Clinical trial NCT01310816: A Safety and Efficacy Study of Patients With Metastatic or Locally Advanced (Unresectable) Chondrosarcoma
Official title: A Phase 2, Double-Blind, Placebo-Controlled Study Evaluating the Safety and Efficacy of IPI-926 in Patients With Metastatic or Locally Advanced (Unresectable) Chondrosarcoma
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Conventional Chondrosarcoma
Keywords: malignant neoplasms of the bone
bone sarcoma
Lead sponsor: Infinity Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
First posted: 2011-03-09
Enrollment: 105
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: IPI-926 is an inhibitor of the hedgehog pathway. IPI-926 may improve therapeutic outcomes in patients with Chondrosarcoma.

Detailed description: Study IPI-926-04 is a Phase 2, double-blind, placebo-controlled, multicenter, trial evaluating the safety and efficacy of IPI-926 in patients with metastatic or locally advanced (unresectable) chondrosarcoma. The study includes an optional cross-over to open-label IPI-926 for patients randomly assigned to placebo who experience documented disease progression.

Eligibility criteria: Inclusion Criteria:

* At least 18 years of age at the time of signing informed consent.
* Pathologically diagnosed conventional chondrosarcoma. Patients must have tumor sample(s) available or provide tumor samples from a new biopsy
* Metastasis to at least 1 location or locally advanced disease that is deemed unresectable by a surgeon
* At least 1 radiologically measurable target lesion per RECIST 1.1.
* Patients must have documented radiographic progression of disease within the 6-month period prior to screening. (MRI or CT Scan)
* Eastern Cooperative Oncology Group (ECOG) performance status: 0 or 1.
* Life expectancy of at least 3 months
* All women of child-bearing potential (WCBP), all sexually active male patients, and all partners of patients must agree to use adequate methods of birth control throughout the study and for 30 days after the last dose of study drug.
* Ability to adhere to the study visit schedule and all protocol requirements.
* Voluntarily signed an informed consent form.

Exclusion Criteria:

* Other invasive malignancies diagnosed within the last 5 years, except non-melanoma skin cancer and localized cured prostate and cervical cancer.
* Systemic anti-cancer therapy within 21 days prior to the first dose of study drug, or radiotherapy within 14 days prior to the first dose of study drug.
* Prior treatment with a Hedgehog pathway inhibitor
* Medically significant surgical procedures or significant traumatic injury within 28 days before Day 1.
* Inadequate hematologic function defined by:
* Hemoglobin \<8.0 g/dL (80 g/L) (may be increased to this level with transfusion as long as there is no evidence of active bleeding).
* Inadequate hepatic function defined by:
* Aspartate aminotransferase (AST) and/or alanine aminotransferase (ALT) \>2.5 x upper limit of normal (ULN).
* Total bilirubin \>1.5 x ULN (with the exception of patients with Gilbert's disease).
* Cirrhotic liver disease, ongoing alcohol abuse, or known chronic active or acute hepatitis.
* Inadequate renal function defined by serum creatinine \>1.5 x ULN
* Patients with a 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. Examples include, but are not limited to sepsis and recent significant traumatic injury.
* Known human immunodeficiency virus (HIV) positivity.
* Known hypersensitivity to IPI-926, or any of the excipients in IPI-926 or placebo capsules.
* Pregnant or lactating women.
* Current administration of the medications or foods which are known to be moderate or strong inhibitors of CYP3A4 activity

Primary outcomes:
measure: Progression Free Survival (PFS)
timeFrame: estimated 6 months
Secondary outcomes:
measure: Comparison of Time To Progression (TTP)
timeFrame: estimated 6 months
measure: Comparison of Overall Survival (OS)
timeFrame: estimated 6 months
measure: Overall Response Rate (ORR)
timeFrame: estimated 6 months
Other outcomes:
Locations:
city: Scottsdale
country: United States
facility: (TGen) Translational Genomics Research Institute
state: Arizona
city: Santa Monica
country: United States
facility: Sarcoma Oncology Center
state: California
city: Aurora
country: United States
facility: University of Colorado Cancer Center
state: Colorado
city: Miami
country: United States
facility: University of Miami - Sylvester Comprehensive Cancer Center
state: Florida
city: Boston
country: United States
facility: Dana-Farber Cancer Institute
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic
state: Minnesota
city: St. Louis
country: United States
facility: Washington University in St. Louis
state: Missouri
city: New York
country: United States
facility: Columbia University Medical Center
state: New York
city: Portland
country: United States
facility: OHSU Knight Cancer Institute
state: Oregon
city: Philadelphia
country: United States
facility: Pennsylvania Oncology Hematology Associates
state: Pennsylvania
city: Philadelphia
country: United States
facility: Fox Chase Cancer Center
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Houston
country: United States
facility: MD Anderson Cancer Center
state: Texas
city: Seattle
country: United States
facility: University of Washington - Seattle Cancer Care Alliance
state: Washington
city: Camperdown
country: Australia
facility: Sydney Cancer Centre
state: New South Wales
city: East Bentleigh
country: Australia
facility: Monash Medical Centre
state: Victoria
city: Wien
country: Austria
facility: Medizinische Universität Wien
state: Vienna
city: Toronto
country: Canada
facility: Mount Sinai Hospital
state: Ontario
city: Villejuif
country: France
facility: Institut de Cancérologie Gustave Roussy
state: Ile-de-france
city: Saint Herblain cedex
country: France
facility: Centre René Gauducheau
state: Pays de La Loire
city: Lyon
country: France
facility: Centre Léon Bérard, Service d'Oncologie Médicale
state: Rhone-alpes
city: Mannheim
country: Germany
facility: Universitätsmedizin Mannheim
state: Baden-wuerttemberg
city: Bad Saarow
country: Germany
facility: Helios Klinikum Bad Saarow
state: Brandenburg
city: Essen
country: Germany
facility: Universitätsklinikum Essen
state: Nordrhein-westfalen
city: Bologna
country: Italy
facility: IRCCS Istituto Ortopedico Rizzoli
city: Milano
country: Italy
facility: Fondazione IRCCS Istituto Nazionale dei Tumori
city: Leiden
country: Netherlands
facility: Leids Unversitair Medisch Centrum
state: Zuid-Holland
city: Oslo
country: Norway
facility: Radiumhospitalet
city: Warszawa
country: Poland
facility: Centrum Onkologii Instytut im. Marii Sklodowskiej-Curie w Warszawie
state: Mazowieckie
city: Moscow
country: Russian Federation
facility: SI Russian Oncological Research Center
city: Moscow
country: Russian Federation
facility: Moscow n.a. P.A Herzen Oncology Research Institute of Rosmedtechnologies
city: Lund
country: Sweden
facility: Skånes Universitetssjukhus
state: Skane
city: Birmingham
country: United Kingdom
facility: Royal Orthopaedic Hospital
state: England
city: London
country: United Kingdom
facility: University College Hospital
state: England
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle General Hospital
state: England
Study officials:
affiliation: Infinity Pharmaceuticals, Inc.
name: Pedro Santabarbara, MD
role: STUDY_CHAIR
References: