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Primary MyelofibrosisPhase 1/2POMINCNCT01644110

Ruxolitinib and Pomalidomide Combination Therapy in Patients With Primary and Secondary MF

A Phase 1/2 study testing INCB018424/CC-4047 in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by University of Ulm at 17 sites in Germany.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The proposed study is an open-label, single-arm, Phase-Ib/II trial to assess the efficacy of oral drug combination ruxolitinib and pomalidomide in primary and secondary MF patients. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
INCB018424/CC-4047
Could I receive a placebo?
No, everyone receives an active treatment
How many people
96 participants
Where
17 sitesGermany
Study dates
Aug 2013to 27 Apr 2024
Phase
Phase 1/2
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 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Age ≥18 years at the time of voluntarily signing an IRB/IEC-approved informed consent
  • Diagnosis of Myeloproliferative Neoplasms (MPN) either de novo myelofibrosis according to current WHO criteria (PMF), secondary myelofibrosis (post-PV MF and post-ET MF) according to the IWG-MRT consensus terminology) (Appendix I)
  • Anemia with hemoglobin level of <10 g/dl or transfusion-dependent anemia*
  • Splenomegaly (>11 cm total 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
  • Subject must be willing to receive transfusion of blood products
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  • ECOG performance status <3
  • Females of childbearing potential (FCBP) must undergo repetitive pregnancy testing (serum or urine) and pregnancy results must be negative.**
  • Reliable contraception should be maintained throughout the study and for 28 days after study treatment discontinuation*
  • Unless practicing complete abstinence from heterosexual intercourse, sexually active FCBP must agree to use adequate contraceptive methods*
  • Males (including those who have had a vasectomy) must use barrier contraception (condoms) when engaging in sexual activity with FCBP. Males must agree not to donate semen or sperm*
  • All subjects must:
  • understand that the investigational product could have a potential teratogenic risk.
  • be counseled about pregnancy precautions and risks of fetal exposure.
  • agree to abstain from donating blood while taking investigational product.
  • agree not to share study medication with another person and to return all unused study drug to the investigator.

Who cannot join

  • Patients eligible for hematopoietic stem cell transplantation (suitable candidate and suitable donor is available)
  • Patients with response to standard therapy as recommended by the Deutsche Gesellschaft für Hämatologie und Medizinische Onkologie (DGHO/Onkopedia)
  • Pregnant or breast feeding females
  • BCR/ABL-positivity
  • Diagnosis of ET (according to WHO 2016 criteria)
  • Diagnosis of PV (according to WHO 2016 criteria)
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  • >20% blasts in peripheral blood or bone marrow
  • thrombocytopenia <100 /nl or transfusion-dependent thrombocytopenia
  • neutropenia <0.5 /nl
  • Known positive status for HIV, HBV or HCV
  • Prior treatment with IMiDs (thalidomide, lenalidomide, pomalidomide) or with Interferon-alpha within a 3 month time period before Screening-phase
  • Patient treatment with Ruxolitinib within a 14 days time period before Screening-phase
  • History of thrombosis or pulmonary embolism within 6 months prior to study entry
  • 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
  • History of malignancy except for i) adequately treated local basal cell or squamous cell carcinoma of the skin, ii) asymptomatic prostate cancer without known metastatic disease and with no requirement for therapy or requiring only hormonal therapy and with normal prostate-specific antigen for ≥ 1 year prior to randomization, or iii) any other cancer that has been in complete remission for ≥ 5 years
  • Patients undergoing treatment with hematopoietic growth factor receptor agonists (i.e., erythropoietin [Epo], granulocyte colony stimulating factor (GCSF [Neupogen; Neulasta], romiplostim, eltrombopag) within a 4 weeks period prior to screening-phase.
  • Patients receiving any medication listed in the Appendix V "Prohibited Medications" (within 7 days prior to the first dose of study drug).
  • Patients with clinically significant bacterial, fungal, parasitic or viral infection which require therapy. Patients with acute bacterial infections requiring antibiotic use should delay screening/enrollment until the course of antibiotic therapy has been completed.
  • Patients under ongoing treatment with another investigational medication or having been treated with an investigational medication within 28 days of screening.
  • No consent for biobanking.

