NCT06839235招募中1 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 Biotechnologies8 个研究点 分布在 5 个国家目标入组 23 人开始时间: 2025年6月16日最近更新:
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 发起方
- 入组人数
- 23
- 试验地点
- 8
- 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
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.
研究设计
- 研究类型
- Interventional
- 分配方式
- Non Randomized
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 6 Years 至 64 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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
排除标准
- •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)
研究组 & 干预措施
Experimental: Part A: Single Ascending Dose Escalation/Adaptive Design
Experimental
干预措施: ABO-101 (Drug)
Experimental: Part B: Single Dose Expansion
Experimental
干预措施: ABO-101 (Drug)
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)
时间窗: Up to 6 months
次要结局
- Plasma concentrations for LNP lipids, Cas12i2 mRNA, and guide RNA (gRNA)(Up to 6 months)
- Urine concentrations for LNP lipids(Up to 6 months)
- Antidrug antibodies to ABO-101 and anti-Cas protein antibodies(Up to 6 months)
- Percent change in 24-hour urinary oxalate excretion (UOx) from Baseline to Month 6(Up to 6 months)
- Absolute change in UOx corrected for body surface area(Up to 6 months)
- Percent change in plasma glycolate from Baseline to Month 6(Up to 6 months)
- Changes in estimated glomerular filtration rate (eGFR) from Baseline to Month 12 and Month 24(Up to 24 months)
研究者
研究点 (8)
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相关资讯
ABO-101 Gene Editing Therapy for Primary Hyperoxaluria Type 1 Granted Orphan Drug Designation by European Commission- The European Commission has granted Orphan Drug Designation to ABO-101, an investigational gene editing therapy for primary hyperoxaluria type 1 developed by Chiesi Group and Arbor Biotechnologies.
- ABO-101 is a one-time liver-directed CRISPR-based therapy targeting the HAO1 gene to permanently reduce hepatic oxalate production, addressing the root cause of PH1.
- The therapy is currently being evaluated in the global Phase 1/2 redePHine clinical study, with program updates to be presented at the 15th International Hyperoxaluria Workshop on June 26, 2026.
- ABO-101 previously received both Orphan Drug Designation and Rare Pediatric Disease Designation from the U.S. FDA in 2025, underscoring significant unmet medical need in this ultra-rare disease.2 months agoPrimary Hyperoxaluria Pipeline Shows Promise with 6+ Therapies in Development as Gene Editing Advances- Primary hyperoxaluria pipeline features over 6 companies developing more than 6 therapeutic candidates, with notable advances in gene editing and RNA interference therapies.
- Arbor Biotechnologies achieved a significant milestone in July 2025 with the first patient treated in their Phase 1/2 redePHine trial for ABO-101, a gene-editing therapy for PH1.
- The FDA has approved two breakthrough therapies: nedosiran (Rivfloza) in September 2023 for PH1 patients with preserved kidney function, and lumasiran in November 2020 for both adults and children.
- Leading pipeline candidates include CHK-336, BBP-711, BMN 255, Oxabact, and Nedosiran, spanning various development stages from preclinical to Phase III trials.last year