Trial · NCT01148810

Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis

NCT01148810, Phase 2, Terminated, Novartis Pharmaceuticals; results first posted 2019-04-25

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis. Trials, record NCT01148810. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT01148810

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Trial

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

Title: Efficacy and Tolerability of BAF312 in Patients With Polymyositis and Dermatomyositis
NCT number: NCT01148810
Status: Terminated
Phases: Phase 2
Enrollment: 18 participants, actual
Lead sponsor: Novartis Pharmaceuticals
Conditions: Polymyositis, Dermatomyositis
Interventions: BAF312, Placebo
First posted: 2010-06-22
Last updated: 2019-04-25
Other registries' IDs: eudract:2008-006311-21
Results first posted: 2019-04-25
Limitations and caveats: The study was terminated due to new clinical data from overall BAF312 program (\<10 mg/day already have optimal pharmacodynamic effect \& clinical efficacy). There was no safety concern testing 10 mg BAF312 that would have required study termination.

Source ClinicalTrials.gov

Registered outcomes11

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

List	Outcome	Time frame	Description
Primary	Number of Participants Who Responded to BAF312	12 weeks	Preliminary clinical efficacy of BAF312 in patients with Polymyositis and dermatomyositis (PM/DM) using the International Myositis Assessment and Clinical Studies Group (IMACS) core set measures (including manual muscle testing, Physician's Global Activity Assessment (on a horizontal 10 cm visual analogue scale), Patient Global Activity Assessment (on a horizontal 10 cm visual analogue scale), Physical Function (Health Assessment Questionnaire), Muscle-associated Enzymes (CK, LDH, AST, ALT, aldolase) and Extra-Muscular Activity Assessment (Extra-muscular portion of Myositis Disease Activity Assessment Tool).
Secondary	Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	12 weeks
Secondary	Mean Plasma Concentrations of BAF312	baseline to end of trial (day 196)
Secondary	Summary of CRP Levels	12 weeks	Biomarkers reflecting efficacy in reducing systemic inflammatory components of the disease using serum markers such as C-reactive protein (CRP)
Secondary	Efficacy in Modifying Health-related Quality of Life Measured by SF-36	12 weeks	Short Form (36) Health Survey. The SF-36 consists of eight scaled scores, which are the weighted sums of the questions in their section. Each scale is directly transformed into a 0-100 scale on the assumption that each question carries equal weight. The lower the score the more disability. The higher the score the less disability i.e., a score of zero is equivalent to maximum disability.
Secondary	Myositis Disease (MD) Activity Scores	Week 12	Myositis Disease Activity Scores. This is a combined tool that captures the physician's assessment of disease activity of various organ systems via the MYOSITIS INTENTION TO TREAT ACTIVITY INDEX (MITAX) and via the MYOSITIS DISEASE ACTIVITY ASSESSMENT VISUAL ANALOGUE SCALES (MYOACT) It rates the physician's overall assessment of the ongoing current disease activity for various systems by drawing a vertical mark on the 10-cm line for each system according to the following scale: left end of line = no evidence of disease activity, midpoint of line = moderate disease activity, and right end of line = extreme or maximum disease activity.
Secondary	Physician Global Activity Assessment	Baseline, Week 12	Physician's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Secondary	Patient Global Activity Assessment	Baseline, Week 12	Patient's overall assessment on a single 0-10 cm scale, where the higher score indicates higher disease activity.
Secondary	Manual Muscle Testing (MMT) - 8 Score	Baseline, Week 12	Manual Muscle Testing - 8 (MMT-8): Assessment of designated muscles manually by scoring each muscle from 0 to 10 where 0 is no strength and 10 is maximum strength. MMT- 8 includes 7 bilateral muscles (potential score 0-70 x 2) and one unilateral (axial) muscle (0-10 x1) so the total score ranges from 0 to 150 (maximum) where higher score indicates more strength.
Secondary	Health Assessment Questionnaire	Baseline, Week 12	Health Assessment Questionnaire (HAQ): This questionnaire is a patient reported outcome (PRO) which is self-administered by the patient. It is used to assess disability and comprises various categories related to usual daily activities. The patients report the amount of difficulty they have in performing some of these activities. Each question asks on a scale ranging from 0 to 3 if the categories can be performed without any difficulty (scale 0) up to cannot be done at all (scale 3). The total score is derived from these sub-scores and ranges from 0 to 3 where higher HAQ indicates more disability.

1 to 10 of 11Source ClinicalTrials.gov

See all 11 registered outcomes

Outcomes11

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

Type	Outcome	Time frame	Population	Units	Measured as
PRIMARY	Number of Participants Who Responded to BAF312	12 weeks	All patients with evaluable (or complete) PD measurement and no major protocol deviations which impact on PD data were included in the PD analysis set.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	12 weeks	The Safety set included all patients who received at least one dose of study medication.	Participants	COUNT_OF_PARTICIPANTS
SECONDARY	Mean Plasma Concentrations of BAF312	baseline to end of trial (day 196)	PK Analysis set in all disease types	ng/ml	MEAN
SECONDARY	Summary of CRP Levels	12 weeks	The Safety set included all patients who received at least one dose of study medication.	mg/L	MEAN
SECONDARY	Efficacy in Modifying Health-related Quality of Life Measured by SF-36	12 weeks	All patients with evaluable (or complete) PD measurement and no major protocol deviations which impact on PD data were included in the PD analysis set.	score on a scale	MEAN
SECONDARY	Myositis Disease (MD) Activity Scores	Week 12	PD set in all disease types	cm	MEAN
SECONDARY	Physician Global Activity Assessment	Baseline, Week 12	PD set in all disease types	cm	MEAN
SECONDARY	Patient Global Activity Assessment	Baseline, Week 12	PD set in all disease types	cm	MEAN
SECONDARY	Manual Muscle Testing (MMT) - 8 Score	Baseline, Week 12	PD set in all disease types	scores on a scale	MEAN
SECONDARY	Health Assessment Questionnaire	Baseline, Week 12	PD set in all disease types	Score on a scale	MEAN

1 to 10 of 11Source ClinicalTrials.gov

See all 11 outcomes

Results by group48

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

Outcome	Group	Category	Value	Spread	Units
Number of Participants Who Responded to BAF312	BAF312/BAF312		4		Participants
Number of Participants Who Responded to BAF312	Placebo/BAF312		1		Participants
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	BAF312/BAF312	Steroid reduction	3		Participants
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	Placebo/BAF312	Steroid reduction	2		Participants
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	BAF312/BAF312	Steroid increase1	1		Participants
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	Placebo/BAF312	Steroid increase1	2		Participants
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	BAF312/BAF312	Steroid stable	3		Participants
Number of Participants With a Change in Steroids Use After BAF312 Administration -Period 2	Placebo/BAF312	Steroid stable	4		Participants
Mean Plasma Concentrations of BAF312	BAF312	baseline	0	Standard Deviation 0	ng/ml
Mean Plasma Concentrations of BAF312	BAF312	Day 8	29.6	Standard Deviation 25.7	ng/ml

1 to 10 of 48Source ClinicalTrials.gov

See all 48 results by group

Participant flow6

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

Period	Milestone	Group	Participants
Overall Study	STARTED	BAF312/BAF312	8
Overall Study	STARTED	Placebo/BAF312	10
Overall Study	COMPLETED	BAF312/BAF312	7
Overall Study	COMPLETED	Placebo/BAF312	5
Overall Study	NOT COMPLETED	BAF312/BAF312	1
Overall Study	NOT COMPLETED	Placebo/BAF312	5

Source ClinicalTrials.gov

Adverse events98

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	Palpitations	Cardiac disorders	BAF312/BAF312	1	16
other	Palpitations	Cardiac disorders	Placebo/BAF312	0	10
other	Cataract	Eye disorders	BAF312/BAF312	0	16
other	Cataract	Eye disorders	Placebo/BAF312	1	10
other	Conjunctivitis	Eye disorders	BAF312/BAF312	1	16
other	Conjunctivitis	Eye disorders	Placebo/BAF312	0	10
other	Macular oedema	Eye disorders	BAF312/BAF312	0	16
other	Macular oedema	Eye disorders	Placebo/BAF312	1	10
other	Periorbital oedema	Eye disorders	BAF312/BAF312	0	16
other	Periorbital oedema	Eye disorders	Placebo/BAF312	1	10

1 to 10 of 98Source ClinicalTrials.gov

See all 98 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	BAF312/BAF312	0	16
Total, all-cause mortality	Placebo/BAF312	0	10
Total, other adverse events	BAF312/BAF312	12	16
Total, other adverse events	Placebo/BAF312	7	10
Total, serious adverse events	BAF312/BAF312	0	16
Total, serious adverse events	Placebo/BAF312	3	10

Source ClinicalTrials.gov

Papers0

The papers the trial lists on ClinicalTrials.gov.

None in this release.

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 comparisons3

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

Comparison	Source
BAF312/BAF312 vs Placebo/BAF312: Number of Participants Who Responded to BAF312	Trial comparisons
BAF312/BAF312 vs Placebo/BAF312: deaths from any cause	Trial comparisons
BAF312/BAF312 vs Placebo/BAF312: serious adverse events	Trial comparisons

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/NCT01148810

GET https://conductscience.org/api/trials/v1/NCT01148810/results?page=2

API reference For AI assistants