- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02370329
P1101 in Treating Patients With Myelofibrosis
Phase II Study of P1101 in Early Myelofibrosis
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
PRIMARY OBJECTIVE:
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).
EXPLORATORY AND CORRELATIVE RESEARCH 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.
Study Type
Enrollment (Actual)
Phase
- Phase 2
Contacts and Locations
Study Locations
-
-
Arizona
-
Scottsdale, Arizona, United States, 85259
- Mayo Clinic in Arizona
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Description
Inclusion Criteria:
Evaluable myelofibrosis by IWG-MRT criteria including one or more of the following:
- Spleen >= 5 cm below the left costal margin
- MPN-SAF total symptom score (TSS) > 10 at baseline
- Hemoglobin < 10 g/dL
- Confirmed diagnosis of myelofibrosis (primary myelofibrosis or myelofibrosis secondary to essential thrombocythemia or polycythemia vera) by World Health Organization (WHO) diagnostic criteria (3 major and 2 minor criteria: major criteria: megakaryocyte proliferation and atypia with either reticulin and/or collagen fibrosis, not meeting criteria for chronic myelogenous leukemia [CML], polycythemia vera [PV], myelodysplastic syndrome [MDS], or other myeloid neoplasm, JAK2V617F or other clonal marker or no evidence of reactive marrow fibrosis; minor criteria: leukoerythroblastosis, increased lactate dehydrogenase [LDH], anemia, palpable splenomegaly)
- For cohort 1: early stage MF (low or intermediate 1 stage as defined by Dynamic International Prognostic Scoring System [DIPSS]) without currently available treatment options
- For cohort 2: intermediate-2 or high risk MF patients as defined by DIPSS either not eligible for ruxolitinib or having failed under ruxolitinib
- No prior treatment for myelofibrosis (for cohort 1 only)
- Eastern Cooperative Oncology Group (ECOG) performance status (PS) 0, 1 or 2
- Platelet count >= 100,000/mm^3 (obtained =< 14 days prior to registration)
- Absolute neutrophil count (ANC) >= 1000/mm^3 (obtained =< 14 days prior to registration)
- Aspartate transaminase (AST) =< 2.5 x upper limit of normal (ULN) (obtained =< 14 days prior to registration)
- Alanine aminotransferase (ALT) =< 2.5 x ULN (obtained =< 14 days prior to registration)
- Calculated creatinine clearance must be >= 50 ml/min using the Cockcroft-Gault formula (obtained =< 14 days prior to registration)
- Negative pregnancy test done =< 7 days prior to registration, for women of childbearing potential only
- Ability to complete questionnaire(s) by themselves or with assistance
- Provide informed written consent
- Willing to return to enrolling institution for follow-up
- Willing to provide blood samples for correlative research purposes
Exclusion Criteria:
- Patients who have had chemotherapy or radiation =< 2 weeks of registration
- For cohort 1 only: patients with evidence of intermediate 2 or high risk disease (according to DIPSS)
- For cohort 1 only: patients with a bone marrow biopsy with < 15% cellularity, evidence of collagen fibrosis, osteosclerosis, or blasts > 10% in peripheral blood or marrow (demonstrating advanced disease)
- Patients who have received a prior stem cell transplant
- Patients who have received radiation to the spleen within 3 months prior to registration
- Patients with intolerance to compounds similar to pegylated interferon alpha-2b
- Patients with evidence of >= grade 2 peripheral sensory neuropathy
Any of the following:
- Pregnant women
- Nursing women
- Men or women of childbearing potential who are unwilling to employ adequate contraception
- Co-morbid systemic illnesses or other severe concurrent disease which, in the judgment of the investigator, would make the patient inappropriate for entry into this study or interfere significantly with the proper assessment of safety and toxicity of the prescribed regimens
- Immunocompromised patients or patients known to be human immunodeficiency virus (HIV) positive and currently receiving antiretroviral therapy
- Uncontrolled simultaneous illness including, but not limited to, ongoing or active infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, history of depression, or psychiatric illness/social situations that would limit compliance with study requirements
- Receiving any other investigational agent which would be considered as a treatment for the primary neoplasm
- History of myocardial infarction =< 6 months prior to registration, or congestive heart failure requiring use of ongoing maintenance therapy for life-threatening ventricular arrhythmias
- History of significant or major funduscopic findings including, but not limited to, retinal exudates, hemorrhage, detachment, neovascularization, papilledema, optic atrophy, micro-aneurysm or macular changes
- Other active malignancy at time of registration; EXCEPTIONS: non-melanotic skin cancer or carcinoma-in-situ of the cervix
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Treatment (PEG-proline-interferon alpha-2b)
Patients receive PEG-proline-interferon alpha-2b SC on days 1 and 15.
