NCT05196165终止不适用
Clinical Survey Study to Assess Physical Function and the Incidence of Hypoglycemia in Patients 1 Year of Age and Over With GSD III
适应症
试验速览
- 阶段
- 不适用
- 状态
- 终止
- 入组人数
- 14
- 试验地点
- 4
- 主要终点
- Number of Hypoglycemic Events During the 26-week Observation Period
研究概览
简要总结
The primary objective of this study is to evaluate the incidence of hypoglycemia in adult and pediatric participants with glycogen storage disease type III (GSD III).
研究设计
- 研究类型
- Observational
- 观察模型
- Case Only
- 时间视角
- Prospective
入排标准
- 年龄范围
- 1 Year 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Diagnosis of GSD III, confirmed by amylo-alpha-1,6-glucosidase 4-alpha-glucanotransferase (AGL) sequencing or glycogen debranching enzyme (GDE) enzymatic testing
排除标准
- •Participant is unwilling to remain blinded to continuous glucose monitor (CGM) data for the first 13 weeks of the study, or the Investigator determines that blinding would compromise subject safety
- •Presence or history of any condition that, in the view of the Investigator, would interfere with participation or pose undue risk
- •Use of any IP or investigational medical device within 30 days or 5.5 half-lives, whichever is longer, prior to screening, or during the study
- •Note: Other criteria may apply per protocol
结局指标
主要结局
Number of Hypoglycemic Events During the 26-week Observation Period
时间窗: Week 26
次要结局
- Bruininks-Oseretsky Test of Motor Proficiency (BOT-2) Strength and Agility Scores(Up to Week 26)
- Number of Times the Participant can Rise from a Seated to a Standing Position in a 30-second Period, as Measured by the Site-to-Stand (STS) Test(Up to Week 26)
- Bayley Scales of Infant and Toddler Development (Bayley-4) Fine Motor and Gross Motor Domain Scores(Up to Week 26)
- Ankle Dorsiflexion, Measured Bilaterally Using Goniometry(Up to Week 26)
- Percent Predicted Distance Walked as Measured by 6-minute Walk Test (6MWT) and 12-minute Walk Test (12MWT)(Up to Week 26)
- GNE Myopathy Functional Activities Scale (GNEM-FAS) Expanded Version Domain Scores and Total Score(Up to Week 26)
- Percent Predicted Muscle Strength as Measured by Handheld Dynamometry (HHD)(Up to Week 26)
研究者
研究点 (4)
Loading locations...
相似试验
已完成
不适用
Hypoglycemia: Physician and Patient PerspectivesType 2 Diabetes MellitusHypoglycemiaNCT01176656AstraZeneca800
已完成
4 期
Insulin Glargine During and After the Period of Fasting in RamadanDiabetes Mellitus, Type 2NCT00258804Sanofi450
撤回
不适用
Blood Sugars in Children With Idiopathic Seizures.SeizuresHypoglycemiaHyperammonemiaNCT00279851Children's Mercy Hospital Kansas City
已完成
2 期
Measurement of Glucose/Glycogen Metabolism in Humans Using Magnetic Resonance at 4 or 7 TeslaType 1 DiabetesNCT00786825University of Minnesota10
已完成
不适用
A Survey on Low Blod Sugar Among Insulin-treated Patients With DiabetesDiabetesDiabetes Mellitus, Type 1Diabetes Mellitus, Type 2NCT02306681Novo Nordisk A/S7,315