Myeloproliferative NeoplasmsPhase 2NCT00949364
Pomalidomide in Patients With Myeloproliferative Neoplasms in Fibrotic Stage
A Phase 2 study testing Pomalidomide in people with Myeloproliferative Neoplasms, run by University of Ulm at 15 sites in Germany.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 50 years and older
- Conditions the study is for
- Myeloproliferative Neoplasms
- What is being tested?
- Pomalidomide
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 103 participants
- Where
- 15 sitesGermany
- Study dates
- Dec 2009to 14 Dec 2016
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 50 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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:
- Age ≥50 years at the time of voluntarily signing an IRB/IEC-approved informed consent
- 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
- 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
- Splenomegaly (>11 cm diameter) and/or leukoerythroblastosis
- 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
Show all 18Show fewer
- Subject must be willing to receive transfusion of blood products
- ECOG performance status < 3
- Female subjects with non-childbearing potential:
- Agree to have a pregnancy test at baseline
- 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.
- 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.
Who cannot join
- The presence of any of the following will exclude a patient from study enrollment:
- Females of childbearing potentials°, pregnant or breast feeding females
- BCR/ABL-positivity
- Diagnosis of ET (according to WHO 2008 criteria)
- Diagnosis of PV (according to WHO 2008 criteria)
- >20% blasts in peripheral blood or bone marrow
Show all 20Show fewer
- Known positive status for HIV, HBV or HCV
- Prior treatment with IMiDs (thalidomide, lenalidomide) or with Interferon-alpha within a 3 month time period before screening
- History of thrombosis or pulmonary embolism
- Peripheral neuropathy >grade 1 CTC
- 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.
- 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
- Drug or alcohol abuse within the last 6 months
- 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
Trial locations and contacts
15 sites in 1 country.
15 sites in 1 country
Germany15 sites
Universitätsklinikum Aachen
Aachen, 52074
Charité Universitätsmedizin Berlin
Berlin, 13353
Zentrum für Ambulante Hämatologie und Onkologie
Bonn, 53119
BAG Freiberg-Richter, Jacobasch, Wolf, Illmer (Gemeinschaftspraxis)
Dresden, 01307
Universitätsklinikum Düsseldorf
Düsseldorf, 40225
Klinikum der Johann Goethe-Universität Frankfurt
Frankfurt, 60590
Universitätsklinikum Freiburg
Freiburg im Breisgau, 79106
Universitätsklinikum Hamburg Eppendorf
Hamburg Eppendorf, 20246
Universitätsklinikum Jena
Jena, 07740
Universitätsklinikum Magdeburg AöR
Magdeburg, 39120
Universitätsmedizin Mannheim
Mannheim, 68167
Johannes Wesling Klinikum Minden
Minden, 32429
Haematologisch-onkologische Praxis
München, 80331
Stauferklinikum Schwäbisch Gmünd
Mutlangen, 73557
Universitätsklinikum Ulm
Ulm, 89081
About this study
Official title: Multi-Center Phase II Study With Pomalidomide in Patients With Myeloproliferative Neoplasms in Fibrotic Stage
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):
Read more
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.
- Conditions
- Myeloproliferative Neoplasms
- Being tested
- Pomalidomide
- Keywords
- Myeloproliferative Neoplasms, Pomalidomide, Fibrotic Stage, Patients with Myeloproliferative Neoplasms in Fibrotic Stage
- Medical subject headings
- Myeloproliferative Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
PomalidomideDrug
Treatment starts with pomalidomide as single agent therapy: 0.5 mg/day. Prednisolone will be started in the absence of PD as randomized either at start of cycle 4 or start of cycle 7 (starting dose: 30 mg/day for 28 days followed by 15 mg/day and 10 mg/day for 28 days), if no response acc. to IWG-MRT (no CR, PR, CI, TI) was achieved.
If PD: treatment is stopped. Otherwise, continuous treatment at least until end of cycle 12 is intended. For patients responding to the combination treatment (pomalidomide/prednisolone) a concom. treatment after cycle 6 or 9 (depending on the randomization result) with prednisolone in doses equal or below 7.5 mg/day are allowed.
If a patient may benefit from treatment with pomalidomide the invest. and the PI will discuss the possibility of further treatment including maintenance treatment with pomalidomide on a case-by-case decision (max. duration: 12 cycles).
| Study group | Role | What they receive |
|---|---|---|
| Pomalidomide | Experimental | Every patient will remain on treatment until disease progression for at least 12 cycles, withdrawal of patient's informed consent or the occurrence of unacceptable toxicity. If a patient may benefit from treatment with pomalidomide the investigator together with the principle investigator will discuss the possibility of further treatment including maintenance treatment with pomalidomide on a case-by-case decision. This additional treatment will be performed within the follow-up period of the study, data will be collected and duration will be maximally 12 cycles. Receives: Drug: Pomalidomide |
What is being measured
The primary outcome is the main question the study is designed to answer.
