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

Phase 1

Recruiting

• Conditions: Primary Hyperoxaluria Type 1 (PH1)
• Interventions: Drug: ABO-101

• 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

Study Timeline

• Study First Submitted: 2025-02-17
• Study First Submitted QC: 2025-02-17
• Study First Posted: 2025-02-21
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-21
• Last Update Posted: 2025-05-25
• Study Start: 2025-06
• Primary Completion: 2029-03
• Study Completion: 2043-02

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

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

Trial Locations

Locations (2)

Mayo Clinic; 🇺🇸 Rochester, Minnesota, United States

Nucleus Network; 🇺🇸 St. Paul, Minnesota, United States