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A Pilot Trial To Assess The Feasibility And Efficacy Of SCIG In Patients With MG Exacerbation (SCIG-MG) (SCIG-MG)

This study is currently recruiting participants.
Verified August 2017 by Zaeem Siddiqi, University of Alberta
Sponsor:
ClinicalTrials.gov Identifier:
NCT02774239
First Posted: May 17, 2016
Last Update Posted: August 28, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
CSL Behring
Information provided by (Responsible Party):
Zaeem Siddiqi, University of Alberta
  Purpose

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 Intervention Phase
Myasthenia Gravis Drug: Human normal immunoglobulin G (IgG) Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Pilot Trial To Assess The Feasibility And Efficacy Of Subcutaneous Immunoglobulin In Patients With Myasthenia Gravis Exacerbation

Resource links provided by NLM:


Further study details as provided by Zaeem Siddiqi, University of Alberta:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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 ]

Other Outcome Measures:
  • 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.


Estimated Enrollment: 30
Study Start Date: October 2014
Estimated Study Completion Date: December 2018
Estimated Primary Completion Date: October 2018 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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:

  • 1st week: 2-3 SCIG infusions of 10ml per site at four sites (total dose 16 to 24g)*
  • 2nd week: 2-3 SCIG infusions of 15ml per site at four sites (total dose 24 to 36g)*
  • 3rd week: 2-4 SCIG infusions of 20ml per site at four sites (total dose 32 to 64g)*
  • 4th week: 2-4 SCIG infusions of 25ml per site at four sites (total dose 40 to 80g)*

    • Doses indicated are study recommended. Doses may be adjusted depending on tolerance and total dose required by the patient.
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®

  Show Detailed Description

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   18 Years to 80 Years   (Adult, Senior)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patients between18 to 80 years of age, diagnosed with MG (see below) who have worsening myasthenic symptoms - (defined as increasing diplopia, ptosis, dysarthria, dysphagia, difficulty chewing, or limb weakness severe enough to warrant immunoglobulin therapy.
  • MG diagnosis will be based upon the clinical evaluation by a neuromuscular expert and meeting any two of the following supportive criteria:

    1. Abnormal Tensilon test
    2. Abnormal repetitive nerve stimulation studies
    3. Abnormal single fiber electromyography (EMG)
    4. Increased serum acetylcholine receptor or anti-MuSK antibodies
    5. Prior response to immunotherapy

Exclusion Criteria:

  1. Respiratory distress requiring ICU admission or a vital capacity <1 L
  2. Severe swallowing difficulties with a high risk of aspiration
  3. Change in corticosteroid dosage in the 4 weeks prior to screening
  4. Known immunoglobulin A (IgA) deficiency
  5. Pregnant or breast feeding women
  6. Active renal or hepatic insufficiency, clinically significant cardiac disease
  7. Patients with worsening weakness associated with an infectious process
  8. Previous lack of responsiveness to IVIG
  9. History of previous MG crises
  Contacts and Locations
Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02774239


Contacts
Contact: Derrick G Blackmore, B.Sc 780-407-3367 dblackmo@ualberta.ca
Contact: Ashley K Mallon, LPN 780-407-6582 akmallon@ualberta.ca

Locations
Canada, Alberta
University of Alberta Recruiting
Edmonton, Alberta, Canada, T6G 2B7
Contact: Zaeem A Siddiqi, MD, PhD    780-407-3367    zsiddiqi@ualberta.ca   
Contact: Ashley K Mallon, LPN    780-407-6582    akmallon@ualberta.ca   
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   
Sponsors and Collaborators
University of Alberta
CSL Behring
Investigators
Principal Investigator: Zaeem A Siddiqi, MD, PhD University of Alberta
  More Information

Responsible Party: Zaeem Siddiqi, Dr. Zaeem Siddiqi, University of Alberta
ClinicalTrials.gov Identifier: NCT02774239     History of Changes
Other Study ID Numbers: ZS2013-01
First Submitted: April 20, 2015
First Posted: May 17, 2016
Last Update Posted: August 28, 2017
Last Verified: August 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

Additional relevant MeSH terms:
Neuromuscular Junction Diseases
Neuromuscular Diseases
Myasthenia Gravis
Autoimmune Diseases of the Nervous System
Nervous System Diseases
Autoimmune Diseases
Immune System Diseases
Immunoglobulins
Antibodies
Immunoglobulin G
Immunologic Factors
Physiological Effects of Drugs