Clinical trial NCT03011372: A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)
Official title: A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: MPN (Myeloproliferative Neoplasms)
Keywords: Myeloid neoplasm
fibroblast growth factor receptor inhibitor
FGFR1 rearrangement
8p11
eosinophilia
eosinophilic syndrome
Lymphoid neoplasm
Lead sponsor: Incyte Corporation
Sponsor class: INDUSTRY
First posted: 2017-01-05
Enrollment: 47
Sex: ALL
Minimum age (years): 18
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 evaluate the efficacy and safety of pemigatinib (INCB054828) in subjects with myeloid/lymphoid neoplasms with fibroblast growth factor receptor (FGFR) 1 rearrangement.


Eligibility criteria: Inclusion Criteria:

* Documented lymphoid or myeloid neoplasm with 8p11 rearrangement known to lead to FGFR1 activation, based on standard diagnostic cytogenetic evaluation performed locally, before signing informed consent for this study.
* Eligible subjects must:

  * Have relapsed after stem cell transplantation or after other disease modifying therapy, OR
  * Not be current candidates for stem cell transplantation or other disease modifying therapies.
* Note: All relapsed/refractory subjects must have evidence of either cytogenetic or hematological disease and have no evidence of residual toxicity (eg, graft-versus-host disease requiring treatment).
* Life expectancy ≥ 12 weeks.
* Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.

Exclusion Criteria:

* Prior receipt of a selective FGFR inhibitor.
* History and/or current evidence of ectopic mineralization/calcification, including but not limited to soft tissue, kidneys, intestine, myocardia, or lung, except calcified lymph nodes and asymptomatic arterial or cartilage/tendon calcifications.
* Current evidence of corneal disorder/keratopathy, including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis, as confirmed by ophthalmologic examination.
* Use of any potent cytochrome P450 3A4 inhibitors or inducers within 14 days or 5 half-lives (whichever is shorter) before the first dose of study drug.

Primary outcomes:
measure: The proportion of participants who achieve Complete Response (CR) based on response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement
timeFrame: : Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary outcomes:
measure: The proportion of subjects who achieve response, defined as a best response of CR or PR, as determined by investigator assessment according to the response criteria
timeFrame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: The proportion of subjects who achieve a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation
timeFrame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: The proportion of subjects who achieve a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation
timeFrame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause
timeFrame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: Duration of response, defined as the time from first assessment of CR or PR to the earlier of disease progression or death due to any cause
timeFrame: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
measure: Progression-free survival (PFS)
timeFrame: From the date of first study drug dose until the date of disease progression or until death due to any cause, whichever is earlier, assessed up to approximately 24 months.
measure: Overall survival
timeFrame: From date of first study drug dose until death due to any cause, assessed up to approximately 24 months.
measure: Safety and tolerability as assessed by frequency, duration, and severity of adverse events
timeFrame: From baseline through 30-35 days after end of treatment, up to 7 months per individual subject
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Mayo Clinic Arizona
state: Arizona
city: Duarte
country: United States
facility: City of Hope National Medical Center
state: California
city: Stanford
country: United States
facility: Stanford Cancer Institute
state: California
city: Atlanta
country: United States
facility: Emory University - Winship Cancer Institute
state: Georgia
city: Indianapolis
country: United States
facility: Franciscan St. Francis Health
state: Indiana
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: Weill Cornell Medical Centers
state: New York
city: Houston
country: United States
facility: Md Anderson Cancer Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Linz
country: Austria
facility: Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz
city: Wien
country: Austria
facility: Medical University of Vienna
city: Wien
country: Austria
facility: Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus
city: Leuven
country: Belgium
facility: Universitair Ziekenhuis (Uz) Leuven
city: Toronto
country: Canada
facility: Princess Margaret Cancer Center
state: Ontario
city: Lyon
country: France
facility: Centre Leon Berard
city: Nice Cedex 3
country: France
facility: Chu de Nice - Hospital L Archet
city: Paris Cedex 10
country: France
facility: Hospital Saint Louis
city: Toulouse Cedex 9
country: France
facility: Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole
city: Aachen
country: Germany
facility: University Medical Center Rwth Aachen
city: Halle
country: Germany
facility: Universitatsklinikum Halle (Saale)
city: Jena
country: Germany
facility: Universitatsklinikum Jena
city: Leipzig
country: Germany
facility: Universitatsklinikum Leipzig
city: Mannheim
country: Germany
facility: University Hospital Mannheim
city: Minden
country: Germany
facility: Johannes Wesling Klinikum Minden
city: Bergamo
country: Italy
facility: Ospedale Papa Giovanni Xxiii
city: Florence
country: Italy
facility: Azienda Ospedaliero-Universitaria Careggi (Aouc)
city: Osaka
country: Japan
facility: Kindai University Hospital
city: Tokyo
country: Japan
facility: Ntt Medical Center Tokyo
city: Valencia
country: Spain
facility: Hospital Clinico Universitario de Valencia
city: Bern
country: Switzerland
facility: Inselspital - Universitaetsspital Bern
city: Zurich
country: Switzerland
facility: Universitatsspital Zurich
city: London
country: United Kingdom
facility: Guys and St Thomas Nhs Foundation Trust
city: Oxford
country: United Kingdom
facility: Oxford University Hospitals Nhs Foundation Trust
Study officials:
affiliation: Incyte Corporation
name: Philomena Collucci, MD
role: STUDY_DIRECTOR
References: