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Pilot Study of Everolimus in the Treatment of Neoplasms in Patients With Peutz-Jeghers Syndrome

Phase 2
18 Years
Open (Enrolling)
Peutz-Jeghers Syndrome, Neoplastic Processes, Neoplasm Metastasis

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Trial Information

Pilot Study of Everolimus in the Treatment of Neoplasms in Patients With Peutz-Jeghers Syndrome

Inclusion Criteria

Tow cohorts of PJS patients will be included. Cohort 1: Advanced malignancy Cohort 2: High
risk polyps

General inclusion criteria:

1. Known Peutz-Jeghers disease (with LKB1 mutation)

2. No concurrent systemic anti cancer treatment

3. No prior treatment with m-TOR inhibitor

4. Prior malignancies or concurrent second malignancies are allowed

5. Prior systemic therapy is permitted with a washout time of at least 4 weeks

6. ECOG/ WHO performance 0-2

7. Age > 18 years

8. Adequate renal function (defined as creatinine < 150 ╬╝mol/L)

9. Adequate liver function (bilirubin < 1.5 times upper limit of normal, ALAT or ASAT <
5.0 times upper limit of normal in case of liver metastases and < 2.5 the upper limit
of normal in absence of liver metastases

10. Adequate bone marrow function (WBC > 3.0 x 10 9/L, platelets > 100 x 10 9/L)

11. Absence of any psychological, familial, sociological or geographical condition
potentially hampering compliance with the study protocol and follow-up schedule;
those conditions should be discussed with the patient before registration in the

12. No pregnancy or lactating and ifof childbearing potential patients must agree to use
a reliable contraceptive method throughout the study

13. No serious concomitant systemic disorder that would compromise the safety of the
patient,at the discretion of the investigator

14. Signed informed consent according to ICH/GCP.

15. No uncontrolled symptomatic hyperglycaemia

Specific inclusion criteria for cohort 1:

1. Cytological or histological confirmed carcinoma

2. Metastatic or non-resectable disease

3. Patients with clinically and/or radiographically documented measurable lesion
according to

RECIST criteria:

1. X-ray, physical exam > 20 mm

2. Spiral CT scan > 10 mm

3. Non-spiral CT scan > 20 mm

Specific inclusion criteria for cohort 2:

1. Known high risk polyps (definition see page 19)

2. Ability to undergo endoscopies

Specific Exclusion criteria:

Symptomatic PJ-polyps, defined as polyps likely to be responsible/causal for the abdominal
symptoms the patient presents with.

Type of Study:


Study Design:

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To determine the response rate of Everolimus in patients with advanced cancer and PJS.

Outcome Description:

Determined with regular radiological scans once every 9 weeks and measured following RECIST 1.1

Outcome Time Frame:

During treatment, expected avarage of 12 months

Safety Issue:


Principal Investigator

Heinz-Josef Klumpen, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)


Netherlands: The Central Committee on Research Involving Human Subjects (CCMO)

Study ID:




Start Date:

October 2010

Completion Date:

September 2014

Related Keywords:

  • Peutz-Jeghers Syndrome
  • Neoplastic Processes
  • Neoplasm Metastasis
  • Peutz-Jeghers syndrome
  • mTOR inhibition
  • cancer
  • Neoplasms
  • Neoplasm Metastasis
  • Neoplastic Processes
  • Peutz-Jeghers Syndrome