Myasthenia GravisTPENCT01927692
Therapeutic Plasma Exchange in MG
An observational study of Myasthenia Gravis, run by Duke University at 2 sites in United States.
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). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis
- What is being tested?
- Skin biopsy and Single Fiber Electromyography
- How many people
- 11 participants
- Where
- 2 sitesUnited States
- Study dates
- Jul 2013to Oct 2014
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
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.
Who may be able to join
- 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
Who cannot join
- 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
Show all 11Show fewer
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
UNC Chapel Hill
Chapel Hill, North Carolina
Duke University Hospital
Durham, North Carolina, 27710
About this study
Official title: Exploratory Study Of Immunological Profiles In Myasthenia Gravis Subjects That Receive Therapeutic Plasma Exchange
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).
Read the 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.
- Conditions
- Myasthenia Gravis
- Being tested
- Skin biopsy, Single Fiber Electromyography
- Keywords
- MG
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Skin biopsyOther
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 ElectromyographyOther
Also called SFEMG
An optional SFEMG study will be performed at baseline and 2 weeks after therapeutic plasma exchange is completed.
| Study group | Role | What they receive |
|---|---|---|
| MG subjects | MG subjects will have serum acetylcholine receptor (AChR) antibodies Receives: Other: Skin biopsy; Other: Single Fiber Electromyography |
What is being measured
The primary outcome is the main question the study is designed to answer.
Immunoglobulin levels
Immunoglobulin and IgG subtype levels, AChR autoantibody levels, IgG/autoantibody ratio will be listed, summarized and plotted graphically by visit.
Measured: 14 weeks
Secondary outcomes (4)
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.
Measured: 14 weeks
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).
Measured: 14 weeks
Vaccination/protective antibodies
The time course for recovery of selected protective antibody levels removed during TPE will be summarized.
Measured: 14 weeks
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.
Measured: 14 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Aug 2013 (estimated)
- Study startJul 2013
- Primary completionOct 2014
- Study completionOct 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Myasthenia GravisStudies of the same condition, open and under way.
- More trials run by Duke UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 11 participants (actual)
Sponsor and investigators
- Lead sponsor
- Duke University
- Sponsor type
- University, hospital or other
- Collaborators
- UCB Pharma
- Responsible party
- Sponsor
- Investigators
- Jeffrey Guptill, MD, Principal investigator, Duke University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01927692
- Sponsor's protocol ID
- Pro00043906
- Acronym
- TPE
- Status last verified
- Jun 2015
- First submitted
- 20 Aug 2013
- Last updated
- 16 Jul 2015
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2015. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Exploratory Study Of Immunological Profiles In Myasthenia Gravis Subjects That Receive Therapeutic Plasma Exchange
- ClinicalTrials.gov ID
- NCT01927692
- Acronym / sponsor ID
- TPE / Pro00043906
- Conditions
- Myasthenia Gravis
- Keywords
- MG
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 11 participants (actual)
- Lead sponsor
- Duke UniversityUniversity, hospital or other
- Collaborators
- UCB Pharma
- Study start
- Jul 2013
- Primary completion
- Oct 2014 (actual)
- Study completion
- Oct 2014 (actual)
- First posted
- 23 Aug 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MG subjects | – | Other: Skin biopsy, Other: Single Fiber ElectromyographyMG subjects will have serum acetylcholine receptor (AChR) antibodies |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- 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
Key exclusion criteria (11 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Skin biopsyAn optional skin biopsy will be performed in up to 5 subjects at baseline and 2 weeks after therapeutic plasma exchange is completed. | Other | MG subjects |
| Single Fiber ElectromyographyAlso: SFEMGAn optional SFEMG study will be performed at baseline and 2 weeks after therapeutic plasma exchange is completed. | Other | MG subjects |
Primary outcome
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.
4 secondary outcomes
Lymphocyte subpopulations
Time frame: 14 weeks
SFEMG
Time frame: 14 weeks
Vaccination/protective antibodies
Time frame: 14 weeks
Clinical Outcomes Assessments
Time frame: 14 weeks
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2015.
Related trials for Myasthenia Gravis
RecruitingPhase 3
A Phase 3 Study to Evaluate Claseprubart in Adults With Generalized Myasthenia Gravis (EMERGE)
Myasthenia Gravis, Generalized
Active, not recruitingPhase 2
A Phase 2 Study to Evaluate Povetacicept in Adults With Generalized Myasthenia Gravis
Myasthenia Gravis, Generalized
RecruitingPhase 2
A Study to Evaluate the Efficacy and Safety of Maintenance Ublituximab Following Induction With Efgartigimod Administration in Participants With Myasthenia Gravis (MG)
Myasthenia Gravis
Active, not recruitingPhase 4
Phase 4 Study Evaluating Efficacy and Safety of Rozanolixizumab in Adult Chinese Participants With Generalized Myasthenia Gravis
Myasthenia Gravis
Active, not recruitingPhase 2/3
Evaluating the Pharmacokinetics, Pharmacodynamics, and Safety of Efgartigimod Administered Intravenously in Children With Generalized Myasthenia Gravis
Generalized Myasthenia Gravis
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Guptill, MD principal investigator
Duke University
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Myasthenia Gravis, run by Duke University at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2015. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Immunoglobulin levels (time frame: 14 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2015.