A Double-blind, Randomized Investigator-initiated Study to Determine the Safety and the Effect of Diamyd® on the Progression to Type 1 Diabetes in Children With Multiple Islet Cell Autoantibodies
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 50
- 试验地点
- 1
- 主要终点
- Adverse Events
研究概览
简要总结
A double-blind, randomized investigator-initiated study to determine the safety and the effect of Diamyd® on the progression to type 1 diabetes in children with multiple islet cell autoantibodies
Eligible children are 4 years or older, have positive GAD-antibodies and at least one additional autoantibody and not yet diabetes.
Objectives:
DiAPREV-IT is the first prevention study with Diamyd®, where the drug is given before onset of type 1 diabetes.
The primary objective is to demonstrate that Diamyd® is safe in children at risk for type 1 diabetes.
The secondary objective is to evaluate if Diamyd® may delay or stop the autoimmune process leading to clinical type 1 diabetes in children with ongoing persistent beta-cell autoimmunity as indicated by multiple positive islet cell autoantibodies.
详细描述
A double-blind, randomized investigator-initiated study to determine the safety and the effect of Diamyd® on the progression to type 1 diabetes in children with multiple islet cell autoantibodies
Eligible children are 4 years or older, have positive GAD-antibodies and at least one additional autoantibody and not yet diabetes.
Objectives:
DiAPREV-IT is the first prevention study with Diamyd®, where the drug is given before onset of type 1 diabetes.
The primary objective is to demonstrate that Diamyd® is safe in children at risk for type 1 diabetes.
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Prevention
- 盲法
- Triple (Participant, Care Provider, Investigator)
入排标准
- 年龄范围
- 4 Years 至 18 Years（Child, Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Children from four (4) years of age and participating in DiPiS, TEDDY or Trial Net.
- •Positive GAD65Ab and at least one additional type 1 diabetes-associated autoantibody (IA-2Ab, ZnT8R/W/QAb or IAA).
- •Written informed consent from the child and the child's parents or legal acceptable representative(s) according to local regulations.
排除标准
- •Ongoing treatment with immunosuppressant therapy (topical or inhaled steroids are accepted).
- •Treatment with any oral or injected anti-diabetic medications.
- •Significantly abnormal hematology results at screening.
- •Clinically significant history of acute reaction to vaccines or other drugs.
- •Treatment with any vaccine, other than influenza, within one month prior to the first dose of the study drug or planned treatment with vaccine up to two months after the last injection with the study drug.
- •A history of epilepsy, serious head trauma or cerebrovascular accident, or clinical features of continuous motor unit activity in proximal muscles.
- •Participation in other clinical trials with a new chemical entity within the previous 3 months.
- •Significant illness other than diabetes within 2 weeks prior to first dosing.
- •Known human deficiency virus (HIV) or hepatitis.
- •Presence of associated serious disease or condition, including active skin infections that preclude subcutaneous injection, which in the opinion of the investigators makes the patient non-eligible for the study.
- •Diabetes-protective HLA-DQ6-genotype.
研究组 & 干预措施
Placebo comparator
Two doses of placebo day 1 and 30
干预措施: Placebo comparator (Other)
Alum-GAD (Diamyd)
20 microgram Diamyd day 1 and 30
干预措施: Diamyd (Drug)
结局指标
主要结局
Adverse Events
时间窗: During 5 years follow up from treatment
Adverse events, serious adverse events, hematology, chemistry, autoantibody titles by treatment group
次要结局
- AUC Glucose From OGTT Over Time(During 5 year follow-up from treatment)
- Number of Participants With Type 1 Diabetes(During 5 years follow up from treatment)
- Fasting Glucose Over Time(During 5 year follow-up from treatment)
- 120 Min C-peptide on OGTT Over Time(During 5 year follow-up from treatment)
- First-phase Insulin Response From IvGTT Over Time(During 5 year follow-up from treatment)
- Fasting C-peptide Over Time(During 5 year follow-up from treatment)
- 120 Minutes Glucose From OGTT Over Time(During 5 year follow-up from treatment)
- AUC C-peptide From OGTT Over Time(During 5 year follow-up from treatment)
- HbA1c(During 5 year follow-up)
研究者
Helena Elding Larsson
MD PhD
Lund University