A Phase 2, Open-Label, Monotherapy, Multicenter Study to Evaluate the Efficacy and Safety of INCB054828 in Subjects With Myeloid/Lymphoid Neoplasms With FGFR1 Rearrangement

Status

Not yet recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2016-002596-10-GB

Enrollment

Registered

2017-01-24

Start date

2017-01-30

Completion date

Unknown

Last updated

2020-08-31

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Lymphoid or myeloid neoplasm with 8p11 rearrangement known to lead to FGFR1 activation

Interventions

Product Code: INCB054828 Pharmaceutical Form: Tablet INN or Proposed INN: pemigatinib Current Sponsor code: INCB054828 Concentration unit: mg milligram(s) Concentration type: range Concentration numbe

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: • Men and women, aged 18 or older. • 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 a. Have relapsed after stem cell transplantation or after other disease modifying therapy, OR b. 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. • ECOG performance status 0 to 2. Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 20 F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range 26

Exclusion criteria

Exclusion criteria: • Prior receipt of a selective FGFR inhibitor. • History of calcium and phosphate hemostasis disorder or systemic mineral imbalance with ectopic calcification of soft tissues (exception: commonly observed calcifications in soft tissues, such as the skin, kidney, tendons, or vessels due to injury, disease, and aging, in the absence of systemic mineral imbalance). • Current evidence of clinically significant corneal disorder/keratopathy (including but not limited to bullous/band keratopathy, corneal abrasion, inflammation/ulceration, and keratoconjunctivitis, etc) or retinal disorder (including but not limited to macular/retinal degeneration, diabetic retinopathy, retinal detachment, etc) 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.

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective of this study is to evaluate the efficacy of pemigatinib in subjects with myeloid/lymphoid neoplasms with FGFR1 rearrangement.;Secondary Objective: The secondary objective is to evaluate the safety and efficacy of pemigatinib in subjects with myeloid/lymphoid neoplasms with FGFR1 rearrangement.;Primary end point(s): The primary endpoint of this study is the proportion of subjects who achieve CR as determined by investigator assessment according to the response criteria listed in Appendix D of the protocol;Timepoint(s) of evaluation of this end point: Investigators will assess response to treatment by evaluation of local peripheral blood results, bone marrow pathology, cytogenetics, and imaging for EMD at the frequencies listed in section 7.6 of the protocol.	—

Measure

Time frame

Main Objective: The primary objective of this study is to evaluate the efficacy of pemigatinib in subjects with myeloid/lymphoid neoplasms with FGFR1 rearrangement.;Secondary Objective: The secondary objective is to evaluate the safety and efficacy of pemigatinib in subjects with myeloid/lymphoid neoplasms with FGFR1 rearrangement.;Primary end point(s): The primary endpoint of this study is the proportion of subjects who achieve CR as determined by investigator assessment according to the response criteria listed in Appendix D of the protocol;Timepoint(s) of evaluation of this end point: Investigators will assess response to treatment by evaluation of local peripheral blood results, bone marrow pathology, cytogenetics, and imaging for EMD at the frequencies listed in section 7.6 of the protocol.

—

Secondary

Measure	Time frame
Secondary end point(s): The secondary endpoints for this study are as follows: • 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 listed in Appendix D of the protocol. • The proportion of subjects who achieve a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation. • The proportion of subjects who achieve a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation. • Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause. • 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. • Progression-free survival. • Overall survival. • Safety and tolerability, as assessed by evaluating the frequency, duration, and severity of AEs; through review of findings of physical examinations, changes in vital signs, and electrocardiograms (ECGs); and through clinical laboratory blood and urine sample evaluations.;Timepoint(s) of evaluation of this end point: Investigators will assess response to treatment by evaluation of local peripheral blood results, bone marrow pathology, cytogenetics, and imaging for EMD at the frequencies listed in section 7.6 of the protocol.	—

Measure

Time frame

Secondary end point(s): The secondary endpoints for this study are as follows: • 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 listed in Appendix D of the protocol. • The proportion of subjects who achieve a complete cytogenetic response (CCyR) as assessed by local analysis and investigator evaluation. • The proportion of subjects who achieve a partial cytogenetic response (PCyR) as assessed by local analysis and investigator evaluation. • Duration of CR, defined as the time from first assessment of CR to the earlier of disease progression or death due to any cause. • 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. • Progression-free survival. • Overall survival. • Safety and tolerability, as assessed by evaluating the frequency, duration, and severity of AEs; through review of findings of physical examinations, changes in vital signs, and electrocardiograms (ECGs); and through clinical laboratory blood and urine sample evaluations.;Timepoint(s) of evaluation of this end point: Investigators will assess response to treatment by evaluation of local peripheral blood results, bone marrow pathology, cytogenetics, and imaging for EMD at the frequencies listed in section 7.6 of the protocol.

—

Countries

Austria, France, Germany, Italy, Spain, Switzerland, United Kingdom, United States

Contacts

Public ContactClinical Trials Information

Incyte Corporation

RA@incyte.com13024252734

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026