EUCTR2014-002596-28-GB进行中（未招募）1 期
A Phase 2 Study of Ataluren (PTC124) as Treatment for Nonsense Mutation Mucopolysaccharidosis Type I
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 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 clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Evidence of signed and dated informed consent document(s) indicating that the study candidate (and/or a parent/legal guardian) has been informed of all pertinent aspects of the study. Note: If the study candidate is considered a child under local regulation, a parent or legal guardian must provide written consent prior to initiation of study screening procedures and the study candidate may be required to provide written assent. The rules of the responsible institutional review board/independent ethics committee (IRB/IEC) regarding whether one or both parents must provide consent and the appropriate ages for obtaining consent and assent from the patient should be followed.
- •2. Age =2 years.
- •3. Clinical diagnosis of MPS I, confirmed by measurable clinical signs and symptoms of MPS Iand a documented fibroblast or leukocyte a-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
- •4. Documentation of the presence of a nonsense mutation in at least 1 allele of the a L iduronidase (IDUA) gene.
- •5. Verification that a blood sample has been drawn for confirmation of the presence of a nonsense mutation in the IDUA gene.
- •6. Confirmed screening laboratory values within the central laboratory ranges specified in the protocol
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 12
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 6
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Prior BMT/HSCT.
- •2. Treatment with ERT within 2 months prior to enrollment.
- •3. Any change (initiation, change in type of drug, dose modifications, schedule modifications, interruption, discontinuation, or re-initiation) in chronic treatment for MPS within 2 months prior to start of Screening.
- •4. Exposure to another investigational drug within 3 months prior to enrollment.
- •5. Ongoing participation in any other therapeutic clinical trial.
- •6. Is pregnant or lactating. Female patients of childbearing potential must have a negative pregnancy test [ß-human chronic gonadotropin (hCG)] at Screening.
- •7. Ongoing warfarin, phenytoin or tolbutamide therapy.
- •8. Ongoing IV aminoglycoside use.
- •9. Ongoing immunosuppressive therapy (other than corticosteroids).
- •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 patient, makes it unlikely that the course of study drug administration or follow-up would be completed, or could impair the assessment of study results.
- •12. 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).
- •13. Surgery within 30 days prior to enrollment, or anticipated surgery during study participation.
研究者
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