跳至主要内容
临床试验/CTRI/2018/03/012266
CTRI/2018/03/012266招募中未知

Genzyme Rare disease registries (MPS I Disease Registry Protocol).

Sanofi Synthelabo India Private Limited0 个研究点目标入组 0 人开始时间: 待定最近更新:

试验速览

阶段
未知
状态
招募中
发起方

研究概览

简要总结

暂无简介。

研究设计

研究类型
Observational

入排标准

入选标准

  • 1. All age group from new born to old age allowed
  • 2. All patients with a confirmed diagnosis of MPS I disease who are willing and able to provide written informed consent and any additional authorization documents required by local law to send health information to the Registry are eligible for inclusion, regardless of whether they are receiving disease therapy including ERT (such as laronidase) and irrespective of the commercial product with which they are being treated.
  • 3. A confirmed diagnosis of MPS I consists of documented biochemical evidence of a deficiency in α-Liduronidase enzyme activity or mutation(s) in the gene coding for α-L-iduronidase

排除标准

  • No Exclusion Criteria

研究者

发起方
Sanofi Synthelabo India Private Limited

相似试验

Disease Registry for patients with MPS I... | 临床试验