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

Recruitment & Eligibility

Status: ot Recruiting

Sex: All

Target Recruitment: 46

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

• 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.

Study & Design

Study Type: Interventional clinical trial of medicinal product

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
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 E 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 Outcome Measures

Name	Time	Method

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