- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02029274
Safety and Efficacy of BAF312 in Dermatomyositis
December 9, 2020 updated by: Novartis Pharmaceuticals
A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)
This study investigated the dose response relationship for the efficacy and safety of BAF312 compared to placebo in active DM patients over a treatment period of 6+6 months and to determine the minimum dose required for a maximal clinical effect.
The study was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered at the dose of 2 mg daily .
Study Overview
Status
Terminated
Conditions
Intervention / Treatment
Detailed Description
The study was prematurely terminated based on the results of an interim analysis where BAF312 did not demonstrate superior efficacy over placebo and a dose-response relationship was not observed.
There were no safety concerns.
Approximately 56 participants were planned to be randomized.
A total of 17 participants were enrolled and randomized by the time the study was terminated.
Study Type
Interventional
Enrollment (Actual)
17
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Czech Republic
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Prague 2, Czech Republic, Czechia, 128 50
- Novartis Investigative Site
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Chiba
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Chiba-city, Chiba, Japan, 260-8712
- Novartis Investigative Site
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Miyagi
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Sendai city, Miyagi, Japan, 980-8574
- Novartis Investigative Site
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Arizona
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Phoenix, Arizona, United States, 85028
- Novartis Investigative Site
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California
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Los Angeles, California, United States, 90095
- Novartis Investigative Site
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Orange, California, United States, 92868
- Novartis Investigative Site
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Florida
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Miami, Florida, United States, 33136
- Novartis Investigative Site
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Kansas
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Kansas City, Kansas, United States, 66160
- Novartis Investigative Site
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Novartis Investigative Site
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 75 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Key Inclusion Criteria:
Written informed consent must be obtained before any assessment is performed.
- Patients who have been defined as "definite" or "probable" based on the criteria of Bohan and Peter (Bohan and Peter 1975) for dermatomyositis at least 3 months before screening
- Patients must have active disease as defined by muscle weakness
- Patients may be on a stable dose of corticosteroid (up/equal to 20 mg once daily prednisone equivalent)
- Patients currently treated with oral or subcutaneous MTX must have been a stable dose of no more/equal to than 25 mg per week
- Patients currently treated with Azathioprine must have been a stable maintenance dose of no more/equal to 3 mg/kg/day
- Negative cancer screening conducted in the 12 months prior to screening visit
Key Exclusion Criteria
- Dermatomyositis patients having overlap myositis or any other type of myositis including paraneoplastic myositis, drug-induced myopathy, necrotizing myositis
- Preexisting severe cardiac or pulmonary conditions, malignancy of any organ system or significant eye diseases.
- Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.
- Pregnant or nursing (lactating) women
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: BAF312 0.5mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 0.5 mg over a 10 day period.
After, participants continued on 0.5 mg daily for up to 24 weeks.
During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period.
After, participants continued on 2.0 mg daily for up to 24 weeks.
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BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Other Names:
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Experimental: BAF312 2mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period.
After, participants continued on 2.0 mg daily for up to 24 weeks.
During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period.
After, participants continued on 2.0 mg daily for up to 24 weeks.
|
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Other Names:
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Experimental: BAF312 10 mg
During period 1, participants were uptitrated daily from BAF312 0.25 mg to 10.0 mg over a 10 day period.
After, participants continued on 10.0 mg daily for up to 24 weeks.
During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period.
After, participants continued on 2.0 mg daily for up to 24 weeks.
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BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Other Names:
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Placebo Comparator: Placebo
During period 1, participants received matching placebo daily for up to 24 weeks.
During period 2, participants were uptitrated daily from BAF312 0.25 mg to 2.0 mg over a 10 day period.
After, participants continued on 2.0 mg daily for up to 24 weeks.
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Matching placebo to BAF312 as tablets for oral administration.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Time Frame: Baseline, 6 months
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Each muscles tested was evaluated on a 0 - 10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score.
The total MMT24 score ranged from 0 - 240, where an increasing trend in the values indicates improvement.
A positive change from baseline indicates improvement.
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Baseline, 6 months
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
BAF312 Plasma Concentration
Time Frame: 6 months
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6 months
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Peripheral Blood Lymphocyte Counts
Time Frame: baseline, 6 months
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Absolute lymphocyte counts
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baseline, 6 months
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Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Time Frame: baseline, 3 months
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Each muscles tested was evaluated on a 0-10 scale where 0 indicated the weakest muscle score and 10 indicated the strongest muscle score.
the total MMT24 score ranged from 0-240, where an increasing trend in the values indicates improvement.
A positive change from baseline indicates improvement.
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baseline, 3 months
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Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
Time Frame: baseline, 6 months
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baseline, 6 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Publications and helpful links
The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
August 25, 2013
Primary Completion (Actual)
February 17, 2016
Study Completion (Actual)
February 17, 2016
Study Registration Dates
First Submitted
December 2, 2013
First Submitted That Met QC Criteria
January 6, 2014
First Posted (Estimate)
January 7, 2014
Study Record Updates
Last Update Posted (Actual)
January 5, 2021
Last Update Submitted That Met QC Criteria
December 9, 2020
Last Verified
December 1, 2018
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Nervous System Diseases
- Skin Diseases
- Musculoskeletal Diseases
- Connective Tissue Diseases
- Muscular Diseases
- Neuromuscular Diseases
- Polymyositis
- Myositis
- Dermatomyositis
- Physiological Effects of Drugs
- Molecular Mechanisms of Pharmacological Action
- Immunosuppressive Agents
- Immunologic Factors
- Sphingosine 1 Phosphate Receptor Modulators
- Siponimod
Other Study ID Numbers
- CBAF312X2206
- 2013-001799-39 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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