Efficacy and Safety of Rapamycin (Sirolimus) in the Treatment of Symptomatic Uterine Fibroids and Leiomyomatosis

NCT03500367 | Unknown Phase 4

• 1 other identifier: LPMS201802
• Study Type: interventional
• Target: 25
• Locations: 1 country
• Sites: 1

Brief Summary

A prospective non-randomized open label clinical trial to research the efficacy and safety of sirolimus in patients with symptomatic, recurrent uterine fibroids or/and various rare leiomyoma.

Conditions

Uterine Fibroids

Keywords

mammilian target of rapamycin inhibitor; Rapamycin (sirolimus); Treatment; Complications

Outcome Measures

Primary Outcomes (1)

• size of uterus and the myoma

  efficacy assessment

  2 day

Secondary Outcomes (4)

• hormone levels

  2 day

• menstrual blood volume / haemorrhage

  1 day

• adverse event

  2 day

• follicular size

  2 day

Study Arms (1)

rapamycin

EXPERIMENTAL

rapamycin, 2 mg a day, orally ,for 3months

Drug: rapamycin

Interventions

rapamycin DRUG

rapamycin, 2 mg a day, orally ,for 3months

rapamycin

Eligibility Criteria

• Age: 18 Years - 50 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Adult (18-64)

You may qualify if:

• Patients who suffer from menorrhagia and pressure symptoms and whose pelvic ultrasound or pelvic MRI indicates uterine fibroids; or who has underwent myomectomy but suffer from menorrhagia and pressure symptoms again with pelvic ultrasound or pelvic MRI indicating uterine fibroids; or whose other imageological examinations or established surgical diagnosis indicate various rare types of myomatosis and who expect a drug therapy.
• Symptoms of uterine fibroids can be confirmed if one or multiple symptoms below exist:
• MP shows an excessive amount of bleeding during menstruation (\>80.0 mL)
• A subject report shows three excessive amounts of bleeding during menstruation in latest six months
• A subject report indicates pelvic pressure symptoms/pain that are likely related to uterine fibroids.
• According to results of medical history, physical examinations, gynecological examinations and laboratory examinations, patients are in a good overall condition (except uterine fibroids).
• Cervical smears show a normal result or have no clinical significance, where further follow-up is unnecessary. If there's a normal result in the latest six month in the medical record of a subject, the subject can pass the inspection of cervical smears. A HPV test can be applied to subjects with equivocal ASCUS as an auxiliary test. ASCUS subjects who get a negative result of a HPV test can be included into this study.
• Endometrial biopsy should be conducted to eliminate non-endometrial lesions if necessary.
• Subjects didn't receive any drug therapy for uterine fibroids three months before the clinical test.
• Female adults have menstruation (\>18 years old) and are not during pregnancy and lactation.
• Subjects have good organ function and results of their biochemical examinations meet the following conditions:
• AST≤2.5×the upper limit of normal (ULN),
• ALT≤2.5×the upper limit of normal (ULN),
• Serum total bilirubin≤1.5×the upper limit of normal (ULN),
• Creatinine≤1.5×the upper limit of normal (ULN).

You may not qualify if:

• Patients are in a period of pregnancy and lactation (patients delivered, miscarried or breast-feed in three months before the treatment)
• Patients are allergic to any ingredient of the medicine
• Patients suffer from a disease requiring immediate blood transfusion
• Patients suffer from a disease that may impact implementation of the study or explanation of results. This type of diseases includes:
• Known severe blood coagulation disorders
• Known anemia that is not caused by HMB
• Known hemoglobinopathy
• Patients suffered or suffer from cancer of the uterus, cervical carcinoma, ovarian cancer or breast cancer 5) An ultrasonic examination shows one or multiple ovarian cysts with a diameter \>30 mm 6) Ovarian tumors or pelvic mass of unknown origin 7) Known or suspected endometrial polyp \>15 mm
• Alcohol or drug (such as aperient) abuse
• Undiagnosed abnormal bleeding of the reproductive system.
• Patients also participate in another clinical medicine study
• Patients took part in another clinical trial that may influence this study before this study

