Ignite Creation Date:
2025-12-24 @ 4:54 PM
Ignite Modification Date:
2025-12-25 @ 2:37 PM
Study NCT ID:
NCT03781050
Status:
UNKNOWN
Last Update Posted:
2019-01-23
First Post:
2018-12-06
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D010580', 'term': 'Peutz-Jeghers Syndrome'}, {'id': 'D009477', 'term': 'Hereditary Sensory and Autonomic Neuropathies'}], 'ancestors': [{'id': 'D009386', 'term': 'Neoplastic Syndromes, Hereditary'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D044483', 'term': 'Intestinal Polyposis'}, {'id': 'D007410', 'term': 'Intestinal Diseases'}, {'id': 'D005767', 'term': 'Gastrointestinal Diseases'}, {'id': 'D004066', 'term': 'Digestive System Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D007911', 'term': 'Lentigo'}, {'id': 'D008548', 'term': 'Melanosis'}, {'id': 'D017495', 'term': 'Hyperpigmentation'}, {'id': 'D010859', 'term': 'Pigmentation Disorders'}, {'id': 'D012871', 'term': 'Skin Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D020123', 'term': 'Sirolimus'}], 'ancestors': [{'id': 'D018942', 'term': 'Macrolides'}, {'id': 'D007783', 'term': 'Lactones'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2018-12-05', 'size': 295712, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2018-12-06T20:36', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE4'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 10}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'RECRUITING', 'startDateStruct': {'date': '2018-09-16', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-01', 'completionDateStruct': {'date': '2022-07-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2019-01-18', 'studyFirstSubmitDate': '2018-12-06', 'studyFirstSubmitQcDate': '2018-12-17', 'lastUpdatePostDateStruct': {'date': '2019-01-23', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2018-12-19', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-01-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': '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', 'description': 'Lesion load (cm2) = A+B. A = sum of the product of maximum diameter and maximum height of the largest 3 lesions shown by 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). 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.'}], 'secondaryOutcomes': [{'measure': 'Concentration of hemoglobin in blood', 'timeFrame': 'The time from start of therapy to 1 year', 'description': 'The value indicates the amount of gastrointestinal bleeding.'}, {'measure': 'Concentration of albumin in blood', 'timeFrame': 'The time from start of therapy to 1 year', 'description': 'The value indicates the status of nutrition.'}, {'measure': 'Concentration of hsCRP in blood', 'timeFrame': 'The time from start of therapy to 1 year', 'description': 'The value indicates the level of inflammation.'}, {'measure': 'Visual analogue score (VAS)', 'timeFrame': 'The time from start of therapy to 1 year', 'description': 'The value indicates the level of pain.'}, {'measure': 'Dermatology life quality index (DLQI)', 'timeFrame': 'The time from start of therapy to 1 year', 'description': 'The value indicates the status of hyperpigmented macules on the lips and oral mucosa.'}, {'measure': 'Adverse events', 'timeFrame': 'The time from start of therapy to 1 year', 'description': 'To evaluate safety'}]}, 'oversightModule': {'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['Peutz-Jeghers Syndrome (PJS)', 'Rapamycin (sirolimus)', 'Mammalian target of rapamycin (mTOR) inhibitor', 'Treatment'], 'conditions': ['Peutz-Jeghers Syndrome']}, 'referencesModule': {'references': [{'pmid': '28653895', 'type': 'BACKGROUND', 'citation': 'Chen HY, Jin XW, Li BR, Zhu M, Li J, Mao GP, Zhang YF, Ning SB. Cancer risk in patients with Peutz-Jeghers syndrome: A retrospective cohort study of 336 cases. Tumour Biol. 2017 Jun;39(6):1010428317705131. doi: 10.1177/1010428317705131.'}, {'pmid': '15261145', 'type': 'BACKGROUND', 'citation': 'Shaw RJ, Bardeesy N, Manning BD, Lopez L, Kosmatka M, DePinho RA, Cantley LC. The LKB1 tumor suppressor negatively regulates mTOR signaling. Cancer Cell. 2004 Jul;6(1):91-9. doi: 10.1016/j.ccr.2004.06.007.'}, {'pmid': '9425897', 'type': 'BACKGROUND', 'citation': 'Jenne DE, Reimann H, Nezu J, Friedel W, Loff S, Jeschke R, Muller O, Back W, Zimmer M. Peutz-Jeghers syndrome is caused by mutations in a novel serine threonine kinase. Nat Genet. 1998 Jan;18(1):38-43. doi: 10.1038/ng0198-38.'}, {'pmid': '19147279', 'type': 'BACKGROUND', 'citation': 'Wei C, Amos CI, Zhang N, Zhu J, Wang X, Frazier ML. Chemopreventive efficacy of rapamycin on Peutz-Jeghers syndrome in a mouse model. Cancer Lett. 2009 May 18;277(2):149-54. doi: 10.1016/j.canlet.2008.11.036. Epub 2009 Jan 14.'