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Recombinant human C1 esterase inhibitor

Phase 1

Hereditary Angioedema (HAE) | Small molecule | Other |Takeda Pharmaceutical Company Limited|Last Updated: Jun 3, 2021

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_CONTROLLED
Total Trials1
Total Enrollment48
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02663687Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult SubjectsPHASE1 COMPLETED 48Feb 19, 2016Dec 5, 2016Jun 3, 20211 United States
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Study Endpoints
Primary Endpoints
Number of Participants With Treatment-emergent Adverse Events (TEAEs ) Including Serious Adverse Events (SAEs)
From the start of study treatment up to 28 days after the last dose of the study treatment (up to 56 days)

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. An AE was considered to be a TEAE in a specific treatment period of the study if the date and time of onset were after investigational product administration in that period and if it occurred less than equals to (\<=) Day 28 and was both not present at the start of that period and was not a chronic condition that was part of the participant's medical history, or it was present at the start of that period or as part of the participant's medical history but the severity or frequency increased during that period \<= Day 28. An SAE was defined as any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose.

Secondary Endpoints
Maximum Observed Plasma Concentration (Cmax) of SHP623 Occurring at Time of Maximum Observed Concentration During a Dosing Interval (Tmax)
Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.
Time of Maximum Plasma Concentration (Tmax) of SHP623 Sampled During a Dosing Interval
Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.
Terminal Half-life (t1/2) of SHP623
Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Treatment 1- 4EXPERIMENTALTreatment A: 9 Subjects will receive dose level I of SHP623 intravenously (IV). B: 9 Subjects will receive dose level I of SHP623 subcutaneously(SC).
PlaceboPLACEBO_COMPARATOR3 Subjects will receive placebo for each cohort
Interventions
NameTypeDescription
Recombinant human C1 esterase inhibitorDRUGSubjects will receive escalating doses I-IV as both IV and SC injections
PlaceboDRUGSubjects will receive matching placebo
SHP623DRUGSHP623
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Eligibility Criteria
Age Range18 Years — 50 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Must be considered healthy. Healthy status is defined by absence of evidence of any active or chronic disease 2. Male, or non-pregnant, non-lactating female, who agrees to comply with any applicable contraceptive requirements of the protocol, or females of non-child-bearing p...

Countries:United States
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