Dermatomyositis MedDRA version: 19.0 Level: PT Classification code 10012503 Term: Dermatomyositis System Organ Class: 10040785 - Skin and subcutaneous tissue disorders
Conditions
Interventions
Product Name: IMO-8400
Product Code: IMO-8400
Pharmaceutical Form: Lyophilisate for solution for injection
INN or Proposed INN: IMO-8400
Current Sponsor code: IMO-8400
Concentration unit: mg milligram
Sponsors
Idera Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1 Has signed the current, approved Informed Consent Form 2 Is 18 to 75 years of age (inclusive) at the time of consent 3 Has definite or probable DM based on the criteria of Bohan and Peter (Appendix 3; 1975a, 1975b) OR those patients who have all other definite or probable Bohan and Peter criteria but do not have heliotrope rash and Gottron’s signs/papules may still be included if they have one or more of the following: a) DM autoantibody (anti-Mi-2, anti-MDA5, anti-TIF1-gamma, anti-NXP-2, anti-Jo-1, anti-PL-12, anti-PL-7, anti-EJ, anti-KS, anti-OJ); at least 1 autoantibody must be present and documented in the patient’s medical record b) A classic DM associated skin change including at least one of the following: malar rash without sparing nasolabial folds, Shawl sign, V neck rash, periungal erythema, or mechanic’s hands; documented in the patient’s medical record 4 Has a CDASIv2-Activity score =15 at Visit 2 (Baseline) 5 Has evidence of active muscle disease as indicated by clinical evidence of symmetrical proximal muscle weakness involving limb-girdle and/or anterior neck flexors within 12 weeks prior to screening and the presence of one or more of the following: a) Elevation of serum creatine kinase or serum aldolase levels >1.5 times the upper limit of normal during Screening or >1.5 times increase compared to a documented within normal limits level obtained prior to disease onset or while in prior remission b) Electromyography demonstrating one or more of the following within 4 weeks prior to Screening: i Fibrillation potentials (increased insertional irritability and spontaneous activity) ii Positive sharp waves and complex repetitive discharges iii Short duration, small amplitude motor unit action potentials c) Muscle biopsy demonstrating one or more of the following within 12 weeks prior to Screening: i Perimysial and/or perivascular infiltration of mononuclear cells ii Perifascicular atrophy iii Note: Patients with a muscle biopsy demonstrating rimmed vacuoles or other histologic findings of Inclusion Body Myositis are excluded d) Magnetic resonance imaging demonstrating evidence of disease activity and inflammation on the short t inversion recovery image within 12 weeks prior to Screening 6 If on permitted concomitant medications at Screening, the therapy regimen can include one or more of the following: a) Stable dose of prednisone (or equivalent) =20 mg/day (or =140 mg/week) for =4 weeks b) Stable regimen that does not exceed the approved dosages for =12 weeks of no more than 1 of the following non-steroidal immunomodulatory medication(s): intravenous immunoglobulin, mycophenolate mofetil, cyclophosphamide, cyclosporine, leflunomide, tacrolimus, methotrexate, azathioprine c) Stable regimen of topical treatments for scalp involvement for =3 weeks 7 Study participants must have a diagnostic evaluation for cancer if the diagnosis of DM was within 2 years prior to the Screening Visit. The evaluation should include: a) All age- and gender-appropriate screening tests and a computed tomography (CT) of the chest, abdomen, and pelvis; OR b) Positron emission tomography and computerized tomography (PET/CT of the chest, abdomen, and pelvis). Note: If a diagnostic evaluation for cancer has not been performed within 2 years prior to the Screening Visit in patients for whom it is required, a CT of the chest, abdomen, and pelvis should be performed, or a PET/CT of the chest, abdomen, and pelvis if this is standard practice in the parti
Exclusion criteria
