EUCTR2016-002652-25-IT进行中（未招募）1 期
A Phase 2 Open-Label Proof of Concept Study to Assess the Efficacy, Safety, and Pharmacokinetics of ACH-0144471 in Untreated Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) - A treatment study of ACH-0144471 in Untreated Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Currently untreated PNH patients with PNH Type III erythrocyte and/or granulocyte clone size =10% and anemia (Hgb <12 g/dL) with adequate reticulocytosis (as determined by the investigator)
- •2. LDH =1.5× upper limit of normal (ULN)
- •3. Platelet count =50,000/µL without the need for platelet transfusions
- •4. Documentation of vaccination for N. meningitidis, H. influenzae, and S. pneumoniae, or willingness to receive vaccinations as described in Section 6.3
- •5. Age =18 years (or = minimum adult age in accordance with local legal requirements)
- •6. Female participants of childbearing potential must agree to use an acceptable method of contraception (as defined in Section 5.5.5) from the date of signing the informed consent to the first day of dosing (Day 1), and must agree to use a highly effective method of contraception (as defined in Section 5.5.5) from the first day of dosing to 30 days after their last dose of study drug. Female participants of childbearing potential must also have a negative serum pregnancy test during Screening and negative urine pregnancy test on Day 1. Female participants of non-childbearing potential need not employ a method of contraception.
- •7. Non-sterile male participants must agree to use a highly effective method of contraception (as defined in Section 5.5.5) with their partner(s) of childbearing potential from the first day of dosing to 90 days after their last dose of study drug. Males who are surgically sterile need not employ additional contraception. Males must agree not to donate sperm while enrolled in this study and for 90 days after their last dose of study drug.
- •8. Must agree to provide written informed consent
- •9. Must be willing, at all times, to have transportation and telephone access, and to be within one hour of an emergency medical center
- •Are the trial subjects under 18? no
- •Number of subjects for this age range:
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 12
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. History of a major organ transplant (e.g., heart, lung, kidney, liver) or hematopoietic stem cell/marrow transplant
- •2. History of dosing with another investigational agent within 30 days or 5 half-lives of the investigational agent prior to study drug administration, whichever is greater
- •3. History of dosing with eculizumab at any dose or interval within the past 75 days before study drug administration
- •4. Known or suspected complement deficiency
- •5. Contraindication to one or more of the required vaccinations
- •6. Active bacterial infection or clinically significant active viral infection, a body temperature >38°C, or other evidence of infection on Day 1, or history of febrile illness within 14 days prior to first study drug administration
- •7. History of meningococcal infection, or a first-degree relative or household contact with a history of meningococcal infection
- •8. History of hypersensitivity reactions to commonly used antibacterial agents, including beta-lactams, penicillin, aminopenicillins, fluoroquinolones (specifically including ciprofloxacin), cephalosporins, and carbapenems, which in the opinion of the investigator would make it difficult to properly provide either empiric antibiotic therapy or treat an active infection
- •9. History or presence of any clinically relevant co-morbidities that would make the patient inappropriate for the study (for example, is likely to result in deterioration of the patient's condition, affect the patient's safety during the study, or confound the results of the study)
- •10. Laboratory abnormalities at screening, including: • Alkaline phosphatase (ALP) >1.5× upper limit of normal (ULN) • Absolute neutrophil count (ANC) <1,000/µL • Alanine aminotransferase (ALT) > ULN • Any other clinically significant laboratory abnormality that, in the opinion of the Primary Investigator (PI), would make the patient inappropriate for the study or put the patient at undue risk
- •11. Females who are pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration or patients with a female partner who is pregnant, nursing, or planning to become pregnant during the study or within 90 days of study drug administration
- •12. Prior history or current evidence of biliary cholestasis
- •13. Gilbert's syndrome Patients with history or family history suggestive of Gilbert's syndrome should be tested and excluded from study if positive for UGT1A1 genotyping polymorphism or missense change
- •14. Evidence of human immunodeficiency virus (HIV), hepatitis B or hepatitis C infection (positive serology for HIV-1 antibody (HIV Ab), positive hepatitis B surface antigen (HbsAg), or positive anti-HCV antibody (HCV Ab) at Screening or historically)
研究者
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