Clinical trial NCT03181893: A Study In Adults With Moderate To Severe Dermatomyositis
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Dermatomyositis
Lead sponsor: Pfizer
Sponsor class: INDUSTRY
First posted: 2017-06-09
Enrollment: 75
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: CROSSOVER
Primary purpose: TREATMENT

Brief 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:

  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

Primary outcomes:
measure: 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)
timeFrame: Baseline and Week 12
measure: Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3)
timeFrame: Up to Week 40
measure: Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3)
timeFrame: Up to Week 40
measure: Number of Participants With Vital Sign Abnormalities (Stage 3)
timeFrame: Baseline up to Week 40
measure: Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3)
timeFrame: Baseline up to Week 40
Secondary outcomes:
measure: Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2)
timeFrame: Up to Week 28
measure: Number of Participants With TEAEs and SAEs (Amended Stage 2)
timeFrame: Up to Week 40
measure: Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2)
timeFrame: Up to Week 28
measure: Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2)
timeFrame: Up to Week 40
measure: Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2)
timeFrame: Up to Week 28
measure: Number of Participants With Vital Sign Abnormalities (Amended Stage 2)
timeFrame: Up to Week 40
measure: Number of Participants With ECG Abnormalities (Stage 1 and Stage 2)
timeFrame: Up to Week 28
measure: Number of Participants With ECG Abnormalities (Amended Stage 2)
timeFrame: Up to Week 40
measure: Change From Baseline in CDASI Activity Score at at All Scheduled Timepoints Through Week 12 (Stage 1, Stage 2 and Amended Stage 2)
timeFrame: Baseline, Week 1, Week 4, and Week 8 (except for Week 12 which is a primary outcome measure)
measure: Change From Baseline in CDASI Activity Score at All Scheduled Timepoints Through Week 12 (Stage 3)
timeFrame: Baseline, Week 1, Week 4, Week 8 and Week 12
measure: Absolute Values of CDASI Activity Score at All Scheduled Timepoints Through Week 12 (All Stages)
timeFrame: Baseline, Week 1, Week 4, Week 8 and Week 12
measure: Absolute Values of CDASI Damage Score at All Scheduled Timepoints Through Week 12 (All Stages)
timeFrame: Baseline, Week 1, Week 4, Week 8 and Week 12
measure: Absolute Values for Total Improvement Score (TIS) at Week 12 and Intermediate Scheduled Time Points (Stage 3)
timeFrame: Week 4, Week 8 and Week 12
measure: Change From Baseline in the Core Set Measures (CSM) of the TIS (Global Disease Activity [PhGA] and Extramuscular Global Assessment [EmGA]) (Stage 3)
timeFrame: Baseline, Week 4, Week 8 and Week 12
measure: Change From Baseline in the CSM of the TIS (PtGA) (Stage 3)
timeFrame: Baseline, Week 4, Week 8 and Week 12
measure: Change From Baseline in the CSM of the TIS (MMT8 and HAQ01-HAQ-DI) (Stage 3)
timeFrame: Week 4, Week 8 and Week 12
measure: Change From Baseline in the CSM of the TIS (Aldolase and Creatine Kinase) (Stage 3)
timeFrame: Baseline, Week 4, Week 8 and Week 12
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama at Birmingham
state: Alabama
city: Birmingham
country: United States
facility: The University of Alabama at Birmingham
state: Alabama
city: Birmingham
country: United States
facility: The University of Alabama at Birmingham
state: Alabama
city: Phoenix
country: United States
facility: Mayo Clinic Arizona Research Pharmacy
state: Arizona
city: Scottsdale
country: United States
facility: Mayo Clinic
state: Arizona
city: Beverly Hills
country: United States
facility: Attune Health Research Inc.
state: California
city: Palo Alto
country: United States
facility: Freidenrich Center for Translational Research at Stanford University
state: California
city: Jacksonville
country: United States
facility: Mayo Clinic Florida
state: Florida
city: Miami
country: United States
facility: University Of Miami Hospital
state: Florida
city: Miami
country: United States
facility: University of Miami Hospital Clinical Translational Research Site (Infusion site)
state: Florida
city: Fairway
country: United States
facility: KU Clinical Research Center - Clinical and Translational Science Unit (CTSU)
state: Kansas
city: Kansas City
country: United States
facility: The University of Kansas Medical Center
state: Kansas
city: Baltimore
country: United States
facility: Johns Hopkins Bayview Medical Center
state: Maryland
city: Boston
country: United States
facility: Brigham and Women's Hospital - ACC
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital - CTC
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital - CTH
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Minneapolis
country: United States
facility: Clinical Research Unit (CRU)
state: Minnesota
city: Minneapolis
country: United States
facility: Department of Medicine Division of Rheumatic and Autoimmune Disease
state: Minnesota
city: Minneapolis
country: United States
facility: Lillehei Clinical Research Unit (LCRU)
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota Health Rheumatology Clinic
state: Minnesota
city: Minneapolis
country: United States
facility: University of Minnesota, Department of Dermatology
state: Minnesota
city: Saint Louis
country: United States
facility: Center for Outpatient Health
state: Missouri
city: Saint Louis
country: United States
facility: Washington University School of Medicine
state: Missouri
city: New York
country: United States
facility: New York University School of Medicine
state: New York
city: New York
country: United States
facility: NYU Langone Health Clinical Research Center
state: New York
city: New York
country: United States
facility: Mount Sinai Doctors Dermatology
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Portland
country: United States
facility: OHSU, Center for Health and Healing CHH2
state: Oregon
city: Portland
country: United States
facility: Oregon Clinical & Translational Research Institute
state: Oregon
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Dallas
country: United States
facility: University of Texas Southwestern Medical Center
state: Texas
city: Murray
country: United States
facility: University of Utah MidValley Dermatology
state: Utah
city: Salt Lake City
country: United States
facility: Center for Clinical & Translational Science
state: Utah
city: Tuebingen
country: Germany
facility: Universitaetsklinikum Tuebingen
city: Debrecen
country: Hungary
facility: University of Debrecen
state: Hajdú-bihar
city: Bialystok
country: Poland
facility: Nova Reuma spolka partnerska
city: Krakow
country: Poland
facility: Centrum Medyczne Plejady
city: Madrid
country: Spain
facility: Hospital Universitario 12 de Octubre
city: Sevilla
country: Spain
facility: Hospital Quiron Infanta Luisa
Study officials:
affiliation: Pfizer
name: Pfizer CT.gov Call Center
role: STUDY_DIRECTOR
References: