Study of NM8074 in Patients with Dermatomyositis (DM)

Official title

A Proof-of-Concept Phase II, Open-Label Study of NM8074 in Patients with Dermatomyositis (DM)

Overview

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).

Detailed description

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.

Interventions

• Drug NM8074
  NM8074 will be administered as an intravenous infusion at a dose of 20mg/kg

Primary outcome measures

• Change from Baseline or Percent Change from Baseline in the number of patients whose Total Improvement Score (TIS) has increased by ≥ 20 [Time frame: Up to Study Day 78]

Secondary outcome measures (6)

• Change from baseline or Percent Change from Baseline in Cutaneous Dermatomyositis Area and Severity Index (CDASI) score [Time frame: Up to Study Day 78]
• Change from Baseline or Percent Change from Baseline in Manual Muscle Testing (MMT-8) parameter [Time frame: Up to Study Day 78]
• Change from Baseline or Percent Change from Baseline in Physician's Global Activity (PhGA) assessment [Time frame: Up to Study Day 78]
• Change from Baseline or Percent Change from Baseline in Patient's Global Activity (PtGA) assessment [Time frame: Up to Study Day 78]
• Change from Baseline or Percent Change from Baseline Health Assessment Questionnaire (HAQ) score [Time frame: Up to Study Day 78]
• Change from Baseline or Percent Change from Baseline in Extra muscular Activity (MDAAT) score [Time frame: Up to Study Day 78]

Eligibility criteria

Inclusion criteria

• Male and female patients ≥18 years of age at the time of consent.
• A body mass index (BMI) within the range of 15 - 38 kg/m2. BMI = Body weight (kg) / \[Height (m)\]2.
• Subjects diagnosed with a probable or definite DM according to 2017 European League Against Rheumatism/American College of Rheumatology (2017 EULAR/ACR).
• Subjects must have proof of vaccination against Neisseria meningitidis (MenACWY and MenB), Streptococcus pneumoniae (PCV13 or PCV15 and PPSV23), and Haemophilus influenzae type b (Hib) taken at least 2 weeks prior to NM8074 administration as per national and local guidelines. If the window of vaccination is short, then patients will be prophylactically treated with appropriate antibiotics.
• Female partners of child-bearing potential (WOCBP), defined as all women physiologically capable of becoming pregnant, must have a negative pregnancy test at screening and must agree to use highly effective methods of contraception during dosing and for at least 8 weeks after stopping the investigational drug.
• Male patients and partners of child-bearing potential must agree to use contraceptives, and male patients must agree to refrain from donating sperm for the duration of the study and for at least 8 weeks after stopping the investigational drug.

Exclusion criteria

• Subjects with drug-induced myositis
• Subjects who have interstitial lung disease requiring the use of supplemental oxygen.
• Use of other investigational drugs at the time of enrollment, or within 5 half- lives of enrollment or within 3 months to study day 1, whichever is longer.
• History of currently active or suspicion of active bacterial, viral, or fungal infection within 2 weeks prior to first dose, or history of unexplained, recurrent bacterial infections.
• Subjects currently or previously diagnosed with cancer or who finished their cancer treatment within 2 years of the start of the clinical trial.
• Subjects with the history of bone marrow, hematopoietic stem cells, or solid organ transplantation.
• Has a currently active or known history of meningococcal disease or N. meningitidis infection.
• Evidence of active malignant disease or malignancies diagnosed within the previous 5 year
• Clinically significant medical or psychological conditions or risk factors that, as per the Investigator's judgment, could hinder the patient's participation in the study, introduce additional risks for the patient, or complicate the evaluation of the patient or study outcomes.
• Pregnant, planning to become pregnant, or nursing female subjects.
• Females with a positive pregnancy test result at Screening or on Day 1.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation

N/A

Model

Single group

Masking

Open label

Primary purpose

Treatment

Study locations

Center list to be confirmed — check the primary protocol.

Publications

• 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, Ytterber PMID 29079590

Identifiers

NCT: NCT06887738 · NM8074-DM-701

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