Recruiting
Phase 1
Phase 2

ABO-101

Sponsor:

Arbor Biotechnologies

Code:

NCT06839235

Conditions

Primary Hyperoxaluria Type 1 (PH1)

Eligibility Criteria

Sex: All

Age: 6 - 64

Healthy Volunteers: Not accepted

Interventions

ABO-101

Study Details

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)

Study ID

NCT06839235

Start date

Jun 16, 2025

Status verified date

Feb, 2026

Completion date

Feb, 2043

Anticipated

Primary completion date

Mar, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 6 - 64

Healthy Volunteers: Not accepted

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 Design

Enrollment

23 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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

experimental: Experimental: Part B: Single Dose Expansion

Interventions

ABO-101

Intravenous (IV) infusion

Primary outcome measure

  • Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs) [ Time Frame: Up to 6 months ]

Central Contacts and Locations

Central contacts

Daniel Ory, MD

617-500-8941arbortrials@arbor.bio

Locations

Mayo Clinic

Recruiting

Rochester, Minnesota, United States, 55905

Contacts

Clinical Trial Site

800-664-4542

More Information

Sponsor

Arbor Biotechnologies

Last update posted

Feb 12, 2026

Last verified

Feb, 2026

Keywords

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

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Arbor Biotechnologies on 2026-02-12.