Exclusion criteria: 1. Has ongoing severe dysphagia (e.g., requires a feeding tube) for the 3 months prior to Screening 2. Has known hypersensitivity to any oligodeoxynucleotide 3. Has a history of drug abuse within one year of screening, or evidence of drug abuse by urine drug screening 4. Has a history of alcohol abuse within one year of screening 5. Is pregnant (or intends to become pregnant within 6 months of last dose of study medication) or nursing 6. Has body weight >140 kg 7. Has a positive hepatitis B surface antigen test (HBsAg), and/or hepatitis B core antibody test (anti-HBc), or a positive hepatitis C virus antibody test (anti-HCV) 8. Has a history of human immunodeficiency Virus (HIV)-1 or HIV-2 9. Has screening safety laboratory test meeting any of the following criteria: a) Hemoglobin 1.2 mg/dL in female patients and >1.5 mg/dL in male patients. Patients with serum creatinine values exceeding limits may be eligible for the study if their eGFRs are >60 mL/min f) Serum aspartate transaminase (AST) or serum alanine transaminase (ALT) >5 times ULN (unless considered consistent with muscle origin and accompanied by gamma-glutamyl transferase (GGT) 1.5 times ULN h) Prothrombin time (PT) >1.5 times ULN i) C3 or C4 1+ 10. Has a diagnosis of Juvenile DM, IBM, drug-induced toxic myopathy, metabolic myopathy, dystrophy, cancer-associated DM , or connective tissue disease-associated DM (e.g., overlap syndrome) 11. Has received one or more of following prohibited treatments within the interval noted prior to Screening (Visit 1): a) Rituximab within 24 weeks (Note: patients who received rituximab are only eligible for inclusion if B-cell counts are confirmed to be within normal limits) b) Intravenous corticosteroids within 12 weeks c) Intravenous immunosuppressive drugs within 12 weeks d) Any other monoclonal antibody, biologic agent, or investigational agent within 12 weeks or 5 half-lives (whichever is longer) e) Antimalarials (e.g., hydroxychloroquine) within 36 weeks f) Topical corticosteroids (excluding scalp) within 2 weeks 12. Has evidence of or has required treatment for cancer (except for treated, non-invasive carcinoma of the skin or cured cervical carcinoma-in-situ) within 5 years 13. Has other chronic or active significant medical conditions within 6 months prior to Screening including but not limited to: allogeneic organ transplant (e.g., solid organ, bone marrow, or stem cells); cardiac disease (e.g., unstable angina, myocardial infarction, ventricular arrhythmia); congestive heart failure; liver disease; neurological disease; hematological disease; kidney disease; uncontrolled seizure disorder; uncontrolled pulmonary disease; uncontrolled gastrointestinal disease; uncontrolled endocrinological disease; uncontrolled psychiatric disease; or uncontrolled diabetes mellitus 14. Has interstitial lung disease requiring the use of supplemental oxygen 15. Has received or is expected to receive any live viral or bacterial vaccination within 3 months prior to Screening 16. Has received a Bacille Calmette-Guerin (BCG) vaccination within 12 months of Screening or is expected to receive it during the course of the study 17. Has a history of or ongoing active, chronic, or recurrent i
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Primary end point(s): Primary Safety and Tolerability Endpoints •Number of patients discontinuing treatment due to AEs •Frequency and severity of AEs •Physical examination findings, including vital signs •Standard laboratory safety tests including hematology, chemistry, coagulation and urinalysis •Assessment of ISRs •ECG findings •Laboratory safety assessments including CH50, C3, C4, troponin, CRP, albumin, globulin, A:G ratio, proteinuria, eGFR, and platelet count Primary Efficacy Endpoint •Change from baseline in mCDASIv2 Activity score ;Timepoint(s) of evaluation of this end point: Safety and Tolerability Endpoints: AEs reported and observed, assessment of ISRs, Vital Signs: weekly during Week 1 to Week 25, then at end of study visit on Week 29 Physical examination findings standard laboratory safety tests, ECG, laboratory safety assessments: every four weeks during Week 1-Week 25, then at end of study visit on Week 29 Efficacy Endpoint: CDASIv2: every four weeks during Week 1-Week 25.;Main Objective: • To assess the safety and tolerability of IMO-8400 in adult patients with dermatomyositis (DM) with active skin and muscle disease • To assess the effect of IMO-8400 on the cutaneous manifestations of DM;Secondary Objective: Not applicable | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): -;Timepoint(s) of evaluation of this end point: - | — |
Countries
Czech Republic, Hungary, Sweden, United Kingdom, United States
Contacts
Public ContactClinical Trials Mailbox
Idera Pharmaceuticals, Inc.
Outcome results
None listed