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

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ClinicalTrials.gov Identifier: NCT02565576
Recruitment Status : Completed
First Posted : October 1, 2015
Last Update Posted : March 13, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

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).

Condition or disease Intervention/treatment Phase
Myasthenia Gravis, Generalized Drug: Placebo Drug: CFZ533 Phase 2

Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 44 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: A Multi-center, Randomized, Double-blind, Placebo-controlled, Parallel Group Study to Preliminarily Evaluate the Safety, Tolerability, Pharmacokinetics and Efficacy of CFZ533 in Patients With Moderate to Severe Myasthenia Gravis
Actual Study Start Date : August 10, 2015
Actual Primary Completion Date : July 31, 2017
Actual Study Completion Date : December 19, 2017

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Active Comparator: CFZ533
CFZ533
Drug: Placebo
Drug: CFZ533
Placebo Comparator: Placebo
Placebo
Drug: Placebo



Primary Outcome Measures :
  1. Mean change from baseline in the Quantitative Myastenia Gravis (QMG) score [ Time Frame: At week 25 ]

Secondary Outcome Measures :
  1. Mean changes from baseline in the MGC score [ Time Frame: From baseline to week 49 ]
  2. Proportion of patients with improvement or worsening by ≥ 3 points in the QMG score [ Time Frame: From baseline to week 49 ]
  3. Proportion of patients intolerant to steroid taper [ Time Frame: from week 13 to week 49 ]
  4. Proportion of patients who discontinued due to inefficacy or worsening [ Time Frame: from baseline to week 49 ]
  5. Mean change from baseline in the Myasthenia Gravis-specific Activities of Daily Living scale (MG-ADL) [ Time Frame: From baseline to week 24 ]
  6. Mean changes from baseline in the QMG score [ Time Frame: From baseline to week 49 ]
  7. Mean change from baseline in the Myasthenia Gravis Quality of Life (MG QOL-15) [ Time Frame: From baseline to week 24 ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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).

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): NCT02565576


Locations
Canada, Quebec
Novartis Investigative Site
Montreal, Quebec, Canada, H3A 2BA
Novartis Investigative Site
Québec, Quebec, Canada, G1J 1Z4
Denmark
Novartis Investigative Site
Aarhus, Denmark, 8000 C
Novartis Investigative Site
Copenhagen, Denmark, 2100
Germany
Novartis Investigative Site
Berlin, Germany, 10098
Novartis Investigative Site
Halle, Germany, 06120
Novartis Investigative Site
Muenchen, Germany, 81377
Russian Federation
Novartis Investigative Site
Samara, Samara Region, Russian Federation, 443095
Novartis Investigative Site
Barnaul, Russian Federation, 656024
Novartis Investigative Site
Kazan, Russian Federation, 420021
Novartis Investigative Site
Novosibirsk, Russian Federation, 630087
Novartis Investigative Site
S-Petersburg, Russian Federation, 194354
Taiwan
Novartis Investigative Site
Tainan, Taiwan ROC, Taiwan, 70421
Novartis Investigative Site
Taipei, Taiwan, 10002
Novartis Investigative Site
Taipei, Taiwan
Sponsors and Collaborators
Novartis Pharmaceuticals

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02565576     History of Changes
Other Study ID Numbers: CCFZ533X2204
First Posted: October 1, 2015    Key Record Dates
Last Update Posted: March 13, 2018
Last Verified: March 2018

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
CFZ533, Myasthenia Gravis

Additional relevant MeSH terms:
Myasthenia Gravis
Muscle Weakness
Muscular Diseases
Musculoskeletal Diseases
Neuromuscular Manifestations
Neurologic Manifestations
Nervous System Diseases
Pathologic Processes
Signs and Symptoms
Autoimmune Diseases of the Nervous System
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases