NCT03781050

Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome

• Status: Unknown
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 10 (estimated)
• Sponsor: Peking Union Medical College Hospital · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

• Peutz-Jeghers Syndrome

Interventions

Type	Name	Description
DRUG	Rapamycin	For children: rapamycin, 1 mg per square meter of body surface area a day, orally, for at least 6 months For adults: rapamycin, 2 mg a day, orally, for at least 6 months

Timeline

• Start date: 2018-09-16
• Primary completion: 2022-01-01
• Completion: 2022-07-01
• First posted: 2018-12-19
• Last updated: 2019-01-23

Locations

1 site across 1 country: China