Uterine FibroidsPhase 4NCT03500367
Efficacy and Safety of Rapamycin (Sirolimus) in the Treatment of Symptomatic Uterine Fibroids and Leiomyomatosis
A Phase 4 study testing rapamycin in people with Uterine Fibroids, run by Peking Union Medical College Hospital at 1 site in China.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 50 years, women only
- Conditions the study is for
- Uterine Fibroids
- What is being tested?
- rapamycin
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 1 siteChina
- Study dates
- 1 Jan 2018to 31 Dec 2022
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- Women only
- No healthy volunteers
- Adult
Who may be able to join
- 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).
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- 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).
- Patients have signed the informed consent.
Who cannot join
- 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
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- 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
Trial locations and contacts
1 site in 1 country.
Lan Zhu, MD contact
China1 site, 1 recruiting
- Recruiting
Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
Beijing, China/Beiing, 100000
Fengzh Feng, MD, 18612671869 ext. +86, fengfzh@pumch.cn
About this study
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.
Read the detailed description
Recent studies indicate that mTOR pathway is a specific and sensitive tumor target molecule in uterine fibroids, which can be used as a target molecule for interventional therapy and can provide a new cut-in point for nonoperative treatment. However, the application of mTOR inhibitor (sirolimus) in the treatment of uterine fibroids remains blank. Our study was designed as a prospective non-randomized open label clinical trial to research its efficacy and safety.
- Conditions
- Uterine Fibroids
- Being tested
- rapamycin
- Keywords
- mammilian target of rapamycin inhibitor, Rapamycin (sirolimus), Treatment, Complications
- Medical subject headings
- Leiomyoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rapamycinDrug
rapamycin, 2 mg a day, orally ,for 3months
| Study group | Role | What they receive |
|---|---|---|
| rapamycin | Experimental | rapamycin, 2 mg a day, orally ,for 3months Receives: Drug: rapamycin |
What is being measured
The primary outcome is the main question the study is designed to answer.
size of uterus and the myoma
efficacy assessment
Measured: 2 day
Secondary outcomes (4)
hormone levels
ovarian function assessment
Measured: 2 day
menstrual blood volume / haemorrhage
symptom assessment
Measured: 1 day
adverse event
safety assessment
Measured: 2 day
follicular size
safety assessment
Measured: 2 day
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Apr 2018
- Study start1 Jan 2018
- Primary completion30 Jun 2022 (estimated)
- Study completion31 Dec 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (6)Papers the sponsor has linked to this study.
- Other trials for Uterine FibroidsStudies of the same condition, open and under way.
- More trials run by Peking Union Medical College HospitalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Peking Union Medical College Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, zhulan, Peking Union Medical College Hospital
- Investigators
- Lan Zhu, MD, Principal investigator, Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03500367
- Sponsor's protocol ID
- LPMS201802
- Status last verified
- Apr 2018
- First submitted
- 10 Mar 2018
- Last updated
- 18 Apr 2018
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Network platform, and the website will be attached later
Publications
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.
PubMed 26588841Prizant 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.
PubMed 23820898Kashani 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.
PubMed 26796059Suo 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.
PubMed 24784716Prizant 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.
PubMed 27409646Zhang 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.
PubMed 38481209
Study at a glance
From the registry record, last updated by the sponsor on 18 Apr 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Efficacy and Safety of Rapamycin (Sirolimus) in the Treatment of Symptomatic Uterine Fibroids and Leiomyomatosis
- ClinicalTrials.gov ID
- NCT03500367
- Acronym / sponsor ID
- LPMS201802
- Conditions
- Uterine Fibroids
- Keywords
- mammilian target of rapamycin inhibitor, Rapamycin (sirolimus), Treatment, Complications
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Peking Union Medical College HospitalUniversity, hospital or other
- Study start
- 1 Jan 2018 (actual)
- Primary completion
- 30 Jun 2022 (estimated)
- Study completion
- 31 Dec 2022 (estimated)
- First posted
- 18 Apr 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Recent studies indicate that mTOR pathway is a specific and sensitive tumor target molecule in uterine fibroids, which can be used as a target molecule for interventional therapy and can provide a new cut-in point for nonoperative treatment. However, the application of mTOR inhibitor (sirolimus) in the treatment of uterine fibroids remains blank. Our study was designed as a prospective non-randomized open label clinical trial to research its efficacy and safety.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rapamycin | Experimental | Drug: rapamycinrapamycin, 2 mg a day, orally ,for 3months |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
Key inclusion criteria (16 listed)
- 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:
- 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.
Key exclusion criteria (12 listed)
- 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:
- Alcohol or drug (such as aperient) abuse
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rapamycinrapamycin, 2 mg a day, orally ,for 3months | Drug | rapamycin |
Primary outcome
size of uterus and the myoma
Time frame: 2 day
efficacy assessment
4 secondary outcomes
hormone levels
Time frame: 2 day
menstrual blood volume / haemorrhage
Time frame: 1 day
adverse event
Time frame: 2 day
follicular size
Time frame: 2 day
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Apr 2018.
- Background: PubMed 26588841Fritsch 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.
- Background: PubMed 23820898Prizant 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.
- Background: PubMed 26796059Kashani 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.
- Background: PubMed 24784716Suo 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.
- Background: PubMed 27409646Prizant 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.
- Derived from this study: PubMed 38481209Zhang 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.
- Xenograft Model for Predicative Study of Uterine Fibroid DiseaseLink provided by the sponsor.
- Medical Management for Uterine LeiomyomasLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Network platform, and the website will be attached later
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Lan Zhu, MD contact
Lan Zhu, MD principal investigator
Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing rapamycin in people with Uterine Fibroids, run by Peking Union Medical College Hospital at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 16 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 18 Apr 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Fengzh Feng, MD (18612671869 ext. +86, fengfzh@pumch.cn), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
size of uterus and the myoma (time frame: 2 day). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03500367, which also lists 6 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Apr 2018.