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Phase I Study of GC1008 in Patients With Primary Myelofibrosis (PMF), Post-polycythemia Vera/Essential Thrombocythemia Related Myelofibrosis (Post-PV/ET MF)

Phase 1
18 Years
Not Enrolling
Myelofibrosis, Primary Myelofibrosis, Post-polycythemia Vera Related Myelofibrosis, Post-essential Thrombocythemia Related Myelofibrosis

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Trial Information

Phase I Study of GC1008 in Patients With Primary Myelofibrosis (PMF), Post-polycythemia Vera/Essential Thrombocythemia Related Myelofibrosis (Post-PV/ET MF)

Inclusion Criteria:

- Age >18

- ECOG 0-2

- Intermediate-1 or higher by IWG-MRT Post PV/ET MF patients OR intermediate-1 or
higher JAK2V617F negative PMF

- Bone marrow MF-2 or higher as assessed by the European consensus grading score AND
grade 3 or higher by modified Bauermeister scale.

- Patients must be able to give written informed consent to participate. Patients may
not be consented by a durable power of attorney.

- Male and female patients of child-producing potential must agree to use effective
contraception while enrolled on study and receiving the investigational agent, and
for at least 3 months after the last treatment.

- At the time of enrollment, patients must be >4 weeks since major surgery,
radiotherapy, chemotherapy (except hydroxyurea) immunotherapy, or biotherapy/targeted
therapies and recovered from the toxicity of prior treatment to < Grade 1, exclusive
of alopecia. Concurrent cancer therapy is not permitted for the exception of
hydroxyurea if already being used at a stable dose for 3 weeks prior to screening.

- Patients must have negative tests for human immunodeficiency virus (HIV) and for
hepatitis viruses B and C (antibody and/or antigen) unless the result is consistent
with prior vaccination or prior infection with full recovery.

- Marrow: Absolute neutrophil count ≥ 500/mm3, and platelet count ≥50,000/mm3 without
the need for platelet transfusion within 4 weeks

- Hepatic: Serum total bilirubin >1.5 X upper limit of normal (ULN) (Patients with
Gilbert's Disease may be included if their total bilirubin is >3.0 mg/dL); alanine
aminotransferase (ALT), and aspartate aminotransferase (AST) >2.5 X ULN.

- Renal: Serum creatinine of < 1.5 x upper limit of normal (ULN) or, if higher,
estimated or measured creatinine clearance >45 mL/min.

- Coagulation:

1. Prothrombin Time (PT) < 1.5 X ULN

2. Partial thromboplastin time (aPTT) < 1.5 X ULN

Exclusion Criteria:

- Central nervous system (CNS) cancer or metastases, meningeal carcinomatosis,
malignant seizures, or a disease that either causes or threatens neurologic
compromise (e.g., unstable vertebral metastases).

- Pregnant or nursing women, due to the unknown effects of GC1008 on the developing
fetus or newborn infant.

- Patients with known bleeding diathesis or signs of uncontrolled active bleeding
(hematuria, GI bleeding) other than self-limited causes of benign etiology that have
been adequately investigated at the discretion of the investigator.

- Patients requiring anticoagulation with aspirin > 81mg daily, unfractionated heparin,
low molecular weight heparin (LMWH), direct anti-thrombin inhibitors, or vitamin K
antagonists (e.g. warfarin). This does not apply to patients actively receiving
aspirin at a dose of ≤ 81mg a day.

- Patients diagnosed with another malignancy - unless following curative intent
therapy, the patient has been disease free for at least 5 years and the probability
of recurrence of the prior malignancy is >5%. Patients with curatively treated early
stage squamous cell carcinoma of the skin, basal cell carcinoma of the skin, or
cervical intraepithelial neoplasia (CIN) are eligible for this study.

- Patients with an organ transplant, including those that have received an allogeneic
bone marrow transplant.

- Use of investigational agents within 4 weeks prior to study enrollment (within 6
weeks if the treatment was with a long-acting agent such as a monoclonal antibody).

- Significant or uncontrolled medical illness, such as congestive heart failure (CHF),
myocardial infarction, symptomatic coronary artery disease, significant ventricular
arrhythmias within the last 6 months, or significant pulmonary dysfunction. Patients
with a remote history of asthma or active mild asthma may participate.

- Active autoimmune disorders or concurrent immunosuppressive medications such as
prednisone, interferon, cyclosporine, methotrexate or azathiopurine.

- Active infection requiring antibiotics.

- A known allergy to any component of GC1008.

- Patients who, in the opinion of the Investigator, have significant medical or
psychosocial problems that warrant exclusion. Examples of significant problems
include, but are not limited to:

1. Other serious non-malignancy-associated medical conditions that may be expected
to limit life expectancy or significantly increase the risk of SAEs.

2. Any condition, psychiatric, substance abuse, or otherwise, that, in the opinion
of the Investigator, would preclude informed consent, consistent follow-up, or
compliance with any aspect of the study

Type of Study:


Study Design:

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

To assess the safety and tolerability of GC1008 in patients with primary myelofibrosis (PMF) or post-polycythemia vera/essential thrombocythemia myelofibrosis (Post-PV/ET MF).

Outcome Time Frame:

28 days

Safety Issue:


Principal Investigator

John Mascarenhas, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

Icahn School of Medicine at Mount Sinai


United States: Food and Drug Administration

Study ID:

GCO 10-1450



Start Date:

February 2011

Completion Date:

January 2013

Related Keywords:

  • Myelofibrosis
  • Primary Myelofibrosis
  • Post-polycythemia Vera Related Myelofibrosis
  • Post-essential Thrombocythemia Related Myelofibrosis
  • monoclonal antibody
  • Primary Myelofibrosis
  • Polycythemia
  • Polycythemia Vera
  • Thrombocythemia, Essential
  • Thrombocytosis



Mount Sinai School of Medicine New York, New York  10029