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

NCT ID: NCT06839235

Last Updated: 2025-12-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE1/PHASE2

Total Enrollment

23 participants

Study Classification

INTERVENTIONAL

Study Start Date

2025-06-16

Study Completion Date

2043-02-28

Brief Summary

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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.

Detailed Description

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Conditions

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Primary Hyperoxaluria Type 1 (PH1)

Keywords

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PH1 Primary hyperoxaluria AGXT CRISPR Gene Editing Pharmacokinetics Pharmacodynamics ABO-101 redePHine

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SEQUENTIAL

Single ascending dose escalation/adaptive design, followed by single dose expansion
Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design

Group Type EXPERIMENTAL

ABO-101

Intervention Type DRUG

Intravenous (IV) infusion

Experimental: Part B: Single Dose Expansion

Group Type EXPERIMENTAL

ABO-101

Intervention Type DRUG

Intravenous (IV) infusion

Interventions

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ABO-101

Intravenous (IV) infusion

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* 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

Exclusion Criteria

* 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)
Minimum Eligible Age

6 Years

Maximum Eligible Age

64 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Arbor Biotechnologies

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Winston Yan, MD, PhD

Role: STUDY_DIRECTOR

Arbor Biotechnologies

Locations

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Mayo Clinic

Rochester, Minnesota, United States

Site Status RECRUITING

Nucleus Network

Saint Paul, Minnesota, United States

Site Status WITHDRAWN

Hospices Civils de Lyon- Hôpital Femmes Mères Enfants

Lyon, , France

Site Status NOT_YET_RECRUITING

Kindernierenzentrum Bonn

Bonn, , Germany

Site Status NOT_YET_RECRUITING

Queen Elizabeth Hospital Birmingham

Birmingham, , United Kingdom

Site Status RECRUITING

Royal Free Hospital

London, , United Kingdom

Site Status RECRUITING

Countries

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United States France Germany United Kingdom

Central Contacts

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Daniel Ory, MD

Role: CONTACT

Phone: 617-500-8941

Email: [email protected]

Facility Contacts

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Clinical Trial Site

Role: primary

Role: primary

Role: primary

Role: primary

Royal Free Clinical Research

Role: primary

Other Identifiers

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ABO-101-101

Identifier Type: -

Identifier Source: org_study_id