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Safety and Efficacy of BAF312 in Dermatomyositis

Primary Purpose

Active Dermatomyositis

Status
Terminated
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
BAF312
Placebo
Sponsored by
Novartis Pharmaceuticals
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Active Dermatomyositis focused on measuring Dermatomyositis

Eligibility Criteria

18 Years - 75 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site
  • Novartis Investigative Site

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm Type

Experimental

Experimental

Experimental

Placebo Comparator

Arm Label

BAF312 0.5mg

BAF312 2mg

BAF312 10 mg

Placebo

Arm Description

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.

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.

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.

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.

Outcomes

Primary Outcome Measures

Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
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.

Secondary Outcome Measures

BAF312 Plasma Concentration
Peripheral Blood Lymphocyte Counts
Absolute lymphocyte counts
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
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.
Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test

Full Information

First Posted
December 2, 2013
Last Updated
December 9, 2020
Sponsor
Novartis Pharmaceuticals
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1. Study Identification

Unique Protocol Identification Number
NCT02029274
Brief Title
Safety and Efficacy of BAF312 in Dermatomyositis
Official Title
A Double Blind, Randomized, Placebo-controlled Study to Evaluate, Safety, Tolerability, Efficacy and Preliminary Dose-response of BAF312 in Patients With Active Dermatomyositis (DM)
Study Type
Interventional

2. Study Status

Record Verification Date
December 2018
Overall Recruitment Status
Terminated
Why Stopped
The study was terminated prematurely after an interim analysis for futility. The study did not provide any evidence for efficacy of BAF312 in dermatomyositis.
Study Start Date
August 25, 2013 (Actual)
Primary Completion Date
February 17, 2016 (Actual)
Study Completion Date
February 17, 2016 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Novartis Pharmaceuticals

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
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 .
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Active Dermatomyositis
Keywords
Dermatomyositis

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
17 (Actual)

8. Arms, Groups, and Interventions

Arm Title
BAF312 0.5mg
Arm Type
Experimental
Arm Description
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.
Arm Title
BAF312 2mg
Arm Type
Experimental
Arm Description
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.
Arm Title
BAF312 10 mg
Arm Type
Experimental
Arm Description
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.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
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.
Intervention Type
Drug
Intervention Name(s)
BAF312
Other Intervention Name(s)
Siponimod
Intervention Description
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Matching placebo to BAF312 as tablets for oral administration.
Primary Outcome Measure Information:
Title
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Description
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.
Time Frame
Baseline, 6 months
Secondary Outcome Measure Information:
Title
BAF312 Plasma Concentration
Time Frame
6 months
Title
Peripheral Blood Lymphocyte Counts
Description
Absolute lymphocyte counts
Time Frame
baseline, 6 months
Title
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Description
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.
Time Frame
baseline, 3 months
Title
Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
Time Frame
baseline, 6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
75 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Novartis Pharmaceuticals
Organizational Affiliation
Novartis Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Novartis Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85028
Country
United States
Facility Name
Novartis Investigative Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90095
Country
United States
Facility Name
Novartis Investigative Site
City
Orange
State/Province
California
ZIP/Postal Code
92868
Country
United States
Facility Name
Novartis Investigative Site
City
Miami
State/Province
Florida
ZIP/Postal Code
33136
Country
United States
Facility Name
Novartis Investigative Site
City
Kansas City
State/Province
Kansas
ZIP/Postal Code
66160
Country
United States
Facility Name
Novartis Investigative Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Novartis Investigative Site
City
Prague 2
State/Province
Czech Republic
ZIP/Postal Code
128 50
Country
Czechia
Facility Name
Novartis Investigative Site
City
Chiba-city
State/Province
Chiba
ZIP/Postal Code
260-8712
Country
Japan
Facility Name
Novartis Investigative Site
City
Sendai city
State/Province
Miyagi
ZIP/Postal Code
980-8574
Country
Japan

12. IPD Sharing Statement

Links:
URL
https://www.novctrd.com/ctrdweb/patientsummary/patientsummaries?patientSummaryId=126
Description
A Plain Language Trial Summary is available on novartisclinicaltrials.com

Learn more about this trial

Safety and Efficacy of BAF312 in Dermatomyositis

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