| Study summary: |
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A Phase 1B, open label, dose finding study of an investigational medication for patients with primary myelofibrosis with low platelets. |
| Qualified participants must: |
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• Be able to sign an informed consent document
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Be 18 years of age or older
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Be diagnosed with PMF, PPV-MF or PET-MF, according to the investigator’s expert judgement, guided by the criteria outlined in the 2008 World Health Organization criteria (Table 2 in Tefferi and Vardiman, 2008, Appendix I) for PMF, and the proposed criteria for PPV-MF and PET-MF outlined by the IWG-MRT (Barosi et al, 2008, Appendix II) irrespective of JAK2 mutation status. A bone marrow biopsy/aspirate
will provide the relevant information
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Be classified as high risk (3 or more prognostic factors), intermediate risk level 2 (2 prognostic factors) or intermediate risk level 1 (1 prognostic factor). The prognostic factors, defined by the IMG-MRT (IWG, Cervantes et al, 2009) are:
- Age > 65 yrs (at time of Screening visit)
- Presence of constitutional symptoms (weight loss, fever, night sweats) (documented in Medical History)
- Marked anemia (hemoglobin < 10 g/dL)* (at Screening or Baseline Visit) Incyte Corporation Final v 1.0 Protocol INCB 18424-260 11 January 2011
- Leukocytosis (WBC > 25 x 109/L) (at Screening or Baseline Visit)
- Circulating (peripheral blood) blasts ≥ 1% at (Screening or Baseline Visit) * A hemoglobin value < 10 g/dL must be demonstrated during the Screening or Baseline Visit for subjects who are not transfusion dependent. Subjects receiving regular transfusions of packed red blood cells will be considered to have hemoglobin < 10 g/dL for the purpose of evaluation of risk factors.
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Subjects in whom treatment of MF is indicated based on presence of one or more of the following:
- IWG-MRT Prognostic Category of High Risk (3 or more risk factors)
- Palpable spleen at 10 cm or more below the costal margin
- Active symptoms of MF at the Screening Visit as demonstrated by one symptom score of at least 5 (on a zero to 10 scale) on at least one of the following items OR a score of 3 or greater on at least two of the following items on the Screening
- Symptom Form (Appendix VII):
- Early satiety
- Abdominal Discomfort
- Abdominal Pain
- Inactivity
- Night Sweats
- Pruritis
- Bone Pain
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Have discontinued all drugs used to treat underlying MF disease no later than 4 weeks prior to the Baseline visit
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Have an ECOG performance status of 0, 1, 2 or 3 (Appendix V)
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Have a palpable spleen measuring 5 cm, or greater, below the costal margin. The edge of the spleen shall be determined by palpation, and measured in centimeters, using a soft ruler (supplied by Sponsor), from the costal margin to the point of greatest splenic protrusion
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Have peripheral blood blast count of < 10% at the Screening and Baseline visits
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Have not previously received treatment with a JAK inhibitor |
| Participants may receive: |
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All study related medication and healthcare at no cost. |