Therapeutic Plasma Exchange in MG

NCT ID: NCT01927692

Last Updated: 2015-07-16

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

11 participants

Study Classification

OBSERVATIONAL

Study Start Date

2013-07-31

Study Completion Date

2014-10-31

Brief Summary

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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

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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.

Conditions

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Myasthenia Gravis

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

PROSPECTIVE

Study Groups

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MG subjects

MG subjects will have serum acetylcholine receptor (AChR) antibodies

Skin biopsy

Intervention Type OTHER

An optional skin biopsy will be performed in up to 5 subjects at baseline and 2 weeks after therapeutic plasma exchange is completed.

Single Fiber Electromyography

Intervention Type OTHER

An optional SFEMG study will be performed at baseline and 2 weeks after therapeutic plasma exchange is completed.

Interventions

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Skin biopsy

An optional skin biopsy will be performed in up to 5 subjects at baseline and 2 weeks after therapeutic plasma exchange is completed.

Intervention Type OTHER

Single Fiber Electromyography

An optional SFEMG study will be performed at baseline and 2 weeks after therapeutic plasma exchange is completed.

Intervention Type OTHER

Other Intervention Names

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SFEMG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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UCB Pharma

INDUSTRY

Sponsor Role collaborator

Duke University

OTHER

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Jeffrey Guptill, MD

Role: PRINCIPAL_INVESTIGATOR

Duke University

Locations

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UNC Chapel Hill

Chapel Hill, North Carolina, United States

Site Status

Duke University Hospital

Durham, North Carolina, United States

Site Status

Countries

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United States

Other Identifiers

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Pro00043906

Identifier Type: -

Identifier Source: org_study_id

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