Trial document





This study has been imported from ClinicalTrials.gov without additional data checks.
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  DRKS00003751

Trial Description

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Title

Multi-Center Phase II Study With Pomalidomide in Patients With Myeloproliferative Neoplasms in Fibrotic Stage

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Trial Acronym

[---]*

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URL of the Trial

http://www.mpnsg.de

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Brief Summary in Lay Language

This is a phase II, multi-center study of pomalidomide in adult patients with PMF, SMF, and
unclassifiable MPN showing at least grade 1 bone marrow fibrosis and requiring therapy. All
patients will receive per oral pomalidomide on a daily basis.

First cohort (Before Amendment No. 1 ID 1-41):

Treatment starts with a phase of pomalidomide therapy with 2 mg per day. Individual dose
reduction as outlined in the safety section is allowed. If no response was achieved (no
complete remission (CR), partial response (PR), clinical improvement (CI) and no progressive
disease according to the IWG-MRT criteria) after 3 months, prednisolone is added in a
starting dose of 30 mg per day. In the absence of progressive disease, at least 6 months of
treatment with pomalidomide is intended. In patients without disease progression after 6
months and those with response to treatment are intended to receive pomalidomide for at
least 12 months. Additional antiproliferative treatment with hydroxyurea for leukocytosis
(>20 x 109/l) and/or thrombocytosis (>750 x 109/l) and/or symptomatic splenomegaly in a
starting dose of 2g/day with individual dose adjustment is allowed.

Second cohort (After Amendment No. 1 ID > 41):

To evaluate the relative impact of prednisolone to the objective response rate, a
randomization has been integrated into the study concept. The addition of prednisolone is
up-front randomized for the start of prednisolone either after 3 or 6 cycles of treatment
with pomalidomide as single agent if no response occurred during this period. This results
in the following treatment arms:

Treatment Arm A) Pomalidomide 0.5 mg per day + additional prednisolone at start of cycle 4
(day 85), in case no response was achieved until end of cycle 3.

Treatment Arm B) Pomalidomide 0.5 mg per day + additional prednisolone at start of cycle 7
(day 169), if no response was achieved until end of cycle 6.

Treatment for all patients starts with pomalidomide as single agent at a dose of 0.5mg per
day. The addition of prednisolone will be initiated as randomized either at start of cycle 4
or start of cycle 7 (starting dose 30 mg per day). In the absence of progressive disease, at
least 12 cycles of treatment with pomalidomide are intended.

Additional antiproliferative treatment with hydroxyurea for leukocytosis (>20 x 109/l)
and/or thrombocytosis (>750 x 109/l) and/or symptomatic splenomegaly in a starting dose of
2g/day with individual dose adjustment is allowed.

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Brief Summary in Scientific Language

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Organizational Data

  •   DRKS00003751
  •   2012/06/20
  •   2009/07/28
  •   yes
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Secondary IDs

  •   NCT00949364  (ClinicalTrials.gov)
  •   MPN-SG 01-09  (University of Ulm)
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Health Condition or Problem studied

  •   Myeloproliferative Neoplasms
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Interventions/Observational Groups

  •   Drug: Pomalidomide
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Characteristics

  •   Interventional
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  •   Single arm study
  •   Open (masking not used)
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  •   Uncontrolled/Single arm
  •   Treatment
  •   Single (group)
  •   II
  •   [---]*
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Primary Outcome

- Objective disease response, as defined by the IWG-MRT criteria for response in MF patients extended by the criterion RBC-transfusion independence (TI); time frame: one year

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Secondary Outcome

- Overall safety profile of pomalidomide characterized by type, frequency, severity, timing and relatedness of adverse events (AEs) and laboratory abnormalities observed during treatment; time frame: one year; Graded using the National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] Version 3.0
- Event-free survival; time frame: three years
- Relapse-free survival; time frame: three years
- Overall survival; time frame: three years

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Countries of Recruitment

  •   Germany
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Locations of Recruitment

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Recruitment

  •   Actual
  •   2009/12/31
  •   95
  •   Multicenter trial
  •   National
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Inclusion Criteria

  •   Both, male and female
  •   50   Years
  •   no maximum age
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Additional Inclusion Criteria

Both female and male patients meeting the mentioned inclusion and exclusion criteria will
be included in this clinical trial. The risk to get PMF or SMF does not depend on a
patient's gender. Patients must meet all of the following inclusion criteria to be
eligible for enrollment into the study:

1. Age ≥50 years at the time of voluntarily signing an IRB/IEC-approved informed consent

2. Diagnosis of Myeloproliferative Neoplasms (MPN) either de novo myelofibrosis
according to WHO criteria (PMF) [20], secondary myelofibrosis (post-PV MF and post-ET
MF according to the IWG-MRT consensus terminology) [21] or unclassifiable MPN with
biopsy proven myelofibrosis

3. Anemia with hemoglobin level of <10 g/dl or transfusion-dependent anemia and/or
thrombocytopenia <50 /nl or transfusion-dependent thrombocytopenia and/or neutropenia
<1.0 /nl

4. Splenomegaly (>11 cm diameter) and/or leukoerythroblastosis

5. Adequate organ function, i.e. ALT and/or AST <3 x upper limit of normal (ULN), total
bilirubin <3 x ULN, and serum creatinine <2 mg/dl

6. Subject must be willing to receive transfusion of blood products

7. ECOG performance status < 3

8. Female subjects with non-childbearing potential:

- Agree to have a pregnancy test at baseline

9. Male subjects:

- Agree to use condoms throughout study drug therapy, during any dose interruption
and for four weeks after cessation of study therapy if their partner is of
childbearing potential and has no contraception.

- Agree not to donate semen during study drug therapy and for four weeks after end
of study drug therapy.

10. All Subjects:

- Will be counseled about potential teratogenic risks of the study medication.

- Agree to abstain from donating blood while taking study drug therapy and for one
week following discontinuation of study drug therapy.

- Agree not to share study medication with another person and to return all unused
study drug to the investigator

- No more than a 12-weeks-supply of study drug will be dispensed at a time.

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Exclusion Criteria

The presence of any of the following will exclude a patient from study enrollment:

1. Females of childbearing potentials°, pregnant or breast feeding females

2. BCR/ABL-positivity

3. Diagnosis of ET (according to WHO 2008 criteria)

4. Diagnosis of PV (according to WHO 2008 criteria)

5. >20% blasts in peripheral blood or bone marrow

6. Known positive status for HIV, HBV or HCV

7. Prior treatment with IMiDs (thalidomide, lenalidomide) or with Interferon-alpha
within a 3 month time period before screening

8. History of thrombosis or pulmonary embolism

9. Peripheral neuropathy >grade 1 CTC

10. No consent for registration, storage and processing of the individual
disease-characteristics and course as well as information of the family physician
about study participation.

11. Presence of any medical/psychiatric condition or laboratory abnormalities which may
limit full compliance with the study, increase the risk associated with study
participation or study drug administration, or may interfere with the interpretation
of study results and, in the judgment of the Investigator, would make the patient
inappropriate for entry into this study

12. Drug or alcohol abuse within the last 6 months

13. Patients with a "currently active" second malignancy other than nonmelanoma skin
cancers. Patients are not considered to have a "currently active" malignancy if they
have completed therapy and are considered by their physician to be at less than 30%
risk of relapse within one year.

Criteria for women of non-childbearing potential:

A female patient or a female partner of a male patient is considered to have childbearing
potential unless she meets at least one of the following criteria:

- Age ≥ 50 years and naturally amenorrhoeic for ≥ 1 year. Amenorrhoea following cancer
therapy does not rule out childbearing potential

- Premature ovarian failure confirmed by a specialist gynecologist

- Previous bilateral salpingo-oophorectomy, or hysterectomy

- XY genotype, Turner syndrome, uterine agenesis

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Addresses

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    • University of Ulm
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Sources of Monetary or Material Support

  • start of 1:1-Block address materialSupport
    • Bitte wenden Sie sich an den Sponsor / Please refer to primary sponsor
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Status

  •   Recruiting complete, follow-up continuing
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Trial Publications, Results and other Documents

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The parameters in ClinicalTrials.gov and DRKS are not identical. Therefore the data import from ClinicalTrials.gov required adjustments. For full details please see the DRKS FAQs .
  •   8
  •   2013/10/30
* This entry means the parameter is not applicable or has not been set.