NCT03895528Approved For Marketing不适用
A Treatment IND (Investigational New Drug) Protocol for EAP (Expanded Access Program) for the Use of Lonafarnib in Patients With Hutchinson-Gilford Progeria Syndrome (HGPS) or Progeroid Laminopathy
Eiger BioPharmaceuticals0 个研究点开始时间: 2019年3月29日最近更新:
适应症
试验速览
- 阶段
- 不适用
- 状态
- Approved For Marketing
- 发起方
研究概览
简要总结
This treatment IND protocol will allow patients with HGPS and progeroid laminopathies access to lonafarnib, the only compound shown to have an effect on the HGPS disease process resulting in improved outcomes (Gordon et al, 2018). There are no approved treatments for HGPS and progeroid laminopathies.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 12 Months 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Clinical diagnosis of HGPS or progeroid laminopathy by qualified medical doctor (based on common phenotype as described in Gordon 2015 and Merideth 2008). Confirmation with genetic testing is preferred but not required.
- •Adequate hepatic function as defined by SGPT (ALT) and SGOT (AST) ≤ 5 times upper limit of normal range for age
排除标准
- •Taking medications or foods that are known to be moderate or strong inducers or inhibitors of CYP3A4 or sensitive CYP3A substrates; or if a patient is taking one of these drugs and cannot safely discontinue or take an alternative drug, the dose of the inhibitor/inducer must be adjusted per the treating physician
- •Taking digoxin, a P-gp substrate with a narrow therapeutic window.
- •Severe renal impairment (GFR < 30 mL/min/1.73m2).
- •Uncontrolled infection.
- •Presence of any active clinically relevant medical condition that in the opinion of the treating physician would preclude patient from safely participating in the program.
- •Pregnant or breast-feeding or plan to become pregnant while on therapy.
研究者
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