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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/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
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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
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Secondary ID [1]
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2020-004228-41
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Secondary ID [2]
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C0251002
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Dermatomyositis
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Condition category
Condition code
Intervention/exposure
Study type
Interventional
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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
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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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)
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Assessment method [1]
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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.
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Timepoint [1]
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Baseline and Week 12
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Primary outcome [2]
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Number of Participants With Treatment Emergent Adverse Events (TEAEs) and Serious Adverse Events (SAE) (Stage 3)
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Assessment method [2]
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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.
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Timepoint [2]
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Up to Week 40
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Primary outcome [3]
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Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 3)
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Assessment method [3]
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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.
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Timepoint [3]
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Up to Week 40
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Primary outcome [4]
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Number of Participants With Vital Sign Abnormalities (Stage 3)
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Assessment method [4]
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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.
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Timepoint [4]
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Baseline up to Week 40
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Primary outcome [5]
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Number of Participants With Electrocardiogram (ECG) Abnormalities (Stage 3)
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Assessment method [5]
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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.
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Timepoint [5]
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Baseline up to Week 40
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Secondary outcome [1]
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Number of Participants With TEAEs and SAEs (Stage 1 and Stage 2)
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Assessment method [1]
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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 28 that were absent before treatment or that worsened relative to pretreatment state.
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Timepoint [1]
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Up to Week 28
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Secondary outcome [2]
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Number of Participants With TEAEs and SAEs (Amended Stage 2)
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Assessment method [2]
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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.
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Timepoint [2]
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Up to Week 40
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Secondary outcome [3]
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Number of Participants With Clinically Significant Laboratory Abnormalities (Stage 1 and Stage 2)
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Assessment method [3]
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HGB,hematocrit,ery.,HDL chl.\<0.8\*LLN;ret., ret./ery. (%)\<0.5\*LLN,\>1.5\*ULN;EMC volume,EMC HGB,EMC HGB concentration,potassium,chloride,calcium,bicarbonate\<0.9\*LLN,\>1.1\*ULN;platelets\<0.5\*LLN,\>1.75\*ULN;leu.,glucose\<0.6\*LLN,\>1.5\*ULN;lym., lym./leu.(%), neu., neu./leu. (%), protein,albumin \<0.8\*LLN,\>1.2\*ULN;bas., bas./leu.(%), eos., eos./leu., 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. Clinical significance of laboratory parameters was determined at the investigator's discretion.
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Timepoint [3]
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Up to Week 28
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Secondary outcome [4]
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Number of Participants With Clinically Significant Laboratory Abnormalities (Amended Stage 2)
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Assessment method [4]
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HGB,hematocrit,ery.,HDL chl.\<0.8\*LLN;ret., ret./ery. (%)\<0.5\*LLN,\>1.5\*ULN;EMC volume,EMC HGB,EMC HGB concentration,potassium,chloride,calcium,bicarbonate\<0.9\*LLN,\>1.1\*ULN;platelets\<0.5\*LLN,\>1.75\*ULN;leu.,glucose\<0.6\*LLN,\>1.5\*ULN;lym., lym./leu.(%), neu., neu./leu. (%), protein,albumin \<0.8\*LLN,\>1.2\*ULN;bas., bas./leu.(%), eos., eos./leu., 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. Clinical significance of laboratory parameters was determined at the investigator's discretion.
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Timepoint [4]
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Up to Week 40
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Secondary outcome [5]
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Number of Participants With Vital Sign Abnormalities (Stage 1 and Stage 2)
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Assessment method [5]
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Abnormality in vital signs: Sitting pulse rate \<40 bpm to \>120 bpm, sitting DBP \< 50 mmHg, sitting SBP \<90 mmHg.
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Timepoint [5]
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Up to Week 28
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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.
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Minimum age
18
Years
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Maximum age
80
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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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
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people assessing the outcomes
The people analysing the results/data
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Intervention assignment
Crossover
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Completed
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
23/01/2018
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
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Actual
28/11/2022
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Sample size
Target
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Accrual to date
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Final
75
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Recruitment in Australia
Recruitment state(s)
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
Pfizer
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
A Study looking at Investigational drug and Placebo administered to adult Patients with moderate to severe Dermatomyositis
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Trial website
https://clinicaltrials.gov/study/NCT03181893
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Trial related presentations / publications
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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
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Contacts
Principal investigator
Name
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Pfizer CT.gov Call Center
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Address
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Pfizer
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
No information has been provided regarding IPD availability
What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results are available at
https://clinicaltrials.gov/study/NCT03181893
Download to PDF