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CINRYZE 500

Phase 3

Hereditary Angioedema (HAE) | Monoclonal antibody | 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
RandomizedCONTROLLED
Total Trials1
Total Enrollment12
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02052141Safety and Efficacy Study of CINRYZE for Prevention of Angioedema Attacks in Children Ages 6-11 With Hereditary AngioedemaPHASE3 COMPLETED 12Mar 20, 2014May 4, 2017Jun 3, 20218 United States, Germany +3
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Study Endpoints
Primary Endpoints
Normalized Number of Angioedema Attacks Per Month in a Treatment Period
From start of treatment up to 12 weeks during each treatment period

Angioedema attack was defined as the participant-reported indication of symptoms or signs such as swelling or pain at any location following a report of no swelling or pain on the previous day. Manifestations of an attack that progress from one site to another, prior to complete resolution, was considered a single attack. Attacks that began to regress and then worsened before complete resolution was also considered one attack. Attacks that began then appeared to resolve and then reappeared without a symptom-free calendar day reported after the appearance of resolution were considered 1 attack. Any events of swelling due to trauma or symmetrical nonpainful swelling of the lower extremities were not considered an angioedema attack. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Secondary Endpoints
Cumulative Attack-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period
From start of treatment up to 12 weeks during each treatment period
Cumulative Daily-severity Score of Angioedema Attacks Normalized Per Month in a Treatment Period
From start of treatment up to 12 weeks during each intervention period
Normalized Number of Angioedema Attacks Per Month Requiring Acute Treatment in a Treatment Period
From start of treatment up to 12 weeks during each intervention period
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelCROSSOVER
PurposePREVENTION
Treatment Arms
ArmTypeDescription
500/1000EXPERIMENTAL500 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks
1000/500EXPERIMENTAL1000 Units of CINRYZE administered by IV injection twice per week for 12 weeks followed by 500 Units of CINRYZE administered by IV injection twice per week for 12 weeks
Interventions
NameTypeDescription
CINRYZE 500BIOLOGICAL500 Units of CINRYZE administered by IV injection
CINRYZE 1000BIOLOGICAL1000 Units of CINRYZE administered by IV injection
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Eligibility Criteria
Age Range6 Years — 11 Years
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: * Diagnosis of Type I or Type II HAE. * History of angioedema attacks. Exclusion Criteria: * History of bleeding or clotting abnormality. * Diagnosis of acquired angioedema or known to have C1 INH antibodies. * History of allergic reaction to C1 esterase inhibitor or other blo...

Countries:United StatesGermanyIsraelMexicoRomania
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