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Fulvestrant, Palbociclib and Erdafitinib in ER+/HER2-/FGFR-amplified Metastatic Breast Cancer

Phase 1
Completed
Conditions
Metastatic Breast Cancer
Interventions
Registration Number
NCT03238196
Lead Sponsor
Vanderbilt-Ingram Cancer Center
Brief Summary

This is an open-label, multi-institution, phase Ib trial that evaluates the safety and tolerability and preliminary anti-tumor activity of fulvestrant, palbociclib and erdafitinib in patients with ER+/HER2-/FGFR-amplified metastatic breast cancer.

Detailed Description

Primary Objectives

To determine the safety and tolerability of fulvestrant, palbociclib and erdafitinib in patients with ER+/HER2-/FGFR-amplified MBC.

Secondary Objectives

* To determine the anti-tumor effect of fulvestrant, palbociclib and erdafitinib in patients with ER+/HER2-/FGFR-amplified MBC.

* Pharmacokinetic assessments of erdafitinib

Correlative Objectives

* To determine the therapeutic predictive role of FGFR1-4, CCND1-2, CDK4 and CDK6 amplifications, and RB1 and ESR1 mutations on clinical outcome

* To determine if the FGFR1 amplification levels is an early surrogate of response

* To determine if the cfDNA results at disease progression show new genomic alterations potentially associated with resistance to CDK4/6 and FGFR inhibition

