Subcutaneous Immunoglobulin (Hizentra) in Patients With Dermatomyositis: A Proof of Concept Study

NCT02271165 | Terminated Early Phase 1

• 1 other identifier: 13P.192
• Study Type: interventional
• Target: 2
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of the study is to evaluate the effectiveness and safety of human immunoglobulin SCIg in the form of Hizentra (Immune globulin Subcutaneous) in patients with Dermatomyositis. Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body) in patients with primary immunodeficiency. At present, patients with steroid resistant dermatomyositis can only be treated with IVIg (The healthy antibodies in IVIG can block the damaging antibodies that attack muscle and skin in dermatomyositis) treatment. An evaluation can then be made to see if SCIg is a suitable replacement and exerts immunomodulatory effect on complement antibodies.

Conditions

Dermatomyositis

Outcome Measures

Primary Outcomes (1)

• The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.

  The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.

  The primary outcome is a change in strength (based on MRC sumscores) from baseline (enrollment) to the mean change at weeks 16, 20, 24 and 28.

Secondary Outcomes (1)

• The main secondary outcome is the preference of the participant for SCIg compared with IVIg

  The main secondary outcome is the preference of the participant for SCIg compared with IVIg

Study Arms (2)

IVIg naive

ACTIVE COMPARATOR

Patients naïve to IVIg who have active disease responding to corticosteroids or are corticosteroid-dependent, will be also included. Before these patients enter the study, they will receive 3 monthly infusions of IVIg starting with the standard dose of 2gram/kg/month and followed with monthly maintenance of 1 or 2gram/kg according to their response.

Drug: Immunoglobulin (Hizentra)

non-IVIg naive

ACTIVE COMPARATOR

Participants already on IVIg will be observed for 12 weeks under their existing IVIg regimen and will undergo measurements of their muscle strength, skin changes, assessments of their daily activities and quality of life (QoL) every 4 weeks at scheduled visits for monthly maintenance IVIg infusions (weeks 0,4,8,12).

Drug: Immunoglobulin (Hizentra)

Interventions

Immunoglobulin (Hizentra) DRUG

Also known as: Immunoglobulin subcutaneous (human) 20% liquid, Hizentra

IVIg naive; non-IVIg naive

Eligibility Criteria

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

You may qualify if:

• Men or woman aged \>18 years
• Diagnosis of DM based on standard criteria
• Receiving the equivalent of at least 0.4 g/kg IVIg every 4 weeks (IVIg group only)
• Established response to IVIg or dependence on IVIg to maintain status established either by symptomatic worsening of condition at the end of the inter-dose interval for both groups or by worsening after reduction of the dose within the previous 12 months (IVIg group only)
• IVIg regimen stable for 12 weeks while on IVIg (minor changes are permitted provided that the dose change is 15% or less) (IVIg group only)
• Stable dosing with steroids and/or other immunosuppressives for 12 weeks with no changes schedule or intended.

You may not qualify if:

• Pregnancy, planned pregnancy, breast feeding or unwillingness to practice contraception
• Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
• Initiation or immunomodulatory treatment other than IVIg in the past 24 weeks or modification of immunomodulatory treatment other than IVIg in the past 12 weeks.
• Participation in trial of an investigational medicinal product in the past 12 weeks
• Presence of skin infection unrelated to dermatomyositis, severe skin involvement
• Presence of any other medical condition, which in the opinion of the investigator might interfere with performance or interpretation of this study.

Sponsors & Collaborators

• Thomas Jefferson University: lead

Study Sites (1)

Thomas Jefferson University

Philadelphia, Pennsylvania, 19107, United States

Location

MeSH Terms

Conditions

Dermatomyositis

Interventions

Immunoglobulins; Hizentra; Fluid Therapy

Condition Hierarchy (Ancestors)

Polymyositis; Myositis; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Connective Tissue Diseases; Skin and Connective Tissue Diseases; Skin Diseases

Intervention Hierarchy (Ancestors)

Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins; Drug Therapy; Therapeutics

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor of Neurology

Study Record Dates

• First Submitted: October 15, 2014
• First Posted: October 22, 2014
• Study Start: November 1, 2014
• Primary Completion: March 2, 2017
• Study Completion: March 2, 2017
• Last Updated: May 16, 2018

Record last verified: 2018-05

Locations

US (1)