Phase 2 N=75 Randomized Triple-blind Treatment

A Study In Adults With Moderate To Severe Dermatomyositis

Dermatomyositis

Bottom Line

View on ClinicalTrials.gov: NCT03181893 ↗

Enrolled (actual)

Serious AEs

6.5%

Results posted

Sep 2023

Primary outcome: Primary: 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) — -3.44; -19.62; 5.00; -17.40 Units on a scale — p=<0.0001

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: PF-06823859 low (Drug); Placebo Arm (Drug); PF-06823859 high (Drug)
Age: Adult, Older Adult · 18+ yrs
Sex: All
Sponsor: Pfizer
Primary completion: May 2022

Outcome Measures

Outcome	Result	p-value
PRIMARY 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)	-3.44; -19.62; 5.00; -17.40; -26.00; -3.00	<0.0001 sig
PRIMARY Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3)	7; 8; 0; 2	—
PRIMARY Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3)	0; 1	—
PRIMARY Number of Participants With Vital Sign Abnormalities (Stage 3)	0; 0; 0; 0; 0; 0	—
PRIMARY Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3)	0; 0; 0; 0; 0; 0	—
SECONDARY Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2)	8; 20; 1; 5; 3; 1	—
SECONDARY Number of Participants With TEAEs and SAEs (Amended Stage 2)	1; 1; 8; 3; 0; 0	—
SECONDARY Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2)	0; 0; 0; 0; 0	—
SECONDARY Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2)	0; 0; 0; 0	—
SECONDARY Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2)	0; 1; 0; 0; 0; 0	—
SECONDARY Number of Participants With Vital Sign Abnormalities (Amended Stage 2)	0; 0; 0; 0; 0; 0	—
SECONDARY Number of Participants With ECG Abnormalities (Stage 1 and Stage 2)	0; 0; 0; 0; 0; 0	—
SECONDARY Number of Participants With ECG Abnormalities (Amended Stage 2)	0; 0; 0; 0; 0; 0	—
SECONDARY Change From Baseline in CDASI Activity Score at at All Scheduled Timepoints Through Week 12 (Stage 1, Stage 2 and Amended Stage 2)	-2.60; -19.62; 1.00; -9.80; -2.33; -6.50	—
SECONDARY Change From Baseline in CDASI Activity Score at All Scheduled Timepoints Through Week 12 (Stage 3)	-2.00; -2.11; -3.78; -5.11; -5.00; -7.78	—
SECONDARY Absolute Values of CDASI Activity Score at All Scheduled Timepoints Through Week 12 (All Stages)	31.50; 33.23; 23.00; 28.60; 37.00; 26.00	—
SECONDARY Absolute Values of CDASI Damage Score at All Scheduled Timepoints Through Week 12 (All Stages)	4.30; 5.50; 3.00; 4.20; 7.00; 7.50	—
SECONDARY Absolute Values for Total Improvement Score (TIS) at Week 12 and Intermediate Scheduled Time Points (Stage 3)	25.83; 36.67; 36.67; 49.17; 36.94; 56.39	0.1537
SECONDARY Change From Baseline in the Core Set Measures (CSM) of the TIS (Global Disease Activity [PhGA] and Extramuscular Global Assessment [EmGA]) (Stage 3)	-1.00; -1.68; -1.65; -2.76; -2.05; -3.40	—
SECONDARY Change From Baseline in the CSM of the TIS (PtGA) (Stage 3)	-18.29; -17.88; -16.81; -32.60; -14.04; -43.81	—
SECONDARY Change From Baseline in the CSM of the TIS (MMT8 and HAQ01-HAQ-DI) (Stage 3)	7.76; 8.24; 12.14; 15.24; 11.65; 21.24	—
SECONDARY Change From Baseline in the CSM of the TIS (Aldolase and Creatine Kinase) (Stage 3)	-0.19; -3.09; -1.31; -3.57; -0.66; -3.20	—

Summary

A Study looking at Investigational drug and Placebo administered to adult Patients with moderate to severe Dermatomyositis

Eligibility 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:
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)
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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03181893). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.