Lymphatic MalformationPhase 2NCT06437158
Use of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients
A Phase 2 study testing Bleomycin in people with Lymphatic Malformation, run by West China Hospital at 1 site in China.
Trial summary
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. Read more about this study
- Who is it for?
- Ages Up to 14 years
- Conditions the study is for
- Lymphatic Malformation
- What is being tested?
- Bleomycin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 200 participants
- Where
- 1 siteChina
- Study dates
- 8 Mar 2023to 31 Dec 2026
- 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 Up to 14 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
West China Hospital of Sichuan University
Chengdu, Sichuan, 610041
Yi Ji, Ph.D., +8618980606865, jijiyuanyuan@163.com
Min Yang, M.D., +8615928411140, hx2014bsym@163.com
About this study
Official title: 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.
Read the 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.
- Conditions
- Lymphatic Malformation
- Being tested
- Bleomycin
- Keywords
- Lymphatic Malformation, Bleomycin, Sclerotherapy, Intracystic injection, High-dose concentrations, Low-dose concentrations
- Medical subject headings
- Lymphatic Abnormalities
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BleomycinDrug
Also called Zeocin
To validated the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of lymphatic malformations for pediatric patients
| Study group | Role | What they receive |
|---|---|---|
| Low-dose Concentrations (1mg/ml) of Bleomycin | Experimental | In this arm, patients with lymphatic malformations were treated by intracapsular injection with low-dose concentrations (1mg/ml) of Bleomycin. Receives: Drug: Bleomycin |
| High-dose Concentrations (2mg/ml) of Bleomycin | Experimental | In this arm, patients with lymphatic malformations were treated by intracapsular injection with high-dose concentrations (2mg/ml) of Bleomycin. Receives: Drug: Bleomycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes of Volume
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.
Measured: 3 to 6 months post-therapy
Secondary outcomes (5)
Score of Pain
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.
Measured: 3 to 6 months post-therapy
Global Efficacy
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).
Measured: 3 to 6 months post-therapy
Score of Quality of Life
Score of Quality of Life is assessed by the validated Children-Dermatological Life Quality Index (C-DLQI).
Measured: 3 to 6 months post-therapy
Number of Participants with Efficacy
Number of Participants with Efficacy was assessed by 2 independent experts.
Measured: 3 to 6 months post-therapy
Number of Participants with Safety
Number of Participants with Safety was assessed based on physical signs and monitoring of imaging examinations or laboratory test.
Measured: 3 to 6 months post-therapy
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted31 May 2024
- Study start8 Mar 2023
- Primary completion31 Dec 2025 (estimated)
- Study completion31 Dec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Lymphatic MalformationStudies of the same condition, open and under way.
- More trials run by West China 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
- Model description
- The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of lymphatic malformations for pediatric patients.
- Masking
- Double blind
- Who is masked
- Participant, Outcomes assessor
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- West China Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yi Ji, West China Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06437158
- Sponsor's protocol ID
- 2023-12-19
- Status last verified
- May 2024
- First submitted
- 19 Dec 2023
- Last updated
- 3 Jun 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Publications
Sun J, Wang C, Li J, Song D, Guo L. The efficacy of bleomycin sclerotherapy in the treatment of lymphatic malformations: a review and meta-analysis. Braz J Otorhinolaryngol. 2023 Jul-Aug;89(4):101285. doi: 10.1016/j.bjorl.2023.101285. Epub 2023 Jun 29.
PubMed 37423005De Maria L, De Sanctis P, Balakrishnan K, Tollefson M, Brinjikji W. Sclerotherapy for lymphatic malformations of head and neck: Systematic review and meta-analysis. J Vasc Surg Venous Lymphat Disord. 2020 Jan;8(1):154-164. doi: 10.1016/j.jvsv.2019.09.007. Epub 2019 Nov 14.
