Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation

PHASE2CompletedINTERVENTIONAL
Enrollment

39

Participants

Timeline

Start Date

April 23, 2015

Primary Completion Date

February 14, 2018

Study Completion Date

February 14, 2018

Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)Growth DisorderGenetics
Interventions
DRUG

Sirolimus

Low dose sirolimus will be given in daily dosing to achieve trough levels of 2-6 ng/ ml.

Trial Locations (1)

20892

National Institutes of Health Clinical Center, 9000 Rockville Pike, Bethesda

All Listed Sponsors
lead

National Human Genome Research Institute (NHGRI)

NIH

NCT02428296 - Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation | Biotech Hunter | Biotech Hunter