CClinicalTrials.gg
Sign In
Status unknownNCT03781050Updated Jan 23, 2019

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

A Phase 4 interventional study of Rapamycin in Peutz-Jeghers Syndrome, sponsored by Peking Union Medical College Hospital. Status unknown at 1 site in China. Per ClinicalTrials.gov, last updated 2019-01-23.

Sponsored by Peking Union Medical College Hospital · Phase 4, Interventional, and Treatment

The sponsor has not verified this record recently (last verified Jan 2019), so the status shown — last known as Recruiting — may be out of date.
Phase
Phase 4
Study type
Interventional
Enrollment
10
Allocation
Not applicable
Sex
All
01

Study 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.

Read the 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.

02

Conditions studied

  • Peutz-Jeghers Syndrome

Keywords

  • Peutz-Jeghers Syndrome (PJS)
  • Rapamycin (sirolimus)
  • Mammalian target of rapamycin (mTOR) inhibitor
  • Treatment
03

In context

Peutz-Jeghers Syndrome

20 studies on the registry are indexed under Peutz-Jeghers Syndrome; 7 are open to participants now.

Browse Peutz-Jeghers Syndrome studies →

Lead sponsor

Peking Union Medical College Hospital is the lead sponsor of 1,115 studies on the registry; 463 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No

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

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.
05

Study design

Phase
Phase 4
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
10 participants (estimated)

Study arms

  • 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

Interventions

  • DrugRapamycin

    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

    Also known as: Sirolimus

06

What researchers measure

Primary outcomes

  1. Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps Total load of PJS-related intestinal polyps

    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.

    Time frame: The time from start of therapy to 1 year

Secondary outcomes

  1. Concentration of hemoglobin in blood

    The value indicates the amount of gastrointestinal bleeding.

    Time frame: The time from start of therapy to 1 year

  2. Concentration of albumin in blood

    The value indicates the status of nutrition.

    Time frame: The time from start of therapy to 1 year

  3. Concentration of hsCRP in blood

    The value indicates the level of inflammation.

    Time frame: The time from start of therapy to 1 year

  4. Visual analogue score (VAS)

    The value indicates the level of pain.

    Time frame: The time from start of therapy to 1 year

  5. Dermatology life quality index (DLQI)

    The value indicates the status of hyperpigmented macules on the lips and oral mucosa.

    Time frame: The time from start of therapy to 1 year

  6. Adverse events

    To evaluate safety

    Time frame: The time from start of therapy to 1 year

07

Study locations

1 of 1 sites recruiting
  • Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences
    Beijing, China/Beijing 100000, China
    Recruiting
08

References and documents

Publications

  • 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. PubMed 28653895 ↗
  • 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. PubMed 15261145 ↗
  • 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. PubMed 9425897 ↗
  • 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. PubMed 19147279 ↗
  • 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. PubMed 19434632 ↗

Study documents

  • Protocol and statistical analysis plan · Dec 5, 2018

Documents are hosted by the registry — open the source record to download them.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jan 23, 2019, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT03781050
Lead sponsor
Peking Union Medical College Hospital
Collaborators
Air Force General Hospital of the PLA, Cancer Institute and Hospital, Chinese Academy of Medical Sciences
Responsible party
Sponsor
First posted
Dec 19, 2018
Start date
Sep 16, 2018
Primary completion
Jan 1, 2022 (estimated)
Completion
Jul 1, 2022 (estimated)
Last update
Jan 23, 2019

Study contacts

Jiaolin Zhou, MD
Contact
conniezhjl@yahoo.com
13910136704
Jiaolin Zhou, MD
principal investigator · Peking Union Medical College Hospital, Chinese Academy of Medicine Sciences

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is status unknown, as verified in Jan 2019. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Other studies in this condition

Peutz-Jeghers Syndrome→

Other studies from this sponsor

Peking Union Medical College Hospital

Start the discussion