NL-OMON44009已完成不适用
An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, Biological Activity, and Systemic Exposure of ATYR1940 in Adult Patients with Facioscapulohumeral Muscular Dystrophy (FSHD) - ATYR1940-C-005
适应症
试验速览
- 阶段
- 不适用
- 状态
- 已完成
- 入组人数
- 8
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 年龄范围
- 18 至 99（—）
入选标准
- •1. Enrolled in >=Cohort 2 and completed the double-blind treatment period in the parent study.;2. Demonstrated, in the Investigator*s opinion, acceptable tolerability of study drug.;3. In the Investigator*s opinion, patient has shown acceptable compliance with study drug and the study procedures in the parent study and is willing and able to comply with all procedures in the current study.;4. Is, in the opinion of the Investigator and Sponsor, a suitable candidate for continued study drug treatment.;5. Provided written informed consent after the nature of the study has been explained and prior to the performance of any research-related procedures.
排除标准
- •1. At any time during participation in the parent study, met a study drug discontinuation criterion, including, but not limited to:;a. Jo-1 Ab levels >=1.5 U/mL.;b. Clinical evidence of a generalized infusion-related reaction (IRR).;c. Clinical evidence of a National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) (version 4.03) >=1.5 U/mL;d. Pregnancy.;e. Progression of disease that, in the opinion of the Investigator, precluded further participation in the study.;f. Withdrawal of consent.;g. Other findings that, at the discretion of the Investigator and/or Sponsor, indicated that study drug administration should be discontinued.;2. Is expected to require treatment with curcumin or systemic albuterol (intermittent inhaled albuterol is permissible) during study participation; or use of a product that putatively enhances muscle growth (e.g., insulin-like growth factor, growth hormone) or activity (e.g., Coenzyme Q, Coenzyme A, creatine, L-carnitine) on a chronic basis; or statin treatment initiation or significant adjustment to statin regimen (stable, chronic statin use is permissible).;3. Planned to receive any vaccination during study participation.;4. Abnormal baseline findings, medical condition(s), or laboratory findings that, in the Investigator*s opinion, might jeopardize the patient's safety or decrease the chance of obtaining satisfactory data needed to achieve the objectives of the study.;5. 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.;6. If female and of childbearing potential (premenopausal and not surgically sterile), has a positive pregnancy test at entry or is unwilling to use contraception from the time of entry through the 1-month Follow-up visit. Acceptable methods of birth control include abstinence, barrier methods, hormones, or intra-uterine device. ;7. If male, is unwilling to use a condom plus spermicide during sexual intercourse from the time of entry through the 1 month Follow-up visit.
研究者
相似试验
进行中（未招募）
1 期
A Study to Evaluate the Long-Term Safety of Inclacumab Administered to Participants with Sickle Cell DiseaseMedDRA version: 21.0Level: PTClassification code 10040644Term: Sickle cell diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersSickle Cell DiseaseEUCTR2020-005289-32-DEGlobal Blood Therapeutics, Inc.520
进行中（未招募）
1 期
A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a-galactosidase A.MedDRA version: 17.0Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-004800-40-ATAmicus Therapeutics, Inc.100
进行中（未招募）
1 期
A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme alfa-galactosidase A.MedDRA version: 14.1Level: SOCClassification code 10010331Term: Congenital, familial and genetic disordersSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-004800-40-ESGlaxoSmithKline Research & Development Limited100
进行中（未招募）
不适用
A Study using Migalastat to see the safety and usefulness of the drug in patients with Fabry Disease.Fabry disease is a rare X-linked lysosomal storage disorder caused by mutations in the gene (GLA) that encodes the lysosomal enzyme a- galactosidase A.MedDRA version: 14.1Level: PTClassification code 10016016Term: Fabry's diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-004800-40-ITGLAXOSMITHKLINE RESEARCH & DEVELOPMENT LTD.100
进行中（未招募）
1 期
An extension to the phase 2 study A536-03 for the treatment of anemia in patients with myelodysplastic syndrome of low and intermediate risk groupsMyelodysplastic Syndromes (MDS)MedDRA version: 20.0Level: SOCClassification code 10029104Term: Neoplasms benign, malignant and unspecified (incl cysts and polyps)System Organ Class: 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps)MedDRA version: 20.0Level: HLTClassification code 10028536Term: Myelodysplastic syndromesSystem Organ Class: 100000004851EUCTR2014-001280-13-DEAcceleron Pharma Inc.153