Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome (NCT03781050) | Clinical Trial Compass
UnknownPhase 4
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
China10 participantsStarted 2018-09-16
Plain-language 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.
Who can participate
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Patients are diagnosed with PJS.
* Patients have gastrointestinal polyps related syndromes, including abdominal pain, abdominal distension, gastrointestinal bleeding, etc, with imageological examination suggesting intestinal obstruction or intussusception; or whose symptoms recur after previous digestive endoscopic treatment and surgery; or who are inappropriate or unwilling to accept the above treatment again and wish to receive pharmacotherapy.
* Conventional treatment didn't work well in patients combined with PJS-related tumors.
* Physical condition (ECGO): 0\~3
* Organ function is good and biochemical indices meet the following conditions:
* AST≤2.5×upper limit of normal value (ULN),
* ALT≤2.5×upper limit of normal value (ULN),
* Serum total bilirubin (TSB)≤1.5×upper limit of normal value (ULN),
* Creatinine≤1.5×upper limit of normal value (ULN).
* No other medications have been received for intestinal polyps within 3 months prior to the clinical trial.
* Patients participate in the trial voluntarily and have signed the informed consent by the participant or his/her legal guardian.
Exclusion Criteria:
* Patients underwent a surgery within 2 weeks.
* Patients may need emergency surgery in the near future.
* Patients are allergic to any ingredient of rapamycin.
* Patients suffer from a disease requiring immediate blood transfusion.
* Patients suffer from any disease or condition that may impact implementation of the study or interpretation…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps
Timeframe: The time from start of therapy to 1 year