PubMed 31734224Wu Z, Zou Y, Fu R, Jin P, Yuan H. A nomogram for predicting sclerotherapy response for treatment of lymphatic malformations in children. Eur J Med Res. 2022 Oct 21;27(1):209. doi: 10.1186/s40001-022-00844-3.
PubMed 36271467
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Different Concentrations of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients
- ClinicalTrials.gov ID
- NCT06437158
- Acronym / sponsor ID
- 2023-12-19
- Conditions
- Lymphatic Malformation
- Keywords
- Lymphatic Malformation, Bleomycin, Sclerotherapy, Intracystic injection, High-dose concentrations, Low-dose concentrations
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 200 participants (estimated)
- Lead sponsor
- West China HospitalUniversity, hospital or other
- Study start
- 8 Mar 2023 (actual)
- Primary completion
- 31 Dec 2025 (estimated)
- Study completion
- 31 Dec 2026 (estimated)
- First posted
- 31 May 2024 (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.
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.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, outcomes assessor)
- Number of arms
- 2
- Model description
- The investigators aim to compare the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of lymphatic malformations for pediatric patients.
| Arm | Type | Receives |
|---|---|---|
| Low-dose Concentrations (1mg/ml) of Bleomycin | Experimental | Drug: BleomycinIn this arm, patients with lymphatic malformations were treated by intracapsular injection with low-dose concentrations (1mg/ml) of Bleomycin. |
| High-dose Concentrations (2mg/ml) of Bleomycin | Experimental | Drug: BleomycinIn this arm, patients with lymphatic malformations were treated by intracapsular injection with high-dose concentrations (2mg/ml) of Bleomycin. |
Who may qualify
- Ages
- Up to 14 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
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 |
|---|---|---|
| BleomycinAlso: ZeocinTo validated the efficacy and safety of different concentrations of Bleomycin in the sclerotherapy of lymphatic malformations for pediatric patients | Drug | High-dose Concentrations (2mg/ml) of Bleomycin, Low-dose Concentrations (1mg/ml) of Bleomycin |
Primary outcome
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.
5 secondary outcomes
Score of Pain
Time frame: 3 to 6 months post-therapy
Global Efficacy
Time frame: 3 to 6 months post-therapy
Score of Quality of Life
Time frame: 3 to 6 months post-therapy
Number of Participants with Efficacy
Time frame: 3 to 6 months post-therapy
Number of Participants with Safety
Time frame: 3 to 6 months post-therapy
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 3 Jun 2024.
- Results publication: PubMed 37423005Sun J, Wang C, Li J, Song D, Guo L. The efficacy of bleomycin sclerotherapy in the treatment of lymphatic malformations: a review and meta-analysis. Braz J Otorhinolaryngol. 2023 Jul-Aug;89(4):101285. doi: 10.1016/j.bjorl.2023.101285. Epub 2023 Jun 29.
- Results publication: PubMed 31734224De Maria L, De Sanctis P, Balakrishnan K, Tollefson M, Brinjikji W. Sclerotherapy for lymphatic malformations of head and neck: Systematic review and meta-analysis. J Vasc Surg Venous Lymphat Disord. 2020 Jan;8(1):154-164. doi: 10.1016/j.jvsv.2019.09.007. Epub 2019 Nov 14.
- Results publication: PubMed 36271467Wu Z, Zou Y, Fu R, Jin P, Yuan H. A nomogram for predicting sclerotherapy response for treatment of lymphatic malformations in children. Eur J Med Res. 2022 Oct 21;27(1):209. doi: 10.1186/s40001-022-00844-3.
- 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.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Bleomycin in people with Lymphatic Malformation, run by West China Hospital 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 3 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: looking for participants now, as of the sponsor's last update on 3 Jun 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 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 Yi Ji, Ph.D. (+8618980606865, jijiyuanyuan@163.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes of Volume (time frame: 3 to 6 months post-therapy). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06437158, which also lists 3 linked publications.
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 3 Jun 2024.