Clinical trial NCT01179893: Intravenous Immunoglobulin and Plasma Exchange in Myasthenia Gravis
Official title: A Randomized Trial of Plasma Exchange vs. IVIG in the Treatment of Myasthenia Gravis
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Myasthenia Gravid
Keywords: Myasthenia Gravis
IVIG
PLEX
Lead sponsor: University Health Network, Toronto
Sponsor class: OTHER
Collaborators: name: Grifols Therapeutics LLC
First posted: 2010-08-11
Enrollment: 87
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief 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. 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.


Eligibility criteria: Inclusion Criteria:

* \>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

Exclusion Criteria:

* 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
* 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

Primary outcomes:
measure: Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 14 after treatment
timeFrame: QMGS at day 14, and patients followed to day 60
Secondary outcomes:
measure: QMGS Score change at days 21 and 28 from start of treatment.
timeFrame: 28 days
measure: Post intervention status
timeFrame: Day 14, 21 and 28
measure: Single fiber electromyography: jitter, percent abnormal pair, percent blocking
timeFrame: Days 14 and 28 compared to baseline
measure: Repetitive Nerve stimulation studies
timeFrame: Days 14 and 28
measure: Acetylcholine Receptor Antibody titers
timeFrame: Day 28 (if positive at baseline)
measure: AntiMUSK antibody
timeFrame: Day 28 (if positive at baseline)
measure: Need for ICU admission, ventilation, intubation
timeFrame: 60 days
measure: Hospitalization
timeFrame: 60 days
measure: Need for additional myasthenic treatment
timeFrame: Day 60
Other outcomes:
Locations:
city: Toronto
country: Canada
facility: University Health Network
state: Ontario
Study officials:
affiliation: University Health Network, Toronto
name: Vera Bril, BSc, MD, FRCPC
role: PRINCIPAL_INVESTIGATOR
affiliation: University Heatlh Network
name: David Barth, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.