The goal of the redePHine study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in participants with primary hyperoxaluria type 1 (PH1). The trial will consist of 2 Study Periods. During the first Study Period, there will be 2 parts. In Part A, adult participants will be treated with a single ascending dose to identify a recommended dose. In Part B, pediatric participants will be treated with the recommended dose. Following the first Study Period, participants will start Study Period 2, a long-term monitoring program to comply with local and national requirements.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
23
Intravenous (IV) infusion
Mayo Clinic
Rochester, Minnesota, United States
RECRUITINGNucleus Network
Saint Paul, Minnesota, United States
WITHDRAWNHospices Civils de Lyon- Hôpital Femmes Mères Enfants
Lyon, France
NOT_YET_RECRUITINGKindernierenzentrum Bonn
Bonn, Germany
NOT_YET_RECRUITINGHeidi Chaker
Sfax, Tunisia
RECRUITINGQueen Elizabeth Hospital Birmingham
Birmingham, United Kingdom
RECRUITINGRoyal Free Hospital
London, United Kingdom
RECRUITINGIncidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs)
Time frame: Up to 6 months
Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6
Time frame: Up to 6 months
Absolute change in UOx corrected for body surface area
Time frame: Up to 6 months
Percent change in plasma glycolate from Baseline to Month 6
Time frame: Up to 6 months
Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24
Time frame: Up to 24 months
Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)
Time frame: Up to 6 months
Urine concentrations for LNP lipids
Time frame: Up to 6 months
Antidrug antibodies to ABO-101 and anti-Cas protein antibodies
Time frame: Up to 6 months
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