Safety and Efficacy Study of CYT387 in Primary Myelofibrosis (PMF) or Post-polycythemia Vera (PV) or Post-essential Thrombocythemia (ET)

Phase 1

Completed

• Conditions: Primary Myelofibrosis; Post-Polycythemia Vera Myelofibrosis; Post-Essential Thrombocythemia Myelofibrosis; Blood - Haematological diseases

• Registration Number: ACTRN12611000159954
• Lead Sponsor: Gilead Sciences Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2011-02-09
• Study Completion: 2012-04-01
• Last Update Posted: 9 November 2020

Recruitment & Eligibility

• Status: Completed
• Sex: All
• Target Recruitment: 165

Inclusion Criteria

Diagnosis of PMF or post-ETIPV MF as per revised World Health Organization (WHO) criteria.
- High-risk or Intermediate-2 risk MF (as defined by the International Prognostic Scoring System [IPSS]); or intermediate-I risk MF (IPSS) associated with symptomatic splenomegaly/hepatomegaly and/or unresponsive to available therapy.
- Must be at least 18 years of age with life expectancy of >= 12 weeks.
- Must be able to provide informed consent and be willing to sign an informed consent form.
- Must have an Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1 or 2.
- Must have evidence of acceptable organ function within 7 days of initiating study drug as evidenced by the following:
-->SGOT (AST) or SGPT (ALT) <= 2.5 x upper limit of normal (ULN) (or <= 5 x ULN if in the investigator's opinion the elevation is due to extramedullary hematopoiesis)
-->Bilirubin <= 2.0 x ULN or direct bilirubin < 1.0
-->Serum creatinine <= 2.5 x ULN
-->Absolute neutrophil count >= 500/microlitre
-->Platelet count >= 50,000/microlitre
- Females of childbearing potential must have a negative pregnancy test within 4 days of initiating study drug.

Exclusion Criteria

- Any chemotherapy (e.g., hydroxyurea), immunomodulatory drug therapy (e.g., thalidomide), immunosuppressive therapy, corticosteroids >10 mg/day prednisone or equivalent, or growth factor treatment (e.g., erythropoietin) within 14 days prior to initiation of study drug.
- Incomplete recovery from major surgery within four weeks of study entry.
- Radiation therapy within four weeks of study entry.
- Women of childbearing potential, unless surgically sterile for at least 3 months (i.e., hysterectomy), OR postmenopausal for at least 12 months (FSH > 30 U/mL), OR unless they agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through end of study. Permitted methods for preventing pregnancy must be communicated to study subjects and their understanding confirmed.
- Men who partner with a woman of childbearing potential, unless they agree to take appropriate precautions to avoid pregnancy (with at least 99% certainty) from screening through to the end of study. Permitted methods for preventing pregnancy must be communicated to study subjects and their understanding confirmed.
- Females who are pregnant or are currently breastfeeding.
- Known positive status for HIV.
- Clinically active hepatitis B or C.
- Diagnosis of another malignancy unless free of disease for at least three years following therapy with curative intent. Patients with early-stage basal cell or squamous cell skin cancer, cervical intraepithelial neoplasia, cervical carcinoma in situ or superficial bladder cancer may be eligible to participate at the Investigator's discretion.
- Any acute active infection.
- Cardiac dysrhythmias requiring treatment, or prolongation of the QTc (Fridericia) interval to >450 msec for males or >470 msec for females at pre-study screening, unless attributable to pre-existing bundle branch block.
- Presence of >= Grade 2 peripheral neuropathy.
- Uncontrolled congestive heart failure (New York Heart Association Classification 3 or 4), uncontrolled or unstable angina, myocardial infarction, cerebrovascular accident, or pulmonary embolism within 3 months prior to initiation of study drug.
- Uncontrolled inter current illness or any concurrent condition that, in the Investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol.

Study & Design

• Study Type: Interventional
• Study Design: Not specified