A Study of Orally Administered BGC20-0134 (Structured Lipid) in Patients With Relapsing Remitting Multiple Sclerosis (RRMS)

Study Overview
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Brief Summary
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To determine the efficacy and safety of an oral drug (BGC20-0134) in patients with relapsing remitting multiple sclerosis. Specifically, the cumulative number of new gadolinium enhancing lesions after 24 weeks of treatment with BGC20-0134.

Detailed Description
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Primary outcome measure: The cumulative number of new GdE T1 lesions developing while on treatment. Secondary outcome measures: MRI: Cumulative number of total GdE T1 lesions developing while on treatment Cumulative number of new T2 lesions Patients free of GdE (T1-weighted) lesions at week 24 Change in volume of GdE T1 Brain atrophy Cumulative number of new T1 hypointense lesions (black holes) Disease burden, T1 and T2 lesion activity at week 48. Number of clinical relapses from baseline to the end of treatment. • Change on the Expanded Disability Status Scale (EDSS) Number of patients requiring methylprednisolone treatment for a relapse. Serum levels of pro- and anti-inflammatory cytokines. Quality of life (MSQOL-54) Eligibility Criteria MS-Related inclusion criteria Diagnosis of relapsing MS according to the revised 2005 McDonald criteria. Has shown disease activity defined by 1 or more MS attack within the last year which has been documented in prior medical notes and or the presence of active lesions on historical scans being either (based on radiology report or investigator review of MRI): Gd-enhancing on any scan obtained in the last year, or new T2 lesions between two scans both obtained within the last year. A minimum total of 9 T2 lesions reported on a recent MRI obtained within 1 month prior to the screening visit. Baseline EDSS score 0 - 5.5. Has refused to be treated with approved disease modifying therapies available for MS, for any reason and once the investigator has fully informed the patient about the related benefits and potential adverse events associated with such treatments. Also, patients for whom such treatments have proved to be intolerable. Exclusion Criteria: Has experienced an MS relapse or received systemic corticosteroids or adrenocorticotropic hormone (ACTH) in the previous 1 month. Has a secondary progressive (SPMS), progressive relapsing (PRMS), or primary progressive MS (PPMS). Has received any of the following agents to treat MS (approved or unapproved): Within the previous 3 months: interferon beta, glatiramer acetate, intravenous immunoglobulin or plasmapheresis. Within the previous 12 months: natalizumab, daclizumab, cytapheresis, azathioprine, cladribine, cyclophosphamide, methotrexate, mitoxantrone, mycophenolate, pixantrone, sirolimus, tacrolimus, or other agents typically used to prevent transplant rejection or as cancer chemotherapy, excluding hormonal treatments. Ever having received: stem cell or bone marrow transplant, total lymphoid irradiation, vaccine therapy for MS, or monoclonal antibodies whose effects may be longer than 1 year (such as alemtuzumab or rituximab). Within the previous 3 months: any other agents given for the non-symptomatic treatment of MS which are not included above, including over-the-counter, herbal and nutritional supplements. However, if the agent is being taken primarily to treat another medical condition, then it is allowed as long as the dose is unchanged within the previous 3 months and is unlikely to change before week

Official Title
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A Placebo-controlled Phase IIa Study of Orally Administered BGC20-0134/Pleneva TM (Structured Lipid) in Patients With RRMS

Conditions
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Relapsing Remitting Multiple Sclerosis

Intervention / Treatment
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* Drug: Pleneva TM BGC20-0134
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Diagnosis of relapsing MS according to the revised 2005 McDonald criteria Has shown disease activity defined by 1 or more MS attack within the last year which has been documented in prior medical notes and or the presence of active lesions on historical scans being either (based on radiology report or investigator review of MRI): Gd-enhancing on any scan obtained in the last year, or new T2 lesions between two scans both obtained within the last year A minimum total of 9 T2 lesions reported on a recent MRI obtained within 1 month prior to the screening visit Baseline EDSS score 0 - 5.5 Has refused to be treated with approved disease modifying therapies available for MS, for any reason and once the investigator has fully informed the patient about the related benefits and potential adverse events associated with such treatments. Also, patients for whom such treatments have proved to be intolerable Exclusion Criteria: Has experienced an MS relapse or received systemic corticosteroids or adrenocorticotropic hormone (ACTH) in the previous 1 month Has a secondary progressive (SPMS), progressive relapsing (PRMS), or primary progressive MS (PPMS). Has received any of the following agents to treat MS (approved or unapproved): Within the previous 3 months: interferon beta, glatiramer acetate, intravenous immunoglobulin or plasmapheresis Within the previous 12 months: natalizumab, daclizumab, cytapheresis, azathioprine, cladribine, cyclophosphamide, methotrexate, mitoxantrone, mycophenolate, pixantrone, sirolimus, tacrolimus, or other agents typically used to prevent transplant rejection or as cancer chemotherapy, excluding hormonal treatments Ever having received: stem cell or bone marrow transplant, total lymphoid irradiation, vaccine therapy for MS, or monoclonal antibodies whose effects may be longer than 1 year (such as alemtuzumab or rituximab) Within the previous 3 months: any other agents given for the non-symptomatic treatment of MS which are not included above, including over-the-counter, herbal and nutritional supplements. However, if the agent is being taken primarily to treat another medical condition, then it is allowed as long as the dose is unchanged within the previous 3 months and is unlikely to change before week 24.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 65 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: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: BGC20-0134 (Pleneva TM)<br>Structured lipid | Drug: Pleneva TM BGC20-0134<br>* Placebo or 5 g dose<br>|
| Placebo Comparator: Placebo control<br>Placebo - dummy pill | Drug: Placebo<br>* Placebo or 5 g dose<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The cumulative number of new gadolinium-enhanced (GdE) T1 weighted lesions developing while on treatment (specifically the sum of new GdE T1 lesions seen on MRI at weeks 12, 16, 20 and 24). |  | 24 weeks | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Cumulative number of total GdE T1 weighted lesions developing while on treatment |  | 24 weeks | 
| Cumulative number of new T2 weighted lesions |  | 24 weeks | 
| Patients free of GdE (T1-weighted) lesions |  | 24 weeks | 
| Change in volume of GdE T1 weighted lesions |  | 24 weeks | 
| Change in volume of T2 lesions |  | 24 weeks | 
| Brain atrophy |  | 24 weeks | 
| Cumulative number of new T1 hypointense lesions (black holes) |  | 24 weeks | 
| Disease burden, T1 and T2 lesion activity at week 48. |  | 48 weeks | 
| Number of clinical relapses from baseline during the first 24 weeks. |  | 24 weeks | 
| Change on the Expanded Disability Status Scale (EDSS) during the first 24 weeks |  | 48 weeks | 
| Number of patients receiving methylprednisolone treatment for a relapse during the first 24 weeks. |  | 48 weeks | 
| Serum levels of cytokines during the first 24 weeks. |  | 24 weeks | 
| Quality of life (MSQOL-54) assessment |  | 48 weeks | 
| PK for determination of circulating levels of BGC20-0134 and plasma concentrations of dihomo-gamma linolenic acid (DHGLA) during the first 24 weeks. |  | 24 weeks | 
| Overall safety of BGC20-0134 |  | 48 weeks | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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Anti inflammatory, Pro inflammatory, TNF alpha, interleukin 1 beta, interferon gamma, Fayaz Master, Polyunsaturated fatty acid, Cytokine balance, Pleneva TM, BGC20-0134, RRMS, Oral treatment for MS, Oral drug for multiple sclerosis, Oral RRMS, Oral relapsing remitting multiple sclerosis, Gamma Linolenic Acid, GLA, Fatty acid, Triglyceride, Structured lipid, MRI, Magnetic resonance imaging, gadolinium enhancing lesions, expanded disability status scale, EDSS, Demyelination, Remyelination, TGFB1, Transforming growth factor beta 1, cytokines, disease modifying therapy, immunomodulator, Omega 6