NCT02029274

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 .

Trial Health

60
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
17

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Aug 2013

Geographic Reach
3 countries

9 active sites

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

August 25, 2013

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

December 2, 2013

Completed
1 month until next milestone

First Posted

Study publicly available on registry

January 7, 2014

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 17, 2016

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

February 17, 2016

Completed
2.9 years until next milestone

Results Posted

Study results publicly available

January 15, 2019

Completed
Last Updated

January 5, 2021

Status Verified

December 1, 2018

Enrollment Period

2.5 years

First QC Date

December 2, 2013

Results QC Date

November 15, 2016

Last Update Submit

December 9, 2020

Conditions

Active Dermatomyositis

Keywords

Dermatomyositis

Outcome Measures

Primary Outcomes (1)

  • 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.

    Baseline, 6 months

Secondary Outcomes (4)

  • BAF312 Plasma Concentration

    6 months

  • Peripheral Blood Lymphocyte Counts

    baseline, 6 months

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

    baseline, 3 months

  • Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test

    baseline, 6 months

Study Arms (4)

BAF312 0.5mg

EXPERIMENTAL

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.

Drug: BAF312

BAF312 2mg

EXPERIMENTAL

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.

Drug: BAF312

BAF312 10 mg

EXPERIMENTAL

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.

Drug: BAF312

Placebo

PLACEBO COMPARATOR

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.

Drug: Placebo

Interventions

BAF312DRUG

BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.

Also known as: Siponimod
BAF312 0.5mgBAF312 10 mgBAF312 2mg
PlaceboDRUG

Matching placebo to BAF312 as tablets for oral administration.

Placebo

Eligibility Criteria

Age18 Years - 75 Years
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (9)

Novartis Investigative Site

Phoenix, Arizona, 85028, United States

Location

Novartis Investigative Site

Los Angeles, California, 90095, United States

Location

Novartis Investigative Site

Orange, California, 92868, United States

Location

Novartis Investigative Site

Miami, Florida, 33136, United States

Location

Novartis Investigative Site

Kansas City, Kansas, 66160, United States

Location

Novartis Investigative Site

Boston, Massachusetts, 02115, United States

Location

Novartis Investigative Site

Prague, Czech Republic, 128 50, Czechia

Location

Novartis Investigative Site

Chiba, Chiba, 260-8712, Japan

Location

Novartis Investigative Site

Sendai, Miyagi, 980-8574, Japan

Location

Related Links

MeSH Terms

Conditions

Dermatomyositis

Interventions

siponimod

Condition Hierarchy (Ancestors)

PolymyositisMyositisMuscular DiseasesMusculoskeletal DiseasesNeuromuscular DiseasesNervous System DiseasesConnective Tissue DiseasesSkin and Connective Tissue DiseasesSkin Diseases

Results Point of Contact

Title
Study Director
Organization
Novartis Pharmaceuticals
862-778-8300

Study Officials

  • Novartis Pharmaceuticals

    Novartis Pharmaceuticals

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
RANDOMIZED
Masking
TRIPLE
Who Masked
PARTICIPANT, INVESTIGATOR, OUTCOMES ASSESSOR
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 2, 2013

First Posted

January 7, 2014

Study Start

August 25, 2013

Primary Completion

February 17, 2016

Study Completion

February 17, 2016

Last Updated

January 5, 2021

Results First Posted

January 15, 2019

Record last verified: 2018-12

Locations

US(6)CZ(1)JP(2)