Neoplasms

All Conditions

Skin and Connective Tissue Diseases

Leiomyoma

Myofibroma

Leiomyomatosis

Neoplasms, Connective and Soft Tissue

Neoplasms by Histologic Type

Neoplasms, Connective Tissue

Connective Tissue Diseases

Anti-Infective Agents

Antineoplastic Agents

All Drugs and Chemicals

Sirolimus

Temsirolimus

MTOR Inhibitors

Anti-Bacterial Agents

Antibiotics, Antitubercular

Clotrimazole

Miconazole

Antifungal Agents

Immunosuppressive Agents

Immunologic Factors

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

rapamycin

Fengzh Feng, MD

Lan Zhu, MD

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.

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.

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

Network platform, and the website will be attached later

Peking Union Medical College Hospital

professor

Symptoms and General Pathology

Diseases and Abnormalities at or Before Birth

Digestive System Diseases

Rare Diseases

Syndrome

Congenital Abnormalities

Blister

Nevus, Pigmented

Nevus

Gastrointestinal Neoplasms

Digestive System Neoplasms

Skin Neoplasms

Nevus, Blue

Melanoma

Nevi and Melanomas

Gastrointestinal Diseases

Skin Diseases

Blue Rubber Bleb Nevus Syndrome

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

Rapamycin

Jiaolin Zhou, MD

A prospective, nonrandomized, open-label, single-arm clinical trial to study efficacy of rapamycin (sirolimus) in the treatment of Blue Rubber Bleb Nevus Syndrome, hereditary or sporadic venous malformation

Blue rubber bleb nevus syndrome (BRBNS) and venous malformation are mainly caused by somatic mutation of TEK and PIK3CA, which activates the PI3K/AKT signaling pathway. As an important protein kinase downstream of the PI3K/AKT pathway, mTOR can serve as a potential therapeutic target for BRBNS. Experiments of mice have shown that rapamycin inhibited the progression of venous malformation lesions. There are a few human cases reported using rapamycin treatment. The investigator's study is designed to be a prospective, nonrandomized, open-label, single-arm clinical trial to investigate its efficacy and safety.

Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation

Efficacy of Rapamycin (Sirolimus) in the Treatment of Blue Rubber Bleb Nevus Syndrome, Hereditary or Sporadic Venous Malformation

lesion load (cm2) = A + B + C. A = sum of the product of maximum diameter and maximum height of the largest 3 lesions shown by chest, abdomen and pelvis MRI or small bowel CT reconstruction (in cm2) B = sum of the product of maximum diameter and maximum height of the largest 3 lesions shown by digestive endoscope (in cm2) C = sum of the product of maximum diameter and maximum height of the largest 3 lesions shown by ultrasound (in cm2) Remarks: 1. If it is impossible to evaluate 3 or more lesions, results of the actual number of lesions should be taken as valid; 2. Lesions evaluated should be correspondent before and after treatment. If the lesion is difficult to assess after treatment, it should be ruled out from the assessment.

The value indicates the amount of gastrointestinal bleeding.

The value indicates the amount of gastrointestinal bleeding

The value indicates the extent of local coagulation caused by Venous Malformation lesions.

Air Force General Hospital of the PLA

Chinese Academy of Medical Sciences

Gland and Hormone Related Diseases

Urinary Tract, Sexual Organs, and Pregnancy Conditions

Respiratory Tract (Lung and Bronchial) Diseases

Blood and Lymph Conditions

Immune System Diseases

Breast Neoplasms

Pancreatic Neoplasms

Prostatic Neoplasms

Thyroid Neoplasms

Thyroid Diseases

Stomach Neoplasms

Head and Neck Neoplasms

Carcinoma, Renal Cell

Neoplasm Metastasis

Lung Neoplasms

Ovarian Neoplasms

Carcinoma, Ovarian Epithelial

Lymphoma

Urinary Bladder Neoplasms

Liver Neoplasms

Mesothelioma

Mesothelioma, Malignant

Kidney Neoplasms

Neoplastic Processes

Pancreatic Cancer

Ovarian Cancer

Renal Cell Carcinoma

Stomach Cancer

Ovarian Epithelial Cancer

Lymphosarcoma

Malignant Mesothelioma

Hematinics

Gastrointestinal Agents

Herbal and Botanical

Liver Extracts

Pancrelipase

Pancreatin

Grapefruit Seed Extract

Weekly oral doses, dose is assigned at the time of study entry

Rapamycin (sirolimus)

Daily oral doses starting during the second week on study.

Grapefruit Juice

Ezra W Cohen, MD

The purpose of this study is to determine the highest safe dose of rapamycin when given with a fixed amount of grapefruit juice.

Rapamycin With Grapefruit Juice for Advanced Malignancies

A Phase Ib Study Administering Rapamycin (Sirolimus) With Grapefruit Juice in Patients With Advanced Malignancies

University of Chicago

Study title	NCT ID	Conditions	Interventions	Eligibility	Organization
Efficacy and Safety of Rapamycin (Sirolimus) in the Treatment of Symptomatic Uterine Fibroids and Leiomyomatosis	NCT03500367	Uterine Fibroid...	rapamycin	18 Years - 50 Years	Peking Union Medical College Hospital
Efficacy and Safety of Rapamycin (Sirolimus) in the Treatment of Symptomatic Uterine Fibroids and Leiomyomatosis	NCT03500367	Uterine Fibroid...	rapamycin	18 Years - 50 Years	Peking Union Medical College Hospital
Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation	NCT03767660	Blue Rubber Ble... Venous Malforma...	Rapamycin	-	Peking Union Medical College Hospital
Rapamycin With Grapefruit Juice for Advanced Malignancies	NCT00375245	Tumors Neoplasm Metast...	Rapamycin (siro... Grapefruit Juic...	18 Years -	University of Chicago

Spots Global Cancer Trial Database for rapamycin (sirolimus)