Trial · NCT03981744

A Study of Ustekinumab in Participants With Active Polymyositis and Dermatomyositis Who Have Not Adequately Responded to One or More Standard-of-care Treatments

NCT03981744, Phase 3, Terminated, Janssen Pharmaceutical K.K.; results first posted 2024-05-13

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. A Study of Ustekinumab in Participants With Active Polymyositis and Dermatomyositis Who Have Not Adequately Responded to One or More Standard-of-care Treatments. Trials, record NCT03981744. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03981744

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: A Study of Ustekinumab in Participants With Active Polymyositis and Dermatomyositis Who Have Not Adequately Responded to One or More Standard-of-care Treatments
NCT number: NCT03981744
Status: Terminated
Phases: Phase 3
Enrollment: 51 participants, actual
Lead sponsor: Janssen Pharmaceutical K.K.
Conditions: Polymyositis, Dermatomyositis
Interventions: Ustekinumab 6 mg/kg, Ustekinumab 90 mg, Placebo IV, Placebo SC
First posted: 2019-06-11
Last updated: 2025-04-29
Results first posted: 2024-05-13
Limitations and caveats: This study was terminated early due to lack of efficacy found at Week 24.

Source ClinicalTrials.gov

Registered outcomes7

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Percentage of Participants Who Achieved Minimal Improvement in International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 24	Week 24	Minimal improvement was defined as IMACS TIS greater than or equal to (\>=) 20 in participants with polymyositis (PM)/dermatomyositis (DM). Criteria used the 6 IMACS core set measures: physician global activity (PhGA)(0-10), patient global activity (PtGA)(0-10), manual muscle testing-8 (MMT-8)(0-80), muscle enzymes (creatine kinase, Aldolase, lactate dehydrogenase, aspartate aminotransferase, alanine aminotransferase), extramuscular assessment of myositis disease activity assessment tool (MDAAT)(0-10), and health assessment questionnaire disability index (HAQ-DI)(0-3). Absolute percent change in each core set measure was calculated as final value minus baseline value divided by range\*100. Total improvement score was calculated by sum of 6 core set improvement scores. Total improvement score ranged from 0 to 100 where higher scores indicated greater improvement. This was categorized into 3 categories (minimal \[improvement \>=20\], moderate \[improvement \>=40\] and major \[improvement \>=60\]).
Secondary	Change From Baseline in Functional Index-2 (FI-2) at Week 24	Baseline, Week 24	Change from baseline in FI-2 at Week 24 was reported. The FI-2 was a functional outcome developed for participants with adult PM or DM to assess muscle endurance in 7 muscle groups (shoulder flexion \[0-60\], shoulder abduction \[0-60\], head lift \[0-60\], hip flexion \[0-60\], step test \[0-60\], heel lift \[0-120\], and toe lift \[0-120\]). Each muscle group was scored as the number of correctly performed repetitions with 60 or 120 maximal number of repetitions depending on muscle group. The FI-2 was performed unilaterally, preferably on the participant's dominant side for muscle groups of shoulder, hip, and step test. The FI-2 score ranged from 0-60 or 0-120 depending on the muscle group. Higher score indicated better muscle endurance.
Secondary	Percentage of Participants Who Experienced Disease Worsening Up to Week 24 Based on Consensus Criteria for Worsening	Up to Week 24	Percentage of participants who experienced disease worsening up to Week 24 based on consensus criteria for worsening was reported. Criteria for disease worsening in a clinical trial were based on international consensus guideline developed by the IMACS. The worsening of disease was defined as 1 of the following criteria: Worsening of the Physician Global Activity by \>=2 centimeter (cm) on a 10-cm visual analogue scale (VAS) and worsening of findings of MMT-8 by \>= 20 percent (%) from baseline; worsening of MDAAT-global extramuscular organ disease activity (a composite of constitutional, cutaneous, skeletal, gastrointestinal, pulmonary, and cardiac activity) by \>=2 cm on a 10-cm VAS from baseline; worsening of any 3 of 6 IMACS core set (PhGA, PtGA, MMT-8, muscle enzymes, MDAAT, and HAQ-D) activity measures by \>= 30% from baseline.
Secondary	Change From Baseline in Manual Muscle Testing (MMT)-8 Score at Week 24	Baseline, Week 24	Change from baseline in MMT-8 score at Week 24 was reported. Manual Muscle Testing was a partially validated tool to assess muscle strength. MMT-8 total score ranged from 0-80, where maximal score was sum of scores from 8 muscle groups (Deltoid middle \[left/right\], Biceps brachii \[left/right\], Gluteus maximus \[left/right\], Gluteus medius \[left/right\], Quadriceps \[left/right\], Wrist extensors \[left/right\], Ankle dorsiflexors \[left/right\], Neck flexors \[axial\]) and each muscle group was scored on a 0 to 10-point scale. The sides (right or left) used for calculating the total score. Higher score indicated greater muscle strength, that is, less impairment of muscle.
Secondary	Change From Baseline in Physician Global Activity (PhGA) at Week 24	Baseline, Week 24	Change from baseline in PhGA at Week 24 was reported. Physician Global Activity was a partially validated tool to measure the global evaluation by the physician of the participant's overall disease activity at the time of assessment using a 0-10 cm VAS, where 0 cm= no evidence of disease activity and 10 cm= extremely active or severe disease activity. Negative values indicated improvement from baseline.
Secondary	Change From Baseline in Extramuscular Assessment by Myositis Disease Activity Assessment Tool (MDAAT) at Week 24	Baseline, Week 24	Change from baseline in extramuscular assessment by MDAAT at Week 24 was reported. This validated tool measure the degree of disease activity of extramuscular organ systems and muscle on a 0-10 cm VAS. Extramuscular activity ranged between 0 and 10 via VAS where, 0 cm = absent and 10 cm = maximum disease activity.
Secondary	Change From Baseline in Muscle Enzyme Levels at Week 24	Baseline, Week 24	Change from baseline in muscle enzyme levels (creatine kinase, lactate dehydrogenase) at Week 24 was reported.