* To determine pharmacodynamic biomarkers of FGFR inhibition

Recruitment & Eligibility

Status
COMPLETED
Sex
All
Target Recruitment
35
Inclusion Criteria
  • Patients must be able to swallow and retain oral medication
  • Patients must be ≥ 18 years of age
  • Female patients of no childbearing potential must be post-menopausal. Postmenopausal female subjects should be defined prior to protocol enrollment by any of the following:
  • Participants at least 60 years of age; OR
  • Participants under 60 years of age and naturally (spontaneous, no alternative pathologic or physiological cause) amenorrhea for at least 12 months; OR
  • Medical ovarian failure confirmed by follicle-stimulating hormone (FSH) and estradiol levels in the post menopausal range per local institutional normal range; OR
  • Prior bilateral oophorectomy; OR
  • Prior radiation castration with amenorrhea for at least 6 months; OR
  • Treatment with a luteinizing hormone-releasing hormone (LH-RH) agonist (such as goserelin acetate or leuprolide acetate) is permitted for induction of ovarian suppression as long as it has been initiated at least 28 days prior to study enrollment
  • Patients must have ECOG performance status 0 - 1
  • Patients must have clinical stage IV or inoperable locoregional recurrent invasive mammary carcinoma that is:
  • ER+ and/or PgR+ (≥ 1% positive stained cells) by immunohistochemistry (IHC)
  • HER2-negative (by IHC or FISH, per ASCO guidelines)
  • FGFR1 - 4 amplified
  • Patients must have evaluable (may have either measurable or non-measurable) disease
  • Patients must have available tissue for FGFR determination
  • Patients must have had at least one line of therapy in the metastatic setting
  • Current use of any of the drugs listed on the Cautionary Concomitant Med list has to be approved by the Study Chair
  • Patients must have adequate hematologic, hepatic and renal function. All laboratory tests must be obtained within 2 weeks from study drug initiation. These include:
  • ANC ≥ 1,500/mm3
  • Platelet count ≥ 100,000/mm3
  • HgB ≥ 9.0 g/dL
  • Creatinine clearance ≥ 40 mL/min/1.73 m2
  • SGOT, SGPT ≤ 2.5 x ULN if no liver metastasis present; SGOT, SGPT ≤ 4 x ULN if liver metastasis present
  • Albumin ≥ 2.0 g/dL
  • Total serum bilirubin ≤ 1.5 x ULN (≤ 3 x ULN or direct bilirubin ≤ 1.5 x ULN if known Gilbert's syndrome)
  • Potassium within institutional normal limits
  • Phosphorus ≤ institutional upper limit of normal
Exclusion Criteria
  • Prior use of an FGFR inhibitor
  • More than 2 lines of chemotherapy in the metastatic setting. No limit on endocrine therapy lines. Prior exposure to CDK4/6 inhibitor acceptable.
  • Radiation therapy ≤ 2 weeks prior to study entry. Patients who have received prior radiotherapy must have recovered from toxicity (≤ grade 1) induced by this treatment (except for alopecia)
  • Prior cancer therapy (except for endocrine therapy) must have been discontinued for 1 week prior to initiation of study drugs
  • Concurrent anti-cancer therapy other than the ones specified in the protocol is not permitted during study participation. Bisphosphonates or denosumab are allowed
  • Major surgery within 4 weeks of enrollment
  • Herbal preparations are not allowed throughout the study, and should be discontinued 14 days prior to initiation of study treatment
  • Any corneal or retinal abnormality likely to increase the risk of eye toxicity, such as:
  • Current corneal pathology such as keratitis, keratoconjunctivitis, keratopathy, corneal abrasion, inflammation or ulceration
  • Uncontrolled glaucoma despite standard of care therapy
  • Diabetic retinopathy with macular edema
  • Known active wet, age-related macular degeneration (AMD)
  • Known central serous retinopathy (CSR) or retinal vascular occlusion (RVO)
  • Uncontrolled intercurrent illness including, but not limited to:
  • Malabsorption syndrome significantly affecting gastrointestinal function
  • Ongoing or active infection requiring antibiotics/antivirals
  • Impairment of lung function (COPD > grade 2, lung conditions requiring oxygen therapy)
  • Symptomatic congestive heart failure
  • Unstable angina pectoris, angioplasty, stenting, or myocardial infarction within 6 months
  • Clinically significant cardiac arrhythmia (multifocal premature ventricular contractions, bigeminy, trigeminy, ventricular tachycardia that is symptomatic or requires treatment [National Cancer Institute -Common Terminology Criteria for Adverse Events, Version 4.03, grade 3]
  • QTcF ≥ 480 msec on screening EKG
  • Known history of clinically significant QT/QTc prolongation or Torsades de Pointes(TdP)
  • ST depression or elevation of ≥ 1.5 mm in 2 or more leads
  • Diarrhea of any cause ≥ CTCAE grade 2 that does not resolve within a few days when adequately treated with anti-diarrhea medications
  • Psychiatric illness/social situations that would compromise patient safety or limit compliance with study requirements including maintenance of a compliance/pill diary
  • Symptomatic brain metastases (patients with a history of brain metastases must be clinically stable for more than 4 weeks from completion of radiation treatment and be off steroids)
  • Known history of chronic liver or chronic renal failure
  • Poor wound healing capacity

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
EscalationErdafitinibFulvestrant - injection into muscle 1 time per month Palbociclib capsule taken by mouth 1 time per day every 21 days followed by 1 week of rest (no drug taken) Erdafitinib tablet taken by mouth 1 time per day
EscalationPalbociclibFulvestrant - injection into muscle 1 time per month Palbociclib capsule taken by mouth 1 time per day every 21 days followed by 1 week of rest (no drug taken) Erdafitinib tablet taken by mouth 1 time per day
ExpansionFulvestrantFulvestrant - injection into muscle 1 time per month Palbociclib capsule taken by mouth 1 time per day every 21 days followed by 1 week of rest (no drug taken) Erdafitinib tablet taken by mouth 1 time per day
EscalationFulvestrantFulvestrant - injection into muscle 1 time per month Palbociclib capsule taken by mouth 1 time per day every 21 days followed by 1 week of rest (no drug taken) Erdafitinib tablet taken by mouth 1 time per day
ExpansionErdafitinibFulvestrant - injection into muscle 1 time per month Palbociclib capsule taken by mouth 1 time per day every 21 days followed by 1 week of rest (no drug taken) Erdafitinib tablet taken by mouth 1 time per day
ExpansionPalbociclibFulvestrant - injection into muscle 1 time per month Palbociclib capsule taken by mouth 1 time per day every 21 days followed by 1 week of rest (no drug taken) Erdafitinib tablet taken by mouth 1 time per day
Primary Outcome Measures
NameTimeMethod
Number of Participants With Dose Limiting Toxicities (DLT) in the First Cycle for the Determination of the Maximum Tolerated Dose (MTD)From the time of randomization up to 4 weeks of treatment (cycle 1), for each patient

