Clinical trial NCT01927692: Therapeutic Plasma Exchange in MG
Official title: Exploratory Study Of Immunological Profiles In Myasthenia Gravis Subjects That Receive Therapeutic Plasma Exchange
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Myasthenia Gravis
Keywords: MG
Lead sponsor: Duke University
Sponsor class: OTHER
Collaborators: name: UCB Pharma
First posted: 2013-08-23
Enrollment: 11
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Potentially eligible subjects with MG that will receive TPE as part of their clinical care will be identified from clinic encounters, emergency department visits, and hospitalizations.

Brief summary: The primary objective of the study is to longitudinally profile immunoglobulin levels and autoantibody levels in subjects with myasthenia gravis (MG) who receive therapeutic plasma exchange (TPE).

Detailed description: This is a prospective, multi-center, pilot biomarker study in subjects receiving TPE for the treatment of MG. No study medications will be given.

Ten (10) AChR antibody positive MG subjects will be enrolled in the study at 2 sites. Of these 10 MG subjects, up to 5 may be receiving chronic TPE.

The study period will be approximately 3 months and will consist of:

* Screening/baseline visit,
* TPE visit where subjects will undergo clinical evaluations and blood draws for immunological assays,
* End of TPE visit where information on the TPE procedure will be recorded, clinical measurements will be performed, and a blood sample will be drawn.
* Post-TPE period where subjects will undergo clinical evaluations and blood draws for immunological assays at week 1, week 2, week 3, week 6, and week 12 after TPE.

Study procedures performed outside of usual care will include optional single-fiber electromyography (SFEMG) studies, blood draws and optional skin biopsies.

Eligibility criteria: Inclusion Criteria:

* has the capacity to understand and sign an informed consent form
* 18 years or older
* diagnosis of MG based on clinical features
* has detectable serum autoantibodies to AChR
* has a clinical indication for the use of TPE to treat MG

Exclusion Criteria:

* unable or unwilling to comply with study procedures that include multiple venipunctures
* weighs less than 50Kg
* has a contraindication to treatment with TPE (e.g. clinically significant bleeding disorder)
* has muscle specific tyrosine kinase or low-density lipoprotein receptor-related protein 4 (LRP4) antibody positive MG
* has prior or current history of thymoma
* had a thymectomy in the past 6 months
* has received rituximab in the past 12 months
* has another coexisting autoimmune disease that is not clinically controlled or may preclude accurate study assessments according to the judgment of the PI
* has current signs or symptoms of severe, progressive, or uncontrolled renal, hepatic, hematological, gastrointestinal, endocrine, pulmonary, cardiac, or central nervous system disease
* has participated in an interventional clinical trial with a novel therapeutic agent in the past 6 months
* is cognitively impaired, a prisoner, or otherwise institutionalized at the time of screening

Primary outcomes:
measure: Immunoglobulin levels
timeFrame: 14 weeks
Secondary outcomes:
measure: Lymphocyte subpopulations
timeFrame: 14 weeks
measure: SFEMG
timeFrame: 14 weeks
measure: Vaccination/protective antibodies
timeFrame: 14 weeks
measure: Clinical Outcomes Assessments
timeFrame: 14 weeks
Other outcomes:
Locations:
city: Chapel Hill
country: United States
facility: UNC Chapel Hill
state: North Carolina
city: Durham
country: United States
facility: Duke University Hospital
state: North Carolina
Study officials:
affiliation: Duke University
name: Jeffrey Guptill, MD
role: PRINCIPAL_INVESTIGATOR
References: