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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02271165
• Date of registration: 15/10/2014
• Prospective Registration: Yes
• Primary sponsor: Thomas Jefferson University
• Public title: Subcutaneous Immunoglobulin (Hizentra) in Patients With Dermatomyositis: A Proof of Concept Study
• Date of first enrolment: November 2014
• Target sample size: 2
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT02271165
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Early Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Dermatomyositis

Intervention(s)

Drug: Immunoglobulin (Hizentra)

Primary Outcome(s)

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(s)

The main secondary outcome is the preference of the participant for SCIg compared with IVIg [Time Frame: The main secondary outcome is the preference of the participant for SCIg compared with IVIg]

Secondary ID(s)

13P.192

Source(s) of Monetary Support

Please refer to primary and secondary sponsors