Study of safety and efficacy of CFZ533 in type 1 diabetes pediatric and young adult subjects.

Phase 1

• Conditions: Treatment of subjects with type 1 diabetes mellitus with residual beta cell function (RBCF), with the goal of preserving RBCF.; Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]; MedDRA version: 21.1Level: PTClassification code 10067584Term: Type 1 diabetes mellitusSystem Organ Class: 10027433 - Metabolism and nutrition disorders

• Registration Number: EUCTR2018-004553-25-IT
• Lead Sponsor: OVARTIS PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-06-14
• Last Update Posted: 30 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 102

Inclusion Criteria

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 6 and 21 years (inclusive, and enrolled in stages) at screening.
3. Body weight range from 20 to 125 kg (inclusive).
4. Newly diagnosed auto immune type 1 diabetes confirmed by at least one positive auto antibody:glutamic acid decarboxylase (anti-GAD65), protein tyrosine phosphatase-like protein (anti-IA-2); zinc transporter 8 (anti-ZnT8); islet cell (cytoplasmic) (anti-ICA).
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 at least 4 weeks prior to first dose with study drug and in accordance with local immunization guidelines. Immunization with a live vaccine must be done at least 4 months prior to the first dose. 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. Must be willing to comply with the standard of care for diabetes management.
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:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

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), type 2 diabetes 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.
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 excluded.
9. Positive human immune virus test(ELISA and Western Blot) at screening.
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 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.
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.
14. History of severe hypersensitivity reaction or anaphylaxis to biological agents, e.g. human monoclonal antibody.
15. History of malignancy of any organ system(other than localized basal cell carcinoma of the skin), treated or untreated, within 5 years of screening, regardless of whether there is evidence of local recurrence or metastases.
For further exclusion criteria please refer to the protocol.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified