Brief Summary

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

Trial Health

On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment

participants targeted

Target at P50-P75 for phase_2

Timeline

Completed

Started Jan 2018

Longer than P75 for phase_2

Geographic Reach

5 countries

41 active sites

Status

completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

4.8 years study duration

First Submitted

Initial submission to the registry

June 5, 2017

Completed

4 days until next milestone

First Posted

Study publicly available on registry

June 9, 2017

Completed

8 months until next milestone

Study Start

First participant enrolled

January 23, 2018

Completed

4.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

May 27, 2022

Completed

6 months until next milestone

Study Completion

Last participant's last visit for all outcomes

November 28, 2022

Completed

10 months until next milestone

Results Posted

Study results publicly available

September 14, 2023

Completed

Last Updated

September 14, 2023

Status Verified

September 1, 2023

Enrollment Period

4.3 years

First QC Date

June 5, 2017

Results QC Date

May 26, 2023

Last Update Submit

September 8, 2023

Conditions

Dermatomyositis

Outcome Measures

Primary Outcomes (5)

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)
The treatment effect was defined as the difference (mean chg from baseline at Week12 in the active treatment group minus that in the placebo group) in the mean change of CDASI activity score from baseline at Week 12. The score (range: 0-100) consists of the extent score (ES), Gottorn hands score (GHS), peringual score (PS) and allopecia score (AS). ES (range: 0-90) was obtained by summing up scores for the total erythema (ER \[0-45\], redness of the skin or mucous membranes), scaling (SC \[0-30\], peeling of the skin) and erosion/ulceration (EU \[0-15\], presence of the deeper wound). Total ER, SC and EU scores were calculated as a sum of the contributions from 15 individual areas of the body. GHS characterizes papules (swellings) on hand and is a sum of the papule's characterization score (0-6) and ulceration score (0-1). PS (0-2) characterizes abnormalities around nails. The AS (0-1) characterizes hair loss. Higher scores indicate greater disease severity.
Baseline and Week 12
Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3)
Adverse event (AE) was any untoward medical occurrence in a participant temporally associated with the use of study intervention, whether or not considered related to the study intervention. SAE was any untoward medical occurrence that at any dose resulted in any of following outcomes/deemed significant for any other reason: death; initial /prolonged inpatient hospitalization; life-threatening; persistent or significant disability/incapacity; congenital anomaly/birth defect and suspected transmission via a Pfizer product of an infectious agent, pathogenic or non-pathogenic. AEs included both serious (if occurred) and all non-serious adverse events. TEAEs are events between first dose of study drug and up to Week 40 that were absent before treatment or that worsened relative to pretreatment state.
Up to Week 40
Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3)
Hemoglobin(HGB),hematocrit,erythrocytes(ery.),HDL cholesterol(chl.)\<0.8\*lower limit of normal(LLN);reticulocytes (ret.), ret./ery.(%)\<0.5\*LLN,\>1.5\*upper limit of normal (ULN);ery. mean corpuscular(EMC) volume,EMC HGB concentration,potassium,chloride,calcium,bicarbonate\<0.9\*LLN,\>1.1\*ULN;platelets\<0.5\*LLN,\>1.75\*ULN; leukocytes(leu.),glucose\<0.6\*LLN,\>1.5\*ULN;lymphocytes(lym.), lym./leu.(%),neutrophils (neu.), neu./leu.(%), protein,albumin\<0.8\*LLN,\>1.2\*ULN;basophils(bas.), bas./leu.(%), eosinophils(eos.), eos./leu., monocytes(mon.), mon./leu.(%), urate\>1.2\*ULN;bilirubin (total, direct,indirect)\>1.5\*ULN;aspartate/alanine aminotransferase,gamma glutamyl transferase,lactate dehydrogenase,alkaline phosphatase\>3.0\*ULN;urea nitrogen,creatinine,triglycerides, chl.\>1.3\*ULN; sodium \<0.95\*LLN,\>1.05\*ULN; creatine kinase \>2.0\*ULN;Urine: pH\<4.5,\>8;glucose, ketones,protein, HGB,urobilinogen,bilirubin,nitrite,leukocyte esterase\>=1;ery., leu.\>= 20;hyaline casts\>1;bacteria\>20.
Up to Week 40
Number of Participants With Vital Sign Abnormalities (Stage 3)
Abnormality in vital signs: Sitting pulse rate \<40 beats per minute (bpm) to \>120 bpm, sitting diastolic blood pressure (DBP) \< 50 millimeter of mercury (mmHg), sitting systolic blood pressure (SBP) \<90 mmHg.
Baseline up to Week 40
Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3)
ECG abnormalities criteria included: 1) QTc interval adjusted according to Fridericia formula (QTcF) (msec): \>450, \>480, \>500, increase from baseline \>=30, increase from baseline \>=60; 2) Pulse rate (PR) (msec): \>=300, change from baseline (Chg) \>=25% or 50%; 3) QT (msec): \>=500; 4) QRS (msec): \>=200, Chg \>=25% or 50%. Categories, with at least 1 participant having ECG abnormality in any of the reporting arms, were reported in this outcome measure.
Baseline up to Week 40

