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A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis

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ClinicalTrials.gov Identifier: NCT02473952
Recruitment Status : Completed
First Posted : June 17, 2015
Results First Posted : March 5, 2019
Last Update Posted : March 5, 2019
Sponsor:
Information provided by (Responsible Party):
Grifols Therapeutics LLC

Tracking Information
First Submitted Date  ICMJE June 14, 2015
First Posted Date  ICMJE June 17, 2015
Results First Submitted Date  ICMJE January 29, 2019
Results First Posted Date  ICMJE March 5, 2019
Last Update Posted Date March 5, 2019
Actual Study Start Date  ICMJE August 2015
Actual Primary Completion Date January 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: March 1, 2019)
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.
Original Primary Outcome Measures  ICMJE
 (submitted: June 16, 2015)
Improvement in myasthenia gravis (MG) symptoms as measured by the mean change in QMG score. [ Time Frame: from Baseline (Week 0) to Week 24 ]
To measure improvement in MG symptoms by the mean change in Quantitative myasthenia gravis (QMG) score from Baseline (Week 0) to Week 24 as compared to placebo.
Change History Complete list of historical versions of study NCT02473952 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE Not Provided
Original Secondary Outcome Measures  ICMJE Not Provided
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis
Official Title  ICMJE 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
Brief Summary 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 Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Myasthenia Gravis, Generalized
Intervention  ICMJE
  • Drug: IGIV-C
    IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified
  • Drug: Placebo
Study Arms  ICMJE
  • Experimental: IGIV-C

    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).

    Intervention: Drug: IGIV-C
  • Placebo Comparator: Placebo
    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).
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: June 16, 2015)
62
Original Estimated Enrollment  ICMJE Same as current
Actual Study Completion Date  ICMJE January 2018
Actual Primary Completion Date January 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  • 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):

    1. Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening and no immunosuppressants
    2. Cholinesterase inhibitor (pyridostigmine or equivalent) for at least 2 weeks prior to Screening AND/OR only one of the following:

      1. Prednisone (up to 60 mg/day or equivalent) for at least 2 months prior to Screening, OR
      2. Azathioprine for at least 6 months prior to Screening, OR
      3. Mycophenolate mofetil for at least 6 months prior to Screening, OR
      4. Methotrexate for at least 6 months prior to Screening, OR
      5. Cyclosporine or tacrolimus for at least 3 months prior to Screening
    3. 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:

      1. Azathioprine for at least 6 months prior to Screening, OR
      2. Mycophenolate mofetil for at least 6 months prior to Screening, OR
      3. Methotrexate for at least 6 months prior to Screening, OR
      4. Cyclosporine or tacrolimus for at least 3 months prior to Screening

Exclusion Criteria:

  • 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
  • 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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 85 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Belgium,   Canada,   Czechia,   Estonia,   France,   Germany,   Hungary,   Lithuania,   Poland,   United States
Removed Location Countries Czech Republic
 
Administrative Information
NCT Number  ICMJE NCT02473952
Other Study ID Numbers  ICMJE GTI1408
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Grifols Therapeutics LLC
Study Sponsor  ICMJE Grifols Therapeutics LLC
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Grifols Therapeutics LLC
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP