Clinical trial NCT01371617: A Phase 2 Study With IPI-926 in Patients With Myelofibrosis
Official title: A Phase 2 Study of IPI-926 in Patients With Myelofibrosis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Myelofibrosis
Fibrosis, Bone Marrow
Keywords: IPI-926
926
Myelofibrosis
Hedgehog
Infinity Pharmaceuticals
Post Essential thrombocythemia
Post Polycythemia vera
Lead sponsor: Infinity Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2011-06-13
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Maximum age (years): 90
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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 \[post-ET MF\]).


Eligibility criteria: 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.

Primary outcomes:
measure: 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
timeFrame: At least 2 months
Secondary outcomes:
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: Arizona Mayo Clinic
state: Arizona
city: Palo Alto
country: United States
facility: Stanford University School of Medicine, Division of Hematology
state: California
city: Houston
country: United States
facility: The University of Texas MD Anderson Cancer Center
state: Texas
Study officials:
affiliation: M.D. Anderson Cancer Center
name: Srdan Verstovsek, M.D.; Ph.D
role: PRINCIPAL_INVESTIGATOR
References: