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Phase 1/2 Study of ABO-101 in Primary Hyperoxaluria Type 1
EU CTIS ID: 2024-518144-20-00
What this study is testing
To evaluate the safety and tolerability of ABO-101 in participants with PH1
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- 1. Confirmed diagnosis of PH1
- 6. Able to follow directions and provide 24-hour urine collections
- 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
- 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
- 10. Participant must not have received any experimental agent for the treatment of PH1 for at least (CCI) half-lives
- 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
You likely can't join if
- 1. Confirmed diagnosis of PH2 or PH3
- 11. Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)
- 12. History of alcohol or drug abuse within 3 years prior to Screening
- 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
- 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
- 17. Anticipated survival <2 years in the opinion of the Investigator
See the full eligibility criteria
Who can join
- 1. Confirmed diagnosis of PH1
- 6. Able to follow directions and provide 24-hour urine collections
- 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
- 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
- 10. Participant must not have received any experimental agent for the treatment of PH1 for at least (CCI) half-lives
- 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
- 4. History of renal stone events, clinically related symptoms, or history of nephrocalcinosis.
- 9. All available standard of care options (including approved siRNA treatment) were considered according to local medical practice and guidelines
- 12. Weight ≤90 kg
- 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
- 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
- 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
- 16. Must be willing to comply with the requirements of the trial
- 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
- 3. Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase gene (AGXT)
- 5. Mean of (CCI) valid 24-hour UOx ≥0.7 mmol (63 mg)/24 hours/1.73 m2
What rules you out
- 1. Confirmed diagnosis of PH2 or PH3
- 11. Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)
- 12. History of alcohol or drug abuse within 3 years prior to Screening
- 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
- 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
- 17. Anticipated survival <2 years in the opinion of the Investigator
- 18. Unwilling to comply with trial procedures including Long-Term Safety Follow-Up
- 2. History of a liver, kidney, or combined liver/kidney transplant
- 3. Currently on dialysis or anticipated requirement for dialysis within the initial (CCI) months of the trial
- 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
- 4. Participant has previously used (within past (CCI) months) or is currently on an approved or investigational urinary oxalate lowering (CCI) or (CCI) therapy
- 5. Medical history includes clinical evidence of extrarenal systemic oxalosis, as determined by the Investigator
- 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
- 7. History of liver cirrhosis
- 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.
- 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
- 14. Use of antiplatelet (e.g., aspirin, clopidogrel) or antithrombotic therapy (e.g., warfarin, dabigatran, apixaban) within 14 days prior to ABO-101 administration.
- 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).
The study team makes the final eligibility decision.
Where it's taking place
- Qatar
- United Kingdom
- Tunisia
- Israel
- United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Qatar; United Kingdom; Tunisia; Israel; United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.