EUCTR2013-001768-48-AT进行中（未招募）1 期
A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 70
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
- •Willing and able to provide written informed consent, after the nature of the study has been explained, and prior to any study-related procedures.
- •Diagnosed with late-onset Pompe disease based on 2 currently or previously documented GAA gene mutations, and endogenous GAA activity <75% of the lower limit of the normal adult range reported by the testing laboratory, as assessed by dried blood spot or whole blood assay.
- •--Historical data may be used to qualify subjects at Screening but samples will also be obtained for the assays to be performed at a central laboratory during the study to establish baseline demographics. If the historical data is not available, the results from the central laboratory confirming eligibility must be available prior to Day 1.
- •= 18 years of age at the time of enrollment in the study
- •Has received prior treatment with commercial rhGAA as defined by ALL of the following:
- •--has received treatment with commercial rhGAA for = 48 weeks (but no more than 20% of the cohort 2 population can have received treatment for = 6 years);
- •--has received > 80% of all scheduled treatments in the prior 48 weeks and = 4 out of the prior 6 scheduled treatments;
- •--has received and completed the last two infusions without a drug related adverse event resulting in dose interruption;
- •--has received last treatment of commercial rhGAA = 10 and = 31 days prior to anticipated initiation of treatment with BMN 701.
- •Sexually active subjects must be willing to use two known effective methods of contraception while participating in the study and for at least 4 months following the last dose of BMN 701
- •Females of childbearing potential must have a negative pregnancy test at Screening and Baseline visits and be willing to have additional pregnancy tests during the study. Females considered not of
- •childbearing potential include those who have been in menopause at least 2 years, or had tubal ligation at least 1 year prior to Screening, or who have had total hysterectomy.
- •Has = 30% predicted FVC upright and < 80% predicted FVC upright.
- •Has = 60% predicted MIP.
- •Is able to ambulate > 75 meters and < 500 meters on the 6MWT on one day conducted during the Screening visit (use of assistive devices such as walker, cane, or crutches, is permitted with consistent use throughout the study).
- •Is willing and able to comply with all study procedures
- •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 65
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 5
排除标准
- •Use of any investigational product or investigational medical device within 4 weeks prior to Screening, or requirement for any investigational agent other than BMN 701 prior to completion of at least the first 24 weeks of all scheduled study assessments.
- •Received any investigational medication for Pompe disease within the prior 12 months.
- •Has a diagnosis of diabetes and/or is currently being treated with or anticipated to require treatment with hypoglycemic agents during the course of the study.
- •Has been treated with any immunosuppressive medication other than glucocorticosteroids within the prior 12 months.
- •Requires noninvasive ventilatory support while awake and in the upright position.
- •Has previously been enrolled to this study.
- •Breastfeeding at Screening or planning to become pregnant (self or partner) at any time during the study.
- •Concurrent disease, medical condition, or extenuating circumstance that, in the opinion of the Investigator, might compromise subject safety, study treatment compliance and completion of the study, or the integrity of the data collected for the study.
- •Has known hypersensitivity to BMN 701 or its excipients.
研究者
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