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Coenzyme Q10

Phase 1

Charcot Marie Tooth Disease | Small molecule | Other |United Therapeutics Corporation|Last Updated: Jul 15, 2013

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment23
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00541164Effects of Coenzyme Q10 on Charcot-Marie-Tooth DiseasePHASE1 COMPLETED 23Sep 1, 2007Jan 1, 2013Jul 15, 20131 United States
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Study Endpoints
Primary Endpoints
Changes in weakness, fatigue and pain in persons with Charcot-Marie-Tooth disease after supplementation with 600 mgs a day of Coenzyme Q10.
60 weeks
Secondary Endpoints
Improvements in quality of life in subjects with CMT before and after CoQ10 supplementation.
60 weeks
Measure blood serum levels of the oxidized and reduced forms of CoQ10.
60 weeks
Measure liver function tests
visits 1, 6, 12
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposeSUPPORTIVE_CARE
Treatment Arms
ArmTypeDescription
1EXPERIMENTAL300 mg CoQ10 chewable wafer twice a day
2PLACEBO_COMPARATORChewable placebo wafer twice a day for 24 weeks with crossover to 300mg CoQ10 twice a day for weeks 24-48.
Interventions
NameTypeDescription
Coenzyme Q10DRUG300 mg CoQ10 twice a day for 48 weeks
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Subjects must have a diagnosis of CMT, confirmed by review of medical records by the study physician * Subjects can be of either gender * Subjects must be between the ages of 18 and 75 * Subjects must be able to complete all assessments at the designated time intervals * Subje...

Countries:United States
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