ABO-101 for Primary Hyperoxaluria

(redePHine Trial)

Recruiting at 3 trial locations

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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a new treatment called ABO-101, a gene therapy for individuals with primary hyperoxaluria type 1 (PH1), a rare condition that causes excessive oxalate production and kidney problems. Initially, the study will evaluate different doses in adults to determine the optimal amount, then apply that dose in children. Participants will then be monitored over an extended period to assess the treatment's effects. Individuals diagnosed with PH1 and experiencing kidney issues due to excess oxalate may be suitable candidates for this study. As a Phase 1 trial, the research aims to understand how the treatment works in people, offering participants the chance to be among the first to receive this new therapy.

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.

Is there any evidence suggesting that ABO-101 is likely to be safe for humans?

A previous study found that ABO-101 was well tolerated by participants, with no serious side effects or dose-related problems in the 28 days following treatment. This indicates that, so far, the treatment has been safe for recipients. As the trial is in its early stages, this is a promising sign for its safety in humans. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about ABO-101 for treating Primary Hyperoxaluria because it operates differently from current treatments, which typically involve dietary changes and medications to reduce oxalate levels. ABO-101 is a gene therapy that targets the root cause by delivering a functional copy of the gene missing or malfunctioning in patients with this condition. This innovative approach has the potential to address the disease at its genetic source, offering a more effective and long-lasting solution than existing options.

What evidence suggests that ABO-101 might be an effective treatment for primary hyperoxaluria?

Research has shown that ABO-101 has promising results in early studies. In tests with animals similar to humans, ABO-101 proved effective and safe without causing harmful effects. This gene-editing treatment aims to help people with primary hyperoxaluria type 1 (PH1), a condition where the body produces excessive oxalate, potentially leading to kidney issues. Early results suggest that ABO-101 may successfully alter genes linked to this condition, possibly reducing oxalate levels. While research in humans continues, early signs of safety and effectiveness are positive. ^{1 2 3 6 7}

Who Is on the Research Team?

Winston Yan, MD, PhD

Principal Investigator

Arbor Biotechnologies

Are You a Good Fit for This Trial?

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 for a Trial Participant

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

What Are the Treatments Tested in This Trial?

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.

How Is the Trial Designed?

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+

Citations

1. arbor.bio arbor.bio/arbor-biotechnologies-to-present-preclinical-data-for-abo-101-in-ph1-at-the-american-society-of-gene-and-cell-therapy-asgct-28th-annual-meeting/

Arbor Biotechnologies to Present Preclinical Data for ABO ... Data from non-human primate (NHP) studies confirmed the editing efficiency and tolerability of ABO-101 with no clinical signs or adverse events.

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06839235

NCT06839235 | Phase 1/2 Study of ABO-101 in Primary ... These data include demographics, such as age, sex/gender, race and ethnicity, and study-specific measures (for example, systolic blood pressure, prior ...

3. urologytimes.com urologytimes.com/view/trial-launches-of-abo-101-for-primary-hyperoxaluria-type-1

Trial launches of ABO-101 for primary hyperoxaluria type 1 Overall, the data demonstrated the proof-of-pharmacology and efficacy of ABO-101 in non-human primates. 4,5. The investigators found that ABO- ...

4. journals.lww.com journals.lww.com/10.1681/ASN.2025k5bbfe75

ABO-101, a Novel Gene Editing Therapy for Primary... We show that our gene editing therapeutic candidate, ABO-101, has demonstrated signals of efficacy and safety across multiple preclinical models, a critical ...

5. arbor.bio arbor.bio/arbor-biotechnologies-announces-first-patient-dosed-at-mayo-clinic-in-the-redephine-phase-1-2-study-of-abo-101-an-investigational-gene-editing-treatment-for-primary-hyperoxaluria-type-1/

Arbor Biotechnologies Announces First Patient Dosed at ... ABO-101 was well tolerated with no serious adverse events or dose-limiting toxicities reported in the 28-days following dosing.

6. mytomorrows.com mytomorrows.com/trials/redephine-en-us/

A Clinical Study of ABO-101 for Patients with Primary ... The purpose of this study is to evaluate the safety, tolerability, and preliminary efficacy of an investigational gene editing drug in people with Primary ...

7. journals.lww.com journals.lww.com/jasn/fulltext/2024/10001/development_of_abo_101,_a_novel_gene_editing.88.aspx

Development of ABO-101, a Novel Gene Editing Therapy ... Here we present pre-clinical in vivo evidence supporting ABO-101 as a novel CRISPR-Cas-mediated gene editing therapy for the treatment of PH1.