Objective disease response, as defined by the IWG-MRT criteria for response in MF patients extended by the criterion RBC-transfusion independence (TI)
Measured: one year
Secondary outcomes (4)
Overall safety profile of pomalidomide characterized by type, frequency, severity, timing and relatedness of adverse events (AEs) and laboratory abnormalities observed during treatment
Graded using the National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] Version 3.0
Measured: one year
Event-free survival
Measured: three years
Relapse-free survival
Measured: three years
Overall survival
Measured: three years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Jul 2009 (estimated)
- Study startDec 2009
- Primary completion14 Dec 2016
- Study completion14 Dec 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myeloproliferative NeoplasmsStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 103 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Ulm
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Prof. Dr. Richard Schlenk, University of Ulm
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00949364
- Sponsor's protocol ID
- MPN-SG 01-09
- Status last verified
- Sep 2017
- First submitted
- 28 Jul 2009
- Last updated
- 25 Sep 2017
- Results posted
- No
- Data monitoring committee
- No
Publications
Schlenk RF, Stegelmann F, Reiter A, Jost E, Gattermann N, Hebart H, Waller C, Hochhaus A, Platzbecker U, Schafhausen P, Blau IW, Verbeek W, Heidel FH, Werner M, Kreipe H, Teleanu V, Benner A, Dohner H, Griesshammer M, Dohner K. Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis. Leukemia. 2017 Apr;31(4):889-895. doi: 10.1038/leu.2016.299. Epub 2016 Oct 24.
PubMed 27774990
Study at a glance
From the registry record, last updated by the sponsor on 25 Sep 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Multi-Center Phase II Study With Pomalidomide in Patients With Myeloproliferative Neoplasms in Fibrotic Stage
- ClinicalTrials.gov ID
- NCT00949364
- Acronym / sponsor ID
- MPN-SG 01-09
- Conditions
- Myeloproliferative Neoplasms
- Keywords
- Myeloproliferative Neoplasms, Pomalidomide, Fibrotic Stage, Patients with Myeloproliferative Neoplasms in Fibrotic Stage
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 103 participants (actual)
- Lead sponsor
- University of UlmUniversity, hospital or other
- Study start
- Dec 2009
- Primary completion
- 14 Dec 2016 (actual)
- Study completion
- 14 Dec 2016 (actual)
- First posted
- 30 Jul 2009 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Pomalidomide | Experimental | Drug: PomalidomideEvery patient will remain on treatment until disease progression for at least 12 cycles, withdrawal of patient's informed consent or the occurrence of unacceptable toxicity. If a patient may benefit from treatment with pomalidomide the investigator together with the principle investigator will discuss the possibility of further treatment including maintenance treatment with pomalidomide on a case-by-case decision. This additional treatment will be performed within the follow-up period of the study, data will be collected and duration will be maximally 12 cycles. |
Who may qualify
- Ages
- 50 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (18 listed)
- 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:
- Age ≥50 years at the time of voluntarily signing an IRB/IEC-approved informed consent
- 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
- 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
- Splenomegaly (>11 cm diameter) and/or leukoerythroblastosis
Key exclusion criteria (20 listed)
- Females of childbearing potentials°, pregnant or breast feeding females
- BCR/ABL-positivity
- Diagnosis of ET (according to WHO 2008 criteria)
- Diagnosis of PV (according to WHO 2008 criteria)
- >20% blasts in peripheral blood or bone marrow
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| PomalidomideTreatment starts with pomalidomide as single agent therapy: 0.5 mg/day. Prednisolone will be started in the absence of PD as randomized either at start of cycle 4 or start of cycle 7 (starting dose: 30 mg/day for 28 days followed by 15 mg/day and 10 mg/day for 28 days), if no response acc. to IWG-MRT (no CR, PR, CI, TI) was achieved. If PD: treatment is stopped. Otherwise, continuous treatment at least until end of cycle 12 is intended. For patients responding to the combination treatment (pomalidomide/prednisolone) a concom. treatment after cycle 6 or 9 (depending on the randomization result) with prednisolone in doses equal or below 7.5 mg/day are allowed. If a patient may benefit from treatment with pomalidomide the invest. and the PI will discuss the possibility of further treatment including maintenance treatment with pomalidomide on a case-by-case decision (max. duration: 12 cycles). | Drug | Pomalidomide |
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
4 secondary outcomes
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
Event-free survival
Time frame: three years
Relapse-free survival
Time frame: three years
Overall survival
Time frame: three years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Sep 2017.
- Results publication: PubMed 27774990Schlenk RF, Stegelmann F, Reiter A, Jost E, Gattermann N, Hebart H, Waller C, Hochhaus A, Platzbecker U, Schafhausen P, Blau IW, Verbeek W, Heidel FH, Werner M, Kreipe H, Teleanu V, Benner A, Dohner H, Griesshammer M, Dohner K. Pomalidomide in myeloproliferative neoplasm-associated myelofibrosis. Leukemia. 2017 Apr;31(4):889-895. doi: 10.1038/leu.2016.299. Epub 2016 Oct 24.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
15 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Pomalidomide in people with Myeloproliferative Neoplasms, run by University of Ulm at 15 sites in Germany.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 18 inclusion and 20 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 25 Sep 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 15 sites in Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00949364, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Sep 2017; AzTrials last refreshed its copy on 8 Nov 2026.