NL-OMON50200已完成3 期
A PHASE 3, OPEN-LABEL STUDY OF ALXN1210 IN CHILDREN AND ADOLESCENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH) - ALXN1210-PNH-304
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 入组人数
- 3
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 2 至 17（—）
入选标准
- •1. Male and female patients < 18 years of age and weighing * 5 kg at the time
- •of consent.
- •2. Documented diagnosis of PNH, confirmed by high-sensitivity flow cytometry
- •evaluation of red blood cells (RBCs) and white blood cells (WBCs), with
- •granulocyte or monocyte clone size of * 5%.
- •3. For patients not currently treated with complement inhibitor, presence of 1
- •or more of the following PNHrelated signs or symptoms within 3 months of
- •Screening: fatigue, hemoglobinuria, abdominal pain, shortness of breath
- •(dyspnea), anemia, history of a major adverse vascular event (including
- •thrombosis), dysphagia, or erectile dysfunction; or history of packed RBC
- •transfusion due to PNH.
- •4. Lactate dehydrogenase (LDH) values at Screening as follows:
- •a. For patients not currently treated with eculizumab, LDH level * 1.5 × upper
- •limit of normal (ULN).
- •b. For patients who are currently taking eculizumab, LDH * 1.5× ULN (sample
- •must be obtained on a
- •scheduled eculizumab-dosing day prior to dose administration [ie, at trough
- •eculizumab level] and
- •analyzed by the central laboratory).
- •5. To reduce the risk of meningococcal infection (Neisseria meningitidis), all
- •patients must be vaccinated against meningococcal infections within 3 years
- •prior to, or at the time of, initiating study drug. Patients who initiate study
- •drug treatment less than 2 weeks after receiving a meningococcal vaccine must
- •receive treatment with appropriate prophylactic antibiotics until 2 weeks after
- •vaccination. Patients who cannot be vaccinated must receive antibiotic
- •prophylaxis for the entire treatment period and for 8 months following last
- •6. Patients must have been vaccinated against Haemophilus influenzae type b
- •(Hib) and Streptococcus pneumoniae according to national and local vaccination
- •schedule guidelines, as appropriate.
- •7. Female patients of childbearing potential (ie, have achieved menarche) and
- •male patients with female partners of childbearing potential must follow
- •protocol-specified guidance for avoiding pregnancy while on treatment and for 8
- •months after last dose of study drug.
- •8. Patient's legal guardian must be willing and able to give written informed
- •consent and the patient must be willing to give written informed assent (if
- •applicable as determined by the central or local Institutional Review Board
- •[IRB]/Institutional (or Independent) Ethics Committee [IEC]) and comply with
- •the study visit schedule.
排除标准
- •1. Platelet count < 30,000/mm3 (30 × 109/L) at Screening.
- •2. Absolute neutrophil count < 500/*L (0.5 × 109/L) at Screening.
- •3. History of bone marrow transplantation.
- •4. History of N. meningitidis infection.
- •5. History of unexplained, recurrent infection.
- •6. Active systemic bacterial, viral, or fungal infection within 14 days prior
- •to study drug administration on Day 1.
- •7. History of malignancy within 5 years of Screening with the exception of
- •adequately treated nonmelanoma skin cancer or carcinoma in situ of the cervix.
- •8. History of or ongoing major cardiac, pulmonary, renal, endocrine, or hepatic
- •disease (eg, active hepatitis) that, in the opinion of the Investigator or
- •Sponsor, precludes the patient*s participation in an
- •investigational clinical trial.
- •9. Unstable medical conditions (eg, myocardial ischemia, active
- •gastrointestinal bleed, severe congestive heart failure, anticipated need for
- •major surgery within 6 months of Screening, coexisting chronic anemia unrelated
- •to PNH) that would make them unlikely to tolerate the requirements of the
- •10. Concomitant use of anticoagulants is prohibited if not on a stable regimen
- •for at least 2 weeks prior to Day 1.
- •11. History of hypersensitivity to any ingredient contained in the study drug,
- •including hypersensitivity to murine proteins.
- •12. Females who plan to become pregnant or are currently pregnant or
- •breastfeeding.
- •13. Females of childbearing potential who have a positive pregnancy test result
- •at Screening or on Day 1.
- •14. Participation in another interventional treatment study or use of any
- •experimental therapy within 30 days before initiation of study drug on Day 1 in
- •this study or within 5 half-lives of that investigational product,whichever is
- •15. Known or suspected history of drug or alcohol abuse or dependence within 1
- •year prior to the start of Screening.
- •16. Known medical or psychological condition(s) or risk factor that, in the
- •opinion of the Investigator or Sponsor, might interfere with the patient*s full
- •participation in the study, pose any additional risk for the patient, or
- •confound the assessment of the patient or outcome of the study.
研究者
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