2024-518144-20-00招募中2 期
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)
Arbor Biotechnologies Inc.3 个研究点 分布在 3 个国家目标入组 6 人开始时间: 2025年5月15日最近更新:
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 发起方
- 入组人数
- 6
- 试验地点
- 3
- 主要终点
- Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101- related TEAEs and serious adverse events (SAEs)
研究概览
简要总结
To evaluate the safety and tolerability of ABO-101 in participants with PH1
研究设计
- 分配方式
- Not Applicable
- 主要目的
- Study Period 2
- 盲法
- None
入排标准
- 年龄范围
- 0 years 至 64 years（18-64 Years, 0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Confirmed diagnosis of PH1
- •Able to follow directions and provide 24-hour urine collections
- •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
- •Must meet the following laboratory: a. AST, ALT, total bilirubin ≤ULN for age. For participants with documented Gilbert’s syndrome, total bilirubin <2 × ULN b. eGFR ≥30 mL/min/1.73m2 based on CKD-EPI 2021 equation for participants ≥18 years of age at Screening and both the Schwartz equation and the CKD-EPI 2021 equation for participants <18 years of age at Screening c. Platelet count >100,000/mm3 d. Normal PT, aPTT, international normalized ratio (INR), D-dimer, fibrinogen
- •Participant must not have received any experimental agent for the treatment of PH1 for at least (CCI) half-lives
- •Participant must agree to not participate in another interventional trial during the Screening Period and for at least 6 months following ABO-101 administration
- •History of renal stone events, clinically related symptoms, or history of nephrocalcinosis.
- •All available standard of care options (including approved siRNA treatment) were considered according to local medical practice and guidelines
- •Weight ≤90 kg
- •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
- •For male participants: Must agree to use at least 1 highly effective method of contraception from the time of signing the ICF through 4 months after dosing with ABO-101, and not donate sperm through 4 months after trial drug administration
- •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
- •Must be willing to comply with the requirements of the trial
- •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
- •Documentation of PH1 as determined by genetic analysis confirming pathogenic mutations in the alanine-glyoxylate aminotransferase (AGXT) gene
- •Mean of (CCI) valid 24-hour UOx ≥0.7 mmol/24 hours/1.73 m2
排除标准
- •Confirmed diagnosis of PH2 or PH3
- •Participant has previously used (within past (CCI) months) or is currently on an approved or investigational urinary oxalate lowering (CCI) or (CCI) therapy
- •Medical history includes clinical evidence of extrarenal systemic oxalosis, as determined by the Investigator
- •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
- •History of liver cirrhosis
- •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.
- •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
- •Use of antiplatelet (e.g., aspirin, clopidogrel) or antithrombotic therapy (e.g., warfarin, dabigatran, apixaban) within 14 days prior to ABO-101 administration.
- •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).
- •Female participants who are pregnant or breastfeeding (or are planning either during the first 12 months)
- •History of alcohol or drug abuse within 3 years prior to Screening
- •History of active hepatitis B, C or HIV infection; or positive hepatitis B surface antigen. Participants who are hepatitis C virus (HCV) antibody positive must have negative HCV RNA
- •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
- •Anticipated survival <2 years in the opinion of the Investigator
- •Unwilling to comply with trial procedures including Long-Term Safety Follow-Up
- •History of a liver, kidney, or combined liver/kidney transplant
- •Currently on dialysis or anticipated requirement for dialysis within the initial (CCI) months of the trial
结局指标
主要结局
Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101- related TEAEs and serious adverse events (SAEs)
Incidence and severity of treatment-emergent adverse events (TEAEs), including ABO-101- related TEAEs and serious adverse events (SAEs)
次要结局
- Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month (CCI)
- Absolute change in UOx corrected for body surface area
- Percent change in plasma glycolate from Baseline to Month (CCI)
- Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month (CCI) and Month (CCI)
- Plasma concentrations for (CCI), and (CCI)
- Urine concentrations for (CCI) and (CCI)
- Antidrug antibodies to ABO-101 and anti-Cas12i2 protein antibodies
研究者
Tara Naylor
Scientific
Arbor Biotechnologies Inc.
研究点 (3)
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