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Recombinant human C1 inhibitor

Phase 3

Hereditary Angioedema | Monoclonal antibody | Immunology |Pharming Group N.V.|Last Updated: Jun 26, 2018

Success Probability
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Market & Valuation
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Trial Design
RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials3
Total Enrollment184
FDA Designations
No designations recorded
Clinical trial landscape

Recombinant human C1 inhibitor · 4 trials · 3 indications

Phase 3 1Phase 2 3
NCT00262301Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary AngioedemaHereditary Angioedema
COMPLETED75 Analytics
PHASE3COMPLETED
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
Hereditary AngioedemaUnlock trial analytics
Study Endpoints
Primary Endpoints
Time to Beginning of Relief of Symptoms
up to 48 hours after study drug administration

The time to beginning of relief of symptoms has been assessed by using a patient-reported visual analogue scale ("VAS") ranging from 0 mm (no symptoms at all) to 100 mm (extremely disabling). Time to beginning of relief of symptoms at the location that showed first "VAS" score decrease of at least 20 mm from baseline score (t= 0 min) to the next assessment time-point). Assessment time-points were taken on pre-scheduled time-points after drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours, 48 hours. Time to beginning of relief has been calculated as median time, by using the exact time-points on which each assessment was performed.

Number of HAE Attacks
28 days

Average number of HAE attacks normalized to a 28 day period

HAE Attacks/Week
8 weeks

Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).

Secondary Endpoints
Time to Minimal Symptoms
up to 48 hours after study drug administration
Number of Participants With Adverse Events
20 weeks
Percentage of Participants Achieving at Least 50% Reduction in Number of Attacks
28 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
100 IU/kg "rhC1INH"EXPERIMENTAL100 IU/kg recombinant human C1 inhibitor
SalinePLACEBO_COMPARATORSaline solution
rhC1INH twice weeklyEXPERIMENTALrhC1INH administered twice weekly
rhC1INH once weeklyEXPERIMENTALrhC1INH administered once weekly
Placebo (Saline) twice weeklyPLACEBO_COMPARATORPlacebo (Saline) administered twice weekly
Recombinant Human C1 InhibitorOTHERWeekly administration of 50 IU/kg Recombinant Human C1 Inhibitor
100 IU/kg rhC1INHEXPERIMENTAL100 IU/kg Recombinant human C1 inhibitor
50 IU/kg rhC1INHEXPERIMENTAL50 IU/kg Recombinant human C1 inhibitor
Interventions
NameTypeDescription
recombinant human C1 inhibitorDRUGIV
PlaceboDRUGIV
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Eligibility Criteria
Age Range16 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites2

Inclusion Criteria: * Clear clinical and laboratory diagnosis of HAE * Baseline plasma level of functional C1INH of less than 50% of normal * Evidence for exacerbation or development of a severe abdominal, oro-facial/ pharyngeal/ laryngeal, genito-urinary and/or peripheral HAE attack Exclusion Cri...

Countries:NetherlandsRomaniaUnited StatesCanadaCzechiaItalyNorth MacedoniaSerbia
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