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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/ct2/show/NCT03181893




Registration number
NCT03181893
Ethics application status
Date submitted
5/06/2017
Date registered
9/06/2017
Date last updated
14/09/2023

Titles & IDs
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
Secondary ID [1] 0 0
2020-004228-41
Secondary ID [2] 0 0
C0251002
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Dermatomyositis 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Neurological 0 0 0 0
Other neurological disorders
Inflammatory and Immune System 0 0 0 0
Other inflammatory or immune system disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - PF-06823859 low
Treatment: Drugs - Placebo Arm
Treatment: Drugs - PF-06823859 high

Placebo Comparator: Placebo ARM -

Experimental: PF-06823859 ARM high -

Experimental: PF-06823859 ARM low -


Treatment: Drugs: PF-06823859 low
A humanized immunoglobulin neutralizing antibody

Treatment: Drugs: Placebo Arm
Placebo contains histidine, sucrose, PS80, ethylene diamine, and triacetic acid

Treatment: Drugs: PF-06823859 high
A humanized immunoglobulin neutralizing antibody
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
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)
Timepoint [1] 0 0
Baseline and Week 12
Primary outcome [2] 0 0
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3)
Timepoint [2] 0 0
Up to Week 40
Primary outcome [3] 0 0
Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3)
Timepoint [3] 0 0
Up to Week 40
Primary outcome [4] 0 0
Number of Participants With Vital Sign Abnormalities (Stage 3)
Timepoint [4] 0 0
Baseline up to Week 40
Primary outcome [5] 0 0
Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3)
Timepoint [5] 0 0
Baseline up to Week 40
Secondary outcome [1] 0 0
Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2)
Timepoint [1] 0 0
Up to Week 28
Secondary outcome [2] 0 0
Number of Participants With TEAEs and SAEs (Amended Stage 2)
Timepoint [2] 0 0
Up to Week 40
Secondary outcome [3] 0 0
Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2)
Timepoint [3] 0 0
Up to Week 28
Secondary outcome [4] 0 0
Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2)
Timepoint [4] 0 0
Up to Week 40
Secondary outcome [5] 0 0
Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2)
Timepoint [5] 0 0
Up to Week 28
Secondary outcome [6] 0 0
Number of Participants With Vital Sign Abnormalities (Amended Stage 2)
Timepoint [6] 0 0
Up to Week 40
Secondary outcome [7] 0 0
Number of Participants With ECG Abnormalities (Stage 1 and Stage 2)
Timepoint [7] 0 0
Up to Week 28
Secondary outcome [8] 0 0
Number of Participants With ECG Abnormalities (Amended Stage 2)
Timepoint [8] 0 0
Up to Week 40
Secondary outcome [9] 0 0
Change From Baseline in CDASI Activity Score at at All Scheduled Timepoints Through Week 12 (Stage 1, Stage 2 and Amended Stage 2)
Timepoint [9] 0 0
Baseline, Week 1, Week 4, and Week 8 (except for Week 12 which is a primary outcome measure)
Secondary outcome [10] 0 0
Change From Baseline in CDASI Activity Score at All Scheduled Timepoints Through Week 12 (Stage 3)
Timepoint [10] 0 0
Baseline, Week 1, Week 4, Week 8 and Week 12
Secondary outcome [11] 0 0
Absolute Values of CDASI Activity Score at All Scheduled Timepoints Through Week 12 (All Stages)
Timepoint [11] 0 0
Baseline, Week 1, Week 4, Week 8 and Week 12
Secondary outcome [12] 0 0
Absolute Values of CDASI Damage Score at All Scheduled Timepoints Through Week 12 (All Stages)
Timepoint [12] 0 0
Baseline, Week 1, Week 4, Week 8 and Week 12
Secondary outcome [13] 0 0
Absolute Values for Total Improvement Score (TIS) at Week 12 and Intermediate Scheduled Time Points (Stage 3)
Timepoint [13] 0 0
Week 4, Week 8 and Week 12
Secondary outcome [14] 0 0
Change From Baseline in the Core Set Measures (CSM) of the TIS (Global Disease Activity [PhGA] and Extramuscular Global Assessment [EmGA]) (Stage 3)
Timepoint [14] 0 0
Baseline, Week 4, Week 8 and Week 12
Secondary outcome [15] 0 0
Change From Baseline in the CSM of the TIS (PtGA) (Stage 3)
Timepoint [15] 0 0
Baseline, Week 4, Week 8 and Week 12
Secondary outcome [16] 0 0
Change From Baseline in the CSM of the TIS (MMT8 and HAQ01-HAQ-DI) (Stage 3)
Timepoint [16] 0 0
Week 4, Week 8 and Week 12
Secondary outcome [17] 0 0
Change From Baseline in the CSM of the TIS (Aldolase and Creatine Kinase) (Stage 3)
Timepoint [17] 0 0
Baseline, Week 4, Week 8 and Week 12

Eligibility
Key inclusion 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.
Minimum age
18 Years
Maximum age
80 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s

The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Crossover
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
23/01/2018
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
28/11/2022
Sample size
Target
Accrual to date
Final
75
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
Alabama
Country [2] 0 0
United States of America
State/province [2] 0 0
Arizona
Country [3] 0 0
United States of America
State/province [3] 0 0
California
Country [4] 0 0
United States of America
State/province [4] 0 0
Florida
Country [5] 0 0
United States of America
State/province [5] 0 0
Kansas
Country [6] 0 0
United States of America
State/province [6] 0 0
Maryland
Country [7] 0 0
United States of America
State/province [7] 0 0
Massachusetts
Country [8] 0 0
United States of America
State/province [8] 0 0
Minnesota
Country [9] 0 0
United States of America
State/province [9] 0 0
Missouri
Country [10] 0 0
United States of America
State/province [10] 0 0
New York
Country [11] 0 0
United States of America
State/province [11] 0 0
Ohio
Country [12] 0 0
United States of America
State/province [12] 0 0
Oregon
Country [13] 0 0
United States of America
State/province [13] 0 0
Pennsylvania
Country [14] 0 0
United States of America
State/province [14] 0 0
Texas
Country [15] 0 0
United States of America
State/province [15] 0 0
Utah
Country [16] 0 0
Germany
State/province [16] 0 0
Tuebingen
Country [17] 0 0
Hungary
State/province [17] 0 0
Hajdú-bihar
Country [18] 0 0
Poland
State/province [18] 0 0
Bialystok
Country [19] 0 0
Poland
State/province [19] 0 0
Krakow
Country [20] 0 0
Spain
State/province [20] 0 0
Madrid
Country [21] 0 0
Spain
State/province [21] 0 0
Sevilla

Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Name
Pfizer
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
A Study looking at Investigational drug and Placebo administered to adult Patients with
moderate to severe Dermatomyositis
Trial website
https://clinicaltrials.gov/ct2/show/NCT03181893
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Pfizer CT.gov Call Center
Address 0 0
Pfizer
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/ct2/show/NCT03181893