Myasthenia GravidPhase 4NCT01179893
Intravenous Immunoglobulin and Plasma Exchange in Myasthenia Gravis
A Phase 4 study testing IVIG and PLEX in people with Myasthenia Gravid, run by University Health Network, Toronto at 1 site in Canada.
Trial summary
Immunomodulation is effective in treating patients with myasthenia gravis (MG), but prior studies have not adequately defined if plasma exchange (PLEX) in superior to intravenous immunoglobulin (IVIG) in the treatment of myasthenia gravis. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravid
- What is being tested?
- IVIG and PLEX
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 87 participants
- Where
- 1 siteCanada
- Study dates
- Mar 2007to Jul 2010
- Phase
- Phase 4
- 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
Who may be able to join
- >18 years old
- diagnosis of moderate-severe MG (defined as a Quantitative Myasthenia Gravis Score QMGS >10.5)
- worsening weakness requiring a change in therapy judged by a neuromuscular expert
Who cannot join
- Worsening weakness secondary to concurrent medications (e.g. Aminoglycosides)
- Worsening weakness secondary to infection
- Change in corticosteroid dosage in the 2 weeks prior to screening
- Other disorders causing weakness or fatigue
- Known absolute IgA deficiency (risk of anaphylactic reaction to IVIG)
- History of anaphylaxis or severe systemic response to IVIG or albumin
Show all 17Show fewer
- Pregnancy or breastfeeding
- Active renal failure precluding volume of IVIG (risk of volume overload with IVIG) as judged by the investigators
- Clinically significant cardiac disease precluding IVIG volume as judged by the investigators
- Known hyperviscosity or hypercoaguable state (risk of stroke with IVIG)
- Known coagulopathy with bleeding
- On another current study medication or protocol within 4 weeks of screening
- Patients with known refractory status to either IVIG or PLEX
- Poorly controlled or severe hypertension (exacerbation by IVIG)
- Patient refuses treatment with either IVIG or PLEX
- Patient refuses follow-up with electrophysiological studies
- Patient unable or unwilling to give informed consent
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Randomized Trial of Plasma Exchange vs. IVIG in the Treatment of Myasthenia Gravis
Immunomodulation is effective in treating patients with myasthenia gravis (MG), but prior studies have not adequately defined if plasma exchange (PLEX) in superior to intravenous immunoglobulin (IVIG) in the treatment of myasthenia gravis. This study aimed to determine if PLEX was superior to IVIG in the treatment of patients with myasthenia gravis.
Patients with MG requiring immunomodulation are randomized to IVIG or PLEX and treated with a full course of immunomodulation. The quantitative myasthenia gravis score (QMGS) will be evaluated as the primary efficacy parameter at day 14 to determine if PLEX is superior to IVIG.
- Conditions
- Myasthenia Gravid
- Being tested
- IVIG, PLEX
- Keywords
- Myasthenia Gravis, IVIG, PLEX
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IVIGBiological
Intravenous immunoglobulin
PLEXProcedure or surgery
Plasma exchange: removal of pathogenic antibodies and constituents and replacement with albumin.
| Study group | Role | What they receive |
|---|---|---|
| IVIG | Active comparator | Intravenous Immunoglobulin, 2G/Kg, infused over 2 days in the Medical Day Unit of the University Health Network Receives: Biological: IVIG |
| PLEX | Experimental | Patients received one plasma volume plasma exchanges with 5% albumin replacement fluid. Five plasma exchange procedures occurred every second day with breaks over the weekend allowed. Patients treated in the apheresis units at the University Health Network. Receives: Procedure: PLEX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 14 after treatment
QMGS is a validated clinical measure of myasthenia gravis ranging from 0 points (no myasthenic weakness) to a maximum of 39 points, with a defined change of 3.4 units required for clinical significance.
Measured: QMGS at day 14, and patients followed to day 60
Secondary outcomes (9)
QMGS Score change at days 21 and 28 from start of treatment.
Change in QMGS with time to see if effect ad day 14 is sustained.
Measured: 28 days
Post intervention status
Categorical scale of improvement, worsening, or no change for myasthenia gravis.
Measured: Day 14, 21 and 28
Single fiber electromyography: jitter, percent abnormal pair, percent blocking
Electrophysiological assessment of neuromuscular transmission.
Measured: Days 14 and 28 compared to baseline
Repetitive Nerve stimulation studies
Assessment of decrement
Measured: Days 14 and 28
Acetylcholine Receptor Antibody titers
Laboratory assay of pathogenic antibody
Measured: Day 28 (if positive at baseline)
AntiMUSK antibody
Laboratory measure of pathogenic antibody
Measured: Day 28 (if positive at baseline)
Need for ICU admission, ventilation, intubation
Myasthenic deterioration and crisis
Measured: 60 days
Hospitalization
Myasthenic deterioration and crisis
Measured: 60 days
Need for additional myasthenic treatment
Myasthenic deterioration or crisis
Measured: Day 60
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Aug 2010 (estimated)
- Study startMar 2007
- Primary completionJul 2010
- Study completionJul 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myasthenia GravidStudies of the same condition, open and under way.
