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

NCT06839235 | Recruiting Phase 1 DMC FDA Drug

• 1 other identifier: ABO-101-101
• Study Type: interventional
• Target: 23
• Locations: 5 countries
• Sites: 7

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.

Conditions

Primary Hyperoxaluria Type 1 (PH1)

Keywords

PH1; Primary hyperoxaluria; AGXT; CRISPR; Gene Editing; Pharmacokinetics; Pharmacodynamics; ABO-101; redePHine

Outcome Measures

Primary Outcomes (1)

• Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs)

  Up to 6 months

Secondary Outcomes (7)

• 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

Study Arms (2)

Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design

EXPERIMENTAL

Drug: ABO-101

Experimental: Part B: Single Dose Expansion

EXPERIMENTAL

Drug: ABO-101

Interventions

ABO-101 DRUG

Intravenous (IV) infusion

Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design; Experimental: Part B: Single Dose Expansion

Eligibility Criteria

• Age: 6 Years - 64 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• 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
• hour UOx ≥0.7 mmol/24 hours/1.73 m²
• eGFR ≥30 mL/min/1.73m²
• Weight ≤90 kg

You may not qualify if:

• 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)

Sponsors & Collaborators

• Arbor Biotechnologies: lead

Study Sites (7)

Mayo Clinic

Rochester, Minnesota, 55905, United States

RECRUITING

Nucleus Network

Saint Paul, Minnesota, 55114, United States

WITHDRAWN

Hospices Civils de Lyon- Hôpital Femmes Mères Enfants

Lyon, France

NOT YET RECRUITING

Kindernierenzentrum Bonn

Bonn, Germany

NOT YET RECRUITING

Heidi Chaker

Sfax, Tunisia

RECRUITING

Queen Elizabeth Hospital Birmingham

Birmingham, United Kingdom

RECRUITING

Royal Free Hospital

London, United Kingdom

RECRUITING

MeSH Terms

Conditions

Primary hyperoxaluria type 1; Hyperoxaluria, Primary

Condition Hierarchy (Ancestors)

Hyperoxaluria; Kidney Diseases; Urologic Diseases; Female Urogenital Diseases; Female Urogenital Diseases and Pregnancy Complications; Urogenital Diseases; Male Urogenital Diseases; Carbohydrate Metabolism, Inborn Errors; Metabolism, Inborn Errors; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Winston Yan, MD, PhD

  Arbor Biotechnologies

  STUDY DIRECTOR

Central Study Contacts

Daniel Ory, MD

CONTACT

617-500-8941; arbortrials@arbor.bio

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single ascending dose escalation/adaptive design, followed by single dose expansion

Study Record Dates

• First Submitted: February 17, 2025
• First Posted: February 21, 2025
• Study Start: June 16, 2025
• Primary Completion (Estimated): March 1, 2029
• Study Completion (Estimated): February 1, 2043
• Last Updated: February 12, 2026

Record last verified: 2026-02

Locations

GB (2); FR (1); US (2); TU (1); DE (1)