A Phase I Study of Oral Arsenic Trioxide with or without Ascorbic Acid in Adults with Myelofibrosis

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Title A Phase I Study of Oral Arsenic Trioxide with or without Ascorbic Acid in Adults with Myelofibrosis
Contact JGreene
Principal Investigator Meir Wetzler, MD
Study Number 154609
Phase1
Summary

This is a phase I trial for patients with Myelofibrosis. Study agent is arsenic trioxide produced by Cephalon, Inc. and ascorbic acid.

The specific purpose of this study is to:

·         determine the highest dose of arsenic trioxide with or without ascorbic acid (Vitamin C) that may be given safely

·         compare effects, both good and bad, of arsenic trioxide with or without ascorbic acid (Vitamin C)

·         test whether arsenic trioxide with or without ascorbic acid (Vitamin C) helps to treat your myelofibrosis

This is a phase I study. A phase I study tests the safety and possible effectiveness, determines a safe dose range and identifies side effects of an investigational drug.

You will receive the investigational drug in 28 day cycles. Arsenic trioxide with or without ascorbic acid (vitamin C) will be taken daily for 21 days, by mouth with orange juice. After 21 days, there will be a resting period of 7 days. This will complete a 28 day cycle. If you are assigned to an ascorbic acid (vitamin C) group, you will also take ascorbic acid (vitamin C) by mouth at 1000 mg per day for 21 days immediately after taking the dose of arsenic trioxide. After 21 days there will be a resting period of 7 days. If you complete 6 months of treatment, you will be eligible to continue treatment if your doctor feels it will benefit you, and if drug is available.

We will start arsenic trioxide at a dose of 0.15mg/kg daily. This is the approved dose in acute promyelocytic leukemia (APL). There are 3 possible dose levels to be tested and each dose will be given either with or without ascorbic acid. A dose of arsenic trioxide will be given to a group of patients without ascorbic acid. If that dose is determined to be safe, the same dose will then be given with ascorbic acid to another group of patients. Doses will be increased until either the top dose of the study is reached, or severe side effects are seen at a lower dose.

Eligibility
  • Good performance status
  • Evidence of primary myelofibrosis, essential thrombocythemia related myelofibrosis, or polycythemia vera related myelofibrosis requiring therapy, including those previously treated and relapsed or refractory or with symptomatic splenomegaly
  • greater than 18 years of age
  • Serum Bilirubin less than or equal to 2 x ULN
  • ALT less than or equal to 2 x ULN
  • Creatinine less than 1.5 ULN
  • No infections
  • No significant cardiovascular disease
  • No major surgery within 28 days
  • Ability to swallow study medicine by mouth
For more information about the clinical study go to Clinicaltrial.Gov