Source ClinicalTrials.gov

Outcomes7

Each outcome the trial posted results for, in ClinicalTrials.gov's words.

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Percentage of Participants Who Achieved Minimal Improvement in International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 24	Week 24	All randomized analysis set included all participants who were randomized in this study.	Percentage of participants	NUMBER
SECONDARY	Change From Baseline in Functional Index-2 (FI-2) at Week 24	Baseline, Week 24	All randomized analysis set included all participants who were randomized in this study. Here, 'n' (number analyzed) signifies participants who were evaluable for this outcome measure for given categories.	Scores on a scale	MEAN
SECONDARY	Percentage of Participants Who Experienced Disease Worsening Up to Week 24 Based on Consensus Criteria for Worsening	Up to Week 24	All randomized analysis set included all participants who were randomized in this study.	Percentage of participants	NUMBER
SECONDARY	Change From Baseline in Manual Muscle Testing (MMT)-8 Score at Week 24	Baseline, Week 24	All randomized analysis set included all participants who were randomized in this study.	Scores on a scale	MEAN
SECONDARY	Change From Baseline in Physician Global Activity (PhGA) at Week 24	Baseline, Week 24	All randomized analysis set included all participants who were randomized in this study.	Scores on a scale	MEAN
SECONDARY	Change From Baseline in Extramuscular Assessment by Myositis Disease Activity Assessment Tool (MDAAT) at Week 24	Baseline, Week 24	All randomized analysis set included all participants who were randomized in this study.	Scores on a scale	MEAN
SECONDARY	Change From Baseline in Muscle Enzyme Levels at Week 24	Baseline, Week 24	All randomized analysis set included all participants who were randomized in this study. Here, 'n' (number analyzed) signifies participants who were evaluable for this outcome measure for given categories.	Enzyme units per liter (Enzyme U/L)	MEAN

