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Recruiting NCT06839235

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

Phase I / Phase II Interventional Primary Hyperoxaluria Type 1 (PH1)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: ABO-101.
Who it may be relevant to
Registry conditions: Primary Hyperoxaluria Type 1 (PH1). Basic parameters: 6 years — 64 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, France, Germany, Tunisia, United Kingdom
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official 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)

Overview

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.

Interventions

  • Drug ABO-101
    Intravenous (IV) infusion

Primary outcome measures

  • 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 outcome measures (7)
  • Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6 [Time frame: Up to 6 months]
  • Absolute change in UOx corrected for body surface area [Time frame: Up to 6 months]
  • Percent change in plasma glycolate from Baseline to Month 6 [Time frame: Up to 6 months]
  • Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24 [Time frame: Up to 24 months]
  • Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA) [Time frame: Up to 6 months]
  • Urine concentrations for LNP lipids [Time frame: Up to 6 months]
  • Antidrug antibodies to ABO-101 and anti-Cas protein antibodies [Time frame: Up to 6 months]

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)

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Non-randomized
Model
Sequential
Masking
Open label
Primary purpose
Treatment

Study locations

United States · 2 centers
  • Mayo Clinic — Rochester
  • Nucleus Network — Saint Paul
United Kingdom · 2 centers
  • Queen Elizabeth Hospital Birmingham — Birmingham
  • Royal Free Hospital — London
France · 1 center
  • Hospices Civils de Lyon- Hôpital Femmes Mères Enfants — Lyon
Germany · 1 center
  • Kindernierenzentrum Bonn — Bonn
Tunisia · 1 center
  • Heidi Chaker — Sfax

Identifiers

NCT: NCT06839235 · ABO-101-101

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