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临床试验/IRCT20210413050958N5
IRCT20210413050958N5招募中2 期

The effects of N-Acetyl-L-Leucine on the improvement of symptoms in A patient with Multiple Sulfatase Deficiency

Mashhad University of Medical Sciences0 个研究点目标入组 1 人开始时间: 待定最近更新:
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适应症

试验速览

阶段
2 期
状态
招募中
入组人数
1
研究概览入排标准研究者记录与标识符相似试验

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
6 years 至 no limit（—）
性别
All

入选标准

  • •Age over 6 years
  • •Patient with a definitive diagnosis of Multiple Sulfatase Deficiency
  • •Having clinical signs
  • •If the patient is taking any medication, he/she should maintain a constant dose/not change his/her treatment during the study period.

排除标准

  • •Have not taken any forbidden drugs (including any variant of N-acetyl-DL-leucine, aminopyridines, Riluzole, gabapentin, Varenicline, Chlorzoxazone, sulfasalazine, Rosuvastatin at least 4 weeks before visit 1 and throughout the duration of the study
  • •Patient who has any of the following: Chronic diarrhea, Unexplained visual loss, Malignancies, Insulin-dependent diabetes mellitus, Known history of hypersensitivity to the N-Acetyl-Leucine (DL-, L-, D-) or derivatives, History of known hypersensitivity to excipients of Ora-Blend® (namely sucrose, sorbitol, cellulose, carboxymethylcellulose, xanthan gum, carrageenan, dimethicone, methylparaben, and potassium sorbate)
  • •Having severe vision or hearing impairment that interferes with their ability to complete study assessments
  • •Having a definite diagnosis of arthritis or other musculoskeletal disorders that affects patient's mobility and interferes with their ability to complete study assessments

研究者

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