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NCT02428296 · ClinicalTrials.gov registry record · Phase 2
Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation
A Phase 2 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 2
- Development phase
- 39
- Enrollment target
NCT02428296 is a Phase 2 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 39 participants.
The verdict
NCT02428296, a Phase 2 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 39 participants
- Enrollment target
Study Summary
Background: \- PIK3CA-related overgrowth spectrum (PROS) is caused by changes in the PIK3CA gene. This gene makes a protein that communicates with other proteins in the body to cause cells to grow. Alterations in PIK3CA change the chemical signals in the body and cause overgrowth in fatty, vascular and other tissues. Sirolimus is a drug that reduces the signals sent by one of the proteins in this chemical signaling pathway. Researchers want to learn whether the drug sirolimus can reduce or stabilize some of the overgrowth that patients with PROS experience. Objectives: \- To measure how the overgrowth of patients with PROS changes over time and whether taking a drug called sirolimus can reduce or stabilize a person s overgrowth. Eligibility: \- People ages 3 to 65 years old with a confirmed mutation or alteration of the PIK3CA gene in the person s affected tissues (a somatic mutation). Design: * Participants will be screened with medical history and genetic counseling. * First 6 months: Participants will have their overgrowth monitored. * Next 6 months: Participants will take sirolimus once or twice a day. * Participants will have to visit the clinic several times, and stay in the area for 4 to 5 days each time. * Participants will have a one month-long visit to the clinic. * During clinic visits, participants will have: * Blood and urine tests. * Photographs of their physical features. * Scans, including an MRI and DEXA, and possibly x-rays and CT scans. * For the MRI and CT scans, participants will lie in a machine that takes pictures of their body. * The DEXA involves a small amount of radiation. * They may have: * Non-invasive heart function tests. * Lung function tests. * Participants will have several blood and urine tests between visits. * Participants will complete surveys and keep a diary of their treatment and side effects. * Participants may visit other health specialists or undergo other tests based on side effects. * One month after stopping the
Interventions
- DRUG Sirolimus
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 39 participants |
| Start Date | 2015-04-23 |
| Est. Completion | 2018-02-14 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02428296
The ClinicalTrials.gov registry entry for NCT02428296 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 39 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Human Genome Research Institute (NHGRI), which has 185 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Sirolimus is the first listed.
NCT02428296 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT02428296 about?
NCT02428296 is a clinical study titled "Study of Sirolimus Therapy for Segmental Overgrowth Caused by Somatic PI3K Activation". Background: \- PIK3CA-related overgrowth spectrum (PROS) is caused by changes in the PIK3CA gene. This gene makes a protein that communicates with other proteins in the body to cause cells to grow. Alterations in PIK3CA change the chemical signals in the body and cause overgrowth in fatty, vascular...
What is the current status of trial NCT02428296?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 39 participants. The study started on 2015-04-23. Estimated completion is 2018-02-14.
What interventions are being tested in trial NCT02428296?
The interventions under investigation include: Sirolimus (DRUG).
Who is sponsoring clinical trial NCT02428296?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
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