Secondary Outcomes (17)

Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2)
Up to Week 28
Number of Participants With TEAEs and SAEs (Amended Stage 2)
Up to Week 40
Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2)
Up to Week 28
Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2)
Up to Week 40
Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2)
Up to Week 28
+12 more secondary outcomes

Study Arms (3)

Placebo ARM

PLACEBO COMPARATOR

Drug: Placebo Arm

PF-06823859 ARM high

EXPERIMENTAL

Drug: PF-06823859 high

PF-06823859 ARM low

EXPERIMENTAL

Drug: PF-06823859 low

Interventions

PF-06823859 lowDRUG

A humanized immunoglobulin neutralizing antibody

PF-06823859 ARM low

Placebo ArmDRUG

Placebo contains histidine, sucrose, PS80, ethylene diamine, and triacetic acid

Placebo ARM

PF-06823859 highDRUG

A humanized immunoglobulin neutralizing antibody

PF-06823859 ARM high

Eligibility Criteria

Age18 Years - 80 Years

Sexall

Healthy VolunteersNo

Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

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
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.

You may not qualify if:

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
Similar to patients with skin predominant activity; Intake of \>20 mg oral prednisone/day, or equivalent

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Pfizerlead

Study Sites (41)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

The University of Alabama at Birmingham

Birmingham, Alabama, 35249, United States

Location

The University of Alabama at Birmingham

Birmingham, Alabama, 35294, United States

Location

Mayo Clinic Arizona Research Pharmacy

Phoenix, Arizona, 85054, United States

Location

Mayo Clinic

Scottsdale, Arizona, 85259, United States

Location

Attune Health Research Inc.

Beverly Hills, California, 90211, United States

Location

Freidenrich Center for Translational Research at Stanford University

Palo Alto, California, 94304, United States

Location

Mayo Clinic Florida

Jacksonville, Florida, 32224, United States

Location

University Of Miami Hospital

Miami, Florida, 33125, United States

Location

University of Miami Hospital Clinical Translational Research Site (Infusion site)

Miami, Florida, 33136, United States

Location

KU Clinical Research Center - Clinical and Translational Science Unit (CTSU)

Fairway, Kansas, 66205, United States

Location

The University of Kansas Medical Center

Kansas City, Kansas, 66160, United States

Location

Johns Hopkins Bayview Medical Center

Baltimore, Maryland, 21224-6821, United States

Location

Brigham and Women's Hospital - ACC

Boston, Massachusetts, 02115, United States

Location

Brigham and Women's Hospital - CTC

Boston, Massachusetts, 02115, United States

Location

Brigham and Women's Hospital - CTH

Boston, Massachusetts, 02115, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Clinical Research Unit (CRU)