Safety and Efficacy of BAF312 in Dermatomyositis

Novartis

Status and phase: Terminated; Phase 2

Conditions: Active Dermatomyositis

Treatments: Drug: Placebo; Drug: BAF312

Study type: Interventional

Funder types: Industry

Identifiers

NCT02029274

2013-001799-39 (EudraCT Number)

CBAF312X2206

Details and patient eligibility

About

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 .

Full 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.

Enrollment: 17 patients

Sex: All

Ages: 18 to 75 years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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17 participants in 4 patient groups, including a placebo group

BAF312 0.5mg

Experimental group

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.

Treatment:

Drug: BAF312

BAF312 2mg

Experimental group

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.

Treatment:

Drug: BAF312

BAF312 10 mg

Experimental group

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.

Treatment:

Drug: BAF312

Placebo

Placebo Comparator group

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.

Treatment:

Drug: Placebo