EUCTR2014-001753-17-IT进行中（未招募）1 期
A Placebo-Controlled, Randomized, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Biological Activity of ATYR1940 in Adult Patients with Molecularly Defined Genetic Muscular Dystrophies
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 20
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. Patient is a male or female aged 18 to 65 years, inclusive.
- •2. Patient has an established, genetically-confirmed, diagnosis of FSHD with clinical findings meeting existing criteria.
- •3. Patient has provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
- •4. Patient is, in the Investigator’s opinion, willing and able to comply with all study procedures.
- •5. Cohorts =2 only: Patient has imaging findings meeting defined criteria for muscle inflammation in at least 1 skeletal muscle (MRI criteria).
- •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 44
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Patient is currently receiving treatment with an immunomodulatory agent or has a history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) within the 3 months before Baseline; corticosteroids within 4 weeks before Baseline; or non-steroidal anti-inflammatory agents (NSAIDs) within 2 weeks before Baseline.
- •2. Patient has evidence of an alternative diagnosis other than FSHD, based on prior muscle biopsy or genetic test findings.
- •3. Patient has a presumptive diagnosis of FSHD, based on clinical assessment, but does not yet have genetic confirmation of the diagnosis.
- •4. Patient has a severe retinopathy.
- •5. Patient has a history of obstructive or restrictive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence for interstitial lung disease on Screening chest radiograph.
- •6. Patient has a history of anti-synthetase syndrome, prior Jo-1 antibody (Ab)-positivity, or has a positive or equivocally positive test result during Screening.
- •7. Patient has symptomatic cardiomyopathy or severe cardiac arrhythmia that may, in the Investigator's opinion, limit the patient's ability to complete the study protocol.
- •8. Patient has abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator's opinion, might jeopardize patient's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.
- •9. Patient has evidence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematological, metabolic, dermatological, or gastrointestinal disease, or has a condition that requires immediate surgical intervention or other treatment or may not allow safe participation.
- •10. Patient has used any investigational product or device (other than a mobility assistance device) within 30 days before Baseline.
- •11. If female and of childbearing potential (premenopausal and not surgically sterile), patient has a positive pregnancy test at Screening or is unwilling to use contraception from the time of Screening through the 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device.
- •12. If male, patient is unwilling to use a condom plus spermicide during sexual intercourse from the time of Screening through the 1 month Follow-up visit.
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