SIPA-SOS (segmental overgrowth syndrome)
An open label phase II study of sirolimus in patients with segmental overgrowth syndrome (SIPA-SOS)
Rekrutierung beendet
Summary
The study aim is to assess the effect of sirolimus to reduce the size of defined target lesions in patients with segmental overgrowth syndrome until 6 months after start of therapy.
Main inclusion criteria
- Male or female patients aged ≥ 3 years (no upper limit)
- Segmental overgrowth syndrome patients independently of genetic background. These diagnoses include patients with CLOVES syndrome, Klippel-Trenaunay-Syndrome and other PIK3CA related overgrowth spectrum diseases, Proteus syndrome, PTEN hamartoma tumor syndromes including patients with PTEN hamartoma of soft tissue (PHOST), vascular malformations with significant overgrowth
- Identification of at least one measurable target lesion
Sponsor
Universitätsklinikum Freiburg in cooperation with Pfizer GmbH, Germany