EUCTR2015-003681-87-IT进行中（未招募）1 期
A multi center, multi national, open label, extension study to evaluate the long-term efficacy and safety of BMN 044 (PRO044) in subjects with Duchenne muscular dystrophy - NA
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 7
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. Subjects previously treated with BMN 044 or a comparator treatment
- •in a BMN 044 Sponsored Study or Investigator Initiated Trial and who
- •are not eligible for another ongoing BMN 044 study. Subjects who
- •withdrew from any previous BMN 044 study due to meeting laboratory safety
- •stopping criteria may be eligible to enroll if the applicable laboratory
- •parameter has resolved to be within normal limits or parent study baseline value, and benefit of further treatment with BMN 044
- •outweighs the risk to the individual subject, as agreed in consultation
- •with the Medical Monitor.
- •2. Continued use of glucocorticosteroids for a minimum of 60 days prior to study entry with a reasonable expectation that the subject will remain
- •on glucocorticosteroids for the duration of this study. Changes to or
- •cessation of glucocorticosteroids will be at the discretion of the
- •Investigator conducting this study in consultation with the
- •subject/parent and Medical Monitor. Subjects who have discontinued
- •glucocorticosteroids in their previous BMN 044 study, following
- •consultation with the Medical Monitor may be enrolled.
- •3. Willing and able to comply with all study requirements and
- •procedures.
- •4. Willing and able to provide written, signed informed consent, or in the
- •case of subjects under the age of 18 years(or 16 years, depending on the
- •region), provide written assent (if required) and written informed
- •consent by a legally authorized representative after the nature of the
- •study has been explained, and prior to the conduct of any researchrelated
- •procedures.
- •Are the trial subjects under 18? yes
- •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 2
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Subjects who have previously been treated with BMN 044 who
- •had a serious adverse experience or met safety stopping criteria
- •that remains unresolved, which in the opinion of the Investigator could
- •have been attributable to BMN 044. Once resolved, subject may be
- •eligible to enter the study following Investigator consultation with the
- •Medical Monitor.
- •2. History of significant medical disorder which may confound the
- •interpretation of safety data (e.g. current or history of renal or liver
- •disease/impairment, history of inflammatory illness, bleeding
- •complications, mental retardation and/or behavioral problems).
- •3. Acute illness within 4 weeks prior to the first dose of BMN 044 (Week
- •1) which may interfere with the measurements.
- •4. Symptomatic cardiomyopathy. If subject has a left ventricular ejection
- •fraction <45% at start of this study, the Investigator should discuss
- •inclusion of subject in this study with the Medical Monitor.
- •5. Baseline aPTT above the upper limit of normal (ULN). A re test is
- •possible at a later stage, and if within normal range, the subject may
- •enter the study.
- •6. Baseline platelet count below the lower limit of normal (LLN). A re
- •test is possible at a later stage, and if within normal range, the subject
- •may enter the study.
- •7. Use of anti coagulants, anti thrombotics or anti platelet agents within
- •28 days of the baseline visit. Chronic use of anti coagulants, anti
- •thrombotics or anti platelet agents is prohibited during the study. As
- •needed dosing (pro re nata – PRN) may be acceptable (except for
- •aspirin) following discussion with the Medical Monitor.
- •8. Prior use of any investigational product (other than BMN 044) or
- •investigational medical device must be discussed with the Medical
- •Monitor prior to screening.
- •9. Current or history of drug and/or alcohol abuse.
研究者
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