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A Phase 2 Study With IPI-926 in Patients With Myelofibrosis

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT01371617
Recruitment Status : Completed
First Posted : June 13, 2011
Last Update Posted : November 15, 2012
Information provided by (Responsible Party):
Infinity Pharmaceuticals, Inc.

Brief Summary:
The purpose of this study is to determine the safety and efficacy of IPI-926 in patients with myelofibrosis (MF) (primary myelofibrosis [PMF], post-polycythemia vera myelofibrosis [post-PV MF], or post-essential thrombocythemia myelofibrosis [post-ET MF]).

Condition or disease Intervention/treatment Phase
Primary Myelofibrosis Fibrosis, Bone Marrow Drug: IPI-926 Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase 2 Study of IPI-926 in Patients With Myelofibrosis
Study Start Date : October 2011
Actual Primary Completion Date : August 2012
Actual Study Completion Date : August 2012

Arm Intervention/treatment
Experimental: IPI-926
Single Arm, Phase 2 trial evaluating the safety and efficacy of IPI-926 in patients with myelofibrosis
Drug: IPI-926
Single Arm study of oral IPI-926 at 160 mg, 130 mg or 110 mg daily, until progressive disease or intolerability to study treatments or withdrawal of ICF
Other Names:
  • Hedgehog Inhibitor
  • Hedgehog Pathway Inhibitor

Primary Outcome Measures :
  1. To determine the overall response rate of IPI-926, defined as clinical improvement (CI); partial remission (PR); and complete remission (CR), according to the International working group (IWG) criteria in patients with Myelofibrosis [ Time Frame: At least 2 months ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 90 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • ≥18 years of age at the time of signing the ICF.
  • Voluntarily sign an ICF.
  • Pathologically confirmed PMF or post ET/PV MF as per the WHO diagnostic criteria (note that it must include at least Grade 1 marrow fibrosis, see Appendix 3) with intermediate-1, intermediate -2, or high risk disease according to the IWG prognostic scoring system (see Appendix 4). If patients have low risk disease, then they must have symptomatic splenomegaly that is ≥ 10 cm below left costal margin by physical exam.
  • ECOG performance of 0-2.
  • Life expectancy of at least 3 months.
  • Recovery to Grade 1 or baseline of any toxicities due to prior systemic treatments, excluding alopecia.
  • If a woman, be of non-child bearing potential or, for women of child-bearing potential (WCBP) (defined as a sexually mature woman who has not undergone surgical sterilization or who has not been naturally post-menopausal for at least 24 consecutive months for women ≤55 years; for women >55 years 12 consecutive months), must have a negative serum or urine pregnancy test result within 2 weeks of first dose of study drug.
  • All WCBP and all sexually active male patients must agree to use adequate methods of birth control throughout the study. Adequate methods of contraception include use of oral contraceptives with an additional barrier method, double barrier methods (diaphragm with spermicidal gel or condoms with contraceptive foam), Depo-Provera, partner vasectomy, and total abstinence.
  • Ability to adhere to the study visit schedule and all protocol requirements.

Exclusion Criteria:

  • Prior treatment with any inhibitor of the hedgehog pathway (e.g. GDC-0449).
  • Received any treatment for myelofibrosis within 2 weeks of study entry.
  • Other invasive malignancies diagnosed within the last 3 years, except non-melanoma skin cancer and localized cured prostate and cervical cancer.
  • Inadequate hepatic function defined by:
  • Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) >2.5 x upper limit of normal (ULN).
  • Direct bilirubin >1.5 x ULN.
  • Cirrhotic liver disease, ongoing alcohol abuse, or known chronic active or acute hepatitis.
  • Inadequate renal function defined by serum creatinine >2 x ULN.
  • History of stroke, unstable angina, myocardial infarction, or ventricular arrhythmia requiring medication or mechanical control within the last 6 months.
  • Presence of active infection or systemic use of antibiotics within 72 hours of treatment.
  • Significant co-morbid condition or disease, which in the judgment of the Investigator, would place the patient at undue risk or interfere with the study.
  • Known human immunodeficiency virus (HIV) positivity.
  • Known hypersensitivity to IPI-926, or any of the excipients in IPI-926 capsules.
  • Pregnant or lactating women.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT01371617

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United States, Arizona
Arizona Mayo Clinic
Scottsdale, Arizona, United States, 85260
United States, California
Stanford University School of Medicine, Division of Hematology
Palo Alto, California, United States, 94025
United States, Texas
The University of Texas MD Anderson Cancer Center
Houston, Texas, United States, 77030
Sponsors and Collaborators
Infinity Pharmaceuticals, Inc.
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Principal Investigator: Srdan Verstovsek, M.D.; Ph.D M.D. Anderson Cancer Center
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Responsible Party: Infinity Pharmaceuticals, Inc. Identifier: NCT01371617    
Other Study ID Numbers: IPI-926-08
First Posted: June 13, 2011    Key Record Dates
Last Update Posted: November 15, 2012
Last Verified: November 2012
Keywords provided by Infinity Pharmaceuticals, Inc.:
Infinity Pharmaceuticals
Post Essential thrombocythemia
Post Polycythemia vera
Additional relevant MeSH terms:
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Primary Myelofibrosis
Myeloproliferative Disorders
Bone Marrow Diseases
Hematologic Diseases