Subcutaneous Immunoglobulin for Myasthenia Gravis

NCT04728425 | Completed Phase 2 FDA Drug

• 1 other identifier: 19-5378.2
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

This is a prospective open-label, randomized, parallel arm clinical trial. The primary objective of the study is to evaluate the safety and efficacy of Cuvitru 20% subcutaneous immunoglobulin in patients with myasthenia gravis (MG). The secondary objective is to evaluate patient preferences and effects on quality of life when treating MG patients with SCIG. Exploratory objectives are to compare de novo administration starting SCIG directly with those starting with a loading dose of IVIG followed by SCIG administration. Patients over age 18 with moderate to severe MG with MGFA Class II-IV without contraindications to immunoglobulin will be considered for the study. All patients will be eligible to enter either arm of the study, Arm 1: 10% Gammagard IVIG followed by 20% Cuvitry SCIG and Arm 2: Cuvitru 20% SCIG alone.

Conditions

Myasthenia Gravis

Keywords

myasthenia gravis; IVIG; SCIG; subcutaneous immunoglobulin; intravenous immunoglobulin; MG; neuromuscular junction

Outcome Measures

Primary Outcomes (1)

• Myasthenia Gravis Impairment Index Efficacy Outcome

  Change in the Myasthenia Gravis Impairment Index (score 0-84, with 84 being the maximal score indicating the most severe MG status) at baseline compared to end of study

  6 months

Secondary Outcomes (6)

• Quantitative Myasthenia Gravis Score Efficacy Outcome

  6 months

• Myasthenia Gravis Activities of Daily Living Efficacy Outcome

  6 months

• Myasthenia Gravis Composite Efficacy Outcome

  6 months

• Myasthenia gravis Quality of Life 15 Score Efficacy Outcome

  6 months

• Parallel Arm Comparison

  6 months

Study Arms (2)

IVIG + SCIG

ACTIVE COMPARATOR

This group of MG patients will start with 2g/kg of IVIG on month 1, 1 g/kg of IVIG 4 and then 8 weeks later, and within 2 weeks switch to SCIG treatment

Drug: intravenous immunoglobulin + subcutaneous immunoglobulin (SCIG)

SCIG alone

ACTIVE COMPARATOR

This group of MG patients will start with SCIG alone

Drug: subcutaneous immunoglobulin (SCIG)

Interventions

subcutaneous immunoglobulin (SCIG) DRUG

20% subcutaneous immunoglobulin

Also known as: Cuvitru

SCIG alone

intravenous immunoglobulin + subcutaneous immunoglobulin (SCIG) DRUG

10% intravenous immunoglobulin + 20% subcutaneous immunoglobulin

Also known as: Gammanorm + Cuvitru

IVIG + SCIG

Eligibility Criteria

• Age: 18 Years - 100 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients over age 18
• Patients with a confirmed diagnosis of myasthenia gravis based on clinical criteria including fatiguable weakness and supported by either serological (acetylcholine receptor, muscle specific kinase or anti-low-density lipoprotein receptor- related protein 4 antibodies or electrophysiological testing (repetitive nerve stimulation of single fiber electromyography)
• Myasthenia Gravis Federation of America class II-IV
• Moderate to severe myasthenia gravis as defined by a quantitative myasthenia gravis score \>10 or generalized myasthenia gravis impairment index score \> 11
• Patient able to give consent and is able and willing to complete all study procedures and activities

You may not qualify if:

• Patients who are pregnant or breastfeeding
• Patients not able to complete the study procedures or with an alternate diagnosis
• Patients with recent thymectomy in the past 6 months
• Patients receiving another biologic agent such as rituximab, belimubab, cyclophosphamide and eculizumab in the past 6 months prior to study entry
• No IVIG or subcutaneous immunoglobulin within the past month
• Patients on prednisone who have had alterations in prednisone dose over the past month prior to study entry
• Patients will previous known allergy or severe adverse reaction to intravenous or subcutaneous immunoglobulin
• Evidence of renal insufficiency (Cr\>1.5 x elevated) or liver disease (transaminases \> 2.5 x elevation) at screening.

Sponsors & Collaborators

• University Health Network, Toronto: lead

Study Sites (1)

University Health Network

Toronto, Ontario, M5G 2C4, Canada

Location

MeSH Terms

Conditions

Myasthenia Gravis

Interventions

Hizentra; Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Paraneoplastic Syndromes, Nervous System; Nervous System Neoplasms; Neoplasms by Site; Neoplasms; Paraneoplastic Syndromes; Autoimmune Diseases of the Nervous System; Nervous System Diseases; Neurodegenerative Diseases; Neuromuscular Junction Diseases; Neuromuscular Diseases; Autoimmune Diseases; Immune System Diseases

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Hans Katzberg, MD

  University Health Network, Toronto

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Neurologist, Principle Investigator

Study Record Dates

• First Submitted: July 14, 2020
• First Posted: January 28, 2021
• Study Start: August 28, 2020
• Primary Completion: June 30, 2023
• Study Completion: December 30, 2023
• Last Updated: April 27, 2025

Record last verified: 2025-04

Locations

CA (1)