EUCTR2018-004553-25-SI进行中（未招募）1 期
Investigator- and subject-blinded, randomized, placebo-controlled study to evaluate safety, tolerability, pharmacokinetics and efficacy of CFZ533 in pediatric and young adults with new onset type 1 diabetes mellitus (T1DM)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 入组人数
- 44
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. Written informed consent, and if needed assent from the child on the trial, must be obtained before any assessment is performed.
- •2. Males and females aged between 12 and 21 years (inclusive, and enrolled in stages) at screening.
- •3. Body weight range from 30 to 125 kg (inclusive).
- •4. Evidence of one or more type 1 diabetes autoantibody(ies) against:
- •glutamic acid decarboxylase (anti-GAD), protein tyrosine, phosphataselike protein (anti-IA-2); zinc transporter 8 (anti-ZnT8); islet cell (cytoplasmic) (anti-ICA) at screening or baseline in the central laboratory OR historical clinical record of one or more of the T1DM diabetes autoantibodies. As part of the historical record insulin
- •autoantibodies (IAA) may have been used as part of the autoantibody panel but the blood sample must have been obtained prior to or within one week of starting exogenous insulin treatment.
- •5. Able to receive first dose of study drug within 56 days of diagnosis of T1DM (which may be extended to within 100 days of diagnosis in the event a screening assessment needs to be confirmed or vaccine administered).
- •6. Peak stimulated C-peptide levels =0.2 nmol/L (0.6 ng/mL) following standard liquid mixed meal tolerance test (MMTT), to be conducted when the subject is metabolically stable, at least 2 weeks from diagnosis and within 56 days prior to randomization (or within 100 days of diagnosis in the event a screening assessment needs to be confirmed or vaccine is required).
- •7. Study participants are to complete all recommended immunizations with live, attenuated vaccine at least eight weeks prior and killed, inactivated vaccine at least two weeks prior to first dose with study drug and in accordance with local immunization guidelines. In the event a subject has not had all vaccinations recommended according to local guidance, the screening period may be extended beyond 56 days to allow these vaccinations to be administered, but first dose of study drug must be administered within 100 days of diagnosis of T1DM.
- •8. A negative pregnancy test at screening is required for all sexually mature female subjects prior to participation in the study.
- •9. Subject and/or guardian must be able to communicate well with the investigator, to understand and comply with the requirements of the study.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 44
- •F.1.2 Adults (18-64 years) yes
- •F.1.2.1 Number of subjects for this age range 9
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
排除标准
- •1. Diabetes forms other than auto immune type 1 such as maturity-onset
- •diabetes of the young (MODY), latent autoimmune diabetes of the adult
- •(LADA), acquired diabetes (secondary to medications or surgery), T2DM
- •by judgement of the investigator.
- •2. Diabetic ketoacidosis within 2 weeks of the baseline MMTT test.
- •3. Polyglandular auto immune disease, Addison's disease, pernicious
- •anemia, celiac sprue. Treated, stable Hashimoto's thyroiditis is not
- •exclusionary.
- •4. Any of the following abnormal laboratory values at screening
- •total white blood cell count (WBC) outside the range 1,500-
- •15,000/mm3(1.5-15.0 x 109/L)
- •neutrophil count(<1500/mm3)(<1.5 X 109 / L)
- •lymphocyte count<500/mm3(<0.5 X 109 / L)
- •hemoglobin (Hgb)<8.0 g/dL
- •platelets<100,000/mm3(<100 x 109/L)
- •5. History of immunodeficiency disorders, such as HyperIgM syndrome;
- •history of recurrent infections suggestive of immunodeficiency disorders.
- •6. History of or active coagulation disorder with increased
- •thromboembolic risk; aPTT and PT/ INR below lower limit of normal
- •prior to inclusion.
- •7. Tuberculosis infection assessed by positive QuantiFERON TB-Gold
- •test(QFT) at screening. Subjects with a positive QFT test may participate
- •in the study if further work up establishes conclusively that the subject
- •has no evidence of active tuberculosis. If presence of latent tuberculosis
- •is established, then anti tuberculosis treatment must have been initiated
- •and maintained according to local country guidelines
- •8. Chronic infection with Hepatitis B(HBV) or Hepatitis C(HCV). A
- •positive HBV surface antigen(HBsAg) test at screening excludes a
- •subject. Subjects with a positive HCV antibody test should have HCV RNA
- •levels measured. Subjects with positive(detectable) HCV RNA should be
- •9. Positive human immune virus test(ELISA and Western Blot) at
- •10. Evidence of EBV, CMV, HSV, and/or SARS-CoV-2 infection by viral
- •load above laboratory upper limit of normal or only positive IgM serology
- •XML File Identifier: pVDSaL1Uf7DLTzvuNO4OZ13ZNSo=
- •in the absence of positive IgG at screening. Rescreening permitted in
- •persistently asymptomatic or post-symptomatic subjects, but study drug
- •must be able to be administered within 100 days of diagnosis of T1D and
- •viral load must be negative and IgG titers positive.
- •11. Major dental work (tooth extractions or dental surgery with access
- •to dental pulp) within 8 days of first dose; febrile illness within 48 hrs of
- •first dose.
- •12. Use of other investigational drugs or use of immunosuppressive
- •agents at the time of enrollment, or within 5 half-lives of enrollment, or
- •until the expected PD effect has returned to baseline, whichever is
- •longer; or longer if required by local regulations.
- •13. History of multiple and recurring allergies or allergy to the
- •investigational compound/compound class being used in this study.
- •Multiple and recurring allergies refer to known allergies to the
- •investigational compound, to immunoglobulin based therapies, or
- •to multiple drug classes. Dust mites, hay fever, and similar
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研究者
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