A Study to Evaluate the Efficacy and Safety of Pemigatinib (INCB054828) in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement - (FIGHT-203)

MPN (Myeloproliferative Neoplasms) Clinical Trial

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)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT03011372
• Other study ID #: INCB 54828-203
• Status: Active, not recruiting
• Phase: Phase 2
• Start date: April 25, 2017
• Est. completion date: July 31, 2024

Study information

• Verified date: May 2024
• Source: Incyte Corporation
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 47
• Est. completion date: July 31, 2024
• Est. primary completion date: July 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• MPN (Myeloproliferative Neoplasms)
• Myeloproliferative Disorders
• Neoplasms

Intervention

Drug:
• Pemigatinib
Pemigatinib once a day by mouth for 2 consecutive weeks and 1 week off therapy. Participants will receive either the intermittent dose (as written) or continuous dosing.

Locations

Country	Name	City	State
Austria	Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz	Linz
Austria	Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus	Wien
Austria	Medical University of Vienna	Wien
Belgium	Universitair Ziekenhuis (Uz) Leuven	Leuven
Canada	Princess Margaret Cancer Center	Toronto	Ontario
France	Centre Leon Berard	Lyon
France	Chu de Nice - Hospital L Archet	Nice Cedex 3
France	Hospital Saint Louis	Paris Cedex 10
France	Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole	Toulouse Cedex 9
Germany	University Medical Center Rwth Aachen	Aachen
Germany	Universitatsklinikum Halle (Saale)	Halle
Germany	Universitatsklinikum Jena	Jena
Germany	Universitatsklinikum Leipzig	Leipzig
Germany	University Hospital Mannheim	Mannheim
Germany	Johannes Wesling Klinikum Minden	Minden
Italy	Ospedale Papa Giovanni Xxiii	Bergamo
Italy	Azienda Ospedaliero-Universitaria Careggi (Aouc)	Florence
Japan	Kindai University Hospital	Osaka
Japan	Ntt Medical Center Tokyo	Tokyo
Spain	Hospital Clinico Universitario de Valencia	Valencia
Switzerland	Inselspital - Universitaetsspital Bern	Bern
Switzerland	Universitatsspital Zurich	Zurich
United Kingdom	Guys and St Thomas Nhs Foundation Trust	London
United Kingdom	Oxford University Hospitals Nhs Foundation Trust	Oxford
United States	Emory University - Winship Cancer Institute	Atlanta	Georgia
United States	City of Hope National Medical Center	Duarte	California
United States	Md Anderson Cancer Center	Houston	Texas
United States	Franciscan St. Francis Health	Indianapolis	Indiana
United States	Weill Cornell Medical Centers	New York	New York
United States	Mayo Clinic Arizona	Phoenix	Arizona
United States	Washington University School of Medicine	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	Stanford Cancer Institute	Stanford	California

Sponsors (1)

Lead Sponsor	Collaborator
Incyte Corporation

Countries where clinical trial is conducted

United States,  Austria,  Belgium,  Canada,  France,  Germany,  Italy,  Japan,  Spain,  Switzerland,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	The proportion of participants who achieve Complete Response (CR) based on response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement		: Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary	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		Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary	The proportion of subjects who achieve a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation		Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary	The proportion of subjects who achieve a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation		Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary	Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause		Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary	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		Assessed at protocol-defined timepoints through end of study, up to approximately 24 months.
Secondary	Progression-free survival (PFS)	PFS is defined as the time from the first date of taking study drug until the date of disease progression, as measured by response criteria for myeloid/lymphoid neoplasms with FGFR1 rearrangement, or until death due to any cause, whichever is earlier.	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.
Secondary	Overall survival	Overall survival is defined as the time from the first day of taking study drug until death due to any cause. Subjects without death observed at the time of the analysis will be censored at last date known to be alive.	From date of first study drug dose until death due to any cause, assessed up to approximately 24 months.
Secondary	Safety and tolerability as assessed by frequency, duration, and severity of adverse events	A treatment-emergent AE was defined as an event occurring after exposure to at least 1 dose of study drug. A treatment-related AE was defined as an event with a definite, probable, or possible causality to study medication. A serious AE is an event resulting in death, hospitalization, persistent or significant disability/incapacity, or is life threatening, a congenital anomaly/birth defect or requires medical or surgical intervention to prevent 1 of the outcomes above. The intensity of an AE was graded according to the National Cancer Institute common terminology criteria for adverse events (NCI-CTCAE) version 4.03: Grade 1 (Mild); Grade 2 (Moderate); Grade 3 (Severe); Grade 4 (life-threatening).	From baseline through 30-35 days after end of treatment, up to 7 months per individual subject