An open label study to evaluate the safety and efficacy of CFZ533 following 12 weeks treatment in patients with Graves' disease

Key Inclusion Criteria:

• Male and female patients 18 to 65 years of age included.
• Women of child-bearing potential must be willing to use highly effective methods of contraception during the study treatment epoch and for 12 weeks after the last study treatment.
• Graves' hyperthyroidism, with the following labs measured at screening:
• TSH<LLN and either FT3>ULN or FT4> ULN and
• TRAb ≥ 2.5 IU/L
• Patients must weigh at least 40 kg to participate in the study

Key Exclusion Criteria:

• History of treatment of Graves' disease with radio-iodine ablation or thyroidectomy and/or current treatment with anti-thyroid drugs (methimazole or propylthiouracil) within one week of starting the study treatment
• History of hyperthyroidism not caused by Graves' disease (e.g. toxic multinodular goiter, autonomous thyroid nodule, or acute inflammatory thyroiditis) and/or history or presence of thyroid storm (fever, profuse sweating, vomiting, diarrhea, delirium, severe weakness, seizures, markedly irregular heartbeat, yellow skin and eyes (jaundice), severe low blood pressure, and coma).
• Previous treatment with a B cell-depleting biologic agent or any other immune-modulatory biologic agent within 5 half-lives (experimental or approved).
• History of recurrent clinically significant infection or of recurrent bacterial infections with encapsulated organisms.
• History of primary or secondary immunodeficiency, including a positive HIV (ELISA and Western blot) test result.
• History or evidence of tuberculosis by either of the following tests:
• Positive PPD skin test (size of induration measured after 48-72 hours, and a positive result is defined as an induration of ≥ 5mm or according to local practice/guidelines) OR
• Positive QuantiFERON TB-Gold test
• Plans for immunization with a live vaccine within a 2-month period before enrollment or during the study period.
• Treatment with immunomodulatory drugs, such as cyclosporine A, methotrexate, and/or cyclophosphamide within 3 months from baseline. Glucocorticosteroid therapy with prednisolone up to 10 mg daily is permitted if patients are on stable dose for more than 3 months before enrollment in the study.
• Pregnant, breastfeeding females, and women of child bearing potential unless they are using highly effective contraception

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

Adverse events are collected from First Patient First Visit (FPFV) until Last Patient Last Visit (LPLV). All adverse events reported in this record are from date of First Patient First Treatment until Last Patient Last Visit up to 36 weeks Day 253.