Evaluating ABO-101 for treating Primary Hyperoxaluria Type 1

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)

This study is testing a new treatment called ABO-101 to see if it is safe and effective for people with primary hyperoxaluria type 1.

Quick facts

• Phase: PHASE1; PHASE2
• Study type: Interventional
• Enrollment: 23 (estimated)
• Ages: 6 Years to 64 Years
• Sex: All
• Sponsor: Arbor Biotechnologies (industry)
• Locations: 7 sites (Rochester, Minnesota and 6 other locations)
• Trial ID: NCT06839235 on ClinicalTrials.gov

What this trial studies

The redePHine study aims to assess the safety, tolerability, pharmacokinetics, and pharmacodynamics of ABO-101 in individuals with primary hyperoxaluria type 1 (PH1). It consists of two study periods, with the first period including two parts: adult participants will receive a single ascending dose to determine the recommended dose, while pediatric participants will receive this recommended dose. Following this, a long-term monitoring program will be initiated to ensure compliance with local and national regulations.

Who should consider this trial

Good fit: Ideal candidates include individuals aged 6 to 64 years with genetically confirmed primary hyperoxaluria type 1 and specific urinary oxalate levels.

Not a fit: Patients with primary hyperoxaluria type 2 or type 3, those with a history of organ transplants, or individuals currently on dialysis may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could significantly improve the management of primary hyperoxaluria type 1, potentially reducing the risk of kidney damage and related complications.

How similar studies have performed: While gene editing approaches are emerging, this specific application of ABO-101 in PH1 is novel and has not been extensively tested in prior studies.

Eligibility criteria

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)

Where this trial is running

Rochester, Minnesota and 6 other locations

• Mayo Clinic — Rochester, Minnesota, United States (RECRUITING)
• Nucleus Network — Saint Paul, Minnesota, 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)

Study contacts

• Study coordinator: Daniel Ory, MD

Conditions: Primary Hyperoxaluria Type 1, PH1, Primary hyperoxaluria, AGXT, CRISPR, Gene Editing, Pharmacokinetics, Pharmacodynamics