5 year study of 6 children each year with vascular anomalies who have severe complications failing to respond to the usual treatments will be offered a new use of an old medication- sirolimus

Phase 1

Recruiting

• Conditions: Complex vascular malformations; Complex lymphatic malformations; Vascular tumours (tufted angiomas and kaposiform haemangioendotheliomas); Generalised lymphatic anomalies; PIKC3A related overgrowth syndrome (PROS); Cardiovascular - Diseases of the vasculature and circulation including the lymphatic system

• Registration Number: ACTRN12616000943448
• Lead Sponsor: Sydney Children's Hospital, Randwick

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-07-15
• Last Update Posted: 13 January 2020

Recruitment & Eligibility

• Status: Recruiting
• Sex: All
• Target Recruitment: 30

Inclusion Criteria

Inclusion criteria include children in the age group of zero to eighteen years who have complex vascular anomalies. Examples include children with the following:
1. Venous Malformations with localised intravascular coagulopathy or severe pain failing to respond to sclerotherapy and /or surgery;
2. Lymphatic Malformations which compromise the airway or vision or are causing a low albumin level;
3. Newly diagnosed Kaposiform haemangioendothelioma/tufted angioma: in view of the promising data from the US on the use of sirolimus as initial treatment for KHE (1,2,5), sirolimus will be given to newly diagnosed patients and the results compared to our historical controls, namely children who had received vincristine as initial therapy.
4. Relapsed Kaposiform haemangioendothelioma: children who have previously been successfully treated with vincristine therapy and then have a recurrence of their tumour will be treated with sirolimus;
5. Generalised Lymphatic Anomalies with bone erosions, chylous leak or infiltration of lungs and gastrointestinal tract;
6. localised lymphatic anomalies not amenable to an operation;
7. Children with various overgrowth syndromes who have benign tumours, asymmetry, pain and coagulopathy that have failed usual therapeutic options and are causing significant symptoms

Exclusion Criteria

Children who are severely immunocompromised will be excluded from the study.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Pain will be assesed by Wong - Baker FACES rating scale.[This will be assessed at baseline before sirolimus treatment and at 3 monthly intervals.];Coagulopathy will be assessed by regular blood coagulation profile studies, baseline prior to commencement of sirolimus, at weekly intervals during the first month, monthly for 3 months and then every 3 months throughout the period of treatment (12 months).<br>[12 months<br>This will be assessed by regular blood coagulation profile studies, baseline prior to commencement of sirolimus, at weekly intervals during the first month, monthly for 3 months and then every 3 months throughout the period of treatment (12 months).]

Secondary Outcome Measures

Name	Time	Method
MRI of the vascular anomalies will be performed pre-treatment, and every 6 months while the child is on sirolimus treatment (12 months) and until 1 year post end treatment<br>Size of the vascular anomaly will be monitored using MRI. <br>[1-2 years<br>MRI of the vascular anomalies will be performed pretreatment, and every 6 months while the child is on sirolimus treatment (12 months) and until 1 year post end treatment. ]