A Study to Assess the Efficacy and Safety of IGIV-C in Patients With Myasthenia Gravis Exacerbations

NCT02413580 | Completed Phase 3 Results

• 1 other identifier: GTI1305
• Study Type: interventional
• Target: 49
• Locations: 12 countries
• Sites: 31

Brief 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.

Conditions

Myasthenia Gravis Exacerbations

Outcome Measures

Primary Outcomes (1)

• 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.

  From Baseline (Day 0) to Day 14

Secondary Outcomes (3)

• Percentage of Subjects With Clinical Improvement Assessed by QMG

  Baseline (Day 0) to Day 14

• Percentage of Subjects With Clinical Improvement Assessed by MG-Activities of Daily Living (MG-ADL) Scale

  Baseline (Day 0) to Day 14

• Percentage of Subjects With Clinical Improvement Assessed by the MG Composite

  Baseline (Day 0) to Day 14

Study Arms (1)

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.

Biological: IGIV-C

Interventions

IGIV-C BIOLOGICAL

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.

IGIV-C Treatment

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.
• 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.

Sponsors & Collaborators

• Grifols Therapeutics LLC: lead

Study Sites (31)

Hospital Italiano

Buenos Aires, C1181ACH, Argentina

Location

Hospital General de Agudos Dr. J. M.

Buenos Aires, C1221ADC, Argentina

Location

Hospital Cordoba

Córdoba, X5004CDT, Argentina

Location

AZ St Lucas Gent

Ghent, East Flanders, 9000, Belgium

Location

UZ Leuven

Leuven, 3000, Belgium

Location

University of Alberta Hospital

Edmonton, Alberta, T6G 2B7, Canada

Location

London Health Sciences Centre

London, Ontario, N6A 5A5, Canada

Location

University Health Network (UHN) - Toronto General Hospital

Toronto, Ontario, M5G 2C4, Canada

Location

Fakultni nemocnice Brno, Neurologicka klinika

Brno, Czechia

Location

Fakultni nemocnice Ostrava, Neurologická klinika

Ostrava, 70800, Czechia

Location

Vseobecna fakultni nemocnice v Praze

Prague, 12808, Czechia

Location

East Tallinn Central Hospital

Tallinn, 10138, Estonia

Location

Hopital Neurologique Pierre Wertheimer

Bron, Lyon, 69677, France

Location

Hôpital Albert Michallon

Grenoble, 38700, France

Location

Hopital Roger Salengro

Lille, 59037, France

Location

Hôpital de la Timone

Marseille, 13385, France

Location

Hôpital Hautepierre Strasbourg

Strasbourg, 67200, France

Location

CHU de Toulouse - Hôpital Purpan

Toulouse, 31059, France

Location

Jahn Ferenc Del-Pesti Korhaz

Budapest, 1204, Hungary

Location

Pest Megyei Flor Ferenc Korhaz

Kistarcsa, 2143, Hungary

Location

Szabolcs-Szatmár-Bereg Megyei Kórházak és Egyetemi Oktatókórház

Nyíregyháza, Hungary

Location

University of Szeged, Faculty of Medicine

Szeged, 4400, Hungary

Location

Zala Megyei Korhaz

Zalaegerszeg, 8900, Hungary

Location

Riga East Clinical University Hospital

Riga, LV-1038, Latvia

Location

Uniwersyteckie Centrum Kliniczne

Gdansk, Poland

Location

Institutul Clinic Fundeni

Bucharest, 22328, Romania

Location

Spitalul Clinic Judetean de Urgenta Targu-Mures

Târgu Mureş, RO540136, Romania

Location

State Budgetary Institution of Healthcare of Nizhniy Novgorod region. Nizhniy Novgorod Regional Clinical Hospital named after N.A.Semashko

Nizhny Novgorod, 603126, Russia

Location

Saint-Petersburg State Budgetary Institution of Healthcare. City Multi-field Hospital # 2

Saint Petersburg, 357538, Russia

Location

State Budgetary Institution of Healthcare "Samara Regional Clinical Hospital. V.D.Seredavin

Samara, 443095, Russia

Location

Groote Schuur Hospital,

Cape Town, South Africa

Location

Results Point of Contact

• Title: Rhonda Griffin
• Organization: Grifols Therapeutics LLC

Rhonda.Griffin@grifols.com

9193166693

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 7, 2015
• First Posted: April 10, 2015
• Study Start: March 1, 2015
• Primary Completion: April 1, 2018
• Study Completion: April 1, 2018
• Last Updated: April 24, 2020
• Results First Posted: April 24, 2020

Record last verified: 2020-04

Locations

AR (3); ES (1); CA (3); RU (3); FR (6); HU (5); PL (1); BE (2); CZ (3); LA (1); ZA (1); RO (2)