Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04921722
Recruitment Status : Recruiting
First Posted : June 10, 2021
Last Update Posted : July 29, 2021
Sponsor:
Information provided by (Responsible Party):
Children's Hospital of Fudan University

Brief Summary:
In this study, we investigate the safety and efficacy of topical sirolimus in the treatment of superficial complicated vascular anomolies.

Condition or disease Intervention/treatment Phase
Kaposiform Hemangioendothelioma Tufted Angioma Superficial Vascular Anomalies Superficial Lymphatic Malformations Drug: Percutaneous sirolimus Drug: Oral sirolimus Phase 4

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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 75 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Single (Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Percutaneous Administration of Sirolimus in the Treatment of Superficial Complicated Vascular Anomalies: a Randomized Controlled Trial
Estimated Study Start Date : August 30, 2021
Estimated Primary Completion Date : November 1, 2023
Estimated Study Completion Date : November 1, 2024


Arm Intervention/treatment
Experimental: Topical use of 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: Percutaneous sirolimus
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.
Other Name: Rapamycin

Active Comparator: Oral use of 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 sirolimus
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.
Other Name: Rapamycin




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. Changes of resistance coefficient [ Time Frame: From admission to follow-up six months ]
    Measured by ultrosonic doppler flowmetery at follow-up

  2. Incidence of adverse events [ Time Frame: From admission to follow-up six months ]
    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

  3. Platelet count [ Time Frame: From admission to follow-up six months ]
    Platelet count is one of the major indicators of response to treatment. It is supposed to be greater than 100×10^9/L.

  4. Changes of peak blood flow [ Time Frame: From admission to follow-up six months ]
    Measured by ultrosonic doppler flowmetery at follow-up



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   up to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Participant clinically or pathologically diagnosed with KHE, TA or complicated superficial vascular anomolies involving lymphatic components.

    1. The case is initial, with a relatively limited superficial lesion.
    2. 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)

Exclusion Criteria:

  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04921722


Locations
Layout table for location information
China
Children's Hospital of Fudan University Recruiting
Shanghai, China, 210012
Contact: Kai Li, MD, PhD    +86 02164931114    likai2727@163.com   
Principal Investigator: Kai Li, MD, PhD         
Sub-Investigator: Weili Yan, MD,PhD         
Sub-Investigator: Ying Gong, MD,PhD         
Sub-Investigator: Wei Yao, MD, PhD         
Sub-Investigator: Zuopeng Wang, MD,PhD         
Sub-Investigator: Yingjing Ding, MD         
Sub-Investigator: Hanlei Yan, MD         
Sponsors and Collaborators
Children's Hospital of Fudan University
Layout table for additonal information
Responsible Party: Children's Hospital of Fudan University
ClinicalTrials.gov Identifier: NCT04921722    
Other Study ID Numbers: LK210106
First Posted: June 10, 2021    Key Record Dates
Last Update Posted: July 29, 2021
Last Verified: July 2021

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
Hemangioendothelioma
Lymphangioma
Vascular Malformations
Lymphatic Abnormalities
Congenital Abnormalities
Hemangioma
Neoplasms, Vascular Tissue
Neoplasms by Histologic Type
Neoplasms
Lymphatic Vessel Tumors
Lymphatic Diseases
Cardiovascular Abnormalities
Cardiovascular Diseases
Sirolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs