NCT07603401Available不适用
Expanded Access Program Protocol: Bitopertin (DISC-1459) For The Treatment of Erythropoietic Protoporphyria (EPP) and X-Linked Protoporphyria (XLP)
试验速览
- 阶段
- 不适用
- 状态
- Available
- 试验地点
- 5
研究概览
简要总结
The goal of this expanded access program is to provide bitopertin to patients with EPP and XLP who have no satisfactory treatment options available in the US and to learn if bitopertin is safe to treat EPP and XLP.
研究设计
- 研究类型
- Expanded Access
入排标准
- 年龄范围
- 12 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Written informed consent (and assent where applicable).
- •Age ≥12 years at time of request.
- •Diagnosis of erythropoietic protoporphyria (EPP) or X-linked protoporphyria (XLP), confirmed by ferrochelatase (FECH) or aminolevulinic acid synthase 2 (ALAS2) genotyping or by biochemical porphyrin analysis. Prior confirmation is acceptable; re-testing is not required if documentation of prior diagnosis is available.
- •Hemoglobin ≥10 g/dL at baseline.
- •Females of childbearing potential must have a negative pregnancy test prior to enrollment.
排除标准
- •Known hypersensitivity to bitopertin or its excipients.
- •Patients with AST or ALT ≥3 × upper limit of normal (ULN), or total bilirubin ≥2 × ULN (unless due to Gilbert syndrome), or albumin below the lower limit of normal at screening.
- •Patients with a history of liver transplantation or anticipated need for liver transplantation.
- •Pregnancy, planned pregnancy during the program, or breastfeeding.
- •Inability or unwillingness to comply with contraception requirements.
- •Use of any of the following prohibited concomitant therapies:
- •Strong CYP3A4 inhibitors
- •Strong CYP3A4 inducers
- •Grapefruit or Seville orange products
- •Afamelanotide or dersimelagon
- •Chronic opioid therapy (>7 consecutive days)
- •Patients who are currently participating in a bitopertin clinical study.
- •Patients who previously participated in a bitopertin study and discontinued the study due to an adverse event or for reasons that, in the judgement of the treating physician or Disc, would put the patient at unacceptable risk or otherwise preclude participation in the EAP.
- •Patients who have received another investigational therapy within 30 days.
- •History of suicidal ideation with intent (C-SSRS Grade 4 or 5) within the past year or suicidal behavior within the past 5 years.
研究者
研究点 (5)
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