- More trials run by University Health Network, TorontoThe 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
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Investigator, Outcomes assessor
- Enrolment
- 87 participants (actual)
Sponsor and investigators
- Lead sponsor
- University Health Network, Toronto
- Sponsor type
- University, hospital or other
- Collaborators
- Grifols Therapeutics LLC
- Investigators
- Vera Bril, BSc, MD, FRCPC, Principal investigator, University Health Network, TorontoDavid Barth, MD, Principal investigator, University Heatlh Network
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01179893
- Sponsor's protocol ID
- 07-0280-B
- Status last verified
- Aug 2010
- First submitted
- 9 Aug 2010
- Last updated
- 11 Aug 2010
- Results posted
- No
- Data monitoring committee
- No
Publications
Zinman L, Ng E, Bril V. IV immunoglobulin in patients with myasthenia gravis: a randomized controlled trial. Neurology. 2007 Mar 13;68(11):837-41. doi: 10.1212/01.wnl.0000256698.69121.45.
PubMed 17353471
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2010. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Randomized Trial of Plasma Exchange vs. IVIG in the Treatment of Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT01179893
- Acronym / sponsor ID
- 07-0280-B
- Conditions
- Myasthenia Gravid
- Keywords
- Myasthenia Gravis, IVIG, PLEX
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 87 participants (actual)
- Lead sponsor
- University Health Network, TorontoUniversity, hospital or other
- Collaborators
- Grifols Therapeutics LLC
- Study start
- Mar 2007
- Primary completion
- Jul 2010 (actual)
- Study completion
- Jul 2010 (actual)
- First posted
- 11 Aug 2010 (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.
Immunomodulation is effective in treating patients with myasthenia gravis (MG), but prior studies have not adequately defined if plasma exchange (PLEX) in superior to intravenous immunoglobulin (IVIG) in the treatment of myasthenia gravis. This study aimed to determine if PLEX was superior to IVIG in the treatment of patients with myasthenia gravis.
Patients with MG requiring immunomodulation are randomized to IVIG or PLEX and treated with a full course of immunomodulation. The quantitative myasthenia gravis score (QMGS) will be evaluated as the primary efficacy parameter at day 14 to determine if PLEX is superior to IVIG.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IVIG | Active comparator | Biological: IVIGIntravenous Immunoglobulin, 2G/Kg, infused over 2 days in the Medical Day Unit of the University Health Network |
| PLEX | Experimental | Procedure: PLEXPatients received one plasma volume plasma exchanges with 5% albumin replacement fluid. Five plasma exchange procedures occurred every second day with breaks over the weekend allowed. Patients treated in the apheresis units at the University Health Network. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- >18 years old
- diagnosis of moderate-severe MG (defined as a Quantitative Myasthenia Gravis Score QMGS >10.5)
- worsening weakness requiring a change in therapy judged by a neuromuscular expert
Key exclusion criteria (17 listed)
- Worsening weakness secondary to concurrent medications (e.g. Aminoglycosides)
- Worsening weakness secondary to infection
- Change in corticosteroid dosage in the 2 weeks prior to screening
- Other disorders causing weakness or fatigue
- Known absolute IgA deficiency (risk of anaphylactic reaction to IVIG)
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 |
|---|---|---|
| IVIGIntravenous immunoglobulin | Biological | IVIG |
| PLEXPlasma exchange: removal of pathogenic antibodies and constituents and replacement with albumin. | Procedure or surgery | PLEX |
Primary outcome
Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 14 after treatment
Time frame: QMGS at day 14, and patients followed to day 60
QMGS is a validated clinical measure of myasthenia gravis ranging from 0 points (no myasthenic weakness) to a maximum of 39 points, with a defined change of 3.4 units required for clinical significance.
9 secondary outcomes
QMGS Score change at days 21 and 28 from start of treatment.
Time frame: 28 days
Post intervention status
Time frame: Day 14, 21 and 28
Single fiber electromyography: jitter, percent abnormal pair, percent blocking
Time frame: Days 14 and 28 compared to baseline
Repetitive Nerve stimulation studies
Time frame: Days 14 and 28
Acetylcholine Receptor Antibody titers
Time frame: Day 28 (if positive at baseline)
AntiMUSK antibody
Time frame: Day 28 (if positive at baseline)
Need for ICU admission, ventilation, intubation
Time frame: 60 days
Hospitalization
Time frame: 60 days
Need for additional myasthenic treatment
Time frame: Day 60
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 11 Aug 2010.
- Background: PubMed 17353471Zinman L, Ng E, Bril V. IV immunoglobulin in patients with myasthenia gravis: a randomized controlled trial. Neurology. 2007 Mar 13;68(11):837-41. doi: 10.1212/01.wnl.0000256698.69121.45.
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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.
Vera Bril, BSc, MD, FRCPC principal investigator
University Health Network, Toronto
David Barth, MD principal investigator
University Heatlh Network
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing IVIG and PLEX in people with Myasthenia Gravid, run by University Health Network, Toronto at 1 site in Canada.
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 3 inclusion and 17 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 11 Aug 2010. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Canada. 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.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
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?
Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 14 after treatment (time frame: QMGS at day 14, and patients followed to day 60). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01179893, which also lists 1 linked publication.
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 11 Aug 2010.