Myasthenia Gravis ExacerbationsPhase 3NCT02413580
A Study to Assess the Efficacy and Safety of IGIV-C in Patients With Myasthenia Gravis Exacerbations
A Phase 3 study testing IGIV-C in people with Myasthenia Gravis Exacerbations, run by Grifols Therapeutics LLC at 31 sites in 12 countries.
Trial summary
This was a multicenter, prospective, open-label, non-controlled study to assess the efficacy and safety of an IV dose of 2 g/kg of IGIV-C in subjects with MG exacerbations. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Myasthenia Gravis Exacerbations
- What is being tested?
- IGIV-C
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 49 participants
- Where
- 31 sitesin 12 countries
- Study dates
- Mar 2015to Apr 2018
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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
- Was male or female aged ≥18 years.
- Subjects must be willing and able to provide written informed consent (if applicable, a legally authorized representative may provide informed consent on behalf of the subject).
- Subjects who met the clinical criteria for diagnosis of MG with an exacerbation defined as worsening of MG symptoms as defined by an Myasthenia Gravis Foundation of America (MGFA) classification IVb or V.
- Subjects on long-term (8 weeks) corticosteroid treatment for MG.
- Female subjects of child-bearing potential must have a negative test for pregnancy (human chorionic gonadotropin [HCG]-based assay).
- Subjects must be willing to comply with all aspects of the clinical trial protocol, including blood sampling and long-term storage of extra samples, for the entire duration of the study.
Who cannot join
- Subjects who had received immune globulin treatment given by IV, subcutaneous or intramuscular route within the last 30 days.
- Subjects with documentation of a lack of clinical response to intravenous immunoglobulin (IVIg) therapy for MG.
- Subjects documented positive for antibodies directed against Muscle specific kinase (MuSK).
- Subjects with corticosteroid (CS) treatment initiated within the last 8 weeks or modified within the last 2 weeks.
- Subjects with plasma exchange (PLEX) within the last 30 days.
- Subjects with MG exacerbation attributable to change in medication or infection or evident infection as defined by, but not limited to, the presence of at least one of the following diagnostic features: 1) axillary temperature ≥38°C, 2) positive blood culture of infective microorganism, 3) white blood cell count >12×10^9/L and differential white blood cell count of >10% band neutrophils (>1.2×10^9/L), and 4) pulmonary infiltrate with consolidation on chest X-ray. Alternatively, other signs and symptoms may be considered for the diagnosis of evident infection according to the Investigator's judgement.
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- Subjects with inadequate venous access.
- Subjects with a history of anaphylactic reactions or severe reactions to any blood-derived product.
- Subjects with a history of intolerance to any component of the investigational products.
- Subjects with a documented diagnosis of thrombotic complications to polyclonal IVIG therapy in the past.
- Subjects with a history of recent (within the last year) myocardial infarction, stroke or uncontrolled hypertension.
- Subjects who suffered from uncontrolled congestive heart failure, embolism or documented electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation.
- Subjects with current known hyperviscosity or hypercoagulable state.
- Subjects currently receiving anti-coagulation therapy.
- Subjects with a history of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Baseline Visit.
- Subjects currently receiving, or having received within 3 months prior to the Baseline Visit, any investigational medicinal product or device.
- Subjects with a known Immunoglobulin A (IgA) deficiency and anti-IgA serum antibodies.
- Subjects with renal impairment (i.e., serum creatinine exceeds more than 1.5 times the upper limit of normal [ULN] for the expected normal range for the testing laboratory).
- Subjects with aspartate aminotransferase (AST) or alanine aminotransferase (ALT) levels exceeding more than 2.5 times the ULN for the expected normal range for the testing laboratory.
- Subjects with haemoglobin levels <9 g/dL.
Trial locations and contacts
31 sites in 12 countries.
31 sites in 12 countries
France6 sites
Hopital Neurologique Pierre Wertheimer
Bron, Lyon, 69677
Hôpital Albert Michallon
Grenoble, 38700
Hopital Roger Salengro
Lille, 59037
Hôpital de la Timone
Marseille, 13385
Hôpital Hautepierre Strasbourg
Strasbourg, 67200
CHU de Toulouse - Hôpital Purpan
Toulouse, 31059
Hungary5 sites
Jahn Ferenc Del-Pesti Korhaz
Budapest, 1204
Pest Megyei Flor Ferenc Korhaz
Kistarcsa, 2143
Szabolcs-Szatmár-Bereg Megyei Kórházak és Egyetemi Oktatókórház
Nyíregyháza
University of Szeged, Faculty of Medicine
Szeged, 4400
Zala Megyei Korhaz
Zalaegerszeg, 8900
Argentina3 sites
Hospital Italiano
Buenos Aires, C1181ACH
Hospital General de Agudos Dr. J. M.
