Recent Updates
Recently added Catalysts

CINRYZE with rHuPH20

Phase 2

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
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
RandomizedDouble-BlindCONTROLLED
Total Trials2
Total Enrollment56
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01756157Subcutaneous CINRYZE With Recombinant Human Hyaluronidase for Prevention of Angioedema AttacksPHASE2 COMPLETED 47Feb 4, 2013Sep 13, 2013Jun 3, 202123 United States, Germany +2
NCT01095510CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12PHASE2 COMPLETED 9Jun 2, 2010Apr 17, 2012Jun 3, 202111 United States, Germany +1
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Normalized Number of Angioedema Attacks During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) 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. Participants who were dosed but did not have any attacks in the period were assigned a value of zero. The number of attacks was normalized for the number of days participants participated in a given period and expressed as the monthly frequency.

Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom
Within 4 hours following treatment
Secondary Endpoints
Cumulative Attack-severity During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Cumulative Daily-severity During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Cumulative Symptomatic Days During the Treatment Period
From Visit 1 (Week 1) up to Visit 16 (Week 8) during each treatment period
Unlock Study Endpoints
Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelCROSSOVER
PurposePREVENTION
Treatment Arms
ArmTypeDescription
SC CINRYZE with rHuPH20 Dose Level 1 followed by Dose Level 2EXPERIMENTALSC CINRYZE with rHuPH20 Dose Level 1 twice weekly (every 3 or 4 days) for 8 weeks followed by SC CINRYZE with rHuPH20 Dose Level 2 twice weekly (every 3 or 4 days) for 8 weeks.
SC CINRYZE with rHuPH20 Dose Level 2 followed by Dose Level 1EXPERIMENTALSC CINRYZE with rHuPH20 Dose Level 2 twice weekly (every 3 or 4 days) for 8 weeks followed by SC CINRYZE with rHuPH20 Dose Level 1 twice weekly (every 3 or 4 days) for 8 weeks.
500 U CINRYZE (10-25 kg body weight)EXPERIMENTALSingle IV dose of 500 U CINRYZE
1000 U CINRYZE (10-25 kg body weight)EXPERIMENTALSingle IV dose of 1000 U CINRYZE
1000 U CINRYZE (>25 kg body weight)EXPERIMENTALSingle IV dose of 1000 U CINRYZE
1500 U CINRYZE (>25 kg body weight)EXPERIMENTALSingle IV dose of 1500 U CINRYZE
Interventions
NameTypeDescription
CINRYZE with rHuPH20BIOLOGICAL -
CINRYZEBIOLOGICAL -
Unlock Study Design Details
Eligibility Criteria
Age Range12 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites23

Inclusion Criteria: * Be ≥12 years of age. * Have a confirmed diagnosis of Hereditary Angioedema. Exclusion Criteria: * Receipt of any C1 inhibitor (C1 INH) therapy or any blood products for treatment or prevention of an angioedema attack within 7 days before the first dose of study drug. * Be re...

Countries:United StatesGermanySpainSwedenHungary
Unlock Eligibility Criteria