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Trial record 1 of 1 for:    myastenia gravis
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Safety,Tolerability,Pharmacokinetics and Efficacy of CFZ533 in Moderate to Severe Myasthenia Gravis

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2017 by Novartis ( Novartis Pharmaceuticals )
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT02565576
First received: June 23, 2015
Last updated: April 10, 2017
Last verified: April 2017
  Purpose
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 Intervention Phase
Myasthenia Gravis, Generalized
Drug: Placebo
Drug: CFZ533
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: 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

Resource links provided by NLM:


Further study details as provided by Novartis ( Novartis Pharmaceuticals ):

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

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

Estimated Enrollment: 44
Actual Study Start Date: September 29, 2015
Estimated Study Completion Date: December 27, 2017
Estimated Primary Completion Date: July 31, 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: CFZ533
CFZ533
Drug: Placebo Drug: CFZ533
Placebo Comparator: Placebo
Placebo
Drug: Placebo

  Eligibility

Ages Eligible for Study:   18 Years to 85 Years   (Adult, Senior)
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).
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02565576

Contacts
Contact: Novartis Pharmaceuticals +41613241111
Contact: Novartis Pharmaceuticals

Locations
Canada, Ontario
Novartis Investigative Site Recruiting
Toronto, Ontario, Canada, M5G 2C4
Canada, Quebec
Novartis Investigative Site Active, not recruiting
Montreal, Quebec, Canada, H3A 2BA
Novartis Investigative Site Recruiting
Québec, Quebec, Canada, G1J 1Z4
Denmark
Novartis Investigative Site Recruiting
Aarhus, Denmark, 8000 C
Novartis Investigative Site Recruiting
Copenhagen, Denmark, 2100
Germany
Novartis Investigative Site Active, not recruiting
Berlin, Germany, 10098
Novartis Investigative Site Completed
Halle/S., Germany, 06120
Novartis Investigative Site Active, not recruiting
Muenchen, Germany, 81377
Novartis Investigative Site Withdrawn
Regensburg, Germany, 93053
Russian Federation
Novartis Investigative Site Active, not recruiting
Barnaul, Russian Federation, 656024
Novartis Investigative Site Active, not recruiting
Kazan, Russian Federation, 420021
Novartis Investigative Site Active, not recruiting
Novosibirsk, Russian Federation, 630087
Novartis Investigative Site Active, not recruiting
S-Petersburg, Russian Federation, 194354
Novartis Investigative Site Active, not recruiting
Samara, Russian Federation, 443095
Taiwan
Novartis Investigative Site Completed
Tainan, Taiwan ROC, Taiwan, 70421
Novartis Investigative Site Active, not recruiting
Taipei, Taiwan, 10002
Novartis Investigative Site Active, not recruiting
Taipei, Taiwan
Sponsors and Collaborators
Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02565576     History of Changes
Other Study ID Numbers: CCFZ533X2204
Study First Received: June 23, 2015
Last Updated: April 10, 2017

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

ClinicalTrials.gov processed this record on May 25, 2017