Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy
Autologous Co-stimulation-impaired Dendritic Cells for Type 1 Diabetes Mellitus (T1DM) Therapy: A Sequential Open Label Phase IB Safety Assessment/ Randomized, Double-blind Phase IIA Efficacy Trial to Maintain and Improve Functional Beta Cell Mass in New Onset Disease T1DM Patients
Phase IB will evaluate the safety of autologous, ex vivo-engineered, co-stimulation impaired dendtritic cells to maintain and improve functional residual beta cell mass in new onset Type I Diabetes Mellitus (T1DM) patients. Efficacy measures will be collected and summarized.
Phase IIA will evaluate the safety and efficacy of 3 randomized treatment groups in new onset T1DM patients to assess if the antisense DNA-treated co-stimulation-impaired immunoregulatory dendritic cells (iDC) will safely preserve and/or increase B-cell mass resulting in improvement and/or normalization of blood glucose levels and glycated hemoglobin A1c.
研究概览
研究类型
注册 (预期的)
阶段
- 阶段2
- 阶段1
联系人和位置
学习地点
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Pennsylvania
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Pittsburgh、Pennsylvania、美国、15213
- UPMC
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参与标准
资格标准
适合学习的年龄
接受健康志愿者
有资格学习的性别
描述
Inclusion Criteria:(both open label phase IB safety and Phase IIA study):
- Patients with new onset T1DM (>18 years of age for the phase IB and then >16 (first 10 subjects), >12 years of age for the second 10 subjects, > 8 years for the next 10 subjects and, finally, >8 years of age for the remainder of the phase IIA patients) within 6 months of diabetes mellitus diagnosis.
- Evidence of decreased β-cell function as measured by C-peptide and blood glucose levels consistent with impaired glucose tolerance.
- Evidence of at least one high-risk HLA haplotype.
- Evidence of at least one diabetes-related autoantibody (e.g. IA-2, GAD, ZnT8) ,
- Adequate immune competence as assessed by immunoreactivity to alloantigens in mixed leukocyte culture and reactivity to viral antigens (CEF Pool Assay) in vitro.
- Normal hematologic, liver and kidney function.
- Female participants of childbearing potential in this study must agree to use an effective form of birth control during study participation. Reliable and effective forms of birth control include: true abstinence, intrauterine device (IUD), hormonal-based contraception, double-barrier contraception [condom or occlusive cap (diaphragm or cervical cap) with spermicide], or surgical sterilization (vasectomy for male partner, tubal ligation or hysterectomy). Sexually active male participants must agree to use an effective form of birth control such as condoms.
Exclusion Criteria:(both open label phase IB safety and Phase IIA study):
- Enrollment or history of enrollment in a drug, or biologic therapy study sponsored by TrialNet.
- A significant history or current evidence of cardiac disease, uncontrolled hypertension, serious arrhythmias.
- Evidence of active infection requiring antibiotic therapy.
- History of other concurrent significant medical diseases.
- Pregnant or lactating women.
- Patients requiring chronic systemic corticosteroids.
- Any other immune disorder including but not limited to other autoimmune diseases, HIV, HBV, HCV, HPV, HSV positivity.
- Impaired renal function with a creatinine level > 1.5.
- Administration of the following therapies while patients are undergoing treatment on this protocol: i) radiation therapy; ii) chemotherapy; iii) corticosteroids (except when administered in life-threatening circumstances); iv) other particle or cell-based therapies; v) other biologic therapies; vi) other therapies aimed at modulating the immune system; vii) other endocrine-related therapies, hormone replacement (other than thyroxine and contraceptive), glucoregulation.
- A hemaglobinopathy known to interfere with the ability to accurately determine HbA1c.
- No prior radiation therapy, immunotherapy, or chemotherapy.
学习计划
研究是如何设计的?
设计细节
- 主要用途:治疗
- 分配:随机化
- 介入模型:并行分配
- 屏蔽:三倍
武器和干预
参与者组/臂 |
干预/治疗 |
|---|---|
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实验性的:iDC recipients
iDC cells modified by in vitro engineering.
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有源比较器:Control DC recipients
DC cells which have not been modified.
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安慰剂比较:Placebo recipients
Saline injections.
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研究衡量的是什么?
主要结果指标
结果测量 |
大体时间 |
|---|---|
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The incidence of treatment-emergent adverse events.
大体时间:Month 12
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Month 12
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次要结果测量
结果测量 |
大体时间 |
|---|---|
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2-hour area under the curve (AUC) average of C-peptide at 12 months after completion of administration of assigned therapy (Protocol Month 15).
大体时间:12 Months
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12 Months
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合作者和调查者
研究记录日期
研究主要日期
学习开始
初级完成 (预期的)
研究注册日期
首次提交
首先提交符合 QC 标准的
首次发布 (估计)
研究记录更新
最后更新发布 (估计)
上次提交的符合 QC 标准的更新
最后验证
更多信息
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