Dermatomyositis
Conditions
Keywords
dermatomyositis, IMO-8400, Pioneer
Brief summary
The purpose of this study is to determine how safe and effective IMO-8400 is in adults with dermatomyositis.
Detailed description
This study will evaluate the safety and efficacy of IMO-8400 in adults with active dermatomyositis (DM).
Interventions
DRUGIMO-8400 Dose Group 1
IMO-8400 Dose 1 subcutaneous injections once a week for 24 weeks.
DRUGIMO-8400 Dose Group 2
IMO-8400 Dose 2 subcutaneous injections once a week for 24 weeks.
DRUGPlacebo
normal saline subcutaneous injections once a week for 24 weeks.
Sponsors
Idera Pharmaceuticals, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
18 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* Has definite or probable DM based on the criteria of Bohan and Peter * Has a Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI)-Activity score ≥15 * Patients with muscle weakness are eligible; however having muscle weakness is not mandatory. * Study participants must have a diagnostic evaluation for cancer if the diagnosis of DM was within 2 years prior to the Screening Visit
Exclusion criteria
* Has ongoing severe dysphagia (e.g., requires a feeding tube) for the 3 months prior to Screening * Has known hypersensitivity to any oligodeoxynucleotide * Has a history of drug or alcohol abuse within one year of screening, or evidence of drug abuse by urine drug screening * Has body weight \>140 kg * 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) * Has received one or more of following prohibited treatments within the interval noted prior to Screening (Visit 1): 1. 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) 2. Intravenous corticosteroids within 12 weeks 3. Antimalarials (e.g., hydroxychloroquine) within 36 weeks 4. Topical corticosteroids (excluding scalp) within 2 weeks * 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 * Has interstitial lung disease requiring the use of supplemental oxygen
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | 28 weeks (24 weeks treatment + 4 weeks follow up) | Number of participants with different types of Treatment Emergent Adverse Events |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | 28 weeks (24 weeks treatment + 4 weeks follow up) | Change from baseline in mCDASI (Cutaneous Disease and Activity Severity Index) v2-Activity score as measured at Visits 2, 6, 10, 14, 18, 22 and 26 (EOT/ Week 25). Index is Clinician administered one page instrument designed to evaluate the cutaneous manifestations of DM. CDASI yields a total score that captures overall disease state, an activity score (range:0-100) that reflects the current inflammatory state of disease and a damage score (range: 0-32). The CDASI includes separate measurements for disease activity and damage and yields a total score that captures overall disease state, an activity score that reflects the current inflammatory state of disease, and a damage score. Decreases in CDASI scores are indicative of improvement. In this study, Activity Scores were measured. The scores below are averaged. |
Countries
Hungary, United Kingdom, United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Placebo normal saline subcutaneous injections once a week for 24 weeks.
Placebo: normal saline subcutaneous injections once a week for 24 weeks. | 11 |
| IMO-8400 Dose Group 1 IMO-8400 Dose Group 1 subcutaneous injections once a week for 24 weeks.
IMO-8400 Dose Group 1: IMO-8400 Dose 1 subcutaneous injections once a week for 24 weeks. | 9 |
| IMO-8400 Dose Group 2 IMO-8400 Dose Group 2 subcutaneous injections once a week for 24 weeks.