Number of participants with DLT in the first cycle for the determination of the MTD.

Secondary Outcome Measures
NameTimeMethod
Progression-free SurvivalImaging studies will be performed every 8 weeks from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months

Assessment of clinical impact \[anti-tumor effect\] of the combination of erdafitinib, palbociclib and fulvestrant in patients with ER+/ FGFR amplified metastatic breast cancer will be assessed by measuring the interval (in months) between treatment initiation and disease progression. Progression-free survival (PFS) time is defined as the time from treatment initiation to progression date or death (whichever comes first). Those alive without prpgression is censored at the last date of known alive. Median PFS time and 95% confidence intervals are obtained using Kaplan-meier method.

Overall Response RateImaging studies will be performed every 8 weeks from date of randomization until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 48 months

Assessment of clinical impact \[anti-tumor effect\] of the combination of erdafitinib, palbociclib and fulvestrant in patients with ER+/ FGFR amplified metastatic breast cancer by measure the rate (%) of complete and partial responses seen in patients with measurable disease. The response of a patient will be evaluated using Solid Tumor Response Criteria RECIST v1.1.

Clinical Benefit Rate (CBR; Complete Response + Partial Response + Stable Disease Without Disease Progression at 6 Months)From the time of randomization up to 6 months for each patient

Assessment of clinical impact \[anti-tumor effect\] of the combination of erdafitinib, palbociclib and fulvestrant in patients with ER+/ FGFR amplified metastatic breast cancer by measure the rate (%) of complete and partial responses + stability of disease at 6 months seen in patients with measurable disease. Response of a patient was evaluated using Solid Tumor Response Criteria -RECIST v1.1.

Pharmacokinetic Assessment of Erdafitinib - Area Under the Curve (AUC)From the time of randomization up to 4 weeks of treatment for each patient

The area under the plasma concentration-time curve from time zero to the last measurable concentration

Pharmacokinetic Assessment of Erdafitinib - Cmax (Maximum Plasma Concentration)From the time of randomization up to 4 weeks of treatment for each patient

The maximum (peak) observed plasma drug concentration after oral dose administration

Pharmacokinetic Assessment of Erdafitinib - TmaxFrom the time of randomization up to 4 weeks of treatment for each patient

Time to reach maximum (Cmax) plasma drug concentration after oral dose administration (time)

Pharmacokinetic Assessment of Erdafitinib - CL/FFrom the time of randomization up to 4 weeks of treatment for each patient

Apparent total body clearance of drug from the plasma after oral administration

Incidence of Treatment-Emergent Adverse Events [Tolerability]From date of randomization until 28 days post treatment discontinuation from any cause, assessed up to 48 months

Assessment of adverse events throughout the study. The number of patients who had any grade of adverse events were reported.

Trial Locations

Locations (6)

Baptist Memorial Hospital MEMPHIS

🇺🇸

Memphis, Tennessee, United States

University of Alabama

🇺🇸

Birmingham, Alabama, United States

University of Pittsburgh Medical Center

🇺🇸

Pittsburgh, Pennsylvania, United States

Vanderbilt-Ingram Cancer Center

🇺🇸

Nashville, Tennessee, United States

University of Texas Southwestern Simmons Comprehensive Cancer Center

🇺🇸

Dallas, Texas, United States

Memorial Sloan-Kettering Cancer Center

🇺🇸

New York, New York, United States

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