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

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified March 2014 by University of Kansas
Sponsor:
Collaborator:
CSL Behring
Information provided by (Responsible Party):
Mazen Dimachkie, MD, University of Kansas Medical Center Research Institute
ClinicalTrials.gov Identifier:
NCT02100969
First received: March 27, 2014
Last updated: March 31, 2014
Last verified: March 2014
  Purpose

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
Drug: HIZENTRA ®
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:

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: June 2014
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.
Drug: HIZENTRA ®
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.

  Eligibility

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

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 ClinicalTrials.gov identifier: NCT02100969

Contacts
Contact: Gabrielle Rico (913) 588-5703 grico@kumc.edu

Locations
United States, Kansas
University of Kansas Medical Center Not yet recruiting
Kansas City, Kansas, United States, 66160
Sponsors and Collaborators
Mazen Dimachkie, MD
CSL Behring
Investigators
Principal Investigator: Mazen M Dimachkie, MD University of Kansas
  More Information

No publications provided

Responsible Party: Mazen Dimachkie, MD, Professor, University of Kansas Medical Center Research Institute
ClinicalTrials.gov Identifier: NCT02100969     History of Changes
Other Study ID Numbers: STUDY00001041
Study First Received: March 27, 2014
Last Updated: March 31, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by University of Kansas:
MG
Hizentra
autoimmune neuromuscular disorder

Additional relevant MeSH terms:
Myasthenia Gravis
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases
Immunoglobulins
Antibodies
Immunologic Factors
Physiological Effects of Drugs
Pharmacologic Actions

ClinicalTrials.gov processed this record on October 19, 2014