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

Primary Purpose

Dermatomyositis

Status
Terminated
Phase
Early Phase 1
Locations
United States
Study Type
Interventional
Intervention
Immunoglobulin (Hizentra)
Sponsored by
Thomas Jefferson University
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Dermatomyositis

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  1. Men or woman aged >18 years
  2. Diagnosis of DM based on standard criteria
  3. Receiving the equivalent of at least 0.4 g/kg IVIg every 4 weeks (IVIg group only)
  4. 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)
  5. 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)
  6. Stable dosing with steroids and/or other immunosuppressives for 12 weeks with no changes schedule or intended.

Exclusion Criteria:

  1. Pregnancy, planned pregnancy, breast feeding or unwillingness to practice contraception
  2. Severe concurrent medical conditions which would prevent treatment or assessment, including significant hematological, renal or liver dysfunction or malignancies
  3. 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.
  4. Participation in trial of an investigational medicinal product in the past 12 weeks
  5. 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.

Sites / Locations

  • Thomas Jefferson University

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Active Comparator

Arm Label

IVIg naive

non-IVIg naive

Arm Description

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.

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).

Outcomes

Primary Outcome Measures

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 Outcome Measures

The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Secondary outcome measures will be a) patient preference, based on the number of participants who prefer SCIg to IVIg (a home-made questionnaire will be utilized to capture preference in the most unbiased way; b) average change in Quality of life scores between week 12 and 28; c) effect on the complement consumption in serum; d) Immunological parameters on the repeated skin biopsies; and e) adverse events as reported at each visit. Accordingly, information will be obtained on the superiority of SCIg vs. IVIg

Full Information

First Posted
October 15, 2014
Last Updated
May 10, 2018
Sponsor
Thomas Jefferson University
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1. Study Identification

Unique Protocol Identification Number
NCT02271165
Brief Title
Subcutaneous Immunoglobulin (Hizentra) in Patients With Dermatomyositis: A Proof of Concept Study
Study Type
Interventional

2. Study Status

Record Verification Date
May 2018
Overall Recruitment Status
Terminated
Why Stopped
Difficult recruitment
Study Start Date
November 2014 (undefined)
Primary Completion Date
March 2, 2017 (Actual)
Study Completion Date
March 2, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Thomas Jefferson University

4. Oversight

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Dermatomyositis

7. Study Design

Primary Purpose
Treatment
Study Phase
Early Phase 1
Interventional Study Model
Parallel Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Arm Title
IVIg naive
Arm Type
Active Comparator
Arm Description
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.
Arm Title
non-IVIg naive
Arm Type
Active Comparator
Arm Description
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).
Intervention Type
Drug
Intervention Name(s)
Immunoglobulin (Hizentra)
Other Intervention Name(s)
Immunoglobulin subcutaneous (human) 20% liquid, Hizentra
Primary Outcome Measure Information:
Title
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.
Description
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.
Time Frame
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 Outcome Measure Information:
Title
The main secondary outcome is the preference of the participant for SCIg compared with IVIg
Description
Secondary outcome measures will be a) patient preference, based on the number of participants who prefer SCIg to IVIg (a home-made questionnaire will be utilized to capture preference in the most unbiased way; b) average change in Quality of life scores between week 12 and 28; c) effect on the complement consumption in serum; d) Immunological parameters on the repeated skin biopsies; and e) adverse events as reported at each visit. Accordingly, information will be obtained on the superiority of SCIg vs. IVIg
Time Frame
The main secondary outcome is the preference of the participant for SCIg compared with IVIg

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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.
Facility Information:
Facility Name
Thomas Jefferson University
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19107
Country
United States

12. IPD Sharing Statement

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

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