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

Sponsor

Incyte Corporation (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT03011372

Collaborator

(none)

Enrollment

Locations

Arm

73.6

Anticipated Duration (Months)

1.4

Patients Per Site

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
MPN (Myeloproliferative Neoplasms)	Drug: Pemigatinib	Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

47 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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)

Actual Study Start Date :

Apr 25, 2017

Anticipated Primary Completion Date :

Jun 12, 2023

Anticipated Study Completion Date :

Jun 12, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: Pemigatinib	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. Other Names: INCB054828

Arm

Intervention/Treatment

Experimental: Pemigatinib

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.

Other Names:

INCB054828

Outcome Measures

Primary Outcome Measures

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 Outcome Measures

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.]
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.]
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.]
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.]
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.]
Progression-free survival (PFS) [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.]
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.
Overall survival [From date of first study drug dose until death due to any cause, assessed up to approximately 24 months.]
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.
Safety and tolerability as assessed by frequency, duration, and severity of adverse events [From baseline through 30-35 days after end of treatment, up to 7 months per individual subject]
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).

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Mayo Clinic Arizona	Phoenix	Arizona	United States	85054
2	City of Hope National Medical Center	Duarte	California	United States	91010
3	Stanford Cancer Institute	Stanford	California	United States	94305
4	Emory University - Winship Cancer Institute	Atlanta	Georgia	United States	30322
5	Franciscan St. Francis Health	Indianapolis	Indiana	United States	46237
6	Washington University School of Medicine	Saint Louis	Missouri	United States	63110
7	Weill Cornell Medical Centers	New York	New York	United States	10021
8	Md Anderson Cancer Center	Houston	Texas	United States	77030
9	University of Utah	Salt Lake City	Utah	United States	84112
10	Ordensklinikum Krankenhaus Der Barmherzigen Schwestern Linz	Linz		Austria	04010
11	Medical University of Vienna	Wien		Austria	01090
12	Iii Med. Abteilung For Hematologie and Onkologie Hanuscfhkrankenhaus	Wien		Austria	01140
13	Universitair Ziekenhuis (Uz) Leuven	Leuven		Belgium	03000
14	Princess Margaret Cancer Center	Toronto	Ontario	Canada	M5G 2M9
15	Centre Leon Berard	Lyon		France	69373
16	Chu de Nice - Hospital L Archet	Nice Cedex 3		France	06202
17	Hospital Saint Louis	Paris Cedex 10		France	75475
18	Universitaire Du Cancer de Toulouse Institut Claudius Regaud Iuct-Oncopole	Toulouse Cedex 9		France	31059
19	University Medical Center Rwth Aachen	Aachen		Germany	D-52074
20	Universitatsklinikum Halle (Saale)	Halle		Germany	06120
21	Universitatsklinikum Jena	Jena		Germany	07740
22	Universitatsklinikum Leipzig	Leipzig		Germany	04103
23	University Hospital Mannheim	Mannheim		Germany	68167
24	Johannes Wesling Klinikum Minden	Minden		Germany	32429
25	Ospedale Papa Giovanni Xxiii	Bergamo		Italy	24127
26	Azienda Ospedaliero-Universitaria Careggi (Aouc)	Florence		Italy	50134
27	Kindai University Hospital	Osaka		Japan	589-8511
28	Ntt Medical Center Tokyo	Tokyo		Japan	141-8625
29	Hospital Clinico Universitario de Valencia	Valencia		Spain	46010
30	Inselspital - Universitaetsspital Bern	Bern		Switzerland	03010
31	Universitatsspital Zurich	Zurich		Switzerland	08091
32	Guys and St Thomas Nhs Foundation Trust	London		United Kingdom	SE1 9RT
33	Oxford University Hospitals Nhs Foundation Trust	Oxford		United Kingdom	OX3 7LE