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Trial record 19 of 84 for:    Polymyositis AND Myopathy

Efficacy and Tolerability of BAF312 in Patients With Polymyositis

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ClinicalTrials.gov Identifier: NCT01801917
Recruitment Status : Terminated
First Posted : March 1, 2013
Results First Posted : January 4, 2018
Last Update Posted : January 4, 2018
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )

Study Type: Interventional
Study Design: Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Condition: Polymyositis
Interventions: Drug: Placebo
Drug: BAF312

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
BAF312 2mg/BAF312 2mg Patients in Period 1 continue on same 2 mg dose of BAF312 in Period 2
BAF312 10 mg/BAF312 10 mg Patients in Period 1 continue on same 10 mg dose of BAF312 in Period 2
Placebo/BAF312 2 mg Patients on placebo in Period 1 switch to active 2 mg BAF312 in Period 2
Placebo/BAF312 10 mg Patients on placebo in Period 1 switch to active 10 mg BAF312 in Period 2

Participant Flow for 2 periods

Period 1:   Period 1 - Randomized
    BAF312 2mg/BAF312 2mg   BAF312 10 mg/BAF312 10 mg   Placebo/BAF312 2 mg   Placebo/BAF312 10 mg
STARTED   7   2   4   1 
COMPLETED   6   2   3   1 
NOT COMPLETED   1   0   1   0 
Adverse Event                1                0                1                0 

Period 2:   Extension - All Active
    BAF312 2mg/BAF312 2mg   BAF312 10 mg/BAF312 10 mg   Placebo/BAF312 2 mg   Placebo/BAF312 10 mg
STARTED   6   0   3   0 
COMPLETED   6   0   3   0 
NOT COMPLETED   0   0   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
BAF312 2mg/BAF312 2mg Patients in Period 1 continue on same 2 mg dose of BAF312 in Period 2
BAF312 10 mg/BAF312 10 mg Patients in Period 1 continue on same 10 mg dose of BAF312 in Period 2
Placebo/BAF312 2 mg Patients on placebo in Period 1 switch to active 2 mg BAF312 in Period 2
Placebo/BAF312 10 mg Patients on placebo in Period 1 switch to active 10 mg BAF312 in Period 2
Total Total of all reporting groups

Baseline Measures
   BAF312 2mg/BAF312 2mg   BAF312 10 mg/BAF312 10 mg   Placebo/BAF312 2 mg   Placebo/BAF312 10 mg   Total 
Overall Participants Analyzed 
[Units: Participants]
 7   2   4   1   14 
Age 
[Units: Years]
Mean (Standard Deviation)
 50.3  (14.78)   47.0  (21.21)   48.0  (8.83)   53.0  (0.0)   49.4  (12.51) 
Sex: Female, Male 
[Units: Participants]
Count of Participants
         
Female      5  71.4%      2 100.0%      2  50.0%      1 100.0%      10  71.4% 
Male      2  28.6%      0   0.0%      2  50.0%      0   0.0%      4  28.6% 
Race/Ethnicity, Customized 
[Units: Participants]
         
Caucasian   7   1   3   0   11 
Black   0   0   0   1   1 
Asian   0   1   1   0   2 
Disease duration 
[Units: Years]
Mean (Standard Deviation)
 5.6  (4.46)   5.4  (2.74)   2.7  (1.67)   16.9  (0)   5.6  (4.77) 
Baseline MMT24 Score [1] 
[Units: Scores on a scale]
Mean (Standard Deviation)
 202.6  (41.74)   184.0  (19.80)   189.5  (45.65)   166.0  (0)   193.6  (37.90) 
[1] Manual muscle testing in 24 muscle groups (MMT24). The scores range was 0 to 260. Higher scores indicate better outcome.
Taking DMARD at baseline [1] 
[Units: Participants]
 7   2   4   1   14 
[1] Taking a disease-modifying antirheumatic drugs


  Outcome Measures

1.  Primary:   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 ]

2.  Primary:   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 ]

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

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

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


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
The agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Results Point of Contact:  
Name/Title: Study Director
Organization: Novartis Pharmaceuticals
phone: 862-778-8300
e-mail: Novartis.email@novartis.com



Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01801917     History of Changes
Other Study ID Numbers: CBAF312X2205
First Submitted: February 1, 2013
First Posted: March 1, 2013
Results First Submitted: August 1, 2017
Results First Posted: January 4, 2018
Last Update Posted: January 4, 2018