Working...
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 16 for:    "Peutz-Jeghers Syndrome"
Previous Study | Return to List | Next Study

Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03781050
Recruitment Status : Recruiting
First Posted : December 19, 2018
Last Update Posted : January 23, 2019
Sponsor:
Collaborators:
Air Force General Hospital of the PLA
Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences
Information provided by (Responsible Party):
Peking Union Medical College Hospital

Brief Summary:
A prospective non-randomized open label single arm clinical trial to examine the efficacy and safety of sirolimus in patients with Peutz-Jeghers Syndrome.

Condition or disease Intervention/treatment Phase
Peutz-Jeghers Syndrome Drug: Rapamycin Phase 4

Detailed Description:
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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 10 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Efficacy of Rapamycin (Sirolimus) in the Treatment of Peutz-Jeghers Syndrome
Actual Study Start Date : September 16, 2018
Estimated Primary Completion Date : January 1, 2022
Estimated Study Completion Date : July 1, 2022


Arm Intervention/treatment
Experimental: Rapamycin

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

Drug: Rapamycin

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

Other Name: Sirolimus




Primary Outcome Measures :
  1. Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps [ Time Frame: The time from start of therapy to 1 year ]
    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.


Secondary Outcome Measures :
  1. Concentration of hemoglobin in blood [ Time Frame: The time from start of therapy to 1 year ]
    The value indicates the amount of gastrointestinal bleeding.

  2. Concentration of albumin in blood [ Time Frame: The time from start of therapy to 1 year ]
    The value indicates the status of nutrition.

  3. Concentration of hsCRP in blood [ Time Frame: The time from start of therapy to 1 year ]
    The value indicates the level of inflammation.

  4. Visual analogue score (VAS) [ Time Frame: The time from start of therapy to 1 year ]
    The value indicates the level of pain.

  5. Dermatology life quality index (DLQI) [ Time Frame: The time from start of therapy to 1 year ]
    The value indicates the status of hyperpigmented macules on the lips and oral mucosa.

  6. Adverse events [ Time Frame: The time from start of therapy to 1 year ]
    To evaluate safety



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients are diagnosed with PJS.
  • 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.
  • Conventional treatment didn't work well in patients combined with PJS-related tumors.
  • Physical condition (ECGO): 0~3
  • Organ function is good and biochemical indices meet the following conditions:

    • AST≤2.5×upper limit of normal value (ULN),
    • ALT≤2.5×upper limit of normal value (ULN),
    • Serum total bilirubin (TSB)≤1.5×upper limit of normal value (ULN),
    • Creatinine≤1.5×upper limit of normal value (ULN).
  • No other medications have been received for intestinal polyps within 3 months prior to the clinical trial.
  • Patients participate in the trial voluntarily and have signed the informed consent by the participant or his/her legal guardian.

Exclusion Criteria:

  • Patients underwent a surgery within 2 weeks.
  • Patients may need emergency surgery in the near future.
  • Patients are allergic to any ingredient of rapamycin.
  • Patients suffer from a disease requiring immediate blood transfusion.
  • Patients suffer from any disease or condition that may impact implementation of the study or interpretation of the results. This type of diseases includes:

    • Known severe blood coagulation disorders
    • Known anemia that is not caused by intestinal polyps
    • Known hemoglobinopathy
    • Other gastrointestinal infectious diseases
    • Serious heart, liver, kidney and other concomitant diseases that may endanger lives
  • Patients are in pregnancy and lactation.
  • Alcohol or drug (such as aperient) abuse
  • Patients took part in another clinical trial that may influence this study.
  • The researchers believe that there are other unfavorable reasons for the patient to become a subject.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03781050


Contacts
Layout table for location contacts
Contact: Jiaolin Zhou, MD 13910136704 conniezhjl@yahoo.com

Locations
Layout table for location information
China, China/Beijing
Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences Recruiting
Beijing, China/Beijing, China, 100000
Contact: Jiaolin Zhou, MD    13910136704    conniezhjl@yahoo.com   
Sponsors and Collaborators
Peking Union Medical College Hospital
Air Force General Hospital of the PLA
Institute of Basic Medical Sciences, Chinese Academy of Medical Sciences
Investigators
Layout table for investigator information
Principal Investigator: Jiaolin Zhou, MD Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
  Study Documents (Full-Text)

Documents provided by Peking Union Medical College Hospital:

Publications:
Layout table for additonal information
Responsible Party: Peking Union Medical College Hospital
ClinicalTrials.gov Identifier: NCT03781050     History of Changes
Other Study ID Numbers: HS-1607
First Posted: December 19, 2018    Key Record Dates
Last Update Posted: January 23, 2019
Last Verified: January 2019

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Peking Union Medical College Hospital:
Peutz-Jeghers Syndrome (PJS)
Rapamycin (sirolimus)
Mammalian target of rapamycin (mTOR) inhibitor
Treatment

Additional relevant MeSH terms:
Layout table for MeSH terms
Peutz-Jeghers Syndrome
Syndrome
Disease
Pathologic Processes
Neoplastic Syndromes, Hereditary
Neoplasms
Intestinal Polyposis
Intestinal Diseases
Gastrointestinal Diseases
Digestive System Diseases
Genetic Diseases, Inborn
Lentigo
Melanosis
Hyperpigmentation
Pigmentation Disorders
Skin Diseases
Sirolimus
Everolimus
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Antifungal Agents
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs