The purpose of this study is to assess the safety, tolerability, PK and PD of BBP-671 in healthy volunteers and patients with Propionic Acidemia or Methylmalonic Acidemia.
This is the first-in-human study with BBP-671 and is designed to provide healthy subjects single- and multiple-dose and patient multidose safety, tolerability, PK, and PD data regarding BBP-671 for future clinical studies.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
TRIPLE
Enrollment
79
Community Health Clinic
Topeka, Indiana, United States
UPMC Children's Hospital of Pittsburg
Pittsburgh, Pennsylvania, United States
PPD Development, LP
Austin, Texas, United States
Incidence of adverse events following administration of BBP-671
Time frame: 49 days
BBP-671 concentration dependent change in change from baseline in QTcF
Time frame: 49 days
Pharmacokinetic Assessments: Cmax
Time to maximum concentration (Cmax)
Time frame: 49 days
Pharmacokinetic Assessments: Tmax
Time to reach maximum observed plasma concentration (Tmax)
Time frame: 49 days
Pharmacokinetic Assessments: t1/2
Plasma decay half-life (t1/2)
Time frame: 49 days
Pharmacokinetic Assessments: AUC0-tau
Area under the plasma concentration-time curve (AUC0-tau)
Time frame: 49 days
Pharmacokinetic Assessments: CL/F
Apparent clearance (CL/F)
Time frame: 15 days
Pharmacokinetic Assessments: Vz/F
Apparent volume of distribution (Vz/F)
Time frame: 15 days
Pharmacokinetic Assessments: CLr
Renal clearance (CLr)
Time frame: 15 days
Food Effect: Cmax
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Time to maximum concentration
Time frame: 10 days
Food Effect: Tmax
Time to reach maximum observed plasma concentration
Time frame: 10 days
Food Effect: AUC
Area under the plasma concentration-time curve
Time frame: 10 days
Pharmacodynamic Assessment: Whole blood, plasma, and urine biomarker concentrations will be quantified and summarized using appropriate descriptive parameters
Measurement will be done using liquid chromatography-tandem mass spectrometry
Time frame: 49 days