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Sirolimus Effect on Hypertrophic Syndromes Related Gene PIK3CA

NCT02443818 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 16

Last updated 2025-11-21

No results posted yet for this study

Summary

It was found that people who have an excessive growth in one or more parts of their body and normal growth in other parts, may have a change in genes responsible for controlling cell growth. This genetic discovery identified a possible treatment for this overgrowth, called Sirolimus.

This is a pilot study, which help the investigators prepare a larger international study, which will aim to determine whether a treatment based Sirolimus is able to stop the excessive growth of one or more parties of their body in people who carry a genetic change in the PIK3CA gene (phosphatidylinosilol-3-kinase encoded by the gene PIK3CA).

Conditions

  • Segmental Overgrowth Disorders Due to PIK3CA

Interventions

DRUG

Sirolimus

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

Sponsors & Collaborators

  • Centre Hospitalier Universitaire Dijon

    lead OTHER

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
3 Years
Max Age
65 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-05-31
Primary Completion
2016-11-30
Completion
2016-11-30

Countries

  • France

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02443818 on ClinicalTrials.gov