Clinical trial NCT02029274: 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
Overall status: TERMINATED
Phase: PHASE2
Conditions: Active Dermatomyositis
Keywords: Dermatomyositis
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2014-01-07
Enrollment: 17
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
timeFrame: Baseline, 6 months
Secondary outcomes:
measure: BAF312 Plasma Concentration
timeFrame: 6 months
measure: Peripheral Blood Lymphocyte Counts
timeFrame: baseline, 6 months
measure: Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
timeFrame: baseline, 3 months
measure: Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
timeFrame: baseline, 6 months
Other outcomes:
Locations:
city: Phoenix
country: United States
facility: Novartis Investigative Site
state: Arizona
city: Los Angeles
country: United States
facility: Novartis Investigative Site
state: California
city: Orange
country: United States
facility: Novartis Investigative Site
state: California
city: Miami
country: United States
facility: Novartis Investigative Site
state: Florida
city: Kansas City
country: United States
facility: Novartis Investigative Site
state: Kansas
city: Boston
country: United States
facility: Novartis Investigative Site
state: Massachusetts
city: Prague 2
country: Czechia
facility: Novartis Investigative Site
state: Czech Republic
city: Chiba-city
country: Japan
facility: Novartis Investigative Site
state: Chiba
city: Sendai city
country: Japan
facility: Novartis Investigative Site
state: Miyagi
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: