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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | 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. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| NM8074 | Drug | NM8074 will be administered as an intravenous infusion at a dose of 20mg/kg |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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. | Up to Study Day 78 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Assessment for the presence of myositis-specific autoantibodies (MSA) | Up to Study Day 127 | |
| Change from Baseline or Percent Change from Baseline in Classical Pathway (CP) modulation | NM8074-mediated CP inhibition is measure via a complement CP ELISA-based assay measuring MAC formation. |
Inclusion Criteria:
Exclusion Criteria:
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| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Rekha Bansal | Contact | 216-440-2696 | clinicalsae@novelmed.com |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 29079590 | Background | Lundberg IE, Tjarnlund A, Bottai M, Werth VP, Pilkington C, Visser M, Alfredsson L, Amato AA, Barohn RJ, Liang MH, Singh JA, Aggarwal R, Arnardottir S, Chinoy H, Cooper RG, Danko K, Dimachkie MM, Feldman BM, Torre IG, Gordon P, Hayashi T, Katz JD, Kohsaka H, Lachenbruch PA, Lang BA, Li Y, Oddis CV, Olesinska M, Reed AM, Rutkowska-Sak L, Sanner H, Selva-O'Callaghan A, Song YW, Vencovsky J, Ytterberg SR, Miller FW, Rider LG; International Myositis Classification Criteria Project consortium, The Euromyositis register and The Juvenile Dermatomyositis Cohort Biomarker Study and Repository (JDRG) (UK and Ireland). 2017 European League Against Rheumatism/American College of Rheumatology classification criteria for adult and juvenile idiopathic inflammatory myopathies and their major subgroups. Ann Rheum Dis. 2017 Dec;76(12):1955-1964. doi: 10.1136/annrheumdis-2017-211468. Epub 2017 Oct 27. |
| Label | URL |
|---|---|
| Related Info | View source |
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| ID | Term |
|---|---|
| D003882 | Dermatomyositis |
| ID | Term |
|---|---|
| D017285 | Polymyositis |
| D009220 | Myositis |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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The proposed trial in DM subjects is designed as an open-label study.
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| 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. | 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. | 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. | 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. | 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. | Up to Study Day 78 |
| Up to Study Day 127 |
| Change from Baseline or Percent Change from Baseline in plasma concentration of NM8074 | Up to Study Day 127 |
| Maximum plasma concentration (Cmax) | Up to Study Day 127 |
| Time corresponding to Cmax (tmax) | Up to Study Day 127 |
| Area under the drug concentration-time curves (AUC0-t) | Up to Study Day 127 |
| D009468 |
| Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D012871 | Skin Diseases |