Myasthenia Gravis, GeneralizedPhase 2NCT02473952
A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis
A Phase 2 study testing IGIV-C in people with Myasthenia Gravis, Generalized, run by Grifols Therapeutics LLC at 40 sites in 10 countries.
Trial summary
The primary objective is to evaluate whether IGIV-C improves MG symptoms as compared to placebo in subjects with MG. Read more about this study
- Who is it for?
- Ages 18 to 85 years
- Conditions the study is for
- Myasthenia Gravis, Generalized
- What is being tested?
- IGIV-C
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 62 participants
- Where
- 40 sitesin 10 countries
- Study dates
- Aug 2015to Jan 2018
- Phase
- Phase 2
- 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 to 85 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Anti-acetylcholine receptor (AChR) antibody positive
- Confirmed diagnosis of generalized myasthenia gravis (MG).
- Myasthenia Gravis Foundation of America (MGFA) classification of Class II, III, or IVa inclusive at Screening.
- QMG >= 10 at Screening. Note: Subjects who only have a history of ocular MG may not enroll.
- Receiving standard of care MG treatment at a stable dose consisting of any one of the following for the time intervals delineated below (time intervals apply to medications and maintenance of stable dose level):
- Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and no immunosuppressants
Show all 17Show fewer
- Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening AND/OR only one of the following:
- Prednisone (up to 60 mg/day or equivalent) for at least 2 months prior to Screening, OR
- Azathioprine for at least 6 months prior to Screening, OR
- Mycophenolate mofetil for at least 6 months prior to Screening, OR
- Methotrexate for at least 6 months prior to Screening, OR
- Cyclosporine or tacrolimus for at least 3 months prior to Screening
- Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening AND/OR prednisone (up to 60 mg/day or equivalent) for at least one month prior to Screening and only one of the following:
- Azathioprine for at least 6 months prior to Screening, OR
- Mycophenolate mofetil for at least 6 months prior to Screening, OR
- Methotrexate for at least 6 months prior to Screening, OR
- Cyclosporine or tacrolimus for at least 3 months prior to Screening
Who cannot join
- Have received cyclophosphamide or any other immunosuppressive agent apart from the ones allowed per inclusion criteria within the past 6 months
- Any change in MG treatment regimen between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)
- Greater than two point change in QMG score, increased or decreased, between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)
- Any episode of myasthenic crisis in the one month prior to Screening
- Evidence of malignancy within the past 5 years (non-melanoma skin cancer, carcinoma in situ of cervix is allowed) or thymoma potentially requiring surgical intervention during the course of the trial (intent to perform thymectomy)
- Thymectomy within the preceding 6 months
Show all 17Show fewer
- Rituximab, belimumab, eculizumab or any monoclonal antibody used for immunomodulation within the past 12 months
- Have received immune globulin (Ig) treatment given by intravenous (IV), subcutaneous, or intramuscular route within the last 3 months
- Current known hyperviscosity or hypercoagulable state
- Currently receiving anti-coagulation therapy (vitamin K antagonists, nonvitamin K antagonist oral anticoagulants [e.g., dabigatran etexilate, rivaroxaban, edoxaban, and apixaban], parenteral anticoagulants [e.g., fondaparinux]). Note that oral anti-platelet agents are allowed (e.g., aspirin, clopidogrel, ticlodipine)
- Documented diagnosis of thrombotic complications to polyclonal intravenous immunoglobulin (IVIg) therapy in the past
- History of recent (within the last year) myocardial infarction or stroke
- Uncontrolled congestive heart failure; embolism; or historically documented (within the last year) electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation
- History of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Screening/Week -3 (Visit 0)
- Plasma exchange (PLEX) performed within the last 3 months
- 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).
- Hemoglobin levels less than 9 g per dL
Trial locations and contacts
40 sites in 10 countries.
40 sites in 10 countries
United States15 sites
Georgia Regents University
Augusta, Georgia, 30912
University of Vermont Medical Center
Burlington, Vermont, 05405
Ohio State University Wexner Medical Center
Columbus, Ohio, 43220
Houston Methodist Neurological Institute
Houston, Texas, 77030
Indiana University
Indianapolis, Indiana, 46202
University of Florida Health Science Center
Jacksonville, Florida, 32209
University of Kansas Medical Center Research Institute, Inc.
Kansas City, Kansas, 66160
Yale University School of Medicine
New Haven, Connecticut, 06510
Columbia University Medical Center
New York, New York, 10032
Rutgers New Jersey Medical School
Newark, New Jersey, 07103
University of California-Irvine
Orange, California, 92868
Thomas Jefferson University Hospital
Philadelphia, Pennsylvania, 19107
Phoenix Neurological Associates, Ltd.
