Neoplasms

Nervous System Diseases

Immune System Diseases

All Conditions

Musculoskeletal Diseases

Symptoms and General Pathology

Rare Diseases

Myasthenia Gravis

Neuromuscular Diseases

Muscle Weakness

Paresis

Asthenia

Muscular Diseases

Neuromuscular Manifestations

Neurologic Manifestations

Syndrome

Paraneoplastic Syndromes

Paraneoplastic Syndromes, Nervous System

Nervous System Neoplasms

Autoimmune Diseases

Autoimmune Diseases of the Nervous System

Neurodegenerative Diseases

Neuromuscular Junction Diseases

All Drugs and Chemicals

Antibodies

Immunoglobulins

Immunoglobulins, Intravenous

gamma-Globulins

Rho(D) Immune Globulin

Immunologic Factors

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). Participants will receive Hizentra in a minimum of one infusion per week and a maximum of 4 infusions per week. Dose and rate depend on the visit and how each participant tolerates the drug. Max cc per site is 50 cc per site per hour.

Patients must fulfill inclusion criteria and remain stable at week 0, which means QMG does not increase by 3 points, will enter receive Hizentra for 12 weeks.

HIZENTRA ®

Mazen M Dimachkie, MD

The purpose of this study is to determine whether Hizentra is a safe and effective treatment for people with myasthenia gravis (MG).

Myasthenia gravis (MG) is a rare autoimmune disorder which causes the muscles to become weak because the immune system attacks the connection between the nerves and the muscles.

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). An immunoglobin is a blood protein. Hizentra is being studied for the treatment of patients with MG. Hizentra is administered by an injection into the skin through a portable infusion pump, which may be easier for patients to administer than the current treatments.

Participants will be asked to complete 9 clinic visits and 3 telephone calls. It could take up to 30 weeks to complete all study visits.

Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

The QMG is a 13 item ordinal scale which measures ocular, bulbar, extremity fatigue and strength, along with respiratory function. The scale is from 0 - 3 for each item, with 0 meaning normal and 3 is severe. Total score can range from 0 to 39.

Change in MG severity will be measured using the Quantitative Myasthenia Gravis (QMG) Score for Disease severity. The QMG is a validated clinical composite scale. As mentioned in the protocol, our hypotheses are:

H0: Proportion of patients whose QMG scores are increased by more than 3 points at the end of the SCIg treatment phase ≤ 0.65 HA: Proportion of patients whose QMG scores are increased by no more than 3 points at the end of the SCIg treatment phase \> 0.65

Thus, analysis of the primary outcome is done as a one-sample Z test of proportions. That is, the QMG is a continuous outcome, but analyses results are reported as proportions.

Myasthenia Gravis-specific Activities of Daily Living scale (MG-ADL): Composite measure of scores from measurement scales. The MG-ADL has a scale of 0 - 24 with 0 being the lowest (no symptoms) and 24 being the highest (most severe symptoms. The MG-ADL is a staff-administered, patient-reported questionnaire that measures 8 commons symptoms of myasthenia gravis and grades them on a scale of 0 - 3.

MG Quality of Life (QOL)-15: Composite measure of scores from measurement scales. The MG QOL-15 is a questionnaire answered by the patient that asked about different symptoms of MG. The questionnaire consists of 15 questions that are graded on a scale of 0 - 4. The total score has a range of 0 - 60 with a higher score meaning more severe symptoms or a worse outcome.

The MGC takes scores from the MG-ADL, the QMG, and combines them will manual muscle testing scores to create the MGC. The scale of this score ranges from 0 - 50 with higher scores meaning a worse outcome or more sever symptoms.

Treatment Satisfaction Questionnaire for Medication (TSQM) - Convenience Score measured on a scale of 0 to 100. 0 indicates no treatment convenience satisfaction and 100 indicates highest treatment convenience satisfaction.

Treatment Satisfaction Questionnaire for Medication (TSQM) - Effectiveness Score measured on a scale of 0 to 100. 0 indicates no treatment effectiveness satisfaction and 100 indicates highest treatment effectiveness satisfaction.

Treatment Satisfaction Questionnaire for Medication (TSQM) - Satisfaction Score measured on a scale of 0 to 100. 0 indicates no treatment satisfaction and 100 indicates highest treatment satisfaction.

Measure IgG level (mg/dL) between intravenous and subcutaneous study phases. Normal range equals 762-1488 mg/dL.

Tolerability is assessed as the number of subjects who completed the study and/or did not withdraw due to worsening.

CSL Behring

Mazen Dimachkie, MD

Professor

Experimental Treatment arm: Privigen/Hizentra assigned to subjects who are stable in the screening phase of the study.

Privigen/Hizentra

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). Participants will receive weekly Hizentra. Dose and rate depend on the visit and how each participant tolerates the drug. Max flow rate not to exceed 100 mL per hour.

HIZENTRA ®

Hizentra

Age, Categorical

Age, Continuous

Sex: Female, Male

Race

Race/Ethnicity, Customized

Canada

United States

Region of Enrollment

The QMG is a clinical composite scale administered by a physician or trained clinical evaluator. The QMG scores 13 different assessments that align with common symptoms of MG. Each item is graded on a scale of 0 - 3. The QMG has a total score of 0 - 39 with 0 being the best (no symptoms) and 39 being the worst (severe symptoms).

As per the protocol, only those patients who completed the screening phase are eligible for analysis. In our case this is 22 out of 23 patients.

Quantitative Myasthenia Gravis test at Baseline

Dr. Mazen Dimachkie

Sample size/power calculations are mentioned in the study protocol.

The study hypotheses are as follows:

H0: Proportion of patients whose QMG scores are increased by no more than 3 points at the end of the SCIg treatment phase ≤ 0.65 HA: Proportion of patients whose QMG scores are increased by no more than 3 points at the end of the SCIg treatment phase \> 0.65

23 patients were enrolled in the study. The protocol mentions that only those subjects who have stable QMG in the screening phase will be considered eligible for analyses. In our case 22 out of 23 patients are eligible for the final analyses.

Proportion of Patients Whose Quantitative Myasthenia Gravis Scores Are Increased by no More Than 3 Points at the End of the SCIg Treatment Phase

Week 12

Baseline

Here we consider the 22 participants on whom the MG-ADL scores at either baseline or Week 12 are available for analysis.

Myasthenia Gravis-specific Activities of Daily Living Scale (MG-ADL) Scores

Myasthenia Gravis Quality of Life (MG QOL-15) Scores

Here we consider the 21 participants on whom the MG-Composite scores are available for analysis.

Myasthenia Gravis Composite (MGC) Score

This secondary outcome was recorded for 18 patients. Analysis is done as per the protocol.

Treatment Satisfaction Questionnaire for Medication (TSQM) - Convenience Score

This secondary outcome was recorded for 20 patients. Analysis is done as per the protocol.

Treatment Satisfaction Questionnaire for Medication (TSQM) - Effectiveness Score

Treatment Satisfaction Questionnaire for Medication (TSQM) - Satisfaction Score

Change: Week -10 to Week 0

Change: Week 1 to Week 12

Note: Change in IgG from Week -10 to Week 0 can be assessed for 20 participants. Change in IgG from Week 1 to Week 12 can be assessed for 19 participants.

Immunoglobulin G (IgG) Antibody Levels

Hizentra is a subcutaneous (under the skin) immunoglobin (SCIg). Participants will receive Hizentra in a minimum of one infusion per week and a maximum of 4 infusions per week. Dose and rate depend on the visit and how each participant tolerates the drug. Max cc per site is 50 cc per site per hour.

HIZENTRA ®: Patients must fulfill inclusion criteria and remain stable at week 0, which means QMG does not increase by 3 points, will enter receive Hizentra for 12 weeks.

Tolerabililty

Privigen: 3 rounds of IV privigen was infused every 4 weeks until baseline visit week 0.

Hizentra: Hizentra infused sub cutaneously based on weight and subject prescribed dose.

Spots Global Cancer Trial Database for Open Label Study of Subcutaneous Immunoglobulin (SCIg) in Myasthenia Gravis

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

Useful links and downloads for this trial