A Study In Adults With Moderate To Severe Dermatomyositis
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| ClinicalTrials.gov Identifier: NCT03181893 |
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Recruitment Status :
Active, not recruiting
First Posted : June 9, 2017
Last Update Posted : June 16, 2022
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| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Dermatomyositis | Drug: PF-06823859 low Drug: Placebo Arm Drug: PF-06823859 high | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Actual Enrollment : | 58 participants |
| Allocation: | Randomized |
| Intervention Model: | Crossover Assignment |
| Masking: | Triple (Participant, Investigator, Outcomes Assessor) |
| Primary Purpose: | Treatment |
| Official Title: | A PHASE 2 DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED STUDY TO EVALUATE THE EFFICACY, SAFETY, AND TOLERABILITY OF PF-06823859 IN ADULT SUBJECTS WITH DERMATOMYOSITIS |
| Actual Study Start Date : | January 23, 2018 |
| Actual Primary Completion Date : | May 27, 2022 |
| Estimated Study Completion Date : | December 1, 2022 |
| Arm | Intervention/treatment |
|---|---|
| Placebo Comparator: Placebo ARM |
Drug: Placebo Arm
Placebo contains histidine, sucrose, PS80, ethylene diamine, and triacetic acid |
| Experimental: PF-06823859 ARM high |
Drug: PF-06823859 high
A humanized immunoglobulin neutralizing antibody |
| Experimental: PF-06823859 ARM low |
Drug: PF-06823859 low
A humanized immunoglobulin neutralizing antibody |
- CDASI Activity Score [ Time Frame: Change from baseline activitly, (DAY 1) score at week 12 ]
The Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI), a skin-based outcome measure, was developed to systematically assess the extent of cutaneous disease in patients with DM.
The CDASI scale is a validated DM-specific instrument designed to capture the extent of cutaneous disease. Disease involvement in 15 different anatomical locations is rated using three activity (erythema, scale, erosion/ulceration) and two damage (poikiloderma, calcinosis) measures. The presence and severity of Gottron's papules, periungual changes and alopecia are also captured. The resulting activity and damage scores range from 0 to 100 and 0 to 32, respectively. Higher scores indicate greater disease severity.
- Treatment emergent adverse events and serious adverse events during the study. [ Time Frame: Baseline through Week 12 ]Incidence of treatment emergent adverse events (AEs) and serious adverse events (SAE) occurring in DM participants
- Clinically significant laboratory abnormalities [ Time Frame: Baseline through Week 12 ]Incidence of clinically significant laboratory abnormalities occurring in DM participants.
- Clinically significant changes in participants vital signs [ Time Frame: Baseline through week 12 ]Incidence of clinically significant changes in vital signs in DM participants.
- Clinically significant changes in ECG readings [ Time Frame: Baseline through week 12 ]Incidence of clinically significant ECG changes in DM participants.
- CDASI Activity Scores [ Time Frame: Baseline, (DAY 1) - week 12 ]Absolute values and change from baseline CDASI activity score through week 12
- CDASI Damage score [ Time Frame: Baseline, (DAY 1) - week 12 ]Absolute values and change from baseline CDASI Damage Score through week 12
- Total Improvement Score (TIS) [ Time Frame: Baseline, (DAY 1) - week 12 ]There are 6 core set measures that comprise the total improvement score. These components are the physician global activity assessment, the patient global activity assessment, MMT 8 score, HAQ-DI score, the myositis disease activity assessment tool, (MDAAT), and the participant's most elevated muscle enzymes. The total improvement score is the sum of all 6 improvement scores associated with the change in each core set measure
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years to 80 Years (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria for Patients with Skin Predominant Activity:
- Must have CDASI Activity score of greater than or equal to 14, and have failed at least 1 standard of care systemic treatment, (eg, corticosteroids).
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Confirmation of DM by the investigator and two of the following:
- Gottron's papules;
- Gottron's sign;
- Heliotrope eruption;
- Nailfold changes, (dilated capillary loops, capillary dropout, cuticular hypertrophy and/or rugged cuticles;
- Photodistributed violaceous erythema, (skin that is exposed to sunlight and appears purplish/reddish, and patchy in appearance;
- Positive DM serology -
- Post DM diagnosis; standard of care workup for DM must have been completed prior to entry into this research study.
- Willing to provide 8 biopsies during the course of the research study
Inclusion Criteria for Patients with Muscle Predominant Activity:
- MMT-8 ≤136/150 and PhGA, VAS ≥3 cm (0-10 cm) by visual analog scale (VAS)
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Sum of PhGA, VAS, PtGA, and extramuscular global assessment VAS scores is ≥10 cm (0-10 cm) VAS for each.
- Participant has failed at least two or more adequate courses of an immunosuppressive agent or immunomodulatory agent, including IVIG, at a dose known to be effective for rheumatologic diseases.
Exclusion Criteria for Patients with Skin Predominant Activity:
- Investigator site staff or members of their family.
- Acute and Chronic present medical conditions
- Intake of greater than 15 mg of prednisone or equivalent per day
- Pregnant or breastfeeding females. Fertile men and women who will not comply with the use of 2 effective birth control methods as per the research protocol
- Have required management of acute or chronic infections
- Have pre existing demyelinating disorder such as multiple sclerosis, or other severe neurological deficits.
- Clinically significant lab abnormalities
- Any health condition that may be worsened by immunosuppression
Exclusion Criteria for Patients with Muscle Predominant Activity:
Similar to patients with skin predominant activity; Intake of >20 mg oral prednisone/day, or equivalent
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03181893
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| Study Director: | Pfizer CT.gov Call Center | Pfizer |
| Responsible Party: | Pfizer |
| ClinicalTrials.gov Identifier: | NCT03181893 |
| Other Study ID Numbers: |
C0251002 2020-004228-41 ( EudraCT Number ) |
| First Posted: | June 9, 2017 Key Record Dates |
| Last Update Posted: | June 16, 2022 |
| Last Verified: | June 2022 |
| Individual Participant Data (IPD) Sharing Statement: | |
| Plan to Share IPD: | Yes |
| Plan Description: | Pfizer will provide access to individual de-identified participant data and related study documents (e.g. protocol, Statistical Analysis Plan (SAP), Clinical Study Report (CSR)) upon request from qualified researchers, and subject to certain criteria, conditions, and exceptions. Further details on Pfizer's data sharing criteria and process for requesting access can be found at: https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests. |
| URL: | https://www.pfizer.com/science/clinical_trials/trial_data_and_results/data_requests |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
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Dermatomyositis Polymyositis Myositis Muscular Diseases Musculoskeletal Diseases |
Neuromuscular Diseases Nervous System Diseases Connective Tissue Diseases Skin Diseases |