Trial locations and contacts

17 sites in 1 country.

17 sites in 1 country

Germany17 sites

  • Universitätsklinikum Aachen - Med. Klinik IV

    Aachen, 52074

  • Hämatologisch onkologische Praxis

    Augsburg, 86150

  • Helios Klinikum Bad Saarow

    Bad Saarow, 15526

  • Klinik für Innere Medizin Uniklinik Köln

    Cologne, 50937

  • BAG Freiberg-Richter, Jacobasch, Wolf, Illmer

    Dresden, 01307

  • Universitätsklinikum Düsseldorf

    Düsseldorf, 40225

  • Universitätsklinikum Essen

    Essen, 45147

  • Uniklinikum Freiburg

    Freiburg im Breisgau, 79106

  • Universitätsklinikum Hamburg-Eppendorf

    Hamburg, 20246

  • Universitätsklinikum Jena

    Jena, 07740

  • Universitätsklinikum Magdeburg AöR

    Magdeburg, 39120

  • Universitätsmedizin Mainz

    Mainz, 55131

  • Universitätsklinikum Mannheim

    Mannheim, 68167

  • Johannes Wesling Klinikum Minden

    Minden, 32429

  • Stauferklinikum Schwäbisch Gmünd

    Mutlangen, 73557

  • Universitätsklinikum Tübingen

    Tübingen, 72076

  • University of Ulm

    Ulm, 89081

Other trials in Germany

About this study

Official title: A Phase-Ib/II Study of Ruxolitinib and Pomalidomide Combination Therapy in Patients With Primary and Secondary Myelofibrosis

The proposed study is an open-label, single-arm, Phase-Ib/II trial to assess the efficacy of oral drug combination ruxolitinib and pomalidomide in primary and secondary MF patients.

Read the detailed description

The proposed study is an open-label, single-arm, Phase-Ib/II trial to assess the efficacy of oral drug combination ruxolitinib and pomalidomide in primary and secondary MF patients. Dosages of the drugs are derived from previous Phase-I/II studies; ruxolitinib treatment will be started at 10 mg twice daily, whereas the dose of pomalidomide will be 0.5 mg once daily.

Dose reductions and discontinuations will be allowed in case of myelosuppressive effects.

Intra-patient dose escalation will be permitted for ruxolitinib to optimize efficacy of the therapeutic regimen; pomalidomide will be given in a permanent dosage of 0.5mg per day.

Treatment response will be evaluated continuously after each treatment cycle (1 cycle = 28 days) according to the IWG-MRT criteria expanded by the response criterion RCT-independency.

In case of progressive disease study therapy will be stopped; In patients showing response or stable disease, continuous therapy within the study is intended for a maximum of 12 treatment cycles; After completion of 12 treatment cycles, therapy can be continued if a measurable benefit of treatment is evident. This extension has to be discussed between the local and the principle investigator. Conditions leading to patient withdrawal from the study are detailed in the protocol "PATIENT WITHDRAWAL FROM STUDY PARTICIPATION".

Being tested
INCB018424/CC-4047
Medical subject headings
Primary Myelofibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • INCB018424/CC-4047Drug

    Cohort 1: For patients (1-41) the starting dose of ruxolitinib in this trial is 10 mg twice daily po; pomalidomide will be administered at a permanent dose of 0.5 mg po once daily.

    Cohort 2: For patients (42-90) the starting dose of ruxolitinib in this trial is 10 mg twice daily po; the starting dose of pomalidomide is 0.5 mg po once daily.

