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

Conditions

• Primary Hyperoxaluria Type 1 (PH1)

Eligibility Criteria

Sex

ALL

Age

6 Years - 64 Years

Healthy Volunteers

Not accepted

Interventions

• ABO-101 — DRUG
  Intravenous (IV) infusion

Study Details

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.

Key Dates

Start date

Jun 16, 2025

Status verified

Feb 2026

Primary completion

Mar 31, 2029

Completion

Feb 28, 2043

Study Design

Enrollment

23 participants (estimated)

Allocation

NON_RANDOMIZED

Intervention model

SEQUENTIAL

Primary purpose

TREATMENT

Arms

• Experimental: Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design
• Experimental: Experimental: Part B: Single Dose Expansion

Primary Outcome Measure

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 ]

Central Contacts

• Daniel Ory, MD
  617-500-8941
  [email protected]

Locations (2)

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