Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2016 by University of Kansas Medical Center
CSL Behring
Information provided by (Responsible Party):
Mazen Dimachkie, MD, University of Kansas Medical Center Research Institute Identifier:
First received: March 27, 2014
Last updated: April 20, 2016
Last verified: April 2016
The purpose of this study is to determine whether Hizentra is a safe and effective treatment for people with myasthenia gravis (MG).

Condition Intervention Phase
Myasthenia Gravis
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

Resource links provided by NLM:

Further study details as provided by University of Kansas Medical Center:

Primary Outcome Measures:
  • Change in MG severity [ Time Frame: Change from Baseline to Week 12 ] [ Designated as safety issue: Yes ]
    Change will be measured using the Quantitative Myasthenia Gravis (QMG) Score for Disease severity. The QMG is a validated clinical composite scale.

Secondary Outcome Measures:
  • Efficacy of SCIg in MG [ Time Frame: Change from Baseline to Week 12 ] [ Designated as safety issue: No ]
    Composite measure of scores from measurement scales including Myasthenia Gravis-specific Activities of Daily Living scale (MG-ADL), MG Quality of Life (QOL)-15, MG composite score, and Treatment Satisfaction Questionnaire for Medication (TSQM)

  • Safety and tolerability of SCIg in people with MG [ Time Frame: Change from Week -12 to Week 12 ] [ Designated as safety issue: No ]
    Measure safety profile between intravenous and subcutaneous treatment phases as measured by changes in routine safety laboratory tests. Lab tests used to determine outcome include CBC, differential and comprehensive chemistry profile.

  • Number of Participants with Serious and Non-Serious Adverse Events [ Time Frame: Up to 16 Weeks ] [ Designated as safety issue: Yes ]
    Safety of SCIg in MG patients measured according to adverse event experienced by trial participants

  • Effect of SCIg on IgG antibody levels [ Time Frame: Change from Week -12 to Week 12 ] [ Designated as safety issue: No ]
    Measure IgG level between intravenous and subcutaneous study phases

Estimated Enrollment: 25
Study Start Date: January 2015
Estimated Study Completion Date: May 2017
Estimated Primary Completion Date: May 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Hizentra
Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). Participants will receive weekly Hizentra. Dose and rate depend on the visit and how each participant tolerates the drug. Max flow rate not to exceed 100 mL per hour.
Other Name: Immune Globulin Subcutaneous (Human), 20% Liquid

Detailed Description:

Myasthenia gravis (MG) is a rare autoimmune disorder which causes the muscles to become weak because the immune system attacks the connection between the nerves and the muscles.

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). An immunoglobin is a blood protein. Hizentra is being studied for the treatment of patients with MG. Hizentra is administered by an injection into the skin through a portable infusion pump, which may be easier for patients to administer than the current treatments.

Participants will be asked to complete 9 clinic visits and 3 telephone calls. It could take up to 30 weeks to complete all study visits.


Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Must have MGFA MG grades 2, 3, or 4 generalized MG, according to the MGFA classification system
  • Elevated AChR or MuSK Ab
  • Patient's signs and symptoms should not be better explained by another disease process
  • IVIg maintenance dose of 0.2 to 2 gm/kg/4 weeks or equivalent dose administered every 2-4 weeks
  • Stable IVIg for at least 3 cycles
  • Able to complete the study and return for follow-up visits
  • Able to give written informed consent before participating in the study

Exclusion Criteria:

  • History of chronic degenerative, psychiatric, or neurologic disorder other than MG that can produce weakness or fatigue
  • Other major chronic or debilitating illnesses within six months prior to study entry
  • Female patients who are premenopausal and are (a) pregnant, (b) breastfeeding, or (c) not using an effective method of double barrier birth control
  • Altered levels of consciousness, dementia, or abnormal mental status
  • Thymectomy in the previous three months
  • History of renal insufficiency or liver disease
  • Skin disease that would interfere with assessment of injection site reaction
  • History of severe reactions to IVIg or SCIg
  • Participation in a research study within the last 3 months
  • Treatment with rituximab or other biologics within 12 months of study entry
  • Unable to provide informed consent
  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 identifier: NCT02100969

Contact: Gabrielle Rico (913) 588-5703

United States, Arizona
Phoenix Neurological Associates Recruiting
Phoenix, Arizona, United States, 85018
Contact: Lynette McKinney    602-358-2271      
Principal Investigator: Todd Levine, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, Texas
University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States
Contact: Nina Gorham   
Principal Investigator: Jaya Trivedi, MD         
Canada, Ontario
University of Toronto Recruiting
Toronto, Ontario, Canada
Contact: Eduardo Ng   
Principal Investigator: Veria Bril, MD         
Sponsors and Collaborators
Mazen Dimachkie, MD
CSL Behring
Principal Investigator: Mazen M Dimachkie, MD University of Kansas Medical Center
  More Information

Responsible Party: Mazen Dimachkie, MD, Professor, University of Kansas Medical Center Research Institute Identifier: NCT02100969     History of Changes
Other Study ID Numbers: STUDY00001041 
Study First Received: March 27, 2014
Last Updated: April 20, 2016
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by University of Kansas Medical Center:
autoimmune neuromuscular disorder

Additional relevant MeSH terms:
Myasthenia Gravis
Autoimmune Diseases
Autoimmune Diseases of the Nervous System
Immune System Diseases
Nervous System Diseases
Neuromuscular Diseases
Neuromuscular Junction Diseases
Immunologic Factors
Physiological Effects of Drugs processed this record on May 26, 2016