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Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)

Phase 1
Recruiting
Conditions
Primary Hyperoxaluria Type 1 (PH1)
Interventions
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
Inclusion Criteria

Not provided

Exclusion Criteria

Not provided

Study & Design

Study Type
INTERVENTIONAL
Study Design
SEQUENTIAL
Arm && Interventions
GroupInterventionDescription
Experimental: Part A: Single Ascending Dose Escalation/Adaptive DesignABO-101-
Experimental: Part B: Single Dose ExpansionABO-101-
Primary Outcome Measures
NameTimeMethod
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
NameTimeMethod
Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)Up to 6 months
Urine concentrations for LNP lipidsUp to 6 months
Antidrug antibodies to ABO-101 and anti-Cas protein antibodiesUp to 6 months
Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6Up to 6 months
Absolute change in UOx corrected for body surface areaUp to 6 months
Percent change in plasma glycolate from Baseline to Month 6Up to 6 months
Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24Up to 24 months

Trial Locations

Locations (2)

Mayo Clinic

🇺🇸

Rochester, Minnesota, United States

Nucleus Network

🇺🇸

St. Paul, Minnesota, United States

Mayo Clinic
🇺🇸Rochester, Minnesota, United States
Clinical Trial Site
Contact
800-664-4542

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