Ignite Creation Date:
2025-12-24 @ 4:54 PM
Ignite Modification Date:
2025-12-24 @ 4:54 PM
Study NCT ID:
NCT03781050
Status:
UNKNOWN
Last Update Posted:
2019-01-23
First Post:
2018-12-06
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
Sponsor:
Peking Union Medical College Hospital
Organization:
Study Overview
Official Title:
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
Status:
UNKNOWN
Status Verified Date:
2019-01
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
A prospective non-randomized open label single arm clinical trial to examine the efficacy and safety of sirolimus in patients with Peutz-Jeghers Syndrome.
Detailed Description:
PJS (Peutz-Jeghers Syndrome) is mostly caused by mutations in Lkb1 gene, which leads to an increased activity of mTOR pathway, thus making mTOR inhibitor (sirolimus) a promising candidate in treatment and prevention of PJS. The efficacy of sirolimus (rapamycin) on PJS has been demonstrated in mouse model, but no clinical trials have been reported. Our study was designed as a prospective non-randomized open label single arm clinical trial to examine its efficacy and safety.
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: