A Pilot Trial To Assess The Feasibility And Efficacy Of SCIG In Patients With MG Exacerbation (SCIG-MG) (SCIG-MG)
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|ClinicalTrials.gov Identifier: NCT02774239|
Recruitment Status : Recruiting
First Posted : May 17, 2016
Last Update Posted : August 28, 2017
This is a prospective open-label, uncontrolled, single-blind, pilot clinical trial.
The primary objective is to assess the efficacy, safety, feasibility and tolerability of SCIG in patients with worsening MG.
Participants with moderate worsening of MG symptoms (MGFA Class II and III) who are considered to be appropriate for immunoglobulin therapy will be screened for the study by the treating neurologist.
Patients will be receive 2gm/kg (150gm for a 75kg patient) of 20% SCIG (Hizentra) infused over 4 weeks in a dose escalating manner.
Additionally, this study will be assessing the feasibility of employing SCIG as an alternative therapy to IVIG in patients with MG exacerbation. The cost-effectiveness of SCIG versus IVIG will be evaluated, and the impact of SCIG therapy will be assessed from both a health-resource perspective and from a patient perspective.
|Condition or disease||Intervention/treatment||Phase|
|Myasthenia Gravis||Drug: Human normal immunoglobulin G (IgG)||Phase 3|
Show Detailed Description
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||30 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Pilot Trial To Assess The Feasibility And Efficacy Of Subcutaneous Immunoglobulin In Patients With Myasthenia Gravis Exacerbation|
|Study Start Date :||October 2014|
|Estimated Primary Completion Date :||October 2018|
|Estimated Study Completion Date :||December 2018|
U.S. FDA Resources
Experimental: SC Treatment Period
Participants will receive 2gm/kg of Human normal immunoglobulin G (IgG) infused over 4 weeks in a dose escalating manner as follows:
Drug: Human normal immunoglobulin G (IgG)
Study medication is available in the following forms: 5 mL (1 g IgG) in a 5 mL infusion bottle, 10 mL (2 g IgG) in a 10 mL infusion bottle, 20 mL (4 g IgG) in a 20 mL infusion bottle, 50 mL (10 g IgG) in a 50 mL infusion bottle.
Patients will be receiving 2gm/kg (150gm for a 75kg patient) of 20% SCIG (Hizentra) infused over four weeks in a dose escalating manner.
Other Name: Hizentra®
- Change in Quantitative Myasthenia Gravis Score (QMGS) from baseline to day 42 after treatment. [ Time Frame: Baseline - Day 42 ]QMGS is a validated clinical measure of myasthenia gravis ranging from 0 points (no myasthenic weakness) to a maximum of 39 points, with a defined change of 3.4 units required for clinical significance.
- Quality of life will be assessed through the Quality of Life (QOL) score, a qualitative questionnaire. [ Time Frame: Baseline - Day 42 ]
- Change in Manual Muscle Testing (MMT) score from baseline to day 42 after treatment. [ Time Frame: Baseline - Day 42 ]MMT is a validated clinical measure of myasthenia gravis ranging from 0 points (no myasthenic weakness) to a maximum of 128 points, with a defined change of 25% from baseline required for clinical significance.
- Adverse events related to SCIG infusions will be recorded if/when they occur. [ Time Frame: Baseline - Day 42 ]
- Patient satisfaction with the treatment modality will be assessed using a questionnaire. [ Time Frame: Day 42 ]TQSM: Treatment Satisfaction Questionnaire for Medication. An 11 question ordinal scale measuring responses from "Extremely satisfied" to "Extremely dissatisfied" over a 7 item option list. Questions cover four domains, corresponding to distinct aspects related to the satisfaction of patients with their treatment (Effectiveness; Side effects; Convenience and Global satisfaction). A score can be obtained for each domain by summing of the corresponding items transformed on a 0-100 scale; higher values indicate higher satisfaction, better perceived effectiveness, lower burden associated to side-effects, better convenience
- Serious Adverse Events related to SCIG infusions will be recorded if/when they occur. [ Time Frame: Baseline - Day 42 ]
- Proportion of participants successfully trained will be recorded indicating feasibility based on patient compliance. [ Time Frame: Baseline - Week 2 ]
- Proportion of participants completing will be recorded indicating feasibility based on patient compliance. [ Time Frame: Baseline - Day 42 ]
- Myasthenia Gravis (MG) Composite scores will be used to evaluate disease severity through a number of functional assessments, including muscle strength and ability to complete activities of daily living. [ Time Frame: Baseline - Day 42 ]
- Cost effectiveness of SCIG therapy will be calculated in comparison to standard IVIG therapy. This will indicate health economics. [ Time Frame: Baseline - Day 42 ]
- Change in QOL 36 score from Baseline to day 42) [ Time Frame: Baseline - Day 42 ]Change in Quality of Life (QOL) score from Baseline to day 42.
- Time required for the treatments. [ Time Frame: Baseline - Day 42 ]Average time in minutes required for each infusion session during study. Determined for each participant.
- Preference for treatments (Treatment Satisfaction Questionnaire for Medication (TSQM). [ Time Frame: Day 42 ]11 item scale measuring treatment satisfaction for medication over 3 domains (Effectiveness, Side effects, Convenience, Global Satisfaction). Score from 10 to 43
- Intent to continue using SCIG if necessary (Via pt. interview) [ Time Frame: Day 42 ]Yes= will continue SCIG therapy, No= will not continue SCIG therapy.
- Proportion of participants successfully trained in self-administration of SCIG. [ Time Frame: Day 2, Day 3. ]Proportion of participants who successfully trained in self-administration of SCIG expressed as a percentage (completed training/underwent training x 100).
- Proportion of participants completing the study. [ Time Frame: Day 42 ]Proportion of patients who completed the study expressed as a percentage (finished study/started study x 100).
- Infusion nurse's assessment of this participant population's ability to use this treatment modality (Written impression) [ Time Frame: Day 42 ]Qualitative measure. Narrative reflecting the infusion nurse's overall impression of target population's ability to employ SCIG within the parameters defined by the study. (data is a written narrative. By definition, this has no units nor is it collected by any instrument (questionnaire, scale parameter ect.) ie. QUALITATIVE data)
- Speed of treatment onset of SCIG. [ Time Frame: Day 1 ]The number of days between prescription of SCIG treatment and infusion of first dose.
- Speed of treatment onset of IVIG. [ Time Frame: Day 1 ]The number of days between prescription of IVIG treatment and infusion of first dose, for any study participant who has received IVIG treatments in the past. This interval will be determined for all available previous IVIG infusions for each patient.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02774239
|Contact: Derrick G Blackmore, B.Scfirstname.lastname@example.org|
|Contact: Ashley K Mallon, LPNemail@example.com|
|University of Alberta||Recruiting|
|Edmonton, Alberta, Canada, T6G 2B7|
|Contact: Zaeem A Siddiqi, MD, PhD 780-407-3367 firstname.lastname@example.org|
|Contact: Ashley K Mallon, LPN 780-407-6582 email@example.com|
|Principal Investigator: Zaeem A Siddiqi, MD, PhD|
|Canada, British Columbia|
|Vancouver General Hospital - Gordon & Leslie Diamond Health Care Centre||Recruiting|
|Vancouver, British Columbia, Canada, V5Z 1M9|
|Contact: Deborah Kraus, RN 604-875-4111 ext 22950 Deborah.Kraus@vch.ca|
|Principal Investigator:||Zaeem A Siddiqi, MD, PhD||University of Alberta|