EUCTR2016-004578-16-GB进行中（未招募）1 期
A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 versus Myozyme®/Lumizyme® in Patients with Late-Onset GSD-II (Pompe Disease) Followed by Open-Label Treatment with VAL-1221 in all Patients
相关药物
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 12
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. Male or female patients =18 years
- •2. Patient is able and willing to provide informed consent prior to any study procedures are performed
- •3. Diagnosis of Glycogen Storage Disease II (GSD-II) based on one of the following:
- •a. Endogenous cultured skin fibroblast acid alpha glucosidase (GAA) activity <40% of adult normal level
- •b. Endogenous whole blood or dried blood spot GAA activity in deficiency range
- •c. Genetic analysis showing pathogenic variants in both alleles
- •4. Onset of Pompe disease-related symptoms after 1 year of age
- •5. Previously treated with Myozyme or Lumizyme for at least 12 months and on a stable regimen for the past 6 months
- •6. Sexually active patients willing to use an acceptable method of contraception (abstinence (defined as refraining from heterosexual intercourse during the entire period of the study and is the preferred and usual lifestyle of the patient), oral contraceptives, barrier method with spermicide, surgical sterilization, implanted or injectable contraceptives [including intrauterine devices with or without hormone release systems]) with a stable dose or insertion, as applicable, at least 1 month prior to Baseline, during the study and for 30 days after completion of treatment
- •a. If patient is female and not considered to be of childbearing potential, she is at least 2 years post-menopause, or has undergone a tubal ligation, a total hysterectomy or bilateral oophorectomy
- •b. If patient is female and of childbearing potential, she has a negative serum pregnancy test during screening and baseline and must be willing to undergo pregnancy testing at specific intervals during the study
- •7. Patient meets at least one of the following criteria:
- •a. >30% and <80% predicted upright forced volume capacity (FVC)
- •b. Able to walk >20% but <80% predicted normal on 6-minute walk test (6MWT) with or without use of assistive devices
- •8. Patient is willing and able to comply with protocol requirements
- •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 12
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Cardiac involvement in first year of life
- •2. Anti-GAA antibody titers >1:51,200 at two time points
- •3. Prior use of chaperone therapy for GSD-II within the last 12 months
- •4. Use of immunosuppressive medication other than glucocorticoids within 6 months prior to study enrollment
- •5. Use of invasive ventilatory assistance other than BiPAP at night or during periods of rest
- •6. Has received any investigational medication or has enrolled in any study involving investigational drugs or therapies within 30 days prior to first dose of study drug
- •7. Start of or change in usual regimen of albuterol or respiratory muscle training within 30 days prior to first dose of study drug
- •8. History of sensitivity to any of the constituents of the study drug
- •9. Patient is planning to become pregnant or to breastfeed during the study or is currently breastfeeding
- •10. Patient has a medical condition or circumstance that, in the opinion of the investigator, might compromise the patient’s ability to comply with the protocol or the patient’s well-being or safety
- •11. Patient has any condition that, in the view of the investigator, places the patient at high risk of poor treatment compliance or of not completing the study
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