MRNA-002, GRNA-001 for Primary hyperoxaluria type 1

Inclusion criteria

• {"criterion_text":"- 1. Confirmed diagnosis of PH1"}
• {"criterion_text":"- 6. Able to follow directions and provide 24-hour urine collections"}
• {"criterion_text":"- 7. If taking pyridoxine (vitamin B6) for the treatment of PH1, must have been on a stable regimen for at least 90 days prior to ABO-101 administration, and be willing to remain on this stable regimen during Study Period 1"}
• {"criterion_text":"- 8. Must meet the following laboratory: a. Within laboratory reference range or deemed clinically non-significant by the Investigator: AST, ALT, and total bilirubin b. eGFR ≥30 mL/min/1.73m2 based on CKD-EPI 2021 equation for participants ≥18 years of age at Screening and the Schwartz equation for participants <18 years of age at Screening c. Platelet count >100,000/mm3 d. Within laboratory reference range or deemed clinically non-significant by the Investigator: PT, aPTT, INR, D-dimer, and fibrinogen"}
• {"criterion_text":"- 10. Participant must not have received any experimental agent for the treatment of PH1 for at least (CCI) half-lives"}
• {"criterion_text":"- 11. Participant must agree to not participate in another interventional trial during the Screening Period and for at least 6 months following ABO-101 administration"}
• {"criterion_text":"- 4. History of renal stone events, clinically related symptoms, or history of nephrocalcinosis."}
• {"criterion_text":"- 9. All available standard of care options (including approved siRNA treatment) were considered according to local medical practice and guidelines"}
• {"criterion_text":"- 12. Weight ≤90 kg"}
• {"criterion_text":"- 13. For female participants: a. If of childbearing potential, must agree to use at least 1 highly effective method of contraception from the time of signing the ICF through 12 months after dosing with ABO-101; b. Be postmenopausal c. Be surgically sterile at least 1 month prior to Screening"}
• {"criterion_text":"- 14. For male participants: Must agree to use condoms with spermicide or abstain from sexual intercourse, and their female partners of childbearing potential must use a highly effective method of contraception from the time of signing the ICF through 4 months after dosing with ABO-101. Male participants must agree to not donate sperm through 4 months after trial drug administration"}
• {"criterion_text":"- 15. Capable of providing signed informed consent. In case of participants who are below the legal age, informed consent must be obtained from the participant’s parent/LAR and the participant must provide assent per local and national requirements"}
• {"criterion_text":"- 16. Must be willing to comply with the requirements of the trial"}
• {"criterion_text":"- 2. Age at time of signing the ICF/assent form: a. Cohorts 1-3: ≥18 years to ≤64 years b. Cohort 4: ≥6 years to <18 years"}
• {"criterion_text":"- 3. Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase gene (AGXT)"}
• {"criterion_text":"- 5. Mean of (CCI) valid 24-hour UOx ≥0.7 mmol (63 mg)/24 hours/1.73 m2"}

Exclusion criteria

• {"criterion_text":"- 1. Confirmed diagnosis of PH2 or PH3"}
• {"criterion_text":"- 11. Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)"}
• {"criterion_text":"- 12. History of alcohol or drug abuse within 3 years prior to Screening"}
• {"criterion_text":"- 13. History of active hepatitis B, C or human immunodeficiency virus (HIV) infection; or positive hepatitis B surface antigen. Participants who are hepatitis C virus (HCV) antibody positive must have negative HCV RNA"}
• {"criterion_text":"- 16. Any condition or laboratory abnormality that in the opinion of the Investigator could pose undue risk, confound the ability to interpret trial results, or preclude compliance with the trial"}
• {"criterion_text":"- 17. Anticipated survival <2 years in the opinion of the Investigator"}
• {"criterion_text":"- 18. Unwilling to comply with trial procedures including Long-Term Safety Follow-Up"}
• {"criterion_text":"- 2. History of a liver, kidney, or combined liver/kidney transplant"}
• {"criterion_text":"- 3. Currently on dialysis or anticipated requirement for dialysis within the initial (CCI) months of the trial"}
• {"criterion_text":"- 9. History of known gram-negative urinary tract infection or presumptively treated urinary tract infection (i.e., no culture) or gram-negative systemic infection within 90 days prior to ABO-101 administration"}
• {"criterion_text":"- 4. Participant has previously used (within past (CCI) months) or is currently on an approved or investigational urinary oxalate lowering (CCI) or (CCI) therapy"}
• {"criterion_text":"- 5. Medical history includes clinical evidence of extrarenal systemic oxalosis, as determined by the Investigator"}
• {"criterion_text":"- 6. Known hypersensitivity to any LNP component or history of Grade 3 or higher AE following administration of any LNP requiring treatment or discontinuation of treatment, or any LNP treatment-related AE that, in the opinion of the Investigator, may pose undue risk to the participant"}
• {"criterion_text":"- 7. History of liver cirrhosis"}
• {"criterion_text":"- 8. Known or suspected systemic bacterial, viral, or fungal infection, or requirement of systemic anti-infectives either ongoing or within 14 days prior to trial drug administration, or where inclusion in the clinical trial would jeopardize the participant’s health and wellbeing, as determined by the Investigator."}
• {"criterion_text":"- 10. History of active malignancy within 5 years prior to Screening except for adequately treated basal or squamous cell carcinoma of the skin, adequately treated cervical carcinoma in situ or adequately treated organ confined prostate cancer"}
• {"criterion_text":"- 14. Use of antiplatelet (e.g., aspirin, clopidogrel) or antithrombotic therapy (e.g., warfarin, dabigatran, apixaban) within 14 days prior to ABO-101 administration."}
• {"criterion_text":"- 15. History of thrombotic disorders or medical history suggestive of predisposing factors for thromboembolic events (e.g., recent surgery or trauma, or known genetic disorder that increase the propensity for venous thromboembolism)."}

