Clinical Trial: P1101 in Treating Patients With Myelofibrosis

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: Phase II Study of P1101 in Early Myelofibrosis

Brief Summary: This pilot phase II trial studies P1101 (polyethyleneglycol [PEG]-proline-interferon alpha-2b) in treating patients with myelofibrosis. PEG-proline-interferon alpha-2b is a substance that can improve the body's natural response and may slow the growth of myelofibrosis.

Detailed Summary:

PRIMARY OBJECTIVES:

I. To evaluate for clinical response (complete remission [CR], partial remission [PR], or clinical improvement [CI]) as defined by International Working Group-Myeloproliferative Neoplasms Research and Treatment (IWG-MRT) criteria in a cohort of intermediate-2/high risk myelofibrosis (MF) patients. Response in a second cohort of early stage MF patients will also be described.

SECONDARY OBJECTIVES:

I. To evaluate the adverse event profile of P1101 in patients with myelofibrosis by cohort (early vs intermediate-2/high risk).

II. To evaluate the tolerability of P1101 in patients with myelofibrosis by cohort (early vs intermediate-2/high risk).

TERTIARY OBJECTIVES:

I. To evaluate quality of life (QOL) and patient-reported symptoms using the Myeloproliferative Neoplasm Symptom Assessment Form (MPN-SAF) with P1101 for patients with myelofibrosis by cohort (early vs intermediate-2/high risk).

II. To evaluate the impact of P1101 on bone marrow and histological features of myelofibrosis including cytogenetics, blast percentage, fibrosis, and JAK2-V617F allele burden by cohort (early vs intermediate-2/high risk).

OUTLINE:

Patients receive PEG-proline-interferon alpha-2b subcutaneously (SC) on days 1 and 15. Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.

After completion of study treatment, patients are followed up every 3-6 months for 3 years.


Sponsor: Mayo Clinic

Current Primary Outcome: Best overall response (CR, PR, or CI) as determined by International Working Group Criteria [ Time Frame: Up to 3 years ]

The proportion of successes will be estimated by the number of successes divided by the total number of evaluable patients. Confidence intervals for the true success proportion will be calculated according to the approach of Duffy and Santner.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Incidence of adverse events, as measured by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCI CTCAE v4) [ Time Frame: Up to 3 years ]
    The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.
  • Survival time [ Time Frame: Time from registration to death due to any cause, assessed up to 3 years ]
    The distribution of survival time will be estimated using the method of Kaplan-Meier.


Original Secondary Outcome:

  • Survival time [ Time Frame: Time from registration to death due to any cause, assessed up to 3 years ]
    The distribution of survival time will be estimated using the method of Kaplan-Meier.
  • Incidence of adverse events, as measured by National Cancer Institute Common Terminology Criteria for Adverse Events version 4.0 (NCI CTCAE v4) [ Time Frame: Up to 3 years ]
    The maximum grade for each type of adverse event will be recorded for each patient, and frequency tables will be reviewed to determine patterns. Additionally, the relationship of the adverse event(s) to the study treatment will be taken into consideration.


Information By: Mayo Clinic

Dates:
Date Received: February 17, 2015
Date Started: August 2015
Date Completion:
Last Updated: October 6, 2016
Last Verified: July 2016