EUCTR2011-003197-84-IT进行中（未招募）1 期
A Phase 2, Open-label, Multinational Clinical Study to Evaluate the Safety and Efficacy of BMN 110 in Pediatric Patients Less Than 5 Years of Age with Mucopolysaccharidosis IVA (Morquio A Syndrome)
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 15
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- Less than 5 years of age. 2-Documented clinical diagnosis of MPS IVA based on clinical signs and symptoms of MPS IVA and documented reduced fibroblast or leukocyte GALNS enzyme activity or genetic testing confirming diagnosis of MPS IVA. 3-Written informed consent provided by parent or legally authorized representative after the nature of the study has been explained and prior to any research-related procedures.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 15
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •Previous hematopoietic stem cell transplant (HSCT). • Previous treatment with BMN 110. • Known hypersensitivity to any of the components of BMN 110. • Major surgery within 3 months prior to study entry or planned major surgery during the 52-week treatment period. • Use of any investigational product or investigational medical device within 30 days prior to Screening, or requirement for any investigational agent prior to completion of all scheduled study assessments. • Concurrent disease or condition, including but not limited to symptomatic cervical spine instability, clinically significant spinal cord compression, or severe cardiac disease that would interfere with study participation or safety as determined by the Investigator. • 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.
研究者
相似试验
进行中（未招募）
1 期
A Study to Evaluate the Safety and Efficacy of BMN 110 in MPS IVA Patients Less Than 5 Years of AgeMucopolysaccharidosis Type IVAMedDRA version: 14.1 Level: PT Classification code 10028095 Term: Mucopolysaccharidosis IV System Organ Class: 10010331 - Congenital, familial and genetic disordersEUCTR2011-003197-84-GBBioMarin Pharmaceutical Inc.15
进行中（未招募）
不适用
A Global, Multi-center, Open-label, Phase 2 Study of AGS-1C4D4 Given in Combination With Gemcitabine in Subjects with Metastatic Pancreatic Cancer?Estudio global, multicéntrico, abierto, de fase II de AGS-1C4D4 administrado en combinación con gemcitabina en sujetos con cáncer de páncreas metastásico.Metastatic Pancreatic Cancer. To evaluate AGS-1C4D4 administered in combination with gemcitabine as measured by the survival rate at 6 months post-randomizationCancer de páncreas metástasico. Evaluar AGS-1C4D4 administrado en combinación con gemcitabina determinado mediante la tasa de supervivencia a los 6 meses después de la aleatorizaciónEUCTR2009-009194-99-ESAgensys Inc185
招募中
2 期
A Clinical Phase II, multicenter, Open-label study evaluating iNduction, consolidation and maintenance treatment with Isatuximab (SAR650984), Carfilzomib, LEnalidomide and Dexamethasone (I-KRd) in Primary diagnosed high-risk multiple myeloma paTients
GMMG-CONCEPTMultiple Myeloma2024-511791-32-00University Medical Center Hamburg-Eppendorf246
进行中（未招募）
1 期
ot Available.EUCTR2015-005576-22-ITBAXALTA INNOVATIONS GMBH10
进行中（未招募）
不适用
An open label, multicenter, pilot phase II study of SOM230 s.c. in patients with duodeno-pancreatic (neuro) endocrine tumors and different pituitary diseases (Nelson?s syndrome, non-functioning adenoma, TSH-adenoma, Gonadotroph adenoma, and PRL-adenoma) with potential sensitivity to somatostatin analogues. - NDduodeno-pancreatic (neuro) endocrine tumors and different pituitary diseases (Nelson?s syndrome, non-functioning adenoma, TSH-adenoma,Gonadotroph adenoma, and PRL-adenoma)MedDRA version: 6.1Level: HLTClassification code 10035098EUCTR2006-002877-30-ITOVARTIS FARMA70