ABO-101 for Primary Hyperoxaluria

(redePHine Trial)

Overseen By Daniel Ory, MD

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Arbor Biotechnologies

Must not be taking: RNAi, siRNA therapies

Disqualifiers: PH2, PH3, Transplant, Dialysis, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

23 Participants Needed

Trial Summary

What is the purpose of this trial?

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.

Will I have to stop taking my current medications?

The trial protocol does not specify whether you need to stop taking your current medications. However, if you are currently using or have used urinary oxalate lowering RNA interference (RNAi) or siRNA therapy in the past 24 months, you would not be eligible to participate.

Research Team

Winston Yan, MD, PhD

Principal Investigator

Arbor Biotechnologies

Eligibility Criteria

The redePHine study is for individuals with primary hyperoxaluria type 1 (PH1), a rare condition leading to kidney stones. Adults will first receive varying doses to find the best one, then children will be treated with that dose. Participants must meet certain health criteria not specified here.

Inclusion Criteria

24-hour UOx ≥0.7 mmol/24 hours/1.73 m²

eGFR ≥30 mL/min/1.73m²

Weight ≤90 kg

Exclusion Criteria

I have been diagnosed with primary hyperoxaluria type 2 or 3.

I have had a liver, kidney, or liver/kidney transplant.

I am currently receiving dialysis.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment Part A

Adult participants receive a single ascending dose to identify a recommended dose

4-6 weeks

Treatment Part B

Pediatric participants receive the recommended dose

4-6 weeks

Long-term Monitoring

Participants are monitored long-term to comply with local and national requirements

Long-term

Treatment Details

Interventions

• ABO-101

Trial Overview ABO-101 is being tested in this trial. It's given in two parts: adults get different amounts at first, and once the right dose is found, kids get it too. Afterward, everyone enters a long-term monitoring phase to track safety and effects over time.

Participant Groups

Group I: Experimental: Part B: Single Dose Expansion Experimental Treatment 1 Intervention

Group II: Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design Experimental Treatment 1 Intervention

Who Is Running the Clinical Trial?

Arbor Biotechnologies

Lead Sponsor

Trials

1

Recruited

20+