Subcutaneous Ig Maintenance Therapy for Myasthenia Gravis

NCT01828294 | Terminated Phase 1 DMC

• 1 other identifier: 17005
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 2

Brief Summary

The study is being done with patients with Myasthenia Gravis (MG), age 18-80 years, positive acetylcholine receptor antibody, receiving greater than 30mg of prednisone daily. Patients may or may not be receiving anticholinesterase agents. A common treatment for patients with this disease includes the administration of intravenous immunoglobulin (IVIG), which is a plasma protein that is given to help maintain adequate antibody levels to prevent infections and decrease the symptoms of the disease of Myasthenia Gravis. This study is being done to test if giving this medication in a subcutaneous form (into the fat of the abdomen, legs and thighs) will be better tolerated for patients with Myasthenia Gravis.

Conditions

Myasthenia Gravis

Keywords

MG

Outcome Measures

Primary Outcomes (1)

• To monitor number of participants completing the study for the six months period

  To evaluate feasibility and tolerability of IGSC as a maintenance treatment of generalized MG as measured by treatment adherence and adverse events experienced by the patient. Patients will be asked if they have experienced any of the most commonly known side effects of Hizentra. A case report form detailing any adverse events will be included with every study visit and reviewed with the patient at each visit.

  Baseline to 6 months

Secondary Outcomes (7)

• To monitor number of adverse events in participants

  Baseline to 6 months

• Number of participants able to decrease prednisone dose below 30 mgs

  Baseline to 6 months

• To monitor effect on manual muscle testing

  Baseline to 6 months

• To measure changes on SF-36 quality of life measurement tool before and after completion of study

  Baseline to 6 months

• To measure and correlate levels of serum IgG with clinical response of the participants

  Baseline to 6 months

Study Arms (1)

Study Population

OTHER

Study population will include patients (18-80 years old) with non-thymomatous myasthenia gravis MGFA Class II-IV receiving a minimum of 30mg of Prednisone daily and no other immunosuppression and no more 240 mgs per day of Cholinesterase inhibitor. Patients will receive Subcutaneous immunoglobulins weekly for 6 months.

Drug: Subcutaneous immunoglobulins

Interventions

Subcutaneous immunoglobulins DRUG

Immunoglobulins used subcutaneously for maintenance of other immune mediated disorders.

Also known as: Hizentra

Study Population

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• AChR Ab positive myasthenia gravis (acetylcholine receptor antibody).
• Age 18-80 years.
• MGFA Classification II-IV (The scale used to determine the severity of symptoms of MG).
• Receiving \> or equal 30mg of Prednisone per day.
• No new MG-specific treatments in prior 3 months.
• Willingness to participate in study protocol.
• QMG \> 10 (quantitative myasthenia gravis score: the sum of grades given for symptoms of MG).
• Treatment with any immunomodulator \> than or equal to 3 months prior to trial initiation.

You may not qualify if:

• IgA deficiency (a major class of immunoglobulins found in serum and external body secretions such as saliva, tears, and sweat as well as in the gastrointestinal, respiratory, and genitourinary tracts).
• Previous thromboembolic events, including deep vein thrombosis, stroke and myocardial infarction
• MGFA Class I, IV (if patient requires hospitalization) or V
• History of thymoma
• Thymectomy in previous year or planning to undergo thymectomy in next six months
• Pregnancy or lactation; unwillingness to avoid pregnancy
• Serious concurrent medical, neurological or psychiatric condition that would interfere with IGSC administration or subsequent clinical assessments
• Unwillingness or incapacity to participate, agree to necessary follow-up visits, or give written and informed consent
• Patients who have had an anaphylactic or severe systemic reaction to the administration of human immune globulin or to components of Hizentra, such as polysorbate 80, or patients with hyperprolinemia because it contains the stabilizer L-proline
• Cholinesterase inhibitor no more than 240 mg/day
• Body weight greater than 120 kg.

Sponsors & Collaborators

• St. Louis University: lead
• CSL Behring: collaborator

Study Sites (2)

George Washington University

Washington D.C., District of Columbia, 20037, United States

Location

Saint Louis University

St Louis, Missouri, 63110, United States

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Hizentra

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Study Officials

• Ghazala Hayat, M.D.

  St. Louis University

  PRINCIPAL INVESTIGATOR

• Jafar Kafaie, M.D.

  St. Louis University

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: November 1, 2011
• First Posted: April 10, 2013
• Study Start: October 1, 2011
• Primary Completion: December 1, 2017
• Study Completion: December 1, 2017
• Last Updated: April 3, 2019

Record last verified: 2019-04

Locations

US (2)