Phoenix, Arizona, 85018
University of Washington Medical Center
Seattle, Washington, 98195
University of South Florida
Tampa, Florida, 33612
Germany7 sites
Universitaetsklinikum Koeln, Neurologie und Psychiatrie
Cologne, North Rhine-Westphalia, 50937
Universitaetsklinikum Carl Gustav Carus TU Dresden
Dresden, Saxony, 1307
Universitaetsmedizin Göttingen, Parent
Göttingen, Lower Saxony, 37075
Krankenhaus Martha-Maria Halle-Doelau, Klinik fuer Neurologie
Halle, Saxony-Anhalt, 6120
Universitaetsklinikum Hamburg-Eppendorf, Klinik und Poliklinik fuer Neurologie
Hamburg, 20246
Universitaetsklinikum Jena, Klinik fuer Neurologie
Jena, Thuringia, 7747
Universitaetsklinikum Regensburg, Parent
Regensburg, Bavaria, 93053
France4 sites
Hopital Neurologique Pierre Wertheimer, Neuro-musculaire - Electromyographie
Bron, Rhone, 69677
CHU Nice - Hôpital de l'Archet 1, Ctre de Réf Maladies Neuromusculaires et SLA
Nice, Alpes Maritimes, 6202
CHU Strasbourg - Nouvel Hôpital Civil, Clinique Neurologique
Strasbourg, Bas Rhin, 67091
CHU de Toulouse - Hôpital Purpan, Service de Neurologie Générale
Toulouse, Haute Garonne, 31059
Poland4 sites
Uniwersyteckie Centrum Kliniczne, Dept of Neurology
Gdansk, 80-952
Krakowska Akademia Neurologii Sp z o.o. Centrum Neurologii Klinicznej
Krakow, 31-505
III Szpital Miejski w Lodzi im. Dr K. Jonschera
Lodz, 93-113
Samodzielny Publiczny Centralny Szpital Kliniczny, Dept of Neurology
Warsaw, 02-097
Hungary3 sites
Jahn Ferenc Del-pesti Korhaz es Rendelointezet, Neurologiai Osztaly
Budapest, 1204
Pest Megyei Flor Ferenc Korhaz, Neurologia es Stroke Osztaly
Kistarcsa, 2143
Szegedi Tudomanyegyetem Szent-Gyorgyi Albert Klinikai Kozpont
Szeged, 6725
Canada2 sites
London Health Sciences Centre - University Hospital
London, Ontario, N6A 5A5
University Health Network (UHN) - Toronto General Hospital
Toronto, Ontario, M5G 2C4
Czechia2 sites
Fakultni nemocnice Brno, Dept of Neurologicka klinika
Brno, 625 00
Fakultni nemocnice Ostrava
Ostrava - Poruba, 708 52
Lithuania1 site
Hospital of Lithuanian University of Health Sciences Kaunas Clinics
Kaunas, 50009
About this study
Official title: A Multi-center, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in Symptomatic Subjects With Generalized Myasthenia Gravis
The primary objective is to evaluate whether IGIV-C improves MG symptoms as compared to placebo in subjects with MG.
Read the detailed description
The primary objective is to evaluate the efficacy of IGIV-C in subjects with generalized myasthenia gravis (MG) on standard of care treatment at study entry in terms of improvement in MG symptoms as measured by the mean change in Quantitative Myasthenia Gravis (QMG) score from Baseline (Week 0) to Week 24 as compared to placebo.
The safety objective of this study is to evaluate the safety and tolerability of IGIV-C loading dose of 2 g/kg followed by 7 maintenance dosages of 1 g/kg every 3 weeks through Week 21 in subjects with MG.
- Conditions
- Myasthenia Gravis, Generalized
- Being tested
- IGIV-C, Placebo
- Medical subject headings
- Myasthenia Gravis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IGIV-CDrug
IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified
PlaceboDrug
| Study group | Role | What they receive |
|---|---|---|
| IGIV-C | Experimental | IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified.
An initial loading dose of 2 g/kg of body weight will be administered at Baseline (Week 0, Visit 1) followed by maintenance doses of 1 g/kg of body weight administered every third week through Week 21 (Visit 8). Receives: Drug: IGIV-C |
| Placebo | Placebo comparator | Placebo: Sterile 0.9% sodium chloride injection or equivalent. Placebo will be infused at the Baseline/Week 0 Visit (Visit 1) using the same volume as would be required for the IGIV-C loading dose. Subsequent placebo maintenance doses will be matched in volume to the IGIV-C maintenance doses and administered every third week until Week 21 (Visit 8). Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score.
To measure improvement in MG symptoms by the mean change in QMG total score from Baseline (Week 0) to Week 24 as compared to placebo. Evaluators score 13 individual items (range from 0=best to 3=worst) and the individual scores are added together for the total score (range 0-39). An average 3-point improvement in QMG score indicates clinically meaningful improvement.
Measured: Baseline (Week 0) to Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jun 2015 (estimated)
- Study startAug 2015
- Primary completionJan 2018
- Study completionJan 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.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Myasthenia Gravis, GeneralizedStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 62 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
- NCT02473952
- Sponsor's protocol ID
- GTI1408
- Status last verified
- Mar 2019
- First submitted
- 14 Jun 2015
- Last updated
- 5 Mar 2019
- Results posted
- Yes, on ClinicalTrials.gov
Publications
Dalakas MC, Meisel A. Immunomodulatory effects and clinical benefits of intravenous immunoglobulin in myasthenia gravis. Expert Rev Neurother. 2022 Apr;22(4):313-318. doi: 10.1080/14737175.2022.2057223. Epub 2022 Apr 5.
PubMed 35350948
Study at a glance
From the registry record, last updated by the sponsor on 5 Mar 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Multi-center, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Immune Globulin (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in Symptomatic Subjects With Generalized Myasthenia Gravis
- ClinicalTrials.gov ID
- NCT02473952
- Acronym / sponsor ID
- GTI1408
- Conditions
- Myasthenia Gravis, Generalized
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 62 participants (actual)
- Lead sponsor
- Grifols Therapeutics LLCIndustry (pharma, biotech, devices)
- Study start
- Aug 2015 (actual)
- Primary completion
- Jan 2018 (actual)
- Study completion
- Jan 2018 (actual)
- First posted
- 17 Jun 2015 (estimated)
- Results
- Posted 5 Mar 2019
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 is to evaluate whether IGIV-C improves MG symptoms as compared to placebo in subjects with MG.
Detailed description
The primary objective is to evaluate the efficacy of IGIV-C in subjects with generalized myasthenia gravis (MG) on standard of care treatment at study entry in terms of improvement in MG symptoms as measured by the mean change in Quantitative Myasthenia Gravis (QMG) score from Baseline (Week 0) to Week 24 as compared to placebo.
The safety objective of this study is to evaluate the safety and tolerability of IGIV-C loading dose of 2 g/kg followed by 7 maintenance dosages of 1 g/kg every 3 weeks through Week 21 in subjects with MG.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IGIV-C | Experimental | Drug: IGIV-CIGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified. An initial loading dose of 2 g/kg of body weight will be administered at Baseline (Week 0, Visit 1) followed by maintenance doses of 1 g/kg of body weight administered every third week through Week 21 (Visit 8). |
| Placebo | Placebo comparator | Drug: PlaceboPlacebo: Sterile 0.9% sodium chloride injection or equivalent. Placebo will be infused at the Baseline/Week 0 Visit (Visit 1) using the same volume as would be required for the IGIV-C loading dose. Subsequent placebo maintenance doses will be matched in volume to the IGIV-C maintenance doses and administered every third week until Week 21 (Visit 8). |
Who may qualify
- Ages
- 18 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (17 listed)
- Anti-acetylcholine receptor (AChR) antibody positive
- Confirmed diagnosis of generalized myasthenia gravis (MG).
- Myasthenia Gravis Foundation of America (MGFA) classification of Class II, III, or IVa inclusive at Screening.
- QMG >= 10 at Screening. Note: Subjects who only have a history of ocular MG may not enroll.
- Receiving standard of care MG treatment at a stable dose consisting of any one of the following for the time intervals delineated below (time intervals apply to medications and maintenance of stable dose level):
Key exclusion criteria (17 listed)
- Have received cyclophosphamide or any other immunosuppressive agent apart from the ones allowed per inclusion criteria within the past 6 months
- Any change in MG treatment regimen between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)
- Greater than two point change in QMG score, increased or decreased, between Screening (Week -3, Visit 0) and Baseline (Week 0, Visit 1)
- Any episode of myasthenic crisis in the one month prior to Screening
- Evidence of malignancy within the past 5 years (non-melanoma skin cancer, carcinoma in situ of cervix is allowed) or thymoma potentially requiring surgical intervention during the course of the trial (intent to perform thymectomy)
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-CIGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified | Drug | IGIV-C |
| Placebo | Drug | Placebo |
Primary outcome
Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score.
Time frame: Baseline (Week 0) to Week 24
To measure improvement in MG symptoms by the mean change in QMG total score from Baseline (Week 0) to Week 24 as compared to placebo. Evaluators score 13 individual items (range from 0=best to 3=worst) and the individual scores are added together for the total score (range 0-39). An average 3-point improvement in QMG score indicates clinically meaningful improvement.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Mar 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 35350948Dalakas MC, Meisel A. Immunomodulatory effects and clinical benefits of intravenous immunoglobulin in myasthenia gravis. Expert Rev Neurother. 2022 Apr;22(4):313-318. doi: 10.1080/14737175.2022.2057223. Epub 2022 Apr 5.
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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.
40 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing IGIV-C in people with Myasthenia Gravis, Generalized, run by Grifols Therapeutics LLC at 40 sites in 10 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 17 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 5 Mar 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 40 sites in United States, Germany, France, Poland, Hungary and 5 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. Some participants in this study may receive a placebo. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 5 Mar 2019). 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?
Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score. (time frame: Baseline (Week 0) to Week 24). 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 NCT02473952, 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 5 Mar 2019.