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

A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants With Primary Hyperoxaluria Type 1 (PH1)

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 Overview

• Status: Recruiting
• Conditions: Primary Hyperoxaluria Type 1 (PH1)
• Intervention / Treatment: Drug: ABO-101

• Study Type: Interventional
• Enrollment (Estimated): 23
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Daniel Ory, MD; Phone Number: 617-500-8941; Email: arbortrials@arbor.bio

Study Locations

• France
  • Lyon, France
    • Not yet recruiting
    • Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
    • Contact: Phone Number: 04 27 85 61 28
• Germany
  • Bonn, Germany
    • Not yet recruiting
    • Kindernierenzentrum Bonn
    • Contact: Phone Number: 0228 6883860; Email: info@knz-bonn.de
• Tunisia
  • Sfax, Tunisia
    • Recruiting
    • Heidi Chaker
    • Contact: Mohamed Ben Hmida, MD; Phone Number: +216 74 244 511
• United Kingdom
  • Birmingham, United Kingdom
    • Recruiting
    • Queen Elizabeth Hospital Birmingham
    • Contact: Phone Number: 0121 371 3170; Email: ClinicalResearchFacilityEnquiries@uhb.nhs.uk
  • London, United Kingdom
    • Recruiting
    • Royal Free Hospital
    • Contact: Royal Free Clinical Research; Phone Number: 32038 020 7794 0500; Email: rf.crf@nhs.net
• United States
  • Minnesota
    • Rochester, Minnesota, United States, 55905
      • Recruiting
      • Mayo Clinic
      • Contact: Clinical Trial Site; Phone Number: 800-664-4542
    • Saint Paul, Minnesota, United States, 55114
      • Withdrawn
      • Nucleus Network

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

Key Inclusion Criteria for Parts A and B

• Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase (AGXT) gene (valid historical laboratory data will be reviewed and approved by the Sponsor)

• Age at time of signing the informed consent/assent form:

  • Part A: ≥18 years to ≤64 years
  • Part B: ≥6 years to <18 years
• 24-hour UOx ≥0.7 mmol/24 hours/1.73 m²
• eGFR ≥30 mL/min/1.73m²
• Weight ≤90 kg

Key Exclusion Criteria for Parts A and B

• Confirmed diagnosis of primary hyperoxaluria type 2 or type 3
• History of a liver, kidney or combined liver/kidney transplant
• Currently on dialysis
• Participant has previously used (within past 24 months) or is currently receiving an approved or investigational urinary oxalate lowering RNA interference (RNAi) or siRNA therapy
• Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Sequential Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design	Drug: ABO-101; Intravenous (IV) infusion
Experimental: Experimental: Part B: Single Dose Expansion	Drug: ABO-101; Intravenous (IV) infusion

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
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

Outcome Measure	Time Frame
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
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

Collaborators and Investigators

• Sponsor: Arbor Biotechnologies
• Investigators: Study Director: Winston Yan, MD, PhD, Arbor Biotechnologies

Study record dates

Study Major Dates

• Study Start (Actual): June 16, 2025
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): February 1, 2043

Study Registration Dates

• First Submitted: February 17, 2025
• First Submitted That Met QC Criteria: February 17, 2025
• First Posted (Actual): February 21, 2025

Study Record Updates

• Last Update Posted (Actual): February 12, 2026
• Last Update Submitted That Met QC Criteria: February 11, 2026
• Last Verified: February 1, 2026

More Information

Terms related to this study

• Keywords: Gene Editing; Pharmacokinetics; Pharmacodynamics; PH1; CRISPR; Primary hyperoxaluria; AGXT; ABO-101; redePHine
• Additional Relevant MeSH Terms: Urogenital Diseases; Male Urogenital Diseases; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Metabolic Diseases; Carbohydrate Metabolism, Inborn Errors; Hyperoxaluria; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Nutritional and Metabolic Diseases; Hyperoxaluria, Primary; Primary hyperoxaluria type 1
• Other Study ID Numbers: ABO-101-101

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No