}, {'pmid': '19434632', 'type': 'BACKGROUND', 'citation': 'Robinson J, Lai C, Martin A, Nye E, Tomlinson I, Silver A. Oral rapamycin reduces tumour burden and vascularization in Lkb1(+/-) mice. J Pathol. 2009 Sep;219(1):35-40. doi: 10.1002/path.2562.'}]}, 'descriptionModule': {'briefSummary': 'A prospective non-randomized open label single arm clinical trial to examine the efficacy and safety of sirolimus in patients with Peutz-Jeghers Syndrome.', 'detailedDescription': 'PJS (Peutz-Jeghers Syndrome) is mostly caused by mutations in Lkb1 gene, which leads to an increased activity of mTOR pathway, thus making mTOR inhibitor (sirolimus) a promising candidate in treatment and prevention of PJS. The efficacy of sirolimus (rapamycin) on PJS has been demonstrated in mouse model, but no clinical trials have been reported. Our study was designed as a prospective non-randomized open label single arm clinical trial to examine its efficacy and safety.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Patients are diagnosed with PJS.\n* 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.\n* Conventional treatment didn't work well in patients combined with PJS-related tumors.\n* Physical condition (ECGO): 0\\~3\n* Organ function is good and biochemical indices meet the following conditions:\n\n * AST≤2.5×upper limit of normal value (ULN),\n * ALT≤2.5×upper limit of normal value (ULN),\n * Serum total bilirubin (TSB)≤1.5×upper limit of normal value (ULN),\n * Creatinine≤1.5×upper limit of normal value (ULN).\n* No other medications have been received for intestinal polyps within 3 months prior to the clinical trial.\n* Patients participate in the trial voluntarily and have signed the informed consent by the participant or his/her legal guardian.\n\nExclusion Criteria:\n\n* Patients underwent a surgery within 2 weeks.\n* Patients may need emergency surgery in the near future.\n* Patients are allergic to any ingredient of rapamycin.\n* Patients suffer from a disease requiring immediate blood transfusion.\n* Patients suffer from any disease or condition that may impact implementation of the study or interpretation of the results. This type of diseases includes:\n\n * Known severe blood coagulation disorders\n * Known anemia that is not caused by intestinal polyps\n * Known hemoglobinopathy\n * Other gastrointestinal infectious diseases\n * Serious heart, liver, kidney and other concomitant diseases that may endanger lives\n* Patients are in pregnancy and lactation.\n* Alcohol or drug (such as aperient) abuse\n* Patients took part in another clinical trial that may influence this study.\n* The researchers believe that there are other unfavorable reasons for the patient to become a subject."}, 'identificationModule': {'nctId': 'NCT03781050', 'briefTitle': 'Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome', 'organization': {'class': 'OTHER', 'fullName': 'Peking Union Medical College Hospital'}, 'officialTitle': 'Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome', 'orgStudyIdInfo': {'id': 'HS-1607'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Rapamycin', 'description': 'For children: rapamycin, 1 mg per square meter of body surface area a day, orally, for at least 6 months\n\nFor adults: rapamycin, 2 mg a day, orally, for at least 6 months', 'interventionNames': ['Drug: Rapamycin']}], 'interventions': [{'name': 'Rapamycin', 'type': 'DRUG', 'otherNames': ['Sirolimus'], 'description': 'For children: rapamycin, 1 mg per square meter of body surface area a day, orally, for at least 6 months\n\nFor adults: rapamycin, 2 mg a day, orally, for at least 6 months', 'armGroupLabels': ['Rapamycin']}]}, 'contactsLocationsModule': {'locations': [{'zip': '100000', 'city': 'Beijing', 'state': 'China/Beijing', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Jiaolin Zhou, MD', 'role': 'CONTACT', 'email': 'conniezhjl@yahoo.com', 'phone': '13910136704'}], 'facility': 'Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences', 'geoPoint': {'lat': 39.9075, 'lon': 116.39723}}], 'centralContacts': [{'name': 'Jiaolin Zhou, MD', 'role': 'CONTACT', 'email': 'conniezhjl@yahoo.com', 'phone': '13910136704'}], 'overallOfficials': [{'name': 'Jiaolin Zhou, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Peking Union Medical College Hospital', 'class': 'OTHER'}, 'collaborators': [{'name': 'Air Force General Hospital of the PLA', 'class': 'OTHER_GOV'}, {'name': 'Cancer Institute and Hospital, Chinese Academy of Medical Sciences', 'class': 'OTHER'}], 'responsibleParty': {'type': 'SPONSOR'}}}}