Safety,Tolerability,Pharmacokinetics and Efficacy of CFZ533 in Moderate to Severe Myasthenia Gravis

NCT ID: NCT02565576

Last Updated: 2021-01-05

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 44 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-09-29
• Study Completion Date: 2017-12-19

Brief Summary

The purpose of this study is to evaluate safety, tolerability, pharmacokinetics/pharmacodynamics and efficacy of CFZ533 as an add-on therapy to standard of care in patients with moderate to severe myasthenia gravis (MG).

Conditions

Myasthenia Gravis, Generalized

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: DOUBLE

Study Groups

CFZ533

CFZ533

Group Type: ACTIVE_COMPARATOR

Placebo

Intervention Type: DRUG

CFZ533

Intervention Type: DRUG

Placebo

Placebo

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Interventions

Placebo

Intervention Type: DRUG

CFZ533

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Diagnosis of MG class IIa to IVa inclusive (Myasthenia Gravis Foundation of America Clinical Classification).

2. Quantitative Myasthenia Gravis (QMG) score of 10 or greater. If the QMG score is < 15 no more than 4 points may be derived from items 1 or 2 (ocular motility disturbance and ptosis).

3. Documented history of acetylcholine receptor (AChR) or Muscle Specific Kinase (MuSK) antibody positive.

4. Only one immunosuppressant or immunomodulatory drug at a stable dose is allowed during the study (i) azathioprine and mycophenolate mofetil must be stable for at least 4 months prior to randomization (ii) cyclosporine must be stable for at least 3 months prior to randomization.

5. If the patient is on oral corticosteroids, methotrexate or tacrolimus at screening, the dose must be stable for at least 1 month prior to randomization.

6. If the patient is on cholinesterase inhibitors at screening, the dose must be stable for at least 2 weeks prior to randomization.

7. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, may be included in the study if they are using highly effective methods of contraception during the study and for 12 weeks after study treatment.

Exclusion Criteria

1. MGFA grade I, IVb, or V disease.

2. Documented presence of unresected thymoma.

3. Patients having undergone thymectomy or thymo thymectomy (resection of thymoma) within 6 months of screening.

4. Patients having received any of the following treatments prior to randomization:

1. IVIg or plasma exchange within 8 weeks;

2. oral or IV cyclosphosphamide treatment within 3 months;

3. IV corticosteroid bolus (dose higher than 1 mg/kg) within 3 months;

4. belimumab within 6 months. For patients who received belimumab earlier, B cell count should be within normal range;

5. rituximab within 12 months. For patients who received rituximab earlier, B cell count should be within normal range;

6. any other biologic or an investigational drug within 1 month or five times thehalf-life, whichever is longer.

7. Live vaccines within 4 weeks of study drug infusion.

5. Patients who are at significant risk for TE as judged by the investigator or have any one of the following:

1. History of either thrombosis or 3 or more spontaneous abortions with or without the presence of anti-cardiolipin autoantibodies;

2. Presence of prolonged partial thromboplastin time (PTT).

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Novartis Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Novartis Investigative Site

Montreal, Quebec, Canada

Novartis Investigative Site

Québec, Quebec, Canada

Novartis Investigative Site

Aarhus, , Denmark

Novartis Investigative Site

Copenhagen, , Denmark

Novartis Investigative Site

Berlin, , Germany

Novartis Investigative Site

Halle/S, , Germany

Novartis Investigative Site

München, , Germany

Novartis Investigative Site

Samara, Samara Oblast, Russia

Novartis Investigative Site

Barnaul, , Russia

Novartis Investigative Site

Kazan', , Russia

Novartis Investigative Site

Novosibirsk, , Russia

Novartis Investigative Site

S-Petersburg, , Russia

Novartis Investigative Site

Tainan City, Taiwan ROC, Taiwan

Novartis Investigative Site

Taipei, , Taiwan

Novartis Investigative Site

Taipei, , Taiwan

Countries

Canada; Denmark; Germany; Russia; Taiwan

Provided Documents

Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Related Links

https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=415

A Plain Language Trial Summary is available on novartisclinicatrials.com

Other Identifiers

CCFZ533X2204

Identifier Type: -

Identifier Source: org_study_id