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Study of Safety and Efficacy of CFZ533 in Type 1 Diabetes Pediatric and Young Adult Subjects (CCFZ533X2207)

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ClinicalTrials.gov Identifier: NCT04129528
Recruitment Status : Recruiting
First Posted : October 16, 2019
Last Update Posted : August 17, 2020
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Brief Summary:
The study is a Phase 2, multicounty, multicenter, non-confirmatory, investigator- and subject masked, randomized, placebo-controlled study to evaluate the safety, tolerability, pharmacokinetics, and efficacy of CFZ533 on preservation of residual pancreatic β-cell function in new onset T1DM in pediatric and young adult subjects.

Condition or disease Intervention/treatment Phase
Type 1 Diabetes Mellitus Drug: CFZ533 Other: Placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 102 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Investigator- and Subject-blinded, Randomized, Placebo-controlled Study to Evaluate Safety, Tolerability, Pharmacokinetics and Efficacy Trial of CFZ533 in Pediatric and Young Adult Subjects With New Onset Type 1 Diabetes (T1DM)
Actual Study Start Date : November 8, 2019
Estimated Primary Completion Date : October 3, 2022
Estimated Study Completion Date : October 4, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Diabetes Type 1

Arm Intervention/treatment
Active Comparator: CFZ533
Randomized in a 2:1 ratio: 2 Active / 1 Placebo
Drug: CFZ533
First dose is administered via intravenous infusion, subsequent doses are administered subcutaneously.
Other Name: Iscalimab

Placebo Comparator: Placebo
Similar in appearance to active study drug
Other: Placebo
Placebo for active drug




Primary Outcome Measures :
  1. Proportion of subjects with adverse events (AE)/serious adverse events (SAE) in treatment groups. [ Time Frame: at 16 months ]
    To evaluate safety and tolerability of CFZ533 in new onset T1DM.

  2. Stimulated C-peptide AUC by mixed meal tolerance test (MMTT). [ Time Frame: at 12 months ]
    To evaluate the treatment effect of CFZ533 on pancreatic beta cell function.


Secondary Outcome Measures :
  1. Free CFZ533 plasma concentration. [ Time Frame: at day 1 ]
    To evaluate the pharmacokinetics (PK) of CFZ533.

  2. Free CFZ533 plasma concentration. [ Time Frame: at 1 week ]
    To evaluate the pharmacokinetics (PK) of CFZ533.

  3. Free CFZ533 plasma concentration. [ Time Frame: at 12 months ]
    To evaluate the pharmacokinetics (PK) of CFZ533.

  4. Proportion of subjects with full or partial remission. [ Time Frame: at 12 months ]
    To evaluate the treatment effect of CFZ533 on full or partial remission.

  5. Stimulated C-peptide AUC by MMTT. [ Time Frame: at 3 years ]
    To evaluate durability of effects of CFZ533 on pancreatic beta cell function.



Information from the National Library of Medicine

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Ages Eligible for Study:   6 Years to 21 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

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 of 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; a PTT 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 (according to local practice/guidelines) 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 HIV 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 is permitted in persistently asymptomatic or postsymptomatic subjects, but study drug must be able to be administered within 100 days of diagnosis of T1D.
  11. Major dental work (e.g. 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.
  16. Active serious psychiatric disorders (diagnosed or treated by a psychiatrist), such as eating disorders and psychosis or history thereof.
  17. Any complicating medical issues or clinically abnormal laboratory results that may cause an increased safety risk to the subject as judged by the investigator.
  18. Ongoing, and up to 2 weeks prior to screening, use of medications that may affect glucose control (e.g, systemic steroids, thiazides, beta blockers). A short course of oral steroids <10 days if medically required is permissible with sponsor notification.
  19. History of drug abuse, nicotine or harmful alcohol use within 12 months prior to first dose, or evidence (as determined by the investigators) of such abuse at screening. For example, harmful alcohol use in adults is defined as five or more drinks per day for 5 or more days in the past 30 days. Harmful alcohol use by adolescents (age 13-18 years) is to be determined by the investigator, based on local culture and laws. Any alcohol use by children (age 6-12) is a disqualifier.
  20. Use of any prescription drugs other than those allowed by this protocol herbal supplements, prescribed medicinal use of cannabis/marijuana and/or over-the-counter (OTC) medication, dietary supplements (vitamins included) within two weeks prior to first dose. If needed, (i.e. an incidental and limited need) paracetamol/acetaminophen is acceptable, but must be documented in the Concomitant medications / Significant non-drug therapies page of the CRF.
  21. Taking medications prohibited by the protocol
  22. 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.
  23. Women of child-bearing potential, defined as all women, who are sexually active, physiologically capable of becoming pregnant (e.g. menstruating), unless they are using highly effective methods of contraception during dosing and for 14 weeks after stopping the investigational drug. Highly effective contraception methods include:

    • Total abstinence from heterosexual intercourse (when this is in line with the preferred and usual lifestyle of the subject). Periodic abstinence (e.g. calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception.
    • Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy), total hysterectomy or tubal ligation at least six weeks prior to first dose. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment.
    • Male sterilization in the sexual partner of female study participant (at least 6 months prior to screening). For female subjects on the study the vasectomized male partner should be the sole partner for that subject.
    • Use of oral (estrogen and progesterone), injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception.
    • In case of use of oral contraception, women should be stable on the same pill for a minimum of 3 months prior to first dose.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04129528


Contacts
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Contact: Novartis Pharmaceuticals +41613241111 novartis.email@novartis.com
Contact: Novartis Pharmaceuticals

Locations
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Belgium
Novartis Investigative Site Recruiting
Edegem, Antwerpen, Belgium, 2650
Novartis Investigative Site Recruiting
Jette, Brussel, Belgium, 1090
Novartis Investigative Site Recruiting
Montegnee, Belgium, 4420
Sponsors and Collaborators
Novartis Pharmaceuticals
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Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT04129528    
Other Study ID Numbers: CCFZ533X2207
2018-004553-25 ( EudraCT Number )
First Posted: October 16, 2019    Key Record Dates
Last Update Posted: August 17, 2020
Last Verified: August 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Novartis ( Novartis Pharmaceuticals ):
T1DM
Additional relevant MeSH terms:
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Diabetes Mellitus
Diabetes Mellitus, Type 1
Glucose Metabolism Disorders
Metabolic Diseases
Endocrine System Diseases
Autoimmune Diseases
Immune System Diseases