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NCT Number: NCT06839235

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

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.

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Key information

Conditions

Primary Hyperoxaluria Type 1 (PH1) Carbohydrate Metabolism, Inborn Errors Congenital, Hereditary, and Neonatal Diseases and Abnormalities Female Urogenital Diseases Female Urogenital Diseases and Pregnancy Complications Genetic Diseases, Inborn Hyperoxaluria Hyperoxaluria, Primary Kidney Diseases Male Urogenital Diseases Metabolic Diseases Metabolism, Inborn Errors Nutritional and Metabolic Diseases Primary hyperoxaluria type 1 Urogenital Diseases Urologic Diseases

Age range

6 year–64 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

Hospices Civils de Lyon- Hôpital Femmes Mères Enfants, Lyon, France

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

ABO-101

Drug

Intravenous (IV) infusion

Primary outcomes

  1. 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

Secondary outcomes

  1. Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6

    Time frame: Up to 6 months

  2. Absolute change in UOx corrected for body surface area

    Time frame: Up to 6 months

  3. Percent change in plasma glycolate from Baseline to Month 6

    Time frame: Up to 6 months

  4. Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24

    Time frame: Up to 24 months

  5. Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)

    Time frame: Up to 6 months

  6. Urine concentrations for LNP lipids

    Time frame: Up to 6 months

  7. Antidrug antibodies to ABO-101 and anti-Cas protein antibodies

    Time frame: Up to 6 months

Study contacts

Contact information is provided by the study sponsor or research team.

Daniel Ory, MD

CONTACT

[email protected]

617-500-8941

Sponsors and collaborators

Lead sponsor

Arbor Biotechnologies

Industry

Registry information

Official study title

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)

Important dates

Study start
2025
Primary completion
2029
Study completion
2043
First posted
Feb 21, 2025
Registry last updated
Feb 12, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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