Source ClinicalTrials.gov

Results by group28

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Category	Value	Spread	Units
Percentage of Participants Who Achieved Minimal Improvement in International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 24	Placebo		61.5		Percentage of participants
Percentage of Participants Who Achieved Minimal Improvement in International Myositis Assessment and Clinical Studies Total Improvement Score (IMACS TIS) at Week 24	Ustekinumab		64.0		Percentage of participants
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Placebo	Change in Shoulder Flexion	2.92	Standard Deviation 14.672	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Ustekinumab	Change in Shoulder Flexion	6.88	Standard Deviation 14.284	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Placebo	Change in Head Lift	2.00	Standard Deviation 11.331	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Ustekinumab	Change in Head Lift	5.56	Standard Deviation 13.364	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Placebo	Change in Hip Flexion	2.54	Standard Deviation 6.819	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Ustekinumab	Change in Hip Flexion	5.84	Standard Deviation 13.184	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Placebo	Change in Step Test	4.15	Standard Deviation 10.806	Scores on a scale
Change From Baseline in Functional Index-2 (FI-2) at Week 24	Ustekinumab	Change in Step Test	3.76	Standard Deviation 16.674	Scores on a scale

1 to 10 of 28Source ClinicalTrials.gov

See all 28 results by group

Participant flow24

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
First Intervention (Prior to Week 24)	STARTED	Placebo	26
First Intervention (Prior to Week 24)	STARTED	Ustekinumab	25
First Intervention (Prior to Week 24)	STARTED	Placebo to Ustekinumab	0
First Intervention (Prior to Week 24)	STARTED	Ustekinumab Through End of Study (EOS)	0
First Intervention (Prior to Week 24)	COMPLETED	Placebo	26
First Intervention (Prior to Week 24)	COMPLETED	Ustekinumab	23
First Intervention (Prior to Week 24)	COMPLETED	Placebo to Ustekinumab	0
First Intervention (Prior to Week 24)	COMPLETED	Ustekinumab Through End of Study (EOS)	0
First Intervention (Prior to Week 24)	NOT COMPLETED	Placebo	0
First Intervention (Prior to Week 24)	NOT COMPLETED	Ustekinumab	2

1 to 10 of 24Source ClinicalTrials.gov

See all 24 participant flow

Adverse events114

Each adverse event the trial posted, group by group: the participants affected out of those at risk.

Type	Event	Organ system	Group	Affected	At risk
other	Iron Deficiency Anaemia	Blood and lymphatic system disorders	Placebo	0	26
other	Iron Deficiency Anaemia	Blood and lymphatic system disorders	Placebo to Ustekinumab	0	26
other	Iron Deficiency Anaemia	Blood and lymphatic system disorders	Ustekinumab Through End of Study (EOS)	2	25
other	Vertigo	Ear and labyrinth disorders	Placebo	0	26
other	Vertigo	Ear and labyrinth disorders	Placebo to Ustekinumab	2	26
other	Vertigo	Ear and labyrinth disorders	Ustekinumab Through End of Study (EOS)	2	25
other	Vitreous Floaters	Eye disorders	Placebo	0	26
other	Vitreous Floaters	Eye disorders	Placebo to Ustekinumab	0	26
other	Vitreous Floaters	Eye disorders	Ustekinumab Through End of Study (EOS)	2	25
other	Abdominal Pain	Gastrointestinal disorders	Placebo	1	26

1 to 10 of 114Source ClinicalTrials.gov

See all 114 adverse events

Adverse event totals9

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Placebo	0	26
Total, all-cause mortality	Placebo to Ustekinumab	0	26
Total, all-cause mortality	Ustekinumab Through End of Study (EOS)	0	25
Total, other adverse events	Placebo	16	26
Total, other adverse events	Placebo to Ustekinumab	15	26
Total, other adverse events	Ustekinumab Through End of Study (EOS)	24	25
Total, serious adverse events	Placebo	1	26
Total, serious adverse events	Placebo to Ustekinumab	6	26
Total, serious adverse events	Ustekinumab Through End of Study (EOS)	8	25

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
37652554	DERIVED

Source PubMed

Grants0

The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.

None in this release.

Trial comparisons0

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

None in this release.

Tests0

The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.

None in this release.

Data dates

The day each source was copied for this release.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

Sources 3

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ClinicalTrials.govNo license stated
NIH RePORTER's clinical studies and project filesPublic domain

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ClinicalTrials.govnone stated
NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this trial from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/trials/v1/NCT03981744

GET https://conductscience.org/api/trials/v1/NCT03981744/results?page=2

API reference For AI assistants