2024-518270-14-00尚未招募2 期
A double-blinded, Randomized, Parallel, Placebo-controlled trial of Wharton´s Jelly-derived Allogenic Mesenchymal Stromal Cells to treat Type I Diabetes in Children Adolescents (WJMSC-P01)
Region Uppsala2 个研究点 分布在 1 个国家目标入组 66 人开始时间: 2024年11月12日最近更新:
适应症
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 尚未招募
- 发起方
- 入组人数
- 66
- 试验地点
- 2
- 主要终点
- Safety Safety parameters will be evaluated in the intervention trial at each study visit and recorded as adverse events (AEs). Any grade 3 event (or higher) will be evaluated by DSMB. Efficacy Change in C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at 12 months following WJMSC/Placebo infusion when compared to test performed before the start of treatment (baseline).
研究概览
简要总结
To investigate the safety, tolerance and efficacy after allogeneic infusion of WJMSCs intravenously in children and adolescents recently (<6 months) diagnosed with type 1 diabetes.
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 年龄范围
- 0 years 至 17 years（0-17 Years）
- 接受健康志愿者
- 是
入选标准
- •Written informed consent for participation of the study (for subjects below 18 years of age also from both caregivers), given before undergoing any study-specific procedures
- •Clinical history compatible with type 1 diabetes diagnosed less than 6 months before enrolment
- •In the first part of the study, six subjects, three between 7-11 and three between 12-18 years of age (both groups inclusive at both ends), will be included. The sixty subjects in the second part of the study are stratified by age (12-21 and 7-11 years, respectively) and randomized to one of two treatment arms (active or placebo), with a 6-month safety delay for the younger stratum.
- •Mentally stable and, in the opinion of the investigator, able to comply with the procedures of the study protocol.
- •Fasting plasma C-peptide concentration >0.12 nmol/L.
- •Subjects of child-bearing potential must agree to using adequate contraception until one year after the administration of WJMSC/Placebo. Adequate contraception is as follows: a) oral (except low-dose gestagen (lynestrenol and noretisteron), injectable or implanted hormonal contraceptives. b) intrauterine device c) intrauterine system (for example progestin-releasing coil) d) vasectomized male (with appropriate postvasectomy documentation of the absence of sperm in the ejaculate)
排除标准
- •Subjects with bodyweight >100 kg
- •Subjects with known, or previous, malignancy.
- •Taking oral anti-diabetic therapies or any other concomitant medication which may interfere with glucose regulation other than insulin.
- •Subjects with GFR <60 ml/min/1.73 m2 body surface.
- •Subject with any condition or any circumstance that, in the opinion of the investigator, would make it unsafe to undergo treatment with MSC.
- •Known hypersensitivity against any excipients, i.e., dimethyl sulfoxide (DMSO).
- •Subjects with unstable cardiovascular status incl. NYHA class III/IV or symptoms of angina pectoris.
- •Subjects with uncontrolled hypertension (≥160/105 mmHg).
- •Subjects with active on-going infections.
- •Subjects with latent or previous as well as on-going therapy against tuberculosis, or exposed to tuberculosis or has traveled in areas with a high risk of tuberculosis or mycosis within the last 3 months.
- •Subjects with serological evidence of infection with HIV, Treponema pallidum, hepatitis B antigen (subjects with serology consistent with previous vaccination and a history of vaccination are acceptable), or hepatitis C.
- •Subjects with any systemic immune suppressive treatment
- •Subjects with a known demyelinating disease or with symptoms or physical examination findings consistent with possible demyelinating disease.
- •Pregnant or nursing (lactating) women, where pregnancy is defined as the state of a female after conception and until the termination of gestation, confirmed by a positive hCG laboratory test.
结局指标
主要结局
Safety Safety parameters will be evaluated in the intervention trial at each study visit and recorded as adverse events (AEs). Any grade 3 event (or higher) will be evaluated by DSMB. Efficacy Change in C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at 12 months following WJMSC/Placebo infusion when compared to test performed before the start of treatment (baseline).
Safety Safety parameters will be evaluated in the intervention trial at each study visit and recorded as adverse events (AEs). Any grade 3 event (or higher) will be evaluated by DSMB. Efficacy Change in C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at 12 months following WJMSC/Placebo infusion when compared to test performed before the start of treatment (baseline).
次要结局
- The proportion of study participants independent of insulin (ADA criteria) at 6 and 12 months.
- The proportion of participants with daily insulin needs <0.25U/kg at 6 and 12 months.
- Insulin requirement/kg body weight at 6 and 12 months.
- Glycosylated Hb (HbA1c) and insulin-dose adjusted HbA1c (IDAA1c) at 6 and 12 months.
- · Time-in-target (4-8 mmol/l) and Time-in-range (3.9-10 mmol/l) as measured by flash glucose monitoring for 14 days at 6 and 12 months.
- · Change in C-peptide Area Under the Curve (AUC) (0-120 min) for Mixed Meal Tolerance Test (MMTT) at 6 months following WJMSC/Placebo infusion when compared to test performed before the start of treatment (baseline).
- Change in peak C-peptide concentration during the first 6 months or the first year after treatment
研究者
Department of Endocrin and Diabetes
Scientific
Region Uppsala
研究点 (2)
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