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Different Doses of Sirolimus for the Treatment of Cystic Lymphatic Malformations

ConditionLymphatic Malformation
Trial statusRecruiting
Trial phasePhase 2, Phase 3
Trial typeInterventional
Biological sexAll
Age1-18
SponsorWest China Hospital

About this trial

The purpose of this study is to compare the efficacy and safety of different concentration gradients of sirolimus in the treatment of cystic lymphatic malformation.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Male and female;

Between 0 and 18 years of age;

Disqualifiers

Patients contraindicated for the administration of sirolimus (e.g., those with an allergy to sirolimus or other rapamycin analog)

Exposure to chemotherapy, embolization, corticosteroids, propranolol, sclerotherapy or any other investigational agents within 1 weeks before enrolment on study;

Patients had a history of a major surgery within 2 weeks before enrollment;

Patients who have a history of treatment with sirolimus or other mTOR inhibitor;

Trial design

Design model

Parallel

Treatments tested in this trial

  • Sirolimus (RAPAMUNE)

    Drug

    Use of different doses of the same drug

Treatment groups

150 Participants
are divided into 2 treatment groups
Group A: Low dose of sirolimusExperimental treatment 1 intervention
Group B: High dose of sirolimusActive comparator 1 intervention

Trial outcomes

Primary outcomes

1

The proportion of patients achieving an objective response at month 12

Objective response was defined as ≥20% reduction in LM volume compared to that at baseline.

Time frame
12 months

Secondary outcomes

1

The proportion of patients achieving an objective response at month 6

Objective response was defined as ≥20% reduction in LM volume compared to that at baseline.

Time frame
6 months
2

lesion responses

The primary endpoint was classified as follow: -Complete involution: 100% resolution of the measured LM; -Nearly complete involution was defined as decrease of ≥75% and \<100%; -Partial involution was defined as decrease of ≥20% and \<75%; -No change was defined as \<20% increase and \<20% decrease in the volumes of LM lesions; -Further growth was defined as ≥20% increase in the volume of index LM compared with the baseline volume measured. Lesion responses were overall lesion response rate and good lesion response rate. Overall lesion response comprised complete, nearly complete and partial involutions. Good lesion response comprised complete and nearly complete involutions.

Time frame
6 and 12 months
3

Quality of life (QOL) in patients

Pediatric Quality of Life Inventory (PedsQLTM) 4.0 Genetic Core Infant Scales (\<2 years) or Pediatric Quality of Life Inventory (PedsQLTM) 4.0 Genetic Core Scales (2-18 years) were used.

Time frame
12 months
4

Disease sequelae

Disease sequelae were assessed by site investigators at month 12. The site investigators assessed patients' extremity swelling (if any), general physical activity and exercise levels.

Time frame
12 months

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

West China Hospital

Lead sponsor

Sichuan University

Sponsor institution

Source ClinicalTrials.gov
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