Use of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients

May 31, 2024 updated by: Yi Ji, West China Hospital

Efficacy and Safety of Different Concentrations of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients

Bleomycin has nowadays been more and more widely used in the sclerotherapy of LMs, which has been proven to be primarily dose dependent. The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of LMs for pediatric patients.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

Lymphatic malformations (LMs) are vascular anomalies that arise from abnormal embryonic development of the lymphatic system and might present as dilated lymphatic channels or cysts lined by lymphatic endothelial cells. With an estimated incidence of approximately 1/4000-1/2000, LMs can occur at any site in the lymphatic system, in which head, neck and axilla were mostly detected and have been reported to account for over 75%. Based on the location and size of the lesion and the extent of involvement, LMs may be asymptomatic with incidental detection, or chronic abdominal pain and distension due to their compression of surrounding structures, or critical and even fatal secondary to their volvulus, hemorrhage, infection and rupture. Surgical excision is a definitive treatment for LMs, while it may be difficult at times because of the infiltrative nature of the lesions, leading to a high incidence of complications like vital organ injuries, nerve injuries, bleeding, infection scar formation, and recurrences. Sclerotherapy is a simpler alternative to tedious surgical excision treatment for LMs and avoids the complications related to surgery. As an anticancer drug extracted from Streptomyces verticillus, Bleomycin has been more and more widely used in the sclerotherapy of LMs for pediatric patients, which has been proven to be primarily dose dependent. However, the optimum concentration of Bleomycin in the sclerotherapy of LMs for pediatric patients has not been strictly validated, due to the lack of high-quality RCT studies. The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of LMs for pediatric patients.

Study Type

Interventional

Enrollment (Estimated)

200

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • China
    • Sichuan
      • Chengdu, Sichuan, China, 610041
        • Recruiting
        • West China Hospital of Sichuan University
        • Contact:
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  • Male or female participants less than 14 years of age at the time of informed consent/assent form was signed.
  • Participants whose parents have voluntarily given written consent and participants who provided assent (if applicable) after the study has been explained to them.
  • Participants with LMs of all sites measured and confirmed via imaging at screening, with rapid progression, resluting in obvious symptoms or dysfunction, which could not be radically resected and could be treated by sclerotherapy.

Exclusion Criteria:

  • Penicillin allergy.
  • Vascular tumors or combined vascular malformations.
  • Participants who may have had surgical or sclerotherapy treatment by other hardeners.
  • LMs growing slowly, without obvious symptoms or dysfunction, which does not need to be treated prematurely.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Double

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Low-dose Concentrations (1mg/ml) of Bleomycin
In this arm, patients with lymphatic malformations were treated by intracapsular injection with low-dose concentrations (1mg/ml) of Bleomycin.
Drug: Bleomycin
To validated the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of lymphatic malformations for pediatric patients
Other Names:
  • Zeocin
Experimental: High-dose Concentrations (2mg/ml) of Bleomycin
In this arm, patients with lymphatic malformations were treated by intracapsular injection with high-dose concentrations (2mg/ml) of Bleomycin.
Drug: Bleomycin
To validated the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of lymphatic malformations for pediatric patients
Other Names:
  • Zeocin

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Changes of Volume
Time Frame: 3 to 6 months post-therapy
Changes of Volume is defined as follows: a complete (90%-100% reduction in LMs volume), substantial (60%-89% reduction in LMs volume), intermediate (20%-59% reduction in LMs volume), or no (< 20% reduction in LMs volume) response 3 to 6 months post-therapy as assessed by imaging.
3 to 6 months post-therapy

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Score of Pain
Time Frame: 3 to 6 months post-therapy
Score of Pain is selfassessed at each visit on a 0 to 10 visual analog scale (where 0 indicates no pain and 10 indicates the worst pain imaginable), reported along with period duration.
3 to 6 months post-therapy
Global Efficacy
Time Frame: 3 to 6 months post-therapy
Global efficacy is assessed at each visit beginning at MS by the physician and self-assessed by the participant and proxy (parents) on a 0 to 10 visual analog scale (where 0 indicates no efficacy and 10 indicates complete resolution).
3 to 6 months post-therapy
Score of Quality of Life
Time Frame: 3 to 6 months post-therapy
Score of Quality of Life is assessed by the validated Children-Dermatological Life Quality Index (C-DLQI).
3 to 6 months post-therapy
Number of Participants with Efficacy
Time Frame: 3 to 6 months post-therapy
Number of Participants with Efficacy was assessed by 2 independent experts.
3 to 6 months post-therapy
Number of Participants with Safety
Time Frame: 3 to 6 months post-therapy
Number of Participants with Safety was assessed based on physical signs and monitoring of imaging examinations or laboratory test.
3 to 6 months post-therapy

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

West China Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

March 8, 2023

Primary Completion (Estimated)

December 31, 2025

Study Completion (Estimated)

December 31, 2026

Study Registration Dates

First Submitted

December 19, 2023

First Submitted That Met QC Criteria

May 25, 2024

First Posted (Actual)

May 31, 2024

Study Record Updates

Last Update Posted (Actual)

June 3, 2024

Last Update Submitted That Met QC Criteria

May 31, 2024

Last Verified

May 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 2023-12-19

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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