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Riluzole

Phase 1

Amyotrophic Lateral Sclerosis | Small molecule | Neurology |Third Harmonic Bio, Inc.|Last Updated: Jul 7, 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
ACTIVE_CONTROLLED
Total Trials1
Total Enrollment20
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT04454840Treatment of Intravenous Infusion Plasma in Amyotrophic Lateral SclerosisEARLY_PHASE1 COMPLETED 20May 1, 2016May 1, 2019Jul 7, 20201 China
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Study Endpoints
Primary Endpoints
Amyotrophic lateral sclerosis Functional Rating Scale scores
22 months after intervention

Rate of amyotrophic lateral sclerosis Functional Rating Scale scores changes

Secondary Endpoints
Survival time
22 months after intervention
Forced vital capacity (FVC)
22 months after intervention
Cognitive function evaluation (ECAS score)
22 months after intervention
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Biological+RiluzoleEXPERIMENTALPlasma from healthy young people treatment + Riluzole
RiluzoleACTIVE_COMPARATORRiluzole
Interventions
NameTypeDescription
Plasma from healthy young people treatment + RiluzoleBIOLOGICALIntravenous injection: the subjects received 400ml of intravenous plasma infusion every 2 weeks, with a continuous course of 2 treatments and a course of treatment every 3 months. The course of treatment lasted 10 months, with a total of 3200ml of plasma infusion.
RiluzoleDRUGThe basic treatment is Riluzole 25\~50mg twice daily
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Eligibility Criteria
SexALL
Healthy VolunteersNo
Study Sites1

Inclusion Criteria: * Meet the following diagnosis standard: confirmed, proposed and laboratory supported diagnosis; * Age 50-70 years old ; * 3-18 months course of disease; * Forced vital capacity (FVC) ≥70% predicted value; * Total amyotrophic lateral sclerosis Functional Rating Scale score ≥36, ...

Countries:China
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