A Study In Adults With Moderate To Severe Dermatomyositis

• Register: ClinicalTrials.gov
• Last refreshed on: 18 September 2023
• Main ID: NCT03181893
• Date of registration: 05/06/2017
• Prospective Registration: Yes
• Primary sponsor: Pfizer
• Public title: A Study In Adults With Moderate To Severe Dermatomyositis
• Scientific 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
• Date of first enrolment: January 23, 2018
• Target sample size: 75
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT03181893
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Crossover Assignment. Primary purpose: Treatment. Masking: Triple (Participant, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Australia, Germany, Hungary, Italy, Poland, Spain, United States

Contacts

• Name: Pfizer CT.gov Call Center
• Affiliation: Pfizer

Key inclusion & exclusion criteria

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).

- Confirmation of DM by the investigator and two of the following:

1. Gottron's papules;

2. Gottron's sign;

3. Heliotrope eruption;

4. Nailfold changes, (dilated capillary loops, capillary dropout, cuticular
hypertrophy and/or rugged cuticles;

5. Photodistributed violaceous erythema, (skin that is exposed to sunlight and
appears purplish/reddish, and patchy in appearance;

6. 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)

- 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



Age minimum: 18 Years
Age maximum: 80 Years
Gender: All

Health Condition(s) or Problem(s) studied

Dermatomyositis

Intervention(s)

Drug: PF-06823859 low

Drug: Placebo Arm

Drug: PF-06823859 high

Primary Outcome(s)

Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3) [Time Frame: Up to Week 40]

Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3) [Time Frame: Baseline up to Week 40]

Change From Baseline in Cutaneous Dermatomyositis Disease Area and Severity Index (CDASI) Activity Score at Week 12 (Stage 1, Stage 2 and Amended Stage 2) [Time Frame: Baseline and Week 12]

Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3) [Time Frame: Up to Week 40]

Number of Participants With Vital Sign Abnormalities (Stage 3) [Time Frame: Baseline up to Week 40]

Secondary Outcome(s)

Change From Baseline in CDASI Activity Score at All Scheduled Timepoints Through Week 12 (Stage 3) [Time Frame: Baseline, Week 1, Week 4, Week 8 and Week 12]

Absolute Values for Total Improvement Score (TIS) at Week 12 and Intermediate Scheduled Time Points (Stage 3) [Time Frame: Week 4, Week 8 and Week 12]

Absolute Values of CDASI Activity Score at All Scheduled Timepoints Through Week 12 (All Stages) [Time Frame: Baseline, Week 1, Week 4, Week 8 and Week 12]

Change From Baseline in the Core Set Measures (CSM) of the TIS (Global Disease Activity [PhGA] and Extramuscular Global Assessment [EmGA]) (Stage 3) [Time Frame: Baseline, Week 4, Week 8 and Week 12]

Number of Participants With ECG Abnormalities (Stage 1 and Stage 2) [Time Frame: Up to Week 28]

Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2) [Time Frame: Up to Week 28]

Number of Participants With ECG Abnormalities (Amended Stage 2) [Time Frame: Up to Week 40]

Change From Baseline in the CSM of the TIS (Aldolase and Creatine Kinase) (Stage 3) [Time Frame: Baseline, Week 4, Week 8 and Week 12]

Change From Baseline in the CSM of the TIS (MMT8 and HAQ01-HAQ-DI) (Stage 3) [Time Frame: Week 4, Week 8 and Week 12]

Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2) [Time Frame: Up to Week 40]

Absolute Values of CDASI Damage Score at All Scheduled Timepoints Through Week 12 (All Stages) [Time Frame: Baseline, Week 1, Week 4, Week 8 and Week 12]

Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2) [Time Frame: Up to Week 28]

Change From Baseline in the CSM of the TIS (PtGA) (Stage 3) [Time Frame: Baseline, Week 4, Week 8 and Week 12]

Number of Participants With TEAEs and SAEs (Amended Stage 2) [Time Frame: Up to Week 40]

Number of Participants With Vital Sign Abnormalities (Amended Stage 2) [Time Frame: Up to Week 40]

Change From Baseline in CDASI Activity Score at at All Scheduled Timepoints Through Week 12 (Stage 1, Stage 2 and Amended Stage 2) [Time Frame: Baseline, Week 1, Week 4, and Week 8 (except for Week 12 which is a primary outcome measure)]

Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2) [Time Frame: Up to Week 28]

Secondary ID(s)

2020-004228-41

C0251002

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 14/09/2023
• URL: https://clinicaltrials.gov/ct2/show/results/NCT03181893