The safety and effectiveness of rapamycin (sirolimus) in the treatment of Prader-Willi syndrome

Status

Recruiting

Phases

Early Phase 1

Study type

Interventional

Source

ChiCTR

Registry ID

ChiCTR2000040947

Enrollment

Unknown

Registered

2020-12-16

Start date

2021-01-01

Completion date

Unknown

Last updated

2021-04-12

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Prader-Willi syndrome

Interventions

rapamycin group:rapamycin 1mg qd po

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: (1) Patients with genetically diagnosed Prader Willi syndrome; (2) No liver and kidney failure; (3) Voluntarily participate in the trial and sign the informed consent form.

Exclusion criteria

Exclusion criteria: (1) Complicated infection or trauma; (2) use of weight loss drugs; (3) inability to cooperate; (4) unable to conduct cognitive function assessment; (5) allergy to rapamycin; (6) patients who participated at the same time or within one month in another clinical trial that may affect the purpose of this study; (7) other reasons are not suitable for this research.

Design outcomes

Primary

Measure	Time frame
Body composition;Clinical global impression;Achenbach Child Behavior Checklist;	—

Secondary

Measure	Time frame
BMI SDS;Homeostatic Model Assessment of Insulin Resistance;Blood lipids;	—

Countries

China

Contacts

Public ContactWu Xueyan

Peking Union Medical College Hospital

wsheyan@vip.sina.com+86 13901397470

Outcome results

None listed

Source: ChiCTR (via WHO ICTRP) · Data processed: Feb 4, 2026