Active DermatomyositisPhase 2NCT02029274
Safety and Efficacy of BAF312 in Dermatomyositis
A Phase 2 study testing BAF312 in people with Active Dermatomyositis, run by Novartis Pharmaceuticals at 9 sites in 3 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Active Dermatomyositis
- What is being tested?
- BAF312
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 17 participants
- Where
- 9 sitesin 3 countries
- Study dates
- 25 Aug 2013to 17 Feb 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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 locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
United States6 sites
Novartis Investigative Site
Boston, Massachusetts, 02115
Novartis Investigative Site
Kansas City, Kansas, 66160
Novartis Investigative Site
Los Angeles, California, 90095
Novartis Investigative Site
Miami, Florida, 33136
Novartis Investigative Site
Orange, California, 92868
Novartis Investigative Site
Phoenix, Arizona, 85028
Japan2 sites
Novartis Investigative Site
Chiba, Chiba, 260-8712
Novartis Investigative Site
Sendai, Miyagi, 980-8574
About this study
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)
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 .
Read the 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.
- Conditions
- Active Dermatomyositis
- Being tested
- BAF312, Placebo
- Keywords
- Dermatomyositis
- Medical subject headings
- Dermatomyositis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BAF312Drug
Also called Siponimod
BAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration.
PlaceboDrug
Matching placebo to BAF312 as tablets for oral administration.
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: 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. Receives: 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. Receives: 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. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Baseline, 6 months
Secondary outcomes (4)
BAF312 Plasma Concentration
Measured: 6 months
Peripheral Blood Lymphocyte Counts
Absolute lymphocyte counts
Measured: baseline, 6 months
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.
Measured: baseline, 3 months
Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
Measured: baseline, 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Jan 2014 (estimated)
- Study start25 Aug 2013
- Primary completion17 Feb 2016
- Study completion17 Feb 2016
Why it stopped: The study was terminated prematurely after an interim analysis for futility. The study did not provide any evidence for efficacy of BAF312 in dermatomyositis.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Active DermatomyositisStudies of the same condition, open and under way.
- More trials run by Novartis PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Investigator, Outcomes assessor
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Novartis Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Novartis Pharmaceuticals, Study director, Novartis Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02029274
- Sponsor's protocol ID
- CBAF312X2206
- Other IDs
- 2013-001799-39
- Status last verified
- Dec 2018
- First submitted
- 2 Dec 2013
- Last updated
- 5 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 5 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02029274
- Acronym / sponsor ID
- CBAF312X2206
- Conditions
- Active Dermatomyositis
- Keywords
- Dermatomyositis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: The study was terminated prematurely after an interim analysis for futility. The study did not provide any evidence for efficacy of BAF312 in dermatomyositis.
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Novartis PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 25 Aug 2013 (actual)
- Primary completion
- 17 Feb 2016 (actual)
- Study completion
- 17 Feb 2016 (actual)
- First posted
- 7 Jan 2014 (estimated)
- Results
- Posted 15 Jan 2019
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, investigator, outcomes assessor)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| BAF312 0.5mg | Experimental | Drug: BAF312During 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. |
| BAF312 2mg | Experimental | Drug: BAF312During 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. |
| BAF312 10 mg | Experimental | Drug: BAF312During 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. |
| Placebo | Placebo comparator | Drug: PlaceboDuring 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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
Key exclusion criteria (4 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BAF312Also: SiponimodBAF312 was provided as film-coated tablets in strengths of 0.25, 0,5, 1 and 2 mg for oral administration. | Drug | BAF312 0.5mg, BAF312 10 mg, BAF312 2mg |
| PlaceboMatching placebo to BAF312 as tablets for oral administration. | Drug | Placebo |
Primary outcome
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Time frame: Baseline, 6 months
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.
4 secondary outcomes
BAF312 Plasma Concentration
Time frame: 6 months
Peripheral Blood Lymphocyte Counts
Time frame: baseline, 6 months
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score
Time frame: baseline, 3 months
Change From Baseline in 6 Minutes Walking Distance (6-MWD) Test
Time frame: baseline, 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Trial Summary is available on novartisclinicaltrials.comLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Novartis Pharmaceuticals study director
Novartis Pharmaceuticals
9 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing BAF312 in people with Active Dermatomyositis, run by Novartis Pharmaceuticals at 9 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 5 Jan 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in United States, Japan, Czechia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 15 Jan 2019). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change From Baseline in Manual Muscle Testing - 24 Muscles (MMT-24) Score (time frame: Baseline, 6 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Jan 2021.