Primary endpoints

• {"endpoint_text":"- Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101- related TEAEs and serious adverse events (SAEs)","definition_or_measurement_approach":""}

Secondary endpoints

• {"endpoint_text":"- Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month (CCI)","definition_or_measurement_approach":"Percent change from Baseline to Month (CCI)"}
• {"endpoint_text":"- Absolute change in UOx corrected for body surface area","definition_or_measurement_approach":"Absolute change from Baseline, corrected for body surface area"}
• {"endpoint_text":"- Percent change in plasma glycolate from Baseline to Month (CCI)","definition_or_measurement_approach":"Percent change from Baseline to Month (CCI)"}
• {"endpoint_text":"- Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month (CCI) and Month (CCI)","definition_or_measurement_approach":"Change in eGFR from Baseline to specified months (CCI)"}
• {"endpoint_text":"- Plasma concentrations for (CCI), and (CCI)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Urine concentrations for (CCI) and (CCI)","definition_or_measurement_approach":""}
• {"endpoint_text":"- Antidrug antibodies to ABO-101 and anti-Cas12i2 protein antibodies","definition_or_measurement_approach":"Measurement of antidrug and anti-Cas12i2 antibodies"}

Methods

• Clinical Trial Landing Page (digital) — Clinical Trial Landing Page Copy and Landing Page Screenshots listed among recruitment materials
• Patient Navigator contacts — Patient Navigator Script and 'Patients Navigator' role in third parties indicate use of patient navigators to engage and assist potential participants
• Patient Brochure (printed/PDF) — Patient Brochure documents available for participant information
• Recruitment arrangements documents tailored per country (documents present associated with France, Germany and Netherlands)

France

Earliest CTIS Part Ii Submission Date

26-02-2025

Latest Decision Or Authorization Date

26-01-2026

Processing Time Days

334

Number Of Sites

1

Number Of Participants

2

Germany

Earliest CTIS Part Ii Submission Date

10-04-2025

Latest Decision Or Authorization Date

23-01-2026

Processing Time Days

288

Number Of Sites

1

Number Of Participants

2

Netherlands

Earliest CTIS Part Ii Submission Date

11-04-2025

Latest Decision Or Authorization Date

20-01-2026

Processing Time Days

284

Number Of Sites

1

Number Of Participants

2

Primary sponsor

Full Name

Arbor Biotechnologies Inc.

Organisation Type

Pharmaceutical company

Country Of Registered Address

United States

Contract research organisations

Name

Worldwide Clinical Trials d.o.o.

Responsibilities

Vendor management and multiple operational responsibilities (codes indicate broad vendor/operational roles)

Name

QPS LLC

Responsibilities

PK: anti-cas12i2 and ADA (anit-LNP)

Third parties

• {"country":"France","full_name":"Quipment","duties_or_roles":"Equipment","organisation_type":"Non-Pharmaceutical company"}
• {"country":"United States","full_name":"Labcorp Central Laboratory Services LP","duties_or_roles":"Analysis of the urinary oxalate and plasma glycolate samples","organisation_type":"Pharmaceutical company"}
• {"country":"France","full_name":"Banook Central Imaging","duties_or_roles":"Central Imaging","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United States","full_name":"Cadoret Global Inc.","duties_or_roles":"Privacy and Data Protection Consulting Services","organisation_type":"Industry"}
• {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"PK: PEG Lipid (ALC-0307) and Ionizable Lipid (ALC-0307)","organisation_type":"Pharmaceutical company"}
• {"country":"United Kingdom","full_name":"Labcorp Early Development Laboratories Limited","duties_or_roles":"PK: mRNA, gRNA","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patients Logistics","organisation_type":"Hospital/Clinic/Other health care facility"}
• {"country":"United Kingdom","full_name":"The Doctors Laboratory Limited","duties_or_roles":"UoX analysis","organisation_type":"Laboratory/Research/Testing facility"}
• {"country":"United Kingdom","full_name":"Biotec Services International Limited","duties_or_roles":"IP Management","organisation_type":"Pharmaceutical company"}
• {"country":"Ireland","full_name":"Millmount Healthcare Limited","duties_or_roles":"IP Management","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"QPS LLC","duties_or_roles":"PK: anti-cas12i2 and ADA (anit-LNP)","organisation_type":"Pharmaceutical company"}
• {"country":"Switzerland","full_name":"Labcorp Central Laboratory Services SARL","duties_or_roles":"","organisation_type":"Pharmaceutical company"}
• {"country":"United States","full_name":"Cadoret Global Inc.","duties_or_roles":"Privacy and Data Protection Consulting Services","organisation_type":"Industry"}
• {"country":"Croatia","full_name":"Worldwide Clinical Trials d.o.o.","duties_or_roles":"Vendor management and multiple operational responsibilities (codes indicate broad vendor/operational roles)","organisation_type":"Pharmaceutical company"}
• {"country":"Netherlands","full_name":"Impatients Holland B.V.","duties_or_roles":"Patients Navigator","organisation_type":"Pharmaceutical company"}