Therapeutic Plasma Exchange in MG (TPE)

This study is currently recruiting participants. (see Contacts and Locations)
Verified April 2014 by Duke University
Sponsor:
Collaborator:
UCB, Inc.
Information provided by (Responsible Party):
Duke University
ClinicalTrials.gov Identifier:
NCT01927692
First received: August 20, 2013
Last updated: April 23, 2014
Last verified: April 2014
  Purpose

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).


Condition Intervention
Myasthenia Gravis
Other: Skin biopsy
Other: Single Fiber Electromyography

Study Type: Observational
Study Design: Observational Model: Case-Only
Time Perspective: Prospective
Official Title: Exploratory Study Of Immunological Profiles In Myasthenia Gravis Subjects That Receive Therapeutic Plasma Exchange

Resource links provided by NLM:


Further study details as provided by Duke University:

Primary Outcome Measures:
  • Immunoglobulin levels [ Time Frame: 14 weeks ] [ Designated as safety issue: No ]
    Immunoglobulin and IgG subtype levels, AChR autoantibody levels, IgG/autoantibody ratio will be listed, summarized and plotted graphically by visit.


Secondary Outcome Measures:
  • Lymphocyte subpopulations [ Time Frame: 14 weeks ] [ Designated as safety issue: No ]
    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 [ Time Frame: 14 weeks ] [ Designated as safety issue: No ]
    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 [ Time Frame: 14 weeks ] [ Designated as safety issue: No ]
    The time course for recovery of selected protective antibody levels removed during TPE will be summarized.

  • Clinical Outcomes Assessments [ Time Frame: 14 weeks ] [ Designated as safety issue: No ]
    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.


Estimated Enrollment: 10
Study Start Date: July 2013
Estimated Study Completion Date: October 2014
Estimated Primary Completion Date: August 2014 (Final data collection date for primary outcome measure)
Groups/Cohorts Assigned Interventions
MG subjects
MG subjects will have serum acetylcholine receptor (AChR) antibodies
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.
Other: Single Fiber Electromyography
An optional SFEMG study will be performed at baseline and 2 weeks after therapeutic plasma exchange is completed.
Other Name: SFEMG

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

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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.

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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01927692

Locations
United States, North Carolina
UNC Chapel Hill Recruiting
Chapel Hill, North Carolina, United States
Contact: Manisha Chopra    919-843-7857    chopram@neurology.unc.edu   
Duke University Hospital Recruiting
Durham, North Carolina, United States, 27710
Contact: Amanda Anderson, RN    919-613-0561    ander153@mc.duke.edu   
Sponsors and Collaborators
Duke University
UCB, Inc.
Investigators
Principal Investigator: Jeffrey Guptill, MD Duke University School of Medicine
  More Information

No publications provided

Responsible Party: Duke University
ClinicalTrials.gov Identifier: NCT01927692     History of Changes
Other Study ID Numbers: Pro00043906
Study First Received: August 20, 2013
Last Updated: April 23, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

Keywords provided by Duke University:
MG

Additional relevant MeSH terms:
Myasthenia Gravis
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Neuromuscular Junction Diseases
Neuromuscular Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on July 22, 2014