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

Sponsor

Infinity Pharmaceuticals, Inc. (Industry)

Overall Status

Completed

CT.gov ID

NCT01371617

Collaborator

(none)

Enrollment

Locations

Arm

Duration (Months)

4.7

Patients Per Site

0.5

Patients Per Site Per Month

Study Details

Study Description

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

Study Design

Study Type:

Interventional

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 :

Oct 1, 2011

Actual Primary Completion Date :

Aug 1, 2012

Actual Study Completion Date :

Aug 1, 2012

Arms and Interventions

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

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

Outcome Measures

Primary Outcome Measures

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 [At least 2 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years to 90 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Arizona Mayo Clinic	Scottsdale	Arizona	United States	85260
2	Stanford University School of Medicine, Division of Hematology	Palo Alto	California	United States	94025
3	The University of Texas MD Anderson Cancer Center	Houston	Texas	United States	77030