Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu

Therapeutic Plasma Exchange in MG (TPE)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT01927692
Recruitment Status : Completed
First Posted : August 23, 2013
Last Update Posted : July 16, 2015
Sponsor:
Collaborator:
UCB Pharma
Information provided by (Responsible Party):
Duke University

Tracking Information
First Submitted Date August 20, 2013
First Posted Date August 23, 2013
Last Update Posted Date July 16, 2015
Study Start Date July 2013
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures
 (submitted: August 20, 2013)
Immunoglobulin levels [ Time Frame: 14 weeks ]
Immunoglobulin and IgG subtype levels, AChR autoantibody levels, IgG/autoantibody ratio will be listed, summarized and plotted graphically by visit.
Original Primary Outcome Measures Same as current
Change History
Current Secondary Outcome Measures
 (submitted: August 20, 2013)
  • Lymphocyte subpopulations [ Time Frame: 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 [ Time Frame: 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 [ Time Frame: 14 weeks ]
    The time course for recovery of selected protective antibody levels removed during TPE will be summarized.
  • Clinical Outcomes Assessments [ Time Frame: 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.
Original Secondary Outcome Measures Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title Therapeutic Plasma Exchange in MG
Official Title Exploratory Study Of Immunological Profiles In Myasthenia Gravis Subjects That Receive Therapeutic Plasma Exchange
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 Type Observational
Study Design Observational Model: Case-Only
Time Perspective: Prospective
Target Follow-Up Duration Not Provided
Biospecimen Not Provided
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.
Condition 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.
  • 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
Study Groups/Cohorts MG subjects
MG subjects will have serum acetylcholine receptor (AChR) antibodies
Interventions:
  • Other: Skin biopsy
  • Other: Single Fiber Electromyography
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status Completed
Actual Enrollment
 (submitted: July 15, 2015)
11
Original Estimated Enrollment
 (submitted: August 20, 2013)
10
Actual Study Completion Date October 2014
Actual Primary Completion Date October 2014   (Final data collection date for primary outcome measure)
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
Sex/Gender
Sexes Eligible for Study: All
Ages 18 Years and older   (Adult, Older Adult)
Accepts Healthy Volunteers No
Contacts Contact information is only displayed when the study is recruiting subjects
Listed Location Countries United States
Removed Location Countries  
 
Administrative Information
NCT Number NCT01927692
Other Study ID Numbers Pro00043906
Has Data Monitoring Committee No
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement Not Provided
Responsible Party Duke University
Study Sponsor Duke University
Collaborators UCB Pharma
Investigators
Principal Investigator: Jeffrey Guptill, MD Duke University
PRS Account Duke University
Verification Date June 2015