Buenos Aires, C1221ADC
Hospital Cordoba
Córdoba, X5004CDT
Canada3 sites
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
London Health Sciences Centre
London, Ontario, N6A 5A5
University Health Network (UHN) - Toronto General Hospital
Toronto, Ontario, M5G 2C4
Czechia3 sites
Fakultni nemocnice Brno, Neurologicka klinika
Brno
Fakultni nemocnice Ostrava, Neurologická klinika
Ostrava, 70800
Vseobecna fakultni nemocnice v Praze
Prague, 12808
Russia3 sites
State Budgetary Institution of Healthcare of Nizhniy Novgorod region. Nizhniy Novgorod Regional Clinical Hospital named after N.A.Semashko
Nizhny Novgorod, 603126
Saint-Petersburg State Budgetary Institution of Healthcare. City Multi-field Hospital # 2
Saint Petersburg, 357538
State Budgetary Institution of Healthcare "Samara Regional Clinical Hospital. V.D.Seredavin
Samara, 443095
Belgium2 sites
AZ St Lucas Gent
Ghent, East Flanders, 9000
UZ Leuven
Leuven, 3000
Romania2 sites
Institutul Clinic Fundeni
Bucharest, 22328
Spitalul Clinic Judetean de Urgenta Targu-Mures
Târgu Mureş, RO540136
About this study
Official title: A Multicenter, Prospective, Open-label, Non-controlled Clinical Trial to Assess the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in Patients With Myasthenia Gravis Exacerbations
This was a multicenter, prospective, open-label, non-controlled study to assess the efficacy and safety of an IV dose of 2 g/kg of IGIV-C in subjects with MG exacerbations.
Read the detailed description
The study consisted of a single dose course of IGIV-C treatment followed by 28-days of post-infusion assessments. The total duration of study participation for each subject was up to 28 ± 2 days. Approximately 50 subjects, ages 18 or greater, were planned to be enrolled in the study and receive a single, total dose of 2 g/kg of IGIV-C over 2 consecutive days (dose of 1 g/kg per day) across multiple centers in Argentina, Canada, Europe, and South Africa.
- Conditions
- Myasthenia Gravis Exacerbations
- Being tested
- IGIV-C
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IGIV-CBiological
An IV dose of 2 g/kg of IGIV-C was administered over 2 consecutive days at a dose of 1 g/kg per day.
| Study group | Role | What they receive |
|---|---|---|
| IGIV-C Treatment | Experimental | In this arm, subjects with myasthenia gravis exacerbations were treated with an IV dose of 2 g/kg of IGIV-C, which was administered over 2 consecutive days at a dose of 1 g/kg per day. Receives: Biological: IGIV-C |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Quantitative Myasthenia Gravis (QMG) Scale Score
Mean Change in Quantitative Myasthenia Gravis (QMG) Scale Score from Baseline (Day 0) to Day 14. The minimum and maximum scores of the QMG Scale are 0 and 39, respectively, and a higher score means a worse outcome.
Measured: From Baseline (Day 0) to Day 14
Secondary outcomes (3)
Percentage of Subjects With Clinical Improvement Assessed by QMG
The percentage of subjects with clinical improvement at Day 14 as assessed by the Quantitative Myasthenia Gravis (QMG) scale in the Evaluable population is presented, in which clinical improvement is defined as at least 3-point decrease in QMG score from Baseline (Day 0) to Day 14. The minimum and maximum scores of the QMG scale are 0 and 39, respectively, and a higher score means a worse outcome.
Measured: Baseline (Day 0) to Day 14
Percentage of Subjects With Clinical Improvement Assessed by MG-Activities of Daily Living (MG-ADL) Scale
The percentage of subjects with clinical improvement at Day 14 as assessed by the MG-ADL Scale in the Evaluable population is presented, in which clinical improvement is defined as at least 2-point decrease in the MG-ADL score. The minimum and maximum scores of the MG-DAL scale are 0 and 24, respectively, and a higher score means a worse outcome.
Measured: Baseline (Day 0) to Day 14
Percentage of Subjects With Clinical Improvement Assessed by the MG Composite
The percentage of subjects with clinical improvement at Day 14 as assessed by the MG Composite scale in the Evaluable population is presented in which clinical improvement is defined as at least 3-point decrease in the MG Composite score. The minimum and maximum scores of the MG Composite scale are 0 and 50, respectively, with a higher score meaning a worse outcome.
