NCT03404869Unknown1 期
Study of ORL-1M (D-mannose) in Patients With CDG-Ib
适应症
干预措施
相关药物
试验速览
- 阶段
- 1 期
- 发起方
- Orpha Labs
- 入组人数
- 5
- 试验地点
- 1
- 主要终点
- Improvement in hypoglycemia, diarrhea and vomiting.
研究概览
简要总结
Study of ORL-1M in Patients With CDG-Ib
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
入排标准
- 年龄范围
- — 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of CGD-1b.
- •Less than 18 years old.
排除标准
- •Diagnosis of any other disease that is not a manifestation of CGD-1b.
研究组 & 干预措施
Treatment with ORL-1M - D-mannose
Other
干预措施: ORL-1M - D-mannose (Drug)
结局指标
主要结局
Improvement in hypoglycemia, diarrhea and vomiting.
时间窗: 6 months after treatment started
Decreased frequency of hypoglycemic episode, diarrhea and vomiting frequency.
次要结局
- Improved glycosylation pattern of serum transferrin.(30 days after treatment started)
研究者
研究点 (1)
Loading locations...
相似试验
Unknown
1 期
Study of ORL-1G (D-galactose) in Patients With Glycogen Storage Disease Type 14Glycogen Storage Disease, Type 14NCT03404856Orpha Labs5
尚未招募
不适用
A feasibility CGM trial for patients with type 1 diabetes in MalawiPACTR202102832069874Dexcom45
招募中
4 期
The effect of a GLP-1 agonist in patients with a dual diagnosis of Parkinson's Disease and Type 2 Diabetes MellitusType 2 Diabetes MellitusParkinson's DiseaseACTRN12620000627954St Vincent's Health Network12
进行中（未招募）
2 期
IDH1 (AG 120) Inhibitor in Patients With IDH1 Mutated Myelodysplastic SyndromeMyelodysplastic SyndromesAcute Myeloid LeukemiaNCT03503409Groupe Francophone des Myelodysplasies68
尚未招募
1 期
Study to Determine the Safety and Tolerability of TG-C in Subjects With Back Pain Due to Degenerative Disc DiseaseDegenerative Disc DiseaseNCT06144970Kolon TissueGene, Inc.24