will be graded according to CTCAE v4.0. Adverse event rates will be calculated for both sirolimus and vincristine.
Measured: 6 months; 12 months
Change in the Serum Levels of KHE Biomarkers
The following KHE biomarkers will be evaluated vascular endothelial growth factor A, C, and D (VEGF-A, C, D_, IL-8 (interleukin), Pleiotrophin, IGF-1 (insulin-like growth factor), endothelin-1, thrombospondin and angiopoietin 1 and 2.
Measured: Baseline, 2 months, 6 months, and 12 months
Identify Genetic Variants in Drug Metabolism Enzymes.
Single nucleotide polymorphism array analysis to obtain genetic information on variants in drug metabolism enzymes that affect sirolimus and vincristine metabolism.
Measured: Baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2014 (estimated)
- Study start14 Jun 2017
- Primary completion2 Oct 2019
- Study completion27 Oct 2020
Why it stopped: This study was challenged by the incidence of KHE with KMP as this continued to be extremely rare, and very sporadic. In July of 2019 grant renewal funding ended.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Kaposiform Hemangioendothelioma (KHE)Studies of the same condition, open and under way.
- More trials run by Boston Children's HospitalThe 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 4 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Pfizer
- Responsible party
- Principal investigator, Denise Martin Adams, Boston Children's Hospital
- Investigators
- Denise Adams, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02110069
- Sponsor's protocol ID
- SIR-DA-1202
- Other IDs
- 1R01FD004363-01A1, 2013-2339
- Status last verified
- Aug 2023
- First submitted
- 8 Apr 2014
- Last updated
- 21 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Phase 2 Study of Vincristine Versus Sirolimus to Treat High Risk Kaposiform Hemangioendothelioma (KHE).
- ClinicalTrials.gov ID
- NCT02110069
- Acronym / sponsor ID
- SIR-DA-1202
- Keywords
- KHE, Vascular anomaly, Kasabach-Merritt Syndrome, Tufted angioma, hemangioendothelioma
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: This study was challenged by the incidence of KHE with KMP as this continued to be extremely rare, and very sporadic. In July of 2019 grant renewal funding ended.
- Enrolment
- 4 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Pfizer
- Study start
- 14 Jun 2017 (actual)
- Primary completion
- 2 Oct 2019 (actual)
- Study completion
- 27 Oct 2020 (actual)
- First posted
- 10 Apr 2014 (estimated)
- Results
- Posted 24 May 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
In this research study we want to learn more about which treatment works better for patients diagnosed with a vascular tumor called Kaposiform Hemangioendothelioma (KHE) or other high risk vascular tumors such as Tufted Angioma (TA). In these tumors, the blood cells that help your blood clot called platelets become trapped in the tumor causing swelling, pain, and bruising. Vascular tumors can be life threatening. There are few medical treatments that will work to shrink the vascular tumor. Some doctors will use steroids and vincristine to try and shrink vascular tumors.
In this research study, the study doctor will compare two different drugs to see which one will work better to help shrink your vascular tumor. One of the drugs is vincristine. Vincristine is approved by the Food and Drug Administration (FDA) to treat people with cancer. Vincristine is used to stop the abnormal cells from growing such as cells that make up blood vessels.
The other drug to be used in this study is sirolimus. Sirolimus is currently approved by the Food and Drug Administration (FDA) to prevent transplanted organ rejection. Sirolimus is not approved by the FDA for treatment of vascular abnormalities and is considered experimental. Sirolimus belongs to a class of drugs call 'mTOR inhibitors'. mTOR (mammilian target of rapamycin) helps cells to grow and may also help blood vessels to grow in a more normal fashion. Sirolimus is currently being tested in patients with vascular tumors and cancer. In vascular tumors, we hope sirolimus will stop the blood vessel growth.
Funding Source: FDA - OOPD (Office of Orphan Products Development)
Detailed description
Kaposiform hemangioendotheliomas (KHE) are extremely rare life threatening tumors which can be associated with Kasabach-Merritt Phenomenon consisting of profound thrombocytopenia and hypofibrinogenemia causing a significant risk of bleeding and an associated mortality rate as high as 20% to 30%. Despite the severity of potential complications, we lack uniform guidelines for the treatment and response to treatment of children and young adults with these tumors. KHE patients have been treated with a multitude of aggressive drug regimens without prospective evaluation of response or safety. Presently, vincristine is considered the standard of practice. We have treated a subset of these patients on study SIR-DA-0901 (FDA Grant# 5RO1FD003712-01). This study is a phase II trial assessing the efficacy and safety of sirolimus for the treatment of complicated vascular anomalies. Although the numbers are small, the response has been extremely promising with excellent tolerability. There is pre-clinical and clinical data supporting the essential regulatory function of the PI3 kinase/AKT/mTOR pathway in vascular growth and organization which suggests a therapeutic target for patients with complicated vascular anomalies. The overall goal of this trial is to objectively assess the efficacy of sirolimus compared to vincristine for the treatment of patients with high risk KHE.
Hypothesis: Sirolimus treatment for children and young adults with Kaposiform hemangioendotheliomas will be more effective than vincristine, assessed by time to response in an induction period and provide equivalent safety parameters.
Study Rationale We propose a multi-center, phase II trial with participation from 8 sites. The study will consist of two phases. The first of these is an initial induction phase in which vincristine and steroids will be compared to sirolimus and steroids. Response in the induction phase will be assessed as time to hematologic response. At the end of induction phase, cross over can occur if there is failure to respond. Part 2 is a maintenance phase which will be 1 year in length. Continued safety and efficacy data will be collected during maintenance and there will be cross over at any time for patients who lose their response following induction. Failure will be defined as worsening of hematological parameters on two separate laboratory evaluations at any time during maintenance or if they meet the definition of progressive disease following response assessments. Formal response in maintenance will be evaluated by imaging studies, functional assessment, and quality of life as per study SIR-DA-0901. Present therapies are very limited and new therapies are desperately needed for this devastating disease. Based on our preliminary data, there is a very good rationale for sirolimus therapy in KHE patients and so a phase II trial is urgently needed to determine if this therapy is to become the new standard of care for KHE patients.
