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

Launched by ARBOR BIOTECHNOLOGIES · Feb 17, 2025

Keywords

ClinConnect Summary

The redePHine study is a clinical trial that aims to test a new treatment called ABO-101 for people with Primary Hyperoxaluria Type 1 (PH1), a rare genetic condition that affects how the body processes oxalate, leading to kidney damage. This study will assess how safe the treatment is and how well it works in both adults and children. The trial has two main parts: the first part will involve giving adult participants different doses of the treatment to find the best amount to use, and then children will receive this recommended dose. After this initial phase, there will be a longer period for monitoring the participants to ensure their safety and to gather more information about the treatment.

To be eligible for the trial, participants need to be between 6 and 64 years old and must have confirmed PH1 through genetic testing. They also need to meet certain health criteria, like having enough kidney function. Participants will be closely monitored throughout the study, and those who are pregnant, breastfeeding, or have had recent kidney or liver transplants cannot take part. This research is important as it could lead to better treatments for PH1, helping people manage their condition more effectively.

Gender

ALL

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)