A study investigating Immune Globuline (Human), 10% Caprylate/Chromatography Purified (IGIV-C) for the treatment of patients with Myasthenia Gravis exacerbations. All patients will receive a single dose course of IGIV-C treatment followed by 28-days of post-infusion assessments.

• Register: EUCTR
• Last refreshed on: 23 July 2018
• Main ID: EUCTR2013-005098-28-CZ
• Date of registration: 03/10/2014
• Prospective Registration: Yes
• Primary sponsor: Grifols Therapeutics LLC.
• Public title: A study investigating Immune Globuline (Human), 10% Caprylate/Chromatography Purified (IGIV-C) for the treatment of patients with Myasthenia Gravis exacerbations. All patients will receive a single dose course of IGIV-C treatment followed by 28-days of post-infusion assessments.
• Scientific title: A multicenter, prospective, open-label, non-controlled clinical trial to assess the efficacy and safety of Immune Globuline (Human), 10% Caprylate/Chromatography Purified (IGIV-C) in patients with Myasthenia Gravis exacerbations
• Date of first enrolment: 16/01/2015
• Target sample size: 50
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2013-005098-28
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: no; Randomised: no; Open: yes; Single blind: no; Double blind: no; Parallel group: no; Cross over: no; Other: no; If controlled, specify comparator, Other Medicinial Product: no; Placebo: no; Other: no
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Argentina, Belgium, Canada, Czech Republic, Estonia, France, Hungary, Latvia, Poland, Romania, Russian Federation, South Africa

Contacts

• Name: Rhonda Griffin-Director,ClinDev
• Affiliation: Grifols Therapeutics LLC.

• Name: Rhonda Griffin-Director,ClinDev
• Affiliation: Grifols Therapeutics LLC.

Key inclusion & exclusion criteria

Inclusion criteria:
A subject must meet all the following inclusion criteria to be eligible for participation in this
study:
1. Male or female aged =18 years.
2. 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).
3. Subjects who meet the clinical criteria for diagnosis of MG with an exacerbation defined as worsening of MG symptoms as defined by an MGFA classification IVb or V.
4. Subjects on long-term (8 weeks) corticosteroid treatment for MG.
5. Female subjects of child-bearing potential must have a negative test for pregnancy (human chorionic gonadotropin (HCG)-based assay).
6. 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 50
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
A subject meeting any of the following exclusion criteria is NOT eligible for participation in the study:
1. Subjects who have received immune globulin treatment given by IV, subcutaneous or intramuscular route within the last 30 days.
2. Subjects with documentation of a lack of clinical response to IVIG therapy for MG.
3. Subjects documented positive for antibodies directed against MuSK.
4. Subjects with CS treatment initiated within the last 8 weeks or modified within the last 2 weeks.
5. Subjects with PLEX within the last 30 days.
6. Subjects with MG exacerbation attributable to change in medication 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 >12×109/L and differential white blood cell count of
>10% band neutrophils (>1.2×109/L), and 4) pulmonary infiltrate with
consolidation on chest Xray. Alternatively, other signs and symptoms
may be considered for the diagnosis of evident infection
according to the Investigator's judgement.
7. Subjects with inadequate venous access.
8. Subjects with a history of anaphylactic reactions or severe reactions to any blood-derived product.
9. Subjects with a history of intolerance to any component of the investigational products (IPs).
10. Subjects with a documented diagnosis of thrombotic complications to polyclonal IVIG therapy in the past.
11. Subjects with a history of recent (within the last year) myocardial infarction, stroke or uncontrolled hypertension.
12. Subjects who suffer from uncontrolled congestive heart failure, embolism or documented electrocardiogram (ECG) changes indicative of myocardial ischemia or atrial fibrillation.
13. Subjects with current known hyperviscosity or hypercoagulable state.
14. Subjects currently receiving anti-coagulation therapy.
15. Subjects with a history of chronic alcoholism or illicit drug abuse (addiction) in the 12 months preceding the Baseline Visit.
16. Subjects with active psychiatric illness that interferes with compliance or communication with health care personnel.
17. Females who are pregnant, breastfeeding, or of child-bearing potential and unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device or intrauterine system, condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or true abstinence*) throughout the study.
* True abstinence: When this is in line with the preferred and usual lifestyle of the subject. (Periodic abstinence [e.g., calendar, ovulation, symptothermal, post-ovulation methods], declaration of abstinence for the duration of a trial, and withdrawal are not acceptable methods of contraception.)
18. Subjects with any medical condition which makes the clinical trial participation unadvisable or which is likely to interfere with the evaluation of the study treatment and/or the satisfactory conduct of the clinical trial according to the Investigator’s judgment.
19. Subjects currently receiving, or having received within 3 months prior to the Baseline Visit, any investigational medicinal product or device.
20. Subjects who are unlikely to adhere to the protocol requirements or are likely to be uncooperative or unable to provide a storage serum/plasma sample prior to the first

Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Myasthenia Gravis Exacerbations
MedDRA version: 20.0 Level: PT Classification code 10028417 Term: Myasthenia gravis System Organ Class: 10029205 - Nervous system disorders

Therapeutic area: Diseases [C] - Nervous System Diseases [C10]

Intervention(s)

Trade Name: GAMUNEX 10%
Pharmaceutical Form: Solution for infusion
INN or Proposed INN: Immune Globulin (Human), 10% Caprylate/Chromatography Purified
Other descriptive name: HUMAN NORMAL IMMUNOGLOBULIN (IV)
Concentration unit: g/ml gram(s)/millilitre
Concentration type: equal
Concentration number: 10g/100ml-

Primary Outcome(s)

Secondary Objective: - To evaluate the efficacy of an IV infusion of IGIV-C (total dose of 2 g/kg administered over 2 days at a dose of 1 g/kg per day) in subjects with MG exacerbations by:
• Percentage of subjects who experience a clinical improvement assessed by QMG from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 3 point decrease in QMG
• Percentages of subjects who experience a clinical improvement assessed by MG - Activities of Daily Living (MG-ADL) from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 2-point decrease in MG-ADL
• Percentages of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Day 0) to DAy 14 where clinical improvement is defined as at least 3 point decrease in the MG Composite

Timepoint(s) of evaluation of this end point: 28 ± 2 days

Main Objective: - To evaluate the efficacy of an an intravenous infusion of IGIV-C (total dose of 2g/kg administered over 2 consecutive days at a dose of 1 g/kg per day) in subjects with MG exacerbations by assessing the change in score of MG symptoms as measured by the QMG scale from baseline to Day 14
- To evaluate the safety and tolerability of an IV infusion of IGIV-C (total dose of 2 g/kg administered over 2 consecutive days at a dose of 1 g/kg per day) in subjects with MG exacerbations.

Primary end point(s): - The primary variable to assess efficacy in this study is the change in QMG score from Baseline (Day 0) to Day 14.

Secondary Outcome(s)

Secondary end point(s): Percentage of subjects who experience a clinical improvement assessed by QMG from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 3 point decrease in QMG
• Percentage of subjects who experience a clinical improvement assessed by MG-ADL from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 2 point decrease in MG-ADL
• Percentage of subjects who experience a clinical improvement assessed by the MG Composite from Baseline (Day 0) to Day 14 where clinical improvement is defined as at least 3 point decrease in the MG Composite

Timepoint(s) of evaluation of this end point: 28 ± 2 days

Secondary ID(s)

GTI1305

Source(s) of Monetary Support

Grifols Therapeutics LLC.