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Amiloride

Phase 2

Secondary Progressive Multiple Sclerosis | Small molecule | Immunology |United Therapeutics Corporation|Last Updated: Mar 26, 2020

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment445
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01910259MS-SMART: Multiple Sclerosis-Secondary Progressive Multi-Arm Randomisation TrialPHASE2 COMPLETED 445Dec 18, 2014Jul 4, 2018Mar 26, 202013 United Kingdom
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Study Endpoints
Primary Endpoints
MRI-derived Percentage Brain Volume Change (PBVC).
2 years

To establish whether a drug, from a panel of 3 leading candidate neuroprotective drugs, slows the rate of brain volume loss in SPMS over 96 weeks using MRI-derived percentage brain volume change (PBVC).

Secondary Endpoints
Multi-arm trial strategy assessment
2 years
Count of new and enlarging T2 lesions
2 years
Pseudo-atrophy
6 months
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AmilorideEXPERIMENTALAmiloride 5 mg twice per day (5 mg once per day for first 4 weeks) for 96 weeks
RiluzoleEXPERIMENTALRiluzole 50 mg twice per day (50 mg once per day for first 4 weeks) for 96 weeks
FluoxetineEXPERIMENTALFluoxetine 20 mg twice per day (20 mg once per day for first 4 weeks) for 96 weeks
PlaceboPLACEBO_COMPARATORMatched placebo 1 capsule twice per day (1 capsule a day for first 4 weeks) for 96 weeks
Interventions
NameTypeDescription
AmilorideDRUGComparison with placebo
RiluzoleDRUGComparison with placebo
FluoxetineDRUGComparison with placebo
PlaceboDRUGPlacebo comparator
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Eligibility Criteria
Age Range25 Years — 65 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: * Confirmed diagnosis of SPMS. Steady progression rather than relapse must be the major cause of increasing disability in the preceding 2 years. Evidence of progression, either an increase of at least one point in EDSS or clinical documentation of increasing disability in patien...

Countries:United Kingdom
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