IMO-8400 Dose Group 2: IMO-8400 Dose 2 subcutaneous injections once a week for 24 weeks. | 10 |
| Total | 30 |
Baseline characteristics
| Characteristic | Total | Placebo | IMO-8400 Dose Group 1 | IMO-8400 Dose Group 2 |
|---|---|---|---|---|
| Age, Continuous | 51.5 Years STANDARD_DEVIATION 12.75 | 51.3 Years STANDARD_DEVIATION 10.55 | 48.3 Years STANDARD_DEVIATION 14.23 | 54.6 Years STANDARD_DEVIATION 14.12 |
| Ethnicity (NIH/OMB) Hispanic or Latino | 3 Participants | 1 Participants | 1 Participants | 1 Participants |
| Ethnicity (NIH/OMB) Not Hispanic or Latino | 27 Participants | 10 Participants | 8 Participants | 9 Participants |
| Ethnicity (NIH/OMB) Unknown or Not Reported | 0 Participants | 0 Participants | 0 Participants | 0 Participants |
| Region of Enrollment Hungary | 5 participants | 2 participants | 1 participants | 2 participants |
| Region of Enrollment United Kingdom | 1 participants | 1 participants | 0 participants | 0 participants |
| Region of Enrollment United States | 24 participants | 8 participants | 8 participants | 8 participants |
| Sex: Female, Male Female | 23 Participants | 7 Participants | 7 Participants | 9 Participants |
| Sex: Female, Male Male | 7 Participants | 4 Participants | 2 Participants | 1 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk |
|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 11 | 0 / 9 | 0 / 10 |
| other Total, other adverse events | 11 / 11 | 9 / 9 | 10 / 10 |
| serious Total, serious adverse events | 0 / 11 | 0 / 9 | 1 / 10 |
Outcome results
Primary
To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM
Number of participants with different types of Treatment Emergent Adverse Events
Time frame: 28 weeks (24 weeks treatment + 4 weeks follow up)
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | Serious TEAE | 0 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Mild | 6 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Moderate | 4 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Severe | 1 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Probably Related | 3 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Possibly Related | 3 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Not Related | 5 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | Injection Site Reaction AE | 2 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Leading to Treatment Discontinuation | 1 participants |
| Placebo | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Leading to Death | 0 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Leading to Treatment Discontinuation | 3 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | Serious TEAE | 0 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Possibly Related | 0 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Probably Related | 9 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Mild | 4 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Leading to Death | 0 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | Injection Site Reaction AE | 8 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Moderate | 3 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Not Related | 0 participants |
| IMO-8400 Dose Group 1 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Severe | 2 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | Injection Site Reaction AE | 10 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Severe | 2 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Probably Related | 9 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Possibly Related | 1 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Leading to Treatment Discontinuation | 1 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Causality Not Related | 0 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | Serious TEAE | 1 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Leading to Death | 0 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Mild | 2 participants |
| IMO-8400 Dose Group 2 | To Assess the Safety and Tolerability of IMO-8400 in Adult Subjects With DM | TEAE Severity Moderate | 6 participants |
Secondary
Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score
Change from baseline in mCDASI (Cutaneous Disease and Activity Severity Index) v2-Activity score as measured at Visits 2, 6, 10, 14, 18, 22 and 26 (EOT/ Week 25). Index is Clinician administered one page instrument designed to evaluate the cutaneous manifestations of DM. CDASI yields a total score that captures overall disease state, an activity score (range:0-100) that reflects the current inflammatory state of disease and a damage score (range: 0-32). The CDASI includes separate measurements for disease activity and damage and yields a total score that captures overall disease state, an activity score that reflects the current inflammatory state of disease, and a damage score. Decreases in CDASI scores are indicative of improvement. In this study, Activity Scores were measured. The scores below are averaged.
Time frame: 28 weeks (24 weeks treatment + 4 weeks follow up)
Population: Some patients did not complete all visits
| Arm | Measure | Group | Value (LEAST_SQUARES_MEAN) |
|---|---|---|---|
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Baseline | 30.1 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 6 | 28.0 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 10 | 27.7 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 14 | 28.9 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 18 | 30.5 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 22 | 26.6 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 26 | 26.0 units on a scale |
| Placebo | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Across Visit | 28.3 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 10 | 30.4 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 26 | 22.1 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 14 | 29.4 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 18 | 26.0 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 22 | 25.3 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Baseline | 31.3 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 6 | 28.5 units on a scale |
| IMO-8400 Dose Group 1 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Across Visit | 27.6 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 10 | 25.7 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 6 | 27.8 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Baseline | 30.7 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 14 | 21.6 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 26 | 23.0 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 22 | 24.6 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Visit 18 | 22.4 units on a scale |
| IMO-8400 Dose Group 2 | Change From Baseline in CDASI (Cutaneous Disease and Activity Severity Index) Activity Score | Across Visit | 25.1 units on a scale |