Our secondary aims will be addressing biomarker analysis. There are limited studies describing the biology of these tumors. Per study SIR-DA-0901 there is some preliminary data indicating the importance of VEGF-C and other upregulated markers in the mTor pathway. This needs to be further investigated especially in KHE patients. Furthermore there are no clear objective measurements to determine response data.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Vincristine | Active comparator | Drug: VincristineInduction phase: participants assigned to vincristine will receive vincristine weekly at a dose of 0.05mg/kg/dose IV (for participants less than 10kg) or a dose of 1.5 mg/m2/dose (for participants greater than 10kg) for 2 months. Maintenance: if participants continue to receive vincristine weekly for 2 months, every 2 weeks for 5 months and every 3 weeks for 5 months. |
| Sirolimus | Experimental | Drug: SirolimusSirolimus will be administered at a dose of 0.8mg/m2/dose twice a day on a continuous dosing schedule throughout the trial for participants randomized to Sirolimus or for participants who fail vincristine may cross-over to the sirolimus arm. Sirolimus trough levels will be maintained between 10-15 ng/ml. |
Who may qualify
- Ages
- Up to 31 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (28 listed)
- Diagnosis: All patients must have one of the following vascular anomalies as determined by clinical, radiologic and histologic criteria (when possible). Biopsy strongly recommended (but not required) with suggested immunostains: CD34, PROX-1 or D240, Glut-1 and MIB-1.
- High Risk Stratification: In addition to the above diagnosis, all of the following criteria need to be met:
- Kasabach Merritt Syndrome defined at a platelet counts less than 50,000 K/µl and/or fibrinogen level < 100 mg/dl at the time of diagnosis.
- Age: Patients must be 0 - 31 years of age at the time of study entry. Enrollment includes patients of both genders and all ethnic groups.
- Organ function requirements:
Key exclusion criteria (9 listed)
- Concurrent severe and/or uncontrolled medical disease that could compromise participation in the study (e.g. uncontrolled diabetes, uncontrolled hypertension, severe infection, severe malnutrition, chronic liver or renal disease, active upper GI tract ulceration).
- Patients who require medications that are strong inhibitors/inducers CYP3A4 enzyme activity, including anticonvulsants, (Appendix II) to control concurrent medical conditions are not eligible. Patients who discontinue use of prohibited medications with a one week washout prior to start of study treatment are eligible.
- Known history of HIV seropositivity or known immunodeficiency. Testing is not required unless a condition is suspected.
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of sirolimus (e.g. ulcerative disease, uncontrolled nausea, vomiting, diarrhea, malabsorption syndrome or small bowel resection). A gastric tube or nasogastric tube is allowed.
- Females who are pregnant or breast feeding.
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 |
|---|---|---|
| VincristineAlso: Oncovin, Vincasar® PFS, VincrexVincristine dose dependent upon weight. Weekly for 2 months (Induction); Weekly 2 months; every 2 weeks for next 5 months; every 3 weeks for 5 months (Maintenance) | Drug | Vincristine |
| SirolimusAlso: RapamuneContinuous dosing to maintain trough level of 10-15ng/ml. | Drug | Sirolimus |
Primary outcome
Change in Hematologic Parameters
Time frame: 2 months
Hematologic parameters are defined as a platelet count greater than 100,000/uL (or a 2 times increase in platelet count compared to baseline) and a fibrinogen level greater than 150mg/dl.
Number of Serious and Non-Serious Adverse Events
Time frame: 2 months; 12 months
Serious and non-serious adverse events will be recorded for all participants and graded using CTCAE v4.0 (Common Toxicity Criteria for Adverse Effects). Adverse event rates will be calculated for both sirolimus and vincristine.
4 secondary outcomes
Evaluation of Disease Response - Maintenance
Time frame: 6 months; 12 months
Number of Serious and Non Serious Adverse Events - Maintenance
Time frame: 6 months; 12 months
Change in the Serum Levels of KHE Biomarkers
Time frame: Baseline, 2 months, 6 months, and 12 months
Identify Genetic Variants in Drug Metabolism Enzymes.
Time frame: Baseline
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 21 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Cincinnati Children's Hospital Medical Center Hemangioma and Vascular Malformation CenterLink provided by the sponsor.
- National Organization of Vascular AnomaliesLink provided by the sponsor.
- Individual participant dataSharing planned: No
Related trials for Kaposiform Hemangioendothelioma (KHE)
RecruitingPhase 2/3
Low- vs High-Dose Sirolimus With Prednisolone for KHE and KMP
Kaposiform Hemangioendothelioma (KHE), Kasabach Merritt Phenomenon
RecruitingObservational study
Identification of Biomarkers for Patients with Vascular Anomalies
Vascular Anomaly, Generalized Lymphatic Anomaly
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.
Denise Adams, MD principal investigator
Boston Children's Hospital
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vincristine and Sirolimus in people with Kaposiform Hemangioendothelioma (KHE) and Kasabach-Merritt Syndrome, run by Boston Children's Hospital at 7 sites in United States.
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 28 inclusion and 9 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: stopped early, as of the sponsor's last update on 21 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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 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?
Yes. Results are posted in the registry (first posted 24 May 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Hematologic Parameters (time frame: 2 months). The study lists 2 primary outcomes in all. 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 21 Sep 2023.