A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of IgPro20 (Subcutaneous Immunoglobulin, Hizentra®) in Adults with Dermatomyositis (DM) - The RECLAIIM Study
- Conditions
- dermatomyositismuscle disease1002839310014982
- Registration Number
- NL-OMON55228
- Lead Sponsor
- CSL Behring LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 0
• Capable of providing written informed consent by signing an informed consent
form and willing and able to adhere to all protocol requirements
• Age >= 18 years
• Diagnosis of at least probable idiopathic inflammatory myopathies per
European League
Against Rheumatism/American College of Rheumatology (EULAR/ACR) Classification
Criteria: minimum aggregate score of 5.5 without and 6.7 with muscle biopsy
(historical muscle biopsy is acceptable) which includes confirmation of DM rash
/ skin manifestation (present or by history [historical skin biopsy is required
for amyopathic DM subjects])
• Disease activity defined by:
• Presence of DM rash / skin manifestation (eg, Gottron's papules / sign,
heliotrope rash, periorbital edema, V sign, Shawl sign) at Screening Visit OR
• One objective disease activity measure within 3 months before Baseline:
o Magnetic resonance imaging showing active inflammation (edema) of a proximal
skeletal muscle - OR -
o Electromyogram showing acute changes such as spontaneous activity not
explained by other disease - OR -
o Muscle biopsy with perivascular or perimysial inflammation - OR -
o CK > 4 x upper limit of normal (ULN)
• Disease severity defined by a minimum value of 2 cm on a 10 cm Physician
Global Disease Activity Visual Analog Scale and:
o MMT-8 <= 142 OR
o CDASI total activity score >= 14
• Subject has failed other DM treatment or is on DM treatment such as
immunosuppressants and/or antimalarials on a stable dose >= 3 months before
Baseline; and/or oral corticosteroids (<= 20 mg/day prednisolone equivalent
and/or topical) on a stable dose >= 1 month before Baseline
• Cancer-associated myositis, defined as the diagnosis of myositis within 2
years of the diagnosis of cancer
• Evidence of malignancies diagnosed within the previous 5 years. Note:
Subjects with a history of carcinoma in situ of the cervix that has been
excised and cured with >= 5 years since excision or subjects with documented
history of treated basal or squamous cell skin cancer may be enrolled into the
study..
• Physician Global Damage Assessment >= 3 on a 5-point Likert scale where a
score of 3 represents severe damage
• Clinically relevant improvement between Screening Visit and Baseline, defined
by >= 2 cm improvement on a 10 cm Physician Global Disease Activity Assessment
Visual Analog Scale
• Known or suspected hypersensitivity or other severe reactions to IgPro20 or
to any of its excipients, or other immunoglobulins (Igs)or severe reactions to
blood products
• Other significant medical conditions that could increase the risk to the
subject, eg:
o History of allogeneic bone marrow / stem cell transplant / solid organ
transplant
o Cardiac insufficiency (New York Heart Association Class III or IV) or
unstable ischemic heart disease
o Chronic kidney disease stage IV or V
o Recent surgery requiring general anesthesia within the previous 4 weeks
before Screening
o Known hyperprolinemia type I or type II
o Documented thrombophilic abnormalities including blood hyperviscosity,
protein C or protein S deficiency, anti-thrombin-III deficiency, plasminogen
deficiency, antiphospholipid antibodies, Factor V Leiden mutation,
dysfibrinogenemia, or prothrombin G20210A mutation
o History of documented thrombotic episode, eg, pulmonary embolism, deep vein
thrombosis, myocardial infarction, or thromboembolic stroke at any time
o More than 3 of the following specified risk factors for thromboembolic events
(documented and current conditions) occurring concurrently: atrial
fibrillation, coronary disease, diabetes mellitus, dyslipidemia, hypertension,
obesity (body mass index >= 30 kg/m2), recent significant trauma and immobility
(wheelchairbound or bedridden)
o Uncontrolled, severe, or rapidly progressive interstitial lung disease which
will prevent the subject from successful participation in the study
o Severe skin disease at planned infusion sites that would make subcutaneous
(SC) infusions infeasible
o Medical conditions whose symptoms and effects could alter protein catabolism
and or Immunoglobulin G (IgG) utilization (eg, protein-losing enteropathies,
nephrotic syndrome, known Immunoglobulin A [IgA] deficiency with antibodies to
Immunoglobulin A)
• Other conditions which would prevent correct assessment or lead to impaired
muscle strength (eg, other neurological disorders including, but not limited
to, Parkinson*s disease or severe musculoskeletal conditions like severe
osteoarthritis or deformities)
• Laboratory exclusions at Screening:
o positive result for any of the following human immunodeficiency virus (HIV),
hepatitis B virus (HBV), hepatitis C virus (HCV)
o Creatinine > 1.5 × ULN or Blood Urea Nitrogen (BUN) > 3 × ULN
• Any of the following therapies:
o Within 1 month before Baseline: intramuscular, intravenous, or
intra-articular corticosteroids including adrenocorticotropic hormone (any
dose), doses > 20 mg/day prednisolone equiv
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary endpoint is the responder status based on the total Improvement<br /><br>Score (TIS) assessments at Weeks 17, 21, and 25.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Key secondary endpoints of the study are:<br /><br>* TIS at Week 25<br /><br>* Change from Baseline in Manual Muscle Testing (MMT-8) at Week 25<br /><br>* Change from Baseline in Cutaneous Dermatomyositis Disease Area and Severity<br /><br>Index (CDASI) total activity score at Week 25<br /><br>* Reduction of oral corticosteroid dose at Week 25</p><br>