Proof of Concept Study Evaluating RNS60 in the Treatment of Relapsing Remitting Multiple Sclerosis

Study Overview
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Brief Summary
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The purpose of this study is to determine whether RNS60 is effective in the treatment of RR-MS compared to interferon beta-1a.

Official Title
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A Phase II Study Evaluating RNS60 Compared to Interferon Beta-1a (Avonex) for the Treatment of Relapsing Remitting Multiple Sclerosis

Conditions
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Relapsing Remitting Multiple Sclerosis

Intervention / Treatment
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* Drug: RNS60 125 ml
* Drug: RNS60 250 ml
* Drug: Interferon beta 1a


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Males or females, aged between 18 and 50 years. Diagnosis of RR-MS according to McDonald 2010 diagnostic criteria with a prior brain MRI that demonstrates lesions consistent with RRMS, both within the past year. No evidence of relapse during the 60 days prior to enrollment. EDSS score of 0-5 at screening. Women of childbearing potential who have a negative pregnancy test (serum HCG) at screening. Men or women of reproductive potential who commit to use adequate contraception during the study and for 1 month following the last day of treatment. Subjects must be capable of understanding the purpose and risks of the study and provide written, informed consent. Exclusion Criteria: Diagnosis of Secondary Progressive MS, Primary Progressive MS or Progressive Relapsing MS. Normal baseline brain MRI. History of any clinically significant autoimmune disease: inflammatory bowel disease, diabetes, lupus or severe asthma. Current or prior malignancies (excluding non-melanoma skin carcinoma or in situ carcinoma of the cervix that has been adequately treated.) Significant organ dysfunction, including cardiac, renal (eGFR ≤ 60 ml/min.), liver, central nervous system, pulmonary, vascular, gastrointestinal, endocrine, or metabolic (e.g., creatinine ≥ 1.6 mg/dL; ALT or AST ≥ 1.5x the upper limit of normal), history of myocardial infarction, congestive heart failure, or arrhythmias within 6 months prior to enrollment. Steroid therapy within 60 days prior to enrollment, with the exception of corticosteroids or ACTH for relapse treatment during the course of the study. Known allergy to Gadolinium-DTPA Therapy with any immunomodulatory drugs within 3 months prior to enrollment, including but not limited to interferons, glatiramir acetate, BG-12, teriflunomide, laquinimod and IV immunoglobulin. Treatment at any time with immunosuppressive drugs such as cladribine, total lymphoid irradiation, monoclonal antibody treatment, mitoxantrone, Tysabri, fingolimod, cytoxan, methotrexate. Participation in any investigational therapy within one year prior to enrollment, unless given approval by PI. Known or suspected current or past alcohol or drug abuse within one year prior to enrollment. Any medical, psychiatric or other condition that could result in a subject not being able to comply with protocol requirements.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 50 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: RNS60 125 ml<br>125 ml of RNS60 administered weekly by IV infusion | Drug: RNS60 125 ml<br> <br> |
| Experimental: RNS60 250 ml<br>250 ml of RNS60 administered weekly by IV infusion | Drug: RNS60 250 ml<br> <br> |
| Active Comparator: Interferon beta-1a<br>Weekly dose of 30 mcg Interferon beta-1a (Avonex) administered by intramuscular injection. | Drug: Interferon beta 1a<br> <br> |

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in number of GAD-enhancing lesions from baseline | Cumulative number of GAD-enhancing lesions by MRI at months 3, 4, 5, and 6 | 3, 4, 5, and 6 months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in number of T2 lesions from baseline | Cumulative number of new or newly enlarged T2 lesions over 6 months of treatment | Months 3, 4, 5, and 6 | 
| Brain volume | Brain volume by MRI over 6 months of treatment | 6 months | 
| T2 lesion volume | T2 lesion volume by MRI over 6 months of treatment | 6 month | 
| Annualized Relapse Rate | Annualized Relapse Rate over 6 months | 6 months | 
| Expanded Disability Status Scale (EDSS), change from baseline | Progression of disability as assessed by the Expanded Disability Status Scale at months 3 and 6. | 3, 6 months | 
| Multiple Sclerosis Functional Composite, change from baseline | Progression of disability as assessed by the Multiple Sclerosis Functional Composite tool at months 3 and 6 months. | 3, 6 months | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Relapsing remitting multiple sclerosis, RR-MS