Safety, Tolerability, Efficacy and Optimal Dose Finding Study of BAF312 in Patients With Relapsing-remitting Multiple Sclerosis



Status:Completed
Conditions:Neurology
Therapuetic Areas:Neurology
Healthy:No
Age Range:18 - 55
Updated:4/17/2018
Start Date:March 2009
End Date:May 2011

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A Phase II, Double-blind, Randomized, Multi-center, Adaptive Dose-ranging, Placebo-controlled, Parallel-group Study Evaluating Safety, Tolerability and Efficacy on MRI Lesion Parameters and Determining the Dose Response Curve of BAF312 Given Orally Once Daily in Patients With Relapsing-remitting Multiple Sclerosis.

The purpose of this study is to determine the dose-response curve for the MRI-based efficacy
of BAF312 compared with placebo in patients with Relapsing-Remitting Multiple Sclerosis
(RRMS), and to characterize its safety and tolerability for the selection of an optimal dose
in a later phase III study.

Study Design Rationale An adaptive design was chosen to characterize the dose response curve
of BAF312. In a first period of study ("Period 1"), three doses of BAF312 and placebo are
tested for MRI efficacy. Based on an interim analysis (IA) after 3 months of treatment, two
additional active doses for period 2 have been selected , thus allowing to optimize the
overall determination of the dose response curve with 5 data points of active treatment, and
placebo. The doses are kept blinded. The use of Modeling and Simulation allows to establish
the full range and dynamics of the dose-response curve in silico, and hence the definition of
the optimal dose for later phase III studies.

The choice of placebo as treatment control is essential to obtain information on the specific
compared to non-specific effects of active treatment and provides the best way of evaluating
the efficacy and of assessing the true safety and tolerability profile of BAF312. Short-term
placebo exposure (6 (Period 1) or 3 (Period 2) months, respectively) is unlikely to lead to
longer term differences in outcomes [Polman, 2008]. The use of an adaptive design strategy
contributes to a significant reduction of placebo exposure, both in terms of the number of
patients and duration, as compared to conventional trial models.

Patients having completed the study within the protocol may be eligible for the Extension
Phase study where they receive long-term BAF312 treatment (a separate protocol).


Inclusion Criteria:

- Diagnosis of Multiple Sclerosis (MS) as defined by revised McDonald criteria.

- A relapsing-remitting course of disease with at least 1 documented relapse during the
previous year, or 2 documented relapses during the previous 2 years, or a positive
gadolinium (Gd)-enhanced MRI scan at screening (in case the first MRI scan obtained at
screening is negative, a second scan may be obtained 1 month later.)

- An Expanded Disability Status Scale (EDSS) score of 0-5.0 inclusive at randomization.

- Neurologically stable with no evidence of relapse or corticosteroid treatment within
30 days prior to randomization.

- Patients who decline initiation or continuation of treatment with available disease
modifying drugs for MS, for whatever reason, after having been informed about their
respective benefits and possible adverse events by the investigator.

Exclusion Criteria:

- A manifestation of another type of MS than RRMS

- History of chronic disease of the immune system other than MS

- Malignancies, diabetes, significant cardiovascular, pulmonary and hepatic diseases and
conditions

- Active infections

Other protocol-defined inclusion/exclusion criteria may apply
We found this trial at
17
sites
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Cullman, AL
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Pompano Beach, Florida 33060
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2919 Swann Avenue
Tampa, Florida 33609
813-353-9613
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Tampa, FL
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Akron, OH
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Centennial, Colorado 80112
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Centennial, CO
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Chicago, IL
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Elk Grove Village, Illinois 60007
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Grand Rapids, Michigan 49525
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Grand Rapids, MI
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Greenville, South Carolina 29615
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Greenville, SC
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Miami, FL
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2900 W Oklahoma Ave
Milwaukee, Wisconsin 53215
414-649-6000
Aurora St. Luke's Medical Center At Aurora St. Luke's Medical Center, you'll find remarkable treatment...
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Raleigh, North Carolina 27607
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Raleigh, NC
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San Francisco, California 94143
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San Francisco, CA
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Seattle, Washington 98122
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Seattle, WA
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Tallahassee, Florida 32308
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Vancouver, British Columbia
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Vancouver,
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