NCT06735755招募中1 期
A Phase 1/2, Dose-Exploration Study to Evaluate the Safety and Efficacy of BEAM-301 in Patients With Glycogen Storage Disease Type Ia (GSDIa) Homozygous or Compound Heterozygous for the G6PC1 c.247C>T (p.R83C) Variant
适应症
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 招募中
- 入组人数
- 36
- 试验地点
- 4
- 主要终点
- To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL
研究概览
简要总结
This is a Phase 1/2, multicenter, open-label, single-ascending-dose study to evaluate the safety, tolerability, and efficacy of BEAM-301 in adult patients with GSDIa homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant and to determine the optimal biological dose.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Sequential
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- 18 Years 至 —（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Males or females age ≥18 years of age at the time of consent.
- •Diagnosis of GSDIa and homozygous or compound heterozygous for the G6PC1 c.247C>T (p.R83C) variant (confirmed by genetic testing).
- •History of at least 1 episode of hypoglycemia <60 mg/dL within the 2 years prior to signing the ICF.
排除标准
- •Liver transplant recipient/on waiting list for liver transplant or known history of liver cirrhosis.
- •Presence of liver adenoma >5 cm in size based on liver MRI or liver ultrasound performed at screening or within 6 months prior to screening.
- •Presence of liver adenoma >3 cm and ≤5 cm with a documented annual growth rate of ≥0.5 cm per year.
- •Have aspartate transaminase or alanine transaminase >upper limit of normal (ULN).
- •Total bilirubin levels >ULN; if documented Gilbert's Syndrome, total bilirubin >2 × ULN.
- •Previous treatment with any cell-, gene-, or viral vector-derived therapy Hospitalization for management of hypoglycemia within 4 weeks prior to signing the ICF.
- •Have triglycerides >1000 mg/dL or a history of pancreatitis or a history of acute pancreatitis within 5 years that does not have a known and corrected etiology (eg, cholecystectomy for gallstone pancreatitis).
结局指标
主要结局
To evaluate the safety and tolerability of BEAM-301 in patients with GSDIa with a G6PC1 c.247C>T (p.R83C) variant by measuring incidence of treatment-emergent adverse events (TEAEs) including serious adverse events (SAEs) and dose-limiting toxicities (DL
时间窗: BEAM-301 administration through month 24
次要结局
- Absolute and change from baseline in time to hypoglycemia or metabolic decompensation during a controlled fasting challenge(Baseline to month 24)
- Changes from baseline in starch supplementation dose(Baseline to month 24)
- Level of serum glucose and metabolic parameters (lipid profile and uric acid) over time(Baseline to month 24)
- BEAM-301 PK parameters (Cmax)(Baseline to month 24)
- BEAM-301 PK parameters (AUC)(Baseline to month 24)
- BEAM-301 PK parameters (half-life)(Baseline to month 24)
研究者
研究点 (4)
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