A Phase 2 Study Of PRM-151 In Subjects With Myelofibrosis



Status:Active, not recruiting
Conditions:Cancer, Blood Cancer, Hematology, Hematology
Therapuetic Areas:Hematology, Oncology
Healthy:No
Age Range:18 - Any
Updated:9/13/2018
Start Date:October 1, 2013
End Date:April 2019

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A Phase 2, Prospective Study Of PRM-151 In Subjects With Primary Myelofibrosis (PMF), Post-Polycythemia Vera MF (Post-PV MF), Or Post-Essential Thrombocythemia MF (Post-ET MF)

PRM-151 is an investigational drug that is being developed for possible use in the treatment
of myelofibrosis (MF), a disease in which the bone marrow, which is the organ in the body
that makes blood cells, is replaced by fibrosis, or excess scar tissue.

The purpose of this study is to gather information on whether PRM-151 has an effect on the MF
disease, whether it is safe in patients with MF, and how well it is tolerated.

Stage 2 of this study is ongoing. Stage 2 is a randomized, double-blind Phase 2 study to
determine the efficacy and safety of three different doses of PRM-151 in subjects with PMF
and post ET/PV MF. Subjects will be randomized to one of three doses: 0.3 mg/kg, 3.0 mg/kg or
10 mg/kg of PRM-151. This is the second stage of an adaptive design study as defined in FDA
Draft Guidance for Industry: Adaptive Design Clinical Trials for Drugs and Biologics,
February 2010. Modifications to dose levels, schedule, and regimen have been made in Stage 2
based on data from Stage 1.

Stage 1 of this study has completed. Stage 1 was an open-label, Simon two stage, Phase 2
study to determine the efficacy and safety of two different dose schedules of PRM-151 in
subjects with PMF and post ET/PV MF. There were two treatment cohorts, each assigned to one
of two dose schedules of PRM-151. Subjects were assigned to a weekly or every four week
dosing schedule by the investigator.

Inclusion Criteria:

1. Subjects must be ≥18 years of age at the time of signing the Informed Consent Form
(ICF);

2. Subjects must voluntarily sign an ICF;

3. Subjects must have a pathologically confirmed diagnosis of PMF as per the WHO
diagnostic criteria or post ET/PV MF;

4. At least Grade 2 marrow fibrosis according to the WHO Grading of Bone Marrow Fibrosis;

5. Intermediate-1, intermediate -2, or high risk disease according to the IWG -MRT
Dynamic International Prognostic Scoring System

6. A bone marrow biopsy must be performed within four weeks prior to Cycle 1 Day 1
treatment to establish the baseline fibrosis score;

7. Subjects must not be candidates for ruxolitinib based on EITHER:

1. Platelet count < 50 x 10e9/L, OR

2. Hgb < 100 g/L, have received ≥ 2 units PRBC in the 12 weeks prior to study entry,
and be intolerant of or had inadequate response to ruxolitinib;

8. Subjects must have an Eastern Cooperative Oncology Group (ECOG) Performance Status of
0-2. (Appendix F);

9. Life expectancy of at least twelve months;

10. At least four weeks must have elapsed between the last dose of any MF- directed drug
treatments for myelofibrosis (including investigational therapies) and study
enrollment;

11. Recovery to ≤ Grade 1 or baseline of any toxicities due to prior systemic treatments,
excluding alopecia;

12. Women of child bearing potential (WCBP), defined as a sexually mature woman not
surgically sterilized or not post-menopausal for at least 24 consecutive months if ≤55
years or 12 months if >55 years, must have a negative serum pregnancy test within four
weeks prior to the first dose of study drug and must agree to use adequate methods of
birth control throughout the study. Adequate methods of contraception are outlined in
the protocol.

13. Ability to adhere to the study visit schedule and all protocol requirements;

14. Must have adequate organ function as demonstrated by the following:

- ALT (SGPT) and/or AST (SGOT) ≤ 3x upper limit of normal (ULN), or ≤ 4 x ULN (if
upon judgment of the treating physician, it is believed to be due to
extramedullary hematopoiesis [EMH] related to MF);

- Direct bilirubin ≤ 1.5 x ULN; or ≤ 2x ULN (if upon judgment of the treating
physician, it is believed to be due to EMH related to MF);

- Serum creatinine ≤ 2.5 mg/dL x ULN.

Exclusion Criteria:

1. White blood cell count > 25 x 10e9/L or > 10% peripheral blood blasts;

2. Other invasive malignancies within the last 3 years, except non- melanoma skin cancer
and localized cured prostate and cervical cancer;

3. History of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia
requiring medication or mechanical control within the last 6 months;

4. Presence of active serious infection;

5. Any serious, unstable medical or psychiatric condition that would prevent, (as judged
by the Investigator) the subject from signing the informed consent form or any
condition, including the presence of laboratory abnormalities, which places the
subject at unacceptable risk if he/she were to participate in the study or confounds
the ability to interpret data from the study;

6. Known history of human immunodeficiency virus (HIV), or known active hepatitis A, B,
or C infection;

7. Organ transplant recipients other than bone marrow transplant;

8. Women who are pregnant or lactating.
We found this trial at
12
sites
22 S Greene St
Baltimore, Maryland 21201
(410) 328-8667
Principal Investigator: Maria Baer, MD
Phone: 410-462-3178
University of Maryland Medical Center Founded in 1823 as the Baltimore Infirmary, the University of...
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Baltimore, MD
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1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Principal Investigator: Michael Savona, MD
Phone: 800-811-8480
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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Nashville, TN
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1 Gustave L Levy Pl # 271
New York, New York 10029
 (212) 241-6500
Principal Investigator: John Mascarenhas, MD
Phone: 212-241-9185
Mount Sinai Med Ctr Founded in 1852, The Mount Sinai Hospital is a 1,171-bed, tertiary-care...
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New York, NY
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500 S State St
Ann Arbor, Michigan 48109
(734) 764-1817
Principal Investigator: Moshe Talpaz, MD
Phone: 734-232-0773
University of Michigan The University of Michigan was founded in 1817 as one of the...
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Atlanta, Georgia 30322
Principal Investigator: Vamsi Kota, MD
Phone: 404-778-4334
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Atlanta, GA
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450 Brookline Ave
Boston, Massachusetts 2215
617-632-3000
Principal Investigator: Martha Wadleigh, MD
Phone: 617-632-5157
Dana-Farber Cancer Institute Since it’s founding in 1947, Dana-Farber has been committed to providing adults...
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Boston, MA
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Houston, Texas 77030
Principal Investigator: Srdan Verstovsek, MD
Phone: 713-745-3429
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Houston, TX
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New York, New York 10065
Principal Investigator: Ellen Ritchie, MD
Phone: 646-962-2700
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Palo Alto, California 94304
Principal Investigator: Jason Gotlib, MD
Phone: 650-725-0744
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Phoenix, Arizona 85054
Principal Investigator: Ruben Mesa, MD
Phone: 480-342-6066
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Vancouver, British Columbia
Principal Investigator: Lynda Foltz, MD
Phone: 604-684-5794
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Winston-Salem, North Carolina 27157
Principal Investigator: Dmitriy Berenzon, MD
Phone: 336-713-3539
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