EUCTR2010-020224-22-FR进行中（未招募）1 期
A Phase 2a Study of Ataluren (PTC124) as an Oral Treatment for Nonsense-Mutation-Mediated Hemophilia A and B
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 13
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
- Male
入选标准
- •1. Evidence of signed and dated informed consent document(s) indicating that the subject has been informed of all pertinent aspects of the study.
- •2. Age =18 years.
- •3. Male sex.
- •4. Presence of a nonsense mutation as the sole disease-causing mutation in the f8 or f9 gene
- •5. Verification that a blood sample has been drawn for confirmation of the presence of a nonsense mutation in the f8/f9 gene. Note: A subject who has written documentation of a nonsense mutation as the cause for hemophilia need not wait for confirmatory results to start ataluren as long as the confirmatory genotyping blood sample has been drawn.
- •6. At least 20 exposure-days of treatment with FVIII/FIX concentrate.
- •7. Confirmed screening laboratory values within the ranges specified in Table 1 of page 34 in the protocol. Note: Confirmation should be performed for out-of-range values to determine if the abnormality is real or artifactual. Values used to establish eligibility should be obtained within the screening period, and should generally be the most recent measurement obtained.
- •8. Willingness, if not surgically sterile, to abstain from sexual intercourse or employ a barrier or medical method of contraception during ataluren administration and follow-up periods.
- •9. Willingness and ability to comply with scheduled visits, drug administration plan, study procedures, and study restrictions. Note: Psychological, social, familial, or geographical factors that might preclude adequate study participation should be considered.
- •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
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Known hypersensitivity to any of the ingredients or excipients of the study drug
- •(polydextrose, polyethylene glycol 3350, poloxamer 407, mannitol 25C, crospovidone XL10, hydroxyethyl cellulose, vanilla, colloidal silica, or magnesium stearate).
- •2. Unable or unwilling to forego prophylactic FVIII/FIX concentrate use during the screening and on-study periods. Note: Subjects are allowed use of FVIII/FIX for treatment of bleeding episodes while on study.
- •3. Any history of prior anti-FVIII/FIX inhibitors as determined by Bethesda assay.
- •4. Exposure to another investigational drug within 30 days prior to start of ataluren
- •administration.
- •5. Ongoing coumarin anti-coagulants, phenytoin, or tolbutamide therapy.
- •6. History of anti-hepatitis C therapy within 14 days prior to start of ataluren administration.
- •7. History of major surgical procedure within 30 days prior to start of ataluren administration.
- •8. Receiving study drug in any other therapeutic clinical trial.
- •9. History of coagulation abnormalities other than HA/HB.
- •10. Known portal hypertension.
- •11. Prior or ongoing medical condition (eg, concomitant illness, alcoholism, drug abuse, psychiatric condition), medical history, physical findings, ECG findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the subject, makes it unlikely that the course of study drug administration or follow-up would be completed, or could impair the assessment of study results. Note: Patients with human immunodeficiency virus (HIV), hepatitis B, and/or hepatitis C may be enrolled as long as they meet all entry criteria.
研究者
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