Courses repeat every 28 days in the absence of disease progression or unacceptable toxicity.
|
Correlative studies
Ancillary studies
Other Names:
Given SC
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
Up to 3 years
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
|
Time from registration to death due to any cause, assessed up to 3 years
|
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.
|
Up to 3 years
|
Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Changes in patient-reported symptoms and QOL as measured by MPN-SAF
Time Frame: Baseline to up to 3 years
|
Patient-reported symptoms and QOL will be described at each time point using the mean, confidence interval, median, and range.
Changes in individual symptoms, changes in a symptom scale composed of symptoms specific to MF patients, and changes in the MPN TSS will be investigated.
Graphical procedures will include stream plots of individual patient scores and plots of average values over time.
Correlational analyses will be done to determine the relationships among patients-reported symptoms and QOL, as well as with clinical outcomes and clinician-assessed symptoms.
|
Baseline to up to 3 years
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Jeanne Palmer, Mayo Clinic
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Estimated)
Study Completion (Estimated)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimated)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- MC138F (Other Identifier: Mayo Clinic in Arizona)
- NCI-2015-00183 (Registry Identifier: CTRP (Clinical Trial Reporting Program))
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Primary Myelofibrosis
-
Assaf-Harofeh Medical CenterUnknownMyelofibrosis, Primary | Myelofibrosis, Post PV | Myelofibrosis, Post ETIsrael
-
The University of Hong KongRecruitingMyelofibrosis | Primary Myelofibrosis, Prefibrotic StageHong Kong
-
AbbVieRecruitingMyelofibrosis (MF)United States, Australia, Austria, Belgium, Bulgaria, Canada, Croatia, Denmark, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Poland, Puerto Rico, Russian Federation, Serbia, Spain, Taiwan, Turkey, United... and more
-
AbbVieActive, not recruitingMyelofibrosis (MF)United States, Australia, Austria, Belgium, Bulgaria, Canada, Croatia, France, Germany, Greece, Israel, Italy, Japan, Korea, Republic of, Netherlands, New Zealand, Russian Federation, Serbia, South Africa, Spain, Sweden, Taiwan, Turkey and more
-
AbbVieActive, not recruitingMyelofibrosis (MF)United States, Argentina, Australia, Brazil, Bulgaria, Chile, Hungary, Israel, Italy, Japan, Korea, Republic of, Spain, Sweden, Turkey
-
AbbVieTerminatedMyelofibrosis (MF)United States, Korea, Republic of, South Africa
-
Centre Hospitalier Annecy GenevoisCompleted
-
National Taiwan University HospitalRecruitingPre-fibrotic MyelofibrosisTaiwan
-
Chia Tai Tianqing Pharmaceutical Group Co., Ltd.RecruitingModerate and High Risk MyelofibrosisChina
-
Novartis PharmaceuticalsTerminatedMyelofibrosis With High Molecular Risk MutationsBelgium, Spain, United Kingdom, Hungary, Italy, Japan, Taiwan, Germany, Canada, Singapore, Austria, Australia, France, Israel, Sweden, Switzerland, Hong Kong, Greece, Turkey, Brazil, Russian Federation, Denmark, Portugal, Norway, Poland
Clinical Trials on Laboratory Biomarker Analysis
-
Children's Oncology GroupNational Cancer Institute (NCI)Completed
-
ECOG-ACRIN Cancer Research GroupNational Cancer Institute (NCI)CompletedProstate Cancer
-
Alliance for Clinical Trials in OncologyNational Cancer Institute (NCI)Active, not recruitingLeukemia | Acute Lymphoblastic Leukemia | Acute Promyelocytic LeukemiaUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedUntreated Adult Acute Lymphoblastic Leukemia | Untreated Childhood Acute Lymphoblastic LeukemiaUnited States, Canada, Australia, New Zealand, Puerto Rico, Switzerland
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedChildhood Acute Lymphoblastic Leukemia in Remission | Recurrent Childhood Acute Lymphoblastic LeukemiaUnited States
-
Alliance for Clinical Trials in OncologyNational Cancer Institute (NCI)CompletedLung CancerUnited States
-
Alliance for Clinical Trials in OncologyNational Cancer Institute (NCI)Completed
-
Children's Oncology GroupNational Cancer Institute (NCI)WithdrawnClear Cell Renal Cell Carcinoma | Rhabdoid Tumor of the Kidney | Congenital Mesoblastic Nephroma | Childhood Kidney NeoplasmUnited States
-
Gynecologic Oncology GroupNational Cancer Institute (NCI)WithdrawnBreast Carcinoma | BRCA1 Mutation Carrier | BRCA2 Mutation CarrierUnited States
-
Children's Oncology GroupNational Cancer Institute (NCI)CompletedWilms Tumor and Other Childhood Kidney TumorsUnited States