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FALCON Clinical Trial
FALCON is an international, multi-center, randomized, double-blind, placebo-controlled Phase 3 trial examining the safety, tolerability, and efficacy of bardoxolone methyl in qualified patients with ADPKD.
Due to the rarity of ADPKD, nephrologists may see only a few patients who could qualify for participation. If you have patients who may qualify, please consider referring these patients by directing the patients to this website (PKDstudy.com). Your referral is crucial to the success of FALCON. Every patient counts. Your referral can make a difference.
The study team’s involvement with your patients would be exclusively study-specific. If any additional testing is required to confirm a diagnosis, the cost will be covered by Reata. Eligible participants may receive compensation and/or reimbursement for study-related time and travel, including flight or lodging costs.
Reata’s decision to initiate FALCON was based, in part, on data from Phase 2 study in patients with rare kidney diseases, PHOENIX. Week 12 data from the ADPKD cohort of PHOENIX were released in July 2018 and can be found here.
In summary, the available data demonstrate that bardoxolone methyl significantly improved kidney function in patients with ADPKD as measured by the estimated glomerular filtration rate (eGFR). Reata collected historical eGFR data for 29 of the 31 PHOENIX ADPKD study patients. The historical eGFR data demonstrate that ADPKD patients’ kidney function was declining at an average annual rate of 4.8 mL/min/1.73 m2 prior to study entry. At week 12, which was the primary endpoint of PHOENIX, an increased eGFR of 9.3 mL/min/1.73 m2 improvement (p<0.0001) was observed in bardoxolone treated patients. The observe eGFR improvement represents a recovery of approximately two years of average eGFR loss.
With respect to safety in the Phase 2 ADPKD cohort of PHOENIX, no treatment-related serious adverse events were reported, and the reported adverse events were generally mild to moderate in intensity. The most common adverse event was muscle spasms. Twenty-eight patients were available for the analysis, and only one patient (3%) discontinued for a treatment-related adverse event of fatigue.
Bardoxolone methyl is an oral investigational drug in a class of drugs called Nrf2 activators. Bardoxolone methyl activates molecular pathways that promote the resolution of inflammation by restoring mitochondrial function, reducing oxidative stress, and inhibiting pro-inflammatory signaling. Bardoxolone methyl binds to Keap1 and consequently activates Nrf2, a transcription factor that promotes normal mitochondrial function by making reducing equivalents available for ATP production, and increases cellular antioxidant content. This reduces mitochondrial reactive oxygen species (ROS) production and ROS-mediated activation of inflammatory signaling complexes. Binding to Keap1 and activation of Nrf2 also inhibit NF-κB, the primary transcription factor producing proteins that promote inflammation and the production of ROS.
Bardoxolone methyl has been tested in many clinical studies in different disease indication. Data released from recent clinical trials show significant improvement in kidney function maintained in Alport syndrome patients after 48 Weeks of treatment and in ADPKD patients after 12 weeks of treatment.
Bardoxolone methyl has generally been well-tolerated in most patient populations studied to date. The most common side effects have included muscle cramps and nausea.
In one previous study, a small subset of Stage 4 CKD patients with type 2 diabetes demonstrated a risk for developing fluid overload adverse events. These patients were subsequently determined to have defined risk factors, including a medical history of heart failure and an elevated BNP level, indicating the presence of fluid retention prior to the study. The increased risk of fluid overload has not been observed in subsequent studies in patients with diabetic CKD, pulmonary hypertension, Alport syndrome, IgA nephropathy, or ADPKD that excluded at-risk patients and carefully monitored their fluid status.
In summary, bardoxolone methyl’s mechanism of action targets inflammatory mechanisms that promote loss of kidney function, which ultimately leads to the need for dialysis or kidney transplant. Data from studies in CKD patients suggest that bardoxolone methyl could produce a durable increase in kidney function. If a similar effect is observed in ADPKD patients, this could ultimately prevent or delay end-stage renal disease.
FALCON Study Design
Schema for Study of Bardoxolone Methyl in Patients with ADPKD
Please consider referring your patients with ADPKD to a research center by sharing this website (PKDstudy.com) or by submitting an Inquiry Form. A member of the study team will contact you as soon as possible.