Therapeutic Plasma Exchange in MG

Completed

• Conditions: Myasthenia Gravis
• Interventions: Other: Skin biopsy; Other: Single Fiber Electromyography

• Registration Number: NCT01927692
• Lead Sponsor: Duke University

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.

Study Timeline

• Study Start: 2013-07
• Study First Submitted: 2013-08-20
• Study First Submitted QC: 2013-08-20
• Study First Posted: 2013-08-23
• Primary Completion: 2014-10
• Study Completion: 2014-10
• Status Verified: 2015-06
• Last Update Submitted: 2015-07-15
• Last Update Posted: 2015-07-16

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 11

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

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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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
MG subjects	Single Fiber Electromyography	MG subjects will have serum acetylcholine receptor (AChR) antibodies
MG subjects	Skin biopsy	MG subjects will have serum acetylcholine receptor (AChR) antibodies

Primary Outcome Measures

Name	Time	Method
Immunoglobulin levels	14 weeks	Immunoglobulin and IgG subtype levels, AChR autoantibody levels, IgG/autoantibody ratio will be listed, summarized and plotted graphically by visit.

Secondary Outcome Measures

Name	Time	Method
Lymphocyte subpopulations	14 weeks	Peripheral blood mononuclear cells (PBMC) will be analyzed using highly standardized polychromatic flow cytometric techniques for the expression of a broad array of phenotypic markers. A B-cell and T-cell panel will be used to profile specific marker expression among PBMCs and define lymphocyte subpopulations.
SFEMG	14 weeks	The extensor digitorum communis muscle will be studied in every subject and the muscle tested must have increased jitter at the initial study. If examination of the extensor digitorum communis is not feasible or the jitter is normal at the initial study, the muscle to study will be at the discretion of the investigator performing the SFEMG test, preferably frontalis. In each SFEMG study, jitter from 20 paired muscle fiber potentials will be recorded whenever feasible. Recorded variables will include total number of muscle fiber pairs studied, abnormal muscle fiber pairs, blocking pairs, and mean consecutive difference (MCD).
Vaccination/protective antibodies	14 weeks	The time course for recovery of selected protective antibody levels removed during TPE will be summarized.
Clinical Outcomes Assessments	14 weeks	All clinical outcomes assessments data (MG-Composite, MG-ADL, MG-QOL-15, MG-MMT) will be listed and summarized by visit. Scores may be presented graphically. Immunological measurements will be correlated with clinical scores, if appropriate.

Trial Locations

Locations (2)

Duke University Hospital; 🇺🇸 Durham, North Carolina, United States

UNC Chapel Hill; 🇺🇸 Chapel Hill, North Carolina, United States