Primary Hyperoxaluria Type 1 (PH1)Phase 1/2NCT06839235
Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1 (redePHine)
A Phase 1/2 study testing ABO-101 in people with Primary Hyperoxaluria Type 1 (PH1), run by Arbor Biotechnologies at 7 sites in 5 countries.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 6 to 64 years
- Conditions the study is for
- Primary Hyperoxaluria Type 1 (PH1)
- What is being tested?
- ABO-101
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 23 participants
- Where
- 7 sitesin 5 countries
- Study dates
- 16 Jun 2025to Feb 2043
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 64 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
Who cannot join
- 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)
Trial locations and contacts
7 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
7 sites in 5 countries
United States2 sites, 1 recruiting
- Recruiting
Mayo Clinic
Rochester, Minnesota, 55905
Clinical Trial Site, 800-664-4542
- Withdrawn
Nucleus Network
Saint Paul, Minnesota, 55114
United Kingdom2 sites, 2 recruiting
- Recruiting
Queen Elizabeth Hospital Birmingham
Birmingham
0121 371 3170, ClinicalResearchFacilityEnquiries@uhb.nhs.uk
- Recruiting
Royal Free Hospital
London
Royal Free Clinical Research, 020 7794 0500 ext. 32038, rf.crf@nhs.net
France1 site, 1 recruiting
- Not yet recruiting
Hospices Civils de Lyon- Hôpital Femmes Mères Enfants
Lyon
04 27 85 61 28
Germany1 site, 1 recruiting
- Not yet recruiting
Kindernierenzentrum Bonn
Bonn
0228 6883860, info@knz-bonn.de
Tunisia1 site, 1 recruiting
- Recruiting
Heidi Chaker
Sfax
Kammoun Khawla, MD, +216 74 244 511
About this study
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)
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.
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Being tested
- ABO-101
- Keywords
- PH1, Primary hyperoxaluria, AGXT, CRISPR, Gene Editing, Pharmacokinetics, Pharmacodynamics, ABO-101, redePHine
- Medical subject headings
- Primary hyperoxaluria type 1, Hyperoxaluria, Primary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ABO-101Drug
Intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design | Experimental | Not described Receives: Drug: ABO-101 |
| Experimental: Part B: Single Dose Expansion | Experimental | Not described Receives: Drug: ABO-101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101-related TEAEs and serious adverse events (SAEs)
Measured: Up to 6 months
Secondary outcomes (7)
Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6
Measured: Up to 6 months
Absolute change in UOx corrected for body surface area
Measured: Up to 6 months
Percent change in plasma glycolate from Baseline to Month 6
Measured: Up to 6 months
Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24
Measured: Up to 24 months
Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)
Measured: Up to 6 months
Urine concentrations for LNP lipids
Measured: Up to 6 months
Antidrug antibodies to ABO-101 and anti-Cas protein antibodies
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Feb 2025
- Study start16 Jun 2025
- Primary completionMar 2029 (estimated)
- Study completionFeb 2043 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Primary Hyperoxaluria Type 1 (PH1)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Single ascending dose escalation/adaptive design, followed by single dose expansion
- Masking
- None (open label)
- Enrolment
- 23 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Arbor Biotechnologies
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Daniel Ory, MD, Study director, Arbor Biotechnologies
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06839235
- Sponsor's protocol ID
- ABO-101-101
- Status last verified
- Oct 2026
- First submitted
- 17 Feb 2025
- Last updated
- 19 Oct 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 19 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT06839235
- Acronym / sponsor ID
- ABO-101-101
- Conditions
- Primary Hyperoxaluria Type 1 (PH1)
- Keywords
- PH1, Primary hyperoxaluria, AGXT, CRISPR, Gene Editing, Pharmacokinetics, Pharmacodynamics, ABO-101
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 23 participants (estimated)
- Lead sponsor
- Arbor BiotechnologiesIndustry (pharma, biotech, devices)
- Study start
- 16 Jun 2025 (actual)
- Primary completion
- Mar 2029 (estimated)
- Study completion
- Feb 2043 (estimated)
- First posted
- 21 Feb 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 2
- Model description
- Single ascending dose escalation/adaptive design, followed by single dose expansion
| Arm | Type | Receives |
|---|---|---|
| Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design | Experimental | Drug: ABO-101 |
| Experimental: Part B: Single Dose Expansion | Experimental | Drug: ABO-101 |
Who may qualify
- Ages
- 6 to 64 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (7 listed)
- 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:
- 24-hour UOx ≥0.7 mmol/24 hours/1.73 m²
- eGFR ≥30 mL/min/1.73m²
- Weight ≤90 kg
Key exclusion criteria (5 listed)
- 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)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ABO-101Intravenous (IV) infusion | Drug | Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design, Experimental: Part B: Single Dose Expansion |
Primary outcome
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
7 secondary outcomes
Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6
Time frame: Up to 6 months
Absolute change in UOx corrected for body surface area
Time frame: Up to 6 months
Percent change in plasma glycolate from Baseline to Month 6
Time frame: Up to 6 months
Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24
Time frame: Up to 24 months
Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)
Time frame: Up to 6 months
Urine concentrations for LNP lipids
Time frame: Up to 6 months
Antidrug antibodies to ABO-101 and anti-Cas protein antibodies
Time frame: Up to 6 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Oct 2026.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Daniel Ory, MD study director
Arbor Biotechnologies
7 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing ABO-101 in people with Primary Hyperoxaluria Type 1 (PH1), run by Arbor Biotechnologies at 7 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 19 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States, United Kingdom, France, Germany, Tunisia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Daniel Ory, MD (617-500-8941, arbortrials@arbor.bio), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the 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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Oct 2026.