Therapeutic Plasma Exchange in MG

Myasthenia Gravis Clinical Trial

— TPE  

Official title:

Exploratory Study Of Immunological Profiles In Myasthenia Gravis Subjects That Receive Therapeutic Plasma Exchange

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT01927692
• Other study ID #: Pro00043906
• Status: Completed
• Phase: N/A
• First received: August 20, 2013
• Last updated: July 15, 2015
• Start date: July 2013
• Est. completion date: October 2014

Study information

• Verified date: June 2015
• Source: Duke University
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationUnited States: Institutional Review Board
• Study type: Observational

Clinical Trial 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).

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: October 2014
• Est. primary completion date: October 2014
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Observational Model: Case-Only, Time Perspective: Prospective

Related Conditions & MeSH terms

• Myasthenia Gravis

Intervention

Other:
• 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.
• Single Fiber Electromyography
An optional SFEMG study will be performed at baseline and 2 weeks after therapeutic plasma exchange is completed.

Locations

Country	Name	City	State
United States	UNC Chapel Hill	Chapel Hill	North Carolina
United States	Duke University Hospital	Durham	North Carolina

Sponsors (2)

Lead Sponsor	Collaborator
Duke University	UCB Pharma

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Immunoglobulin levels	Immunoglobulin and IgG subtype levels, AChR autoantibody levels, IgG/autoantibody ratio will be listed, summarized and plotted graphically by visit.	14 weeks	No
Secondary	Lymphocyte subpopulations	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.	14 weeks	No
Secondary	SFEMG	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).	14 weeks	No
Secondary	Vaccination/protective antibodies	The time course for recovery of selected protective antibody levels removed during TPE will be summarized.	14 weeks	No
Secondary	Clinical Outcomes Assessments	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.	14 weeks	No