Measured: Baseline (Day 0) to Day 14
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2015 (estimated)
- Study startMar 2015
- Primary completionApr 2018
- Study completionApr 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Myasthenia Gravis ExacerbationsStudies of the same condition, open and under way.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 49 participants (actual)
Sponsor and investigators
- Lead sponsor
- Grifols Therapeutics LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02413580
- Sponsor's protocol ID
- GTI1305
- Status last verified
- Apr 2020
- First submitted
- 7 Apr 2015
- Last updated
- 24 Apr 2020
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 24 Apr 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multicenter, Prospective, Open-label, Non-controlled Clinical Trial to Assess the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in Patients With Myasthenia Gravis Exacerbations
- ClinicalTrials.gov ID
- NCT02413580
- Acronym / sponsor ID
- GTI1305
- Conditions
- Myasthenia Gravis Exacerbations
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 49 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- Mar 2015
- Primary completion
- Apr 2018 (actual)
- Study completion
- Apr 2018 (actual)
- First posted
- 10 Apr 2015 (estimated)
- Results
- Posted 24 Apr 2020
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This was a multicenter, prospective, open-label, non-controlled study to assess the efficacy and safety of an IV dose of 2 g/kg of IGIV-C in subjects with MG exacerbations.
Detailed description
The study consisted of a single dose course of IGIV-C treatment followed by 28-days of post-infusion assessments. The total duration of study participation for each subject was up to 28 ± 2 days. Approximately 50 subjects, ages 18 or greater, were planned to be enrolled in the study and receive a single, total dose of 2 g/kg of IGIV-C over 2 consecutive days (dose of 1 g/kg per day) across multiple centers in Argentina, Canada, Europe, and South Africa.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| IGIV-C Treatment | Experimental | Biological: IGIV-CIn this arm, subjects with myasthenia gravis exacerbations were treated with an IV dose of 2 g/kg of IGIV-C, which was administered over 2 consecutive days at a dose of 1 g/kg per day. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Was male or female aged ≥18 years.
- Subjects must be willing and able to provide written informed consent (if applicable, a legally authorized representative may provide informed consent on behalf of the subject).
- Subjects who met the clinical criteria for diagnosis of MG with an exacerbation defined as worsening of MG symptoms as defined by an Myasthenia Gravis Foundation of America (MGFA) classification IVb or V.
- Subjects on long-term (8 weeks) corticosteroid treatment for MG.
- Female subjects of child-bearing potential must have a negative test for pregnancy (human chorionic gonadotropin [HCG]-based assay).
Key exclusion criteria (20 listed)
- Subjects who had received immune globulin treatment given by IV, subcutaneous or intramuscular route within the last 30 days.
- Subjects with documentation of a lack of clinical response to intravenous immunoglobulin (IVIg) therapy for MG.
- Subjects documented positive for antibodies directed against Muscle specific kinase (MuSK).
- Subjects with corticosteroid (CS) treatment initiated within the last 8 weeks or modified within the last 2 weeks.
- Subjects with plasma exchange (PLEX) within the last 30 days.
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 |
|---|---|---|
| IGIV-CAn IV dose of 2 g/kg of IGIV-C was administered over 2 consecutive days at a dose of 1 g/kg per day. | Biological | IGIV-C Treatment |
Primary outcome
Change in Quantitative Myasthenia Gravis (QMG) Scale Score
Time frame: From Baseline (Day 0) to Day 14
Mean Change in Quantitative Myasthenia Gravis (QMG) Scale Score from Baseline (Day 0) to Day 14. The minimum and maximum scores of the QMG Scale are 0 and 39, respectively, and a higher score means a worse outcome.
3 secondary outcomes
Percentage of Subjects With Clinical Improvement Assessed by QMG
Time frame: Baseline (Day 0) to Day 14
Percentage of Subjects With Clinical Improvement Assessed by MG-Activities of Daily Living (MG-ADL) Scale
Time frame: Baseline (Day 0) to Day 14
Percentage of Subjects With Clinical Improvement Assessed by the MG Composite
Time frame: Baseline (Day 0) to Day 14
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2020.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Related trials for Myasthenia Gravis Exacerbations
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Treatment of Myasthenia Gravis Exacerbation or Crisis With Efgartigimod
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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.
The record lists no contact. Use the official record to reach the sponsor.
31 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing IGIV-C in people with Myasthenia Gravis Exacerbations, run by Grifols Therapeutics LLC at 31 sites in 12 countries.
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 6 inclusion and 20 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 24 Apr 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in France, Hungary, Argentina, Canada, Czechia and 7 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 24 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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 (QMG) Scale Score (time frame: From Baseline (Day 0) to Day 14). 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 24 Apr 2020; AzTrials last refreshed its copy on 7 Nov 2026.