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Trial record 1 of 1 for:    NCT02100969
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Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

This study is currently recruiting participants.
Verified October 2017 by Mazen Dimachkie, MD, University of Kansas Medical Center
Sponsor:
ClinicalTrials.gov Identifier:
NCT02100969
First Posted: April 1, 2014
Last Update Posted: November 1, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
CSL Behring
Information provided by (Responsible Party):
Mazen Dimachkie, MD, University of Kansas Medical Center
  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: Intervention Model: Single Group Assignment
Masking: None (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 Mazen Dimachkie, MD, University of Kansas Medical Center:

Primary Outcome Measures:
  • Change in MG severity [ Time Frame: Change from Baseline to Week 12 ]
    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 ]
    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 ]
    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 ]
    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 ]
    Measure IgG level between intravenous and subcutaneous study phases


Estimated Enrollment: 25
Actual Study Start Date: January 2015
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: December 2018 (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

Information from the National Library of Medicine

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, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   All
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
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): NCT02100969


Contacts
Contact: Kiley Sims 913-945-9922 ksims2@kumc.edu

Locations
United States, Arizona
Phoenix Neurological Associates Recruiting
Phoenix, Arizona, United States, 85018
Contact: Lynne Flynn    602-258-2863    lynneflynn@pnal.net   
Principal Investigator: Todd Levine, MD         
United States, Kansas
University of Kansas Medical Center Recruiting
Kansas City, Kansas, United States, 66160
United States, New York
University at Buffalo Recruiting
Buffalo, New York, United States, 14203
Contact: Kara Patrick    716-859-7510    kpatrick@buffalo.edu   
Contact: Connie Brand    716-859-7620    conniebr@buffalo.edu   
Principal Investigator: Nicholas Silvestri, MD         
United States, Texas
University of Texas Southwestern Medical Center Recruiting
Dallas, Texas, United States
Contact: Nina Gorham       nina.gorham@utsouthwestern.edu   
Principal Investigator: Jaya Trivedi, MD         
Canada, Ontario
University of Toronto Recruiting
Toronto, Ontario, Canada
Contact: Eduardo Ng       eduardo.ng@uhn.ca   
Principal Investigator: Veria Bril, MD         
Sponsors and Collaborators
Mazen Dimachkie, MD
CSL Behring
Investigators
Principal Investigator: Mazen M Dimachkie, MD University of Kansas Medical Center
  More Information

Responsible Party: Mazen Dimachkie, MD, Professor, University of Kansas Medical Center
ClinicalTrials.gov Identifier: NCT02100969     History of Changes
Other Study ID Numbers: STUDY00001041
First Submitted: March 27, 2014
First Posted: April 1, 2014
Last Update Posted: November 1, 2017
Last Verified: October 2017

Keywords provided by Mazen Dimachkie, MD, University of Kansas Medical Center:
MG
Hizentra
autoimmune neuromuscular disorder

Additional relevant MeSH terms:
Muscle Weakness
Myasthenia Gravis
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
Immunoglobulins
Antibodies
Immunoglobulin G
gamma-Globulins
Immunoglobulins, Intravenous
Rho(D) Immune Globulin
Immunologic Factors
Physiological Effects of Drugs