Efficacy and Tolerability of BAF312 in Patients With Polymyositis

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT01801917
• Date of registration: 01/02/2013
• Prospective Registration: Yes
• Primary sponsor: Novartis Pharmaceuticals
• Public title: Efficacy and Tolerability of BAF312 in Patients With Polymyositis
• Scientific title: A Multi-centre Double-blind, Placebo Controlled, Proof of Concept Study to Evaluate the Efficacy and Tolerability of BAF312 in Patients With Polymyositis
• Date of first enrolment: April 24, 2013
• Target sample size: 14
• Recruitment status: Terminated
• URL: https://clinicaltrials.gov/show/NCT01801917
• Study type: Interventional
• Study design: Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).
• Phase: Phase 2

Countries of recruitment

Belgium, Canada, Czech Republic, Czechia, Hungary, Netherlands, Poland, Switzerland, Taiwan, United States

Contacts

• Name: Novartis Pharmaceuticals
• Affiliation: Novartis Pharmaceuticals

Key inclusion & exclusion criteria

Inclusion Criteria:

- "definite" or "probable" for polymyositis at least three months before Baseline

- active disease as defined by elevated CK levels, or other enzymes, or MRI/biopsy if
enzymes are normal, and persisting muscle weakness

- stable dose of corticosteroid for at least 2 weeks prior to Baseline and should not
have received a medium or high dose in the last 8 weeks prior to study entry.

- patients treated with methotrexate must have been on a stable dose for at least 6
weeks prior to Baseline.

Exclusion Criteria:

- Patients with overlap polymyositis, late-stage polymyositis, or other types of
myositis.

- Preexisting severe cardiac or pulmonary involvement, malignancy of any organ system or
significant eye diseases.

- Uncontrolled diabetes mellitus or diabetes complicated with organ involvement.

- Pregnant or nursing (lactating) women



Age minimum: 18 Years
Age maximum: 75 Years
Gender: All

Health Condition(s) or Problem(s) studied

Polymyositis

Intervention(s)

Drug: BAF312

Drug: Placebo

Primary Outcome(s)

Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) for Combined Efficacy Endpoint: Manual Muscle Testing in 24 Muscles (MMT24) [Time Frame: Baseline, at 12 weeks]

Percent Change From Baseline at Week 12 for BAF312 2 mg, 10 mg or Placebo (Once Daily) Serum Creatine Kinase (CK) Levels [Time Frame: Baseline, at 12 weeks]

Secondary Outcome(s)

Six-minute Walking Distance (6MWD) at Week 24 [Time Frame: Baseline, 24 weeks]

Six-minute Walking Distance (6MWD) at Week 12 [Time Frame: Baseline, 12 weeks]

BAF312 Trough Plasma Concentrations (PK Set) [Time Frame: -7 Baseline, day 28, 56, 84]

Secondary ID(s)

CBAF312X2205

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 04/01/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01801917