EUCTR2015-001910-88-DK进行中（未招募）1 期
An Open-Label, Intrapatient Dose Escalation Study to Evaluate the Safety, Tolerability, Immunogenicity, and Biological Activity of ATYR1940 in Patients with Limb Girdle and Facioscapulohumeral Muscular Dystrophies
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 16
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 aged 18 to 75 years, inclusive.
- •2. Provided written informed consent.
- •3. In the Investigator’s opinion, is willing and able to complete all study procedures and comply with the study visit schedule.
- •4. Established genetically confirmed diagnosis of LGMD2B or FSHD.
- •5. Either the presence of a STIR positive muscle with limited fatty infiltration, as determined by the Fischer scoring system, on MRI (FHSD) or an elevated muscle marker (LGMD2B)
- •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) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •1. Currently receiving treatment with an immunomodulatory agent or history of such treatment, including targeted biological therapies (e.g., etanercept, omalizumab) or corticosteroids within the 3 months before baseline; or high-dose non-steroidal anti-inflammatory agents within 2 weeks before baseline.
- •2. Currently receiving curcumin or albuterol or requires such treatment during study participation; use of a product that putatively enhances muscle growth or activity on a chronic basis within 30 days before baseline; or statin treatment initiation or significant adjustment to statin regimen within 3 months before baseline.
- •3. Use of an investigational product or device (other than a mobility assistance device) within 30 days before baseline.
- •4. History of severe restrictive or obstructive lung disease (including interstitial lung disease, pulmonary fibrosis, or asthma), or evidence of active lung disease on screening chest radiograph.
- •5. History of anti-synthetase syndrome, prior Jo-1 Ab-positivity, or Jo 1 Ab level =0.6 U/mL on screening.
- •6. Acute or clinically relevant Epstein-Barr virus (EBV) or cytomegalovirus (CMV) infection or re-activation.
- •7. Chronic infection, such as hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) or a history of tuberculosis.
- •8. Vaccination within 8 weeks before baseline or vaccination is planned during study participation.
- •9. Symptomatic cardiomyopathy or severe cardiac arrhythmia (including bradyarrythmias), that may, in the Investigator’s opinion, limit the patient’s ability to complete the study protocol.
- •10. Gamma-glutamyl transferase (GGT) or serum creatinine levels >2× the upper limit of normal.
- •11. Muscle biopsy within 30 days before baseline.
研究者
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