Sponsors & Collaborators

• Peking Union Medical College Hospital: lead

Study Sites (1)

Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences

Beijing, China/Beiing, 100000, China

RECRUITING

Related Publications (6)

• Fritsch M, Schmidt N, Groticke I, Frisk AL, Keator CS, Koch M, Slayden OD. Application of a Patient Derived Xenograft Model for Predicative Study of Uterine Fibroid Disease. PLoS One. 2015 Nov 20;10(11):e0142429. doi: 10.1371/journal.pone.0142429. eCollection 2015.

  PMID: 26588841 BACKGROUND

• Prizant H, Sen A, Light A, Cho SN, DeMayo FJ, Lydon JP, Hammes SR. Uterine-specific loss of Tsc2 leads to myometrial tumors in both the uterus and lungs. Mol Endocrinol. 2013 Sep;27(9):1403-14. doi: 10.1210/me.2013-1059. Epub 2013 Jul 2.

  PMID: 23820898 BACKGROUND

• Kashani BN, Centini G, Morelli SS, Weiss G, Petraglia F. Role of Medical Management for Uterine Leiomyomas. Best Pract Res Clin Obstet Gynaecol. 2016 Jul;34:85-103. doi: 10.1016/j.bpobgyn.2015.11.016. Epub 2015 Nov 25.

  PMID: 26796059 BACKGROUND

• Suo G, Sadarangani A, Tang W, Cowan BD, Wang JY. Telomerase expression abrogates rapamycin-induced irreversible growth arrest of uterine fibroid smooth muscle cells. Reprod Sci. 2014 Sep;21(9):1161-70. doi: 10.1177/1933719114532839. Epub 2014 Apr 30.

  PMID: 24784716 BACKGROUND

• Prizant H, Hammes SR. Minireview: Lymphangioleiomyomatosis (LAM): The "Other" Steroid-Sensitive Cancer. Endocrinology. 2016 Sep;157(9):3374-83. doi: 10.1210/en.2016-1395. Epub 2016 Jul 13.

  PMID: 27409646 BACKGROUND

• Zhang G, Fan R, Yang H, Su H, Yu X, Wang Y, Feng F, Zhu L. Safety and efficacy of sirolimus in recurrent intravenous leiomyomatosis, pulmonary benign metastatic leiomyomatosis, and leiomyomatosis peritonealis disseminata: a pilot study. BMC Med. 2024 Mar 13;22(1):119. doi: 10.1186/s12916-024-03344-9.

  PMID: 38481209 DERIVED

Related Links

• Xenograft Model for Predicative Study of Uterine Fibroid Disease
• Medical Management for Uterine Leiomyomas
• Telomerase expression abrogates rapamycin-induced irreversible growth arrest of uterine fibroid smooth muscle cells

MeSH Terms

Conditions

Leiomyoma

Interventions

Sirolimus

Condition Hierarchy (Ancestors)

Neoplasms, Muscle Tissue; Neoplasms, Connective and Soft Tissue; Neoplasms by Histologic Type; Neoplasms

Intervention Hierarchy (Ancestors)

Macrolides; Lactones; Organic Chemicals

Study Officials

• Lan Zhu, MD

  Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Fengzh Feng, MD

CONTACT

18612671869; fengfzh@pumch.cn

Lan Zhu, MD

CONTACT

zhu_julie@sina.com

Study Design

• Study Type: interventional
• Phase: phase 4
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: professor

Study Record Dates

• First Submitted: March 10, 2018
• First Posted: April 18, 2018
• Study Start: January 1, 2018
• Primary Completion: June 30, 2022
• Study Completion: December 31, 2022
• Last Updated: April 18, 2018

Record last verified: 2018-04

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, ANALYTIC CODE
• Time Frame: Within twelve months after the trial complete
• Access Criteria: Data access requests will be reviewed by an external independent review panel. Requestors will be required to sign a Data Access Agreement.

Locations

CN (1)