Study groups in NCT01644110
Study groupRoleWhat they receive
ruxolitinib/pomalidomideExperimentalCohort 1 (Patient 1 - Patient 41): ruxolitinib treatment will be started at 10 mg twice daily up to 25 mg twice daily, whereas the dose of pomalidomide will be 0.5 mg once daily. Cohort 2 (Patient 42 - Patient 90): ruxolitinib treatment will be started at 10 mg twice daily up to 25 mg twice daily, whereas the dose of pomalidomide will be started at 0.5 mg once daily up to 2 mg once daily.
Receives: Drug: INCB018424/CC-4047

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Best response rate within 12 treatment cycles according to the IWG-MRT criteria (including CR, PR, CI) and red cell transfusion (RCT) independency according to Gale et al 2010 and 2011).

    Best response rate within 12 treatment cycles according to the IWG-MRT

    Measured: one year

Secondary outcomes (7)

  1. Overall safety profile of ruxolitinib and pomalidomide combination observed during treatment, as well as cumulative incidence of leukemic transformation

    Overall safety profile of ruxolitinib and pomalidomide combination characterized by type, frequency, severity (graded using the National Cancer Institute Common Terminology Criteria for Adverse Events [NCI CTCAE] Version 3.0), timing and relatedness of adverse events (AEs) and laboratory abnormalities observed during treatment, as well as cumulative incidence of leukemic transformation

    Measured: one year

  2. Progression-free survival

    Progression-free survival

    Measured: three years

  3. duration of response

    duration of response

    Measured: three years

  4. overall survival

    overall survival

    Measured: three years

  5. Quality of life assessed by the Myeloproliferative Neoplasm Symptom

    Quality of life assessed by the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF Protocol 5/25/11), change in ECOG performance status from study entry to each visit where the variable is measured.

    Measured: three years

  6. Clinical Benefit - Assessment of each patient

    Clinical Benefit:

    • Lab / Clinical data: Stable disease (SD) plus hematologic improvement: prolongation of RBC transfusion intervals by ≥50% compared to baseline in transfusion dependent patients or ≥1 g/dL Hb increase in the absence of RBC transfusion dependency and/or
    • Questionaire: Stable disease (SD) plus improvement of MF-associated symptoms: SD plus improvement of at least one MF-associated symptom according to the MPN-SAF / EORTC QLQ-C30 or FACT-Lym by a minimum of 50% and/or SD plus improvement of ≥ two MF-associated symptoms according the MPN-SAF / EORTC QLQ-C30 or FACT-Lym by a minimum of 25% each.

    Measured: three years

  7. Monthly Response assessment

    Response criteria:

    Assessment according to the IWG-MRT (based on lab, clinical data): CR / PR / CI / PD / SD / RD/ RBC-TD / RBC-TI

    Measured: three years

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted18 Jul 2012 (estimated)
  2. Study startAug 2013
  3. Primary completionApr 2024
  4. Study completion27 Apr 2024

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Model description
Cohort 1 (Patient 1 - Patient 41): ruxolitinib treatment will be started at 10 mg twice daily up to 25 mg twice daily, whereas the dose of pomalidomide will be 0.5 mg once daily. Cohort 2 (Patient 42 - Patient 90): ruxolitinib treatment will be started at 10 mg twice daily up to 25 mg twice daily, whereas the dose of pomalidomide will be started at 0.5 mg once daily up to 2 mg once daily.
Masking
None (open label)
Enrolment
96 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01644110
Sponsor's protocol ID
POMINC(MPNSG02-12)
Other IDs
2012-002431-29
Acronym
POMINC
Status last verified
May 2023
First submitted
16 Jul 2012
Last updated
30 Mar 2025
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing INCB018424/CC-4047 in people with Primary Myelofibrosis and Secondary Myelofibrosis, run by University of Ulm at 17 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 16 inclusion and 23 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 30 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 17 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 combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

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?

Best response rate within 12 treatment cycles according to the IWG-MRT criteria (including CR, PR, CI) and red cell transfusion (RCT) independency according to Gale et al 2010 and 2011). (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 NCT01644110.

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 30 Mar 2025.

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