Graves' DiseasePhase 2NCT02713256
A Study to Evaluate the Safety and Effect of CFZ533 on Patients With Graves' Disease
A Phase 2 study testing CFZ533 in people with Graves' Disease, run by Novartis Pharmaceuticals at 3 sites in 2 countries.
Trial summary
An open label study to evaluate the safety and efficacy of CFZ533 following 12 weeks treatment in patients with Graves' disease Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Graves' Disease
- What is being tested?
- CFZ533
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 3 sitesin 2 countries
- Study dates
- 19 Apr 2016to 24 Apr 2017
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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:
Show all 11Show fewer
- 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
Trial locations and contacts
3 sites in 2 countries.
3 sites in 2 countries
United States2 sites
Novartis Investigative Site
Honolulu, Hawaii, 96814
Novartis Investigative Site
Rochester, Minnesota, 55905
About this study
Official title: An Open Label Study to Evaluate the Safety and Efficacy of 12 Week Treatment With CFZ533 in Patients With Graves' Disease
An open label study to evaluate the safety and efficacy of CFZ533 following 12 weeks treatment in patients with Graves' disease
- Conditions
- Graves' Disease
- Being tested
- CFZ533
- Keywords
- Graves' disease, Hyperthyroidism, CFZ533, TSH
- Medical subject headings
- Graves Disease, Hyperthyroidism
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CFZ533Drug
CFZ533 intravenously over approximately one hour
| Study group | Role | What they receive |
|---|---|---|
| CFZ533 10mg/kg | Experimental | CFZ533 intravenously over approximately one hour Receives: Drug: CFZ533 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Participants Whose Thyroid Stimulating Hormone (TSH) Levels Normalize After 12 Week Treatment
Normalization of TSH is defined as TSH level greater than 0.35 mU/L after 12 week treatment (Day 85)
Measured: 12 week (DAY 85)
Percentage of Participants Whose Total Triiodothyronine (Total T3) Levels Decrease After 12 Week Treatment
Percentage of participants whose total triiodothyronine (total T3) levels decrease after 12 week treatment. A decrease is when total T3 level is below Upper limit of normal (ULN) ≤ 2.79 nmol/L
Measured: 12 week (DAY 85)
Percentage of Participants Whose Free Thyroxine (Free T4) Levels Decrease After 12 Week Treatment
Percentage of participants whose free thyroxine (free T4) levels decrease after 12 weeks of treatment (DAY85). A decrease is when free T4 level is below Upper limit of normal (ULN) ≤ 22.7 pmol/L)
Measured: 12 week (DAY 85)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 Mar 2016 (estimated)
- Study start19 Apr 2016
- Primary completion24 Apr 2017
- Study completion24 Apr 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Graves' DiseaseStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02713256
- Sponsor's protocol ID
- CCFZ533X2205
- Other IDs
- 2015-005564-41, 2015
- Status last verified
- Mar 2019
- First submitted
- 15 Mar 2016
- Last updated
- 5 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: 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
Publications
Faustino LC, Kahaly GJ, Frommer L, Concepcion E, Stefan-Lifshitz M, Tomer Y. Precision Medicine in Graves' Disease: CD40 Gene Variants Predict Clinical Response to an Anti-CD40 Monoclonal Antibody. Front Endocrinol (Lausanne). 2021 Jun 4;12:691781. doi: 10.3389/fendo.2021.691781. eCollection 2021.
PubMed 34149627Kahaly GJ, Stan MN, Frommer L, Gergely P, Colin L, Amer A, Schuhmann I, Espie P, Rush JS, Basson C, He Y. A Novel Anti-CD40 Monoclonal Antibody, Iscalimab, for Control of Graves Hyperthyroidism-A Proof-of-Concept Trial. J Clin Endocrinol Metab. 2020 Mar 1;105(3):dgz013. doi: 10.1210/clinem/dgz013.
PubMed 31512728
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open Label Study to Evaluate the Safety and Efficacy of 12 Week Treatment With CFZ533 in Patients With Graves' Disease
- ClinicalTrials.gov ID
- NCT02713256
- Acronym / sponsor ID
- CCFZ533X2205
- Conditions
- Graves' Disease
- Keywords
- Graves' disease, Hyperthyroidism, CFZ533, TSH
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 15 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 19 Apr 2016 (actual)
- Primary completion
- 24 Apr 2017 (actual)
- Study completion
- 24 Apr 2017 (actual)
- First posted
- 18 Mar 2016 (estimated)
- Results
- Posted 21 May 2018
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
An open label study to evaluate the safety and efficacy of CFZ533 following 12 weeks treatment in patients with Graves' disease
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CFZ533 10mg/kg | Experimental | Drug: CFZ533CFZ533 intravenously over approximately one hour |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- 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
Key exclusion criteria (11 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| CFZ533CFZ533 intravenously over approximately one hour | Drug | CFZ533 10mg/kg |
Primary outcome
Percentage of Participants Whose Thyroid Stimulating Hormone (TSH) Levels Normalize After 12 Week Treatment
Time frame: 12 week (DAY 85)
Normalization of TSH is defined as TSH level greater than 0.35 mU/L after 12 week treatment (Day 85)
Percentage of Participants Whose Total Triiodothyronine (Total T3) Levels Decrease After 12 Week Treatment
Time frame: 12 week (DAY 85)
Percentage of participants whose total triiodothyronine (total T3) levels decrease after 12 week treatment. A decrease is when total T3 level is below Upper limit of normal (ULN) ≤ 2.79 nmol/L
Percentage of Participants Whose Free Thyroxine (Free T4) Levels Decrease After 12 Week Treatment
Time frame: 12 week (DAY 85)
Percentage of participants whose free thyroxine (free T4) levels decrease after 12 weeks of treatment (DAY85). A decrease is when free T4 level is below Upper limit of normal (ULN) ≤ 22.7 pmol/L)
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 34149627Faustino LC, Kahaly GJ, Frommer L, Concepcion E, Stefan-Lifshitz M, Tomer Y. Precision Medicine in Graves' Disease: CD40 Gene Variants Predict Clinical Response to an Anti-CD40 Monoclonal Antibody. Front Endocrinol (Lausanne). 2021 Jun 4;12:691781. doi: 10.3389/fendo.2021.691781. eCollection 2021.
- Derived from this study: PubMed 31512728Kahaly GJ, Stan MN, Frommer L, Gergely P, Colin L, Amer A, Schuhmann I, Espie P, Rush JS, Basson C, He Y. A Novel Anti-CD40 Monoclonal Antibody, Iscalimab, for Control of Graves Hyperthyroidism-A Proof-of-Concept Trial. J Clin Endocrinol Metab. 2020 Mar 1;105(3):dgz013. doi: 10.1210/clinem/dgz013.
- A Plain Language Trial Summary is available on novartisclinicaltrials.comLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
3 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing CFZ533 in people with Graves' Disease, run by Novartis Pharmaceuticals at 3 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 21 May 2018). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Percentage of Participants Whose Thyroid Stimulating Hormone (TSH) Levels Normalize After 12 Week Treatment (time frame: 12 week (DAY 85)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02713256, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021; AzTrials last refreshed its copy on 6 Nov 2026.