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Recruiting
NCT06839235
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
Conditions: Primary Hyperoxaluria Type 1 (PH1)
Sex: All
Ages: 6 Years – 64 Years
Healthy volunteers: No
Phase: PHASE1, PHASE2
Enrollment: 23
Sponsor: Arbor Biotechnologies
Location: Mayo Clinic Rochester Minnesota
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.
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 \
Source: ClinicalTrials.gov (NCT06839235). StuddyBuddy aggregates publicly available trial information.