This is a Phase II, open-label, multicenter study to evaluate the safety and efficacy of NM8074 administered via intravenous infusion in patients with Dermatomyositis (DM).
The Proposed Study, NM8074-DM-701 will enroll a planned number of eight (8) DM subjects, with the potential to enroll more patients. The total duration of the study for all subjects will include a 30-day screening period, dosing for a 12-week Treatment Period, followed by an observation period of 6-weeks. All subjects will be administered 20 mg/kg of NM8074 intravenously every week for a total of 12 doses from Day 1 to Day 78 of the Treatment Period.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
8
NM8074 will be administered as an intravenous infusion at a dose of 20mg/kg
Change from Baseline or Percent Change from Baseline in the number of patients whose Total Improvement Score (TIS) has increased by ≥ 20
TIS is a composite measure used in clinical trials that integrates the 6 Core Set Measures (CSM). TIS scores range from 0-100, with 0-19, 20-39, 40-59, and 60-100 indicating no improvement, minimal, moderate, and major improvement respectively.
Time frame: Up to Study Day 78
Change from baseline or Percent Change from Baseline in Cutaneous Dermatomyositis Area and Severity Index (CDASI) score
In DM, CDASI is a visual tool for a physician to assess the activity and damage of skin involvement in DM patients. The total activity score ranges from 0 to 100, and the damage score ranges from 0 to 32, wherein both criteria the higher score indicates more severe activity or damage of the disease in the patient.
Time frame: Up to Study Day 78
Change from Baseline or Percent Change from Baseline in Manual Muscle Testing (MMT-8) parameter
In Manual Muscle testing (MMT-8) eight specific muscle groups strengths are evaluated, each muscle group is scored on a scale from 0 (no movement) to 10 (normal strength), resulting in a maximum total score of 150 with a higher score indicating better muscle strength. Where a score \< 136 would indicate muscle weakness significant enough to classify the patient as at risk of DM. MMT-8 can contribute between 0 to 32.5 points to the TIS: If there is a significant improvement in the patient's condition of more than 30% from baseline, the maximum score of 32.5 points is awarded. If the condition worsens or shows only a slight improvement (up to 2% from baseline), then 0 points are given.
Time frame: Up to Study Day 78
Change from Baseline or Percent Change from Baseline in Physician's Global Activity (PhGA) assessment
The PhGA is scored on a scale from 0 (no evidence of disease activity) to 10 (extremely severe disease), with higher scores indicating greater disease severity.
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Time frame: Up to Study Day 78
Change from Baseline or Percent Change from Baseline in Patient's Global Activity (PtGA) assessment
The PhGA is scored on a scale from 0 (no evidence of disease activity) to 10 (extremely severe disease), with higher scores indicating greater disease severity.
Time frame: Up to Study Day 78
Change from Baseline or Percent Change from Baseline Health Assessment Questionnaire (HAQ) score
HAQ can contribute between 0 to 10 points to the TIS: \- If there is a significant improvement in the patient's condition of more than 40% from baseline, the maximum score of 10 points is awarded. If the condition worsens or shows only a slight improvement (up to 5% from baseline), then 0 points are given.
Time frame: Up to Study Day 78
Change from Baseline or Percent Change from Baseline in Extra muscular Activity (MDAAT) score
Extra-muscular Activity can contribute between 0 to 20 points to the TIS: \- If there is a significant improvement in the patient's condition of more than 40% from baseline, the maximum score of 20 points is awarded. If the condition worsens or shows only a slight improvement (up to 5% from baseline), then 0 points are given.
Time frame: Up to Study Day 78