Mayo Clinic
RecruitingRochester, Minnesota, 55905, United States
Site contacts (1)
Clinical Trial SiteContactPhone: 800-664-4542
CLINICAL STUDY · NCT06839235
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
RecruitingPhase 1 / Phase 2Interventional
Sponsored by Arbor Biotechnologies
- Condition
- Primary Hyperoxaluria Type 1 (PH1)
- Study locations
- 7 reported sites
- Study updated
- 19 Oct 2026
- Source
- ClinicalTrials.gov
Overall study status is shown above. Individual hospitals may have a different recruitment status.
THE STUDY
About this study
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.
Full official title
A Phase 1/2 Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of ABO-101 in Participants With Primary Hyperoxaluria Type 1 (PH1)
PARTICIPATION
Who may participate
- Minimum age
- 6 Years
- Maximum age
- 64 Years
- Sex eligibility
- All sexes
- Healthy volunteers
- Not accepted
Only the study team can confirm eligibility. The registry criteria below are not a medical recommendation.
Full inclusion and exclusion 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 \<18 years
* 24-hour UOx ≥0.7 mmol/24 hours/1.73 m²
* eGFR ≥30 mL/min/1.73m²
* Weight ≤90 kg
Key Exclusion Criteria for Parts A and B
* 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)
* 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
Key Exclusion Criteria for Parts A and B
* 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)
NEAR YOU
Study locations worldwide
7 sitesCheck the individual site’s status and contact the listed study team for current availability.
7 sites shown
Nucleus Network
WithdrawnSaint Paul, Minnesota, 55114, United States
Study team contact · shared across sites
Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
Not yet recruitingLyon, France
Site contacts (1)
Name not reportedContactPhone: 04 27 85 61 28
Kindernierenzentrum Bonn
Not yet recruitingBonn, Germany
Site contacts (1)
Heidi Chaker
RecruitingSfax, Tunisia
Site contacts (1)
Kammoun Khawla, MDContactPhone: +216 74 244 511
Queen Elizabeth Hospital Birmingham
RecruitingBirmingham, United Kingdom
Site contacts (1)
Royal Free Hospital
RecruitingLondon, United Kingdom
Site contacts (1)
No centres match this search. Try another hospital, city, state or country.
WHAT IS BEING STUDIED
Treatments and study groups
Interventions
ABO-101
Intravenous (IV) infusion
Study groups: Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design, Experimental: Part B: Single Dose Expansion
Study groups
Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design
Not reported.
Interventions: Drug: ABO-101
Experimental: Part B: Single Dose Expansion
Not reported.
Interventions: Drug: ABO-101
RESEARCH DETAILS
Study design
- Study type
- Interventional
- Phase
- Phase 1 / Phase 2
- Primary purpose
- Treatment
- Allocation
- Non randomized
- Study model
- Sequential
- Masking
- None (open label)
- Enrolment
- 23 participants · Estimated
Study model details
Single ascending dose escalation/adaptive design, followed by single dose expansion
TIMELINE & MEASURES
Dates and outcomes
- Study started
- 16 Jun 2025 · Actual
- Primary completion
- Mar 2029 · Estimated
- Study completion
- Feb 2043 · Estimated
- First registered
- 21 Feb 2025 · Actual
- Last registry update
- 19 Oct 2026 · Actual
- Status last verified
- Oct 2026
Primary outcomes (1)
Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs)
Not reported.
Time frame: Up to 6 months
Secondary outcomes (7)
Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6
Not reported.
Time frame: Up to 6 months
Absolute change in UOx corrected for body surface area
Not reported.
Time frame: Up to 6 months
Percent change in plasma glycolate from Baseline to Month 6
Not reported.
Time frame: Up to 6 months
Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24
Not reported.
Time frame: Up to 24 months
Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)
Not reported.
Time frame: Up to 6 months
Urine concentrations for LNP lipids
Not reported.
Time frame: Up to 6 months
Antidrug antibodies to ABO-101 and anti-Cas protein antibodies
Not reported.
Time frame: Up to 6 months
STUDY TEAM
Sponsor and contacts
- Lead sponsor
- Arbor Biotechnologies
- Sponsor type
- Industry
- Collaborators
- Not reported
- Organisation study ID
- ABO-101-101
Central study contacts
Study officials
Daniel Ory, MDStudy director · Arbor Biotechnologies
Contacts are provided by the registry for study enquiries. Do not use these details for marketing or promotions.
FURTHER READING
Results, documents and references
No results are posted in this registry record
This does not mean the study has no findings. Results may not yet have been submitted or published here.
No study documents are listed in this record.