Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
- Registration Number
- NCT06839235
- Lead Sponsor
- Arbor Biotechnologies
- Brief Summary
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.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 23
Not provided
Not provided
Study & Design
- Study Type
- INTERVENTIONAL
- Study Design
- SEQUENTIAL
- Arm && Interventions
Group Intervention Description Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design ABO-101 - Experimental: Part B: Single Dose Expansion ABO-101 -
- Primary Outcome Measures
Name Time Method Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs) Up to 6 months
- Secondary Outcome Measures
Name Time Method Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA) Up to 6 months Urine concentrations for LNP lipids Up to 6 months Antidrug antibodies to ABO-101 and anti-Cas protein antibodies Up to 6 months Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6 Up to 6 months Absolute change in UOx corrected for body surface area Up to 6 months Percent change in plasma glycolate from Baseline to Month 6 Up to 6 months Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24 Up to 24 months
Related Research Topics
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Trial Locations
- Locations (2)
Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Nucleus Network
🇺🇸St. Paul, Minnesota, United States
Mayo Clinic🇺🇸Rochester, Minnesota, United StatesClinical Trial SiteContact800-664-4542