Trial Outcomes & Findings for A Study to Evaluate the Efficacy and Safety of IGIV-C in Symptomatic Subjects With Generalized Myasthenia Gravis (NCT NCT02473952)

Last Updated: 2019-03-05

Results Overview

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.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

62 participants

Primary outcome timeframe

Baseline (Week 0) to Week 24

Results posted on

2019-03-05

Participant Flow

Participant milestones

Participant milestones
Measure	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). IGIV-C: IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified	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). Placebo
Overall Study STARTED	30	32
Overall Study COMPLETED	28	24
Overall Study NOT COMPLETED	2	8

Reasons for withdrawal

Reasons for withdrawal
Measure	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). IGIV-C: IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified	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). Placebo
Overall Study Adverse Event	2	2
Overall Study Withdrawal by Subject	0	6

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	IGIV-C n=30 Participants 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). IGIV-C: IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified	Placebo n=32 Participants 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). Placebo	Total n=62 Participants Total of all reporting groups
Ethnicity (NIH/OMB) Not Hispanic or Latino	28 Participants n=5 Participants	29 Participants n=7 Participants	57 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Age, Continuous	54.6 years STANDARD_DEVIATION 17.06 • n=5 Participants	48.0 years STANDARD_DEVIATION 13.66 • n=7 Participants	51.2 years STANDARD_DEVIATION 15.63 • n=5 Participants
Age, Customized <65 years	20 Participants n=5 Participants	29 Participants n=7 Participants	49 Participants n=5 Participants
Age, Customized >=65	10 Participants n=5 Participants	3 Participants n=7 Participants	13 Participants n=5 Participants
Sex: Female, Male Female	14 Participants n=5 Participants	19 Participants n=7 Participants	33 Participants n=5 Participants
Sex: Female, Male Male	16 Participants n=5 Participants	13 Participants n=7 Participants	29 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	3 Participants n=7 Participants	5 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	1 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	0 Participants n=7 Participants	1 Participants n=5 Participants
Race (NIH/OMB) White	29 Participants n=5 Participants	30 Participants n=7 Participants	59 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Region of Enrollment North America	11 participants n=5 Participants	12 participants n=7 Participants	23 participants n=5 Participants
Region of Enrollment Europe	19 participants n=5 Participants	20 participants n=7 Participants	39 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline (Week 0) to Week 24

Population: Modified intent-to-treat population consisting of all randomized subjects who received at least 1 dose of study medication.

Outcome measures

Outcome measures
Measure	IGIV-C n=30 Participants 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). IGIV-C: IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified	Placebo n=32 Participants 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). Placebo
Improvement in Myasthenia Gravis (MG) Symptoms as Measured by the Mean Change in Quantitative Myasthenia Gravis (QMG) Total Score.	-4.6 units on a scale Standard Deviation 5.11	-2.7 units on a scale Standard Deviation 6.23

Adverse Events

IGIV-C

Serious events: 5 serious events

Other events: 22 other events

Deaths: 1 deaths

Placebo

Serious events: 4 serious events

Other events: 21 other events

Deaths: 0 deaths

Serious adverse events

Serious adverse events
Measure	IGIV-C n=30 participants at risk 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). IGIV-C: IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified	Placebo n=32 participants at risk 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). Placebo
Nervous system disorders Myasthenia gravis	10.0% 3/30 • Number of events 3 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Cardiac disorders Atrial fibrillation	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Cardiac disorders Cardiopulmonary failure	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Injury, poisoning and procedural complications Lower limb fracture	0.00% 0/30 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Injury, poisoning and procedural complications Wrist fracture	0.00% 0/30 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Nervous system disorders Ischaemic stroke	0.00% 0/30 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Nervous system disorders Cerebral haemorrhage	0.00% 0/30 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Gastrointestinal disorders Haemorrhoids thrombosed	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Infections and infestations Pneumonia	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Infections and infestations Septic shock	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Psychiatric disorders Panic attack	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.

Other adverse events

Additional Information

Rhonda Griffin, BSN

Grifols Therapeutics, LLC

Phone: 919-316-6693

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee Site may publish results from the study, after providing Sponsor at least thirty days' notice prior to submitting a manuscript or other materials related to the study to any outside party. At Sponsor's request, the site will remove any confidential information (other than study results), and incorporate all reasonable comments by Sponsor, or delay publication or presentation for a period of up to 120 days to allow Sponsor to protect its interests in any Sponsor's Inventions.
Publication restrictions are in place

Restriction type: OTHER

Measure	IGIV-C n=30 participants at risk 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). IGIV-C: IGIV-C: Immune Globulin Injection (Human), 10%, Caprylate/Chromatography Purified	Placebo n=32 participants at risk 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). Placebo
Gastrointestinal disorders Diarrhoea	10.0% 3/30 • Number of events 4 • Adverse event data were collected from screening through Week 24.	6.2% 2/32 • Number of events 2 • Adverse event data were collected from screening through Week 24.
Gastrointestinal disorders Nausea	10.0% 3/30 • Number of events 13 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
General disorders Chills	6.7% 2/30 • Number of events 2 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
General disorders Fatigue	6.7% 2/30 • Number of events 3 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Infections and infestations Nasopharyngitis	10.0% 3/30 • Number of events 3 • Adverse event data were collected from screening through Week 24.	12.5% 4/32 • Number of events 4 • Adverse event data were collected from screening through Week 24.
Infections and infestations Upper respiratory tract infection	0.00% 0/30 • Adverse event data were collected from screening through Week 24.	9.4% 3/32 • Number of events 4 • Adverse event data were collected from screening through Week 24.
Musculoskeletal and connective tissue disorders Arthralgia	6.7% 2/30 • Number of events 2 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Musculoskeletal and connective tissue disorders Back pain	6.7% 2/30 • Number of events 3 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Musculoskeletal and connective tissue disorders Neck pain	6.7% 2/30 • Number of events 2 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Musculoskeletal and connective tissue disorders Pain in extremity	3.3% 1/30 • Number of events 1 • Adverse event data were collected from screening through Week 24.	6.2% 2/32 • Number of events 2 • Adverse event data were collected from screening through Week 24.
Nervous system disorders Dizziness	6.7% 2/30 • Number of events 2 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Nervous system disorders Headache	30.0% 9/30 • Number of events 22 • Adverse event data were collected from screening through Week 24.	12.5% 4/32 • Number of events 6 • Adverse event data were collected from screening through Week 24.
Nervous system disorders Myasthenia gravis	0.00% 0/30 • Adverse event data were collected from screening through Week 24.	6.2% 2/32 • Number of events 2 • Adverse event data were collected from screening through Week 24.
Respiratory, thoracic and mediastinal disorders Catarrh	6.7% 2/30 • Number of events 3 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Respiratory, thoracic and mediastinal disorders Cough	10.0% 3/30 • Number of events 4 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Respiratory, thoracic and mediastinal disorders Dyspnoea	6.7% 2/30 • Number of events 2 • Adverse event data were collected from screening through Week 24.	0.00% 0/32 • Adverse event data were collected from screening through Week 24.
Skin and subcutaneous tissue disorders Rash	6.7% 2/30 • Number of events 2 • Adverse event data were collected from screening through Week 24.	3.1% 1/32 • Number of events 1 • Adverse event data were collected from screening through Week 24.
Vascular disorders Hypertension	10.0% 3/30 • Number of events 5 • Adverse event data were collected from screening through Week 24.	6.2% 2/32 • Number of events 3 • Adverse event data were collected from screening through Week 24.