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rhC1INH

Phase 3

Hereditary Angioedema | Small molecule | Other |Pharming Group N.V.|Last Updated: Mar 29, 2024

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 Trials2
Total Enrollment132
FDA Designations
No designations recorded
Clinical Trials (2)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01188564Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE AttacksPHASE3 COMPLETED 75Jan 1, 2011Mar 1, 2013Aug 7, 201527 United States, Bulgaria +9
NCT01359969Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) PatientsPHASE2 COMPLETED 57Jan 17, 2012Jul 17, 2017Mar 29, 202416 United States, Czechia +8
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Study Endpoints
Primary Endpoints
Time to Beginning of Relief of Symptoms
Patients observed for 24 hours

Time to beginning of relief is the time lapsed from the beginning of the infusion of study medication to the beginning of a beneficial effect based on patient's responses to the Treatmetn Effect Questionnaire (TEQ) for the primary attack location. The beginning of relief is defined as the first timepoint at which * The patient reports any of the following answers for TEQ question 1: "A little better", "Better" or "Much better"; and; * The patient reports the following answer for TEQ question 2: "Yes"; and, * There is persistence in improvement at the next assessment time, i.e.either the same or a better response to Question 1 and "Yes" to Question 2.

Time to Beginning of Relief Based on Visual Analogue Scale (VAS) Was Defined as the Time, in Minutes, From Time of Infusion to the Beginning of Relief.
The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion VAS score decrease of ≥ 20 mm from baseline.

Time to beginning of relief of symptoms that showed the response to treatment based on the overall VAS score decrease of ≥ 20 mm from baseline. Separate VAS forms will be given to express the current feelings considering the severity of angioedema symptoms for five possible anatomical locations. The form will be completed by placing vertical marks on each of the 100 mm horizontal lines. The Abdominal VAS measures the patient's perceptions relating to illness, pain, bloodedness, and nausea; the oro-pharyngeal-laryngeal (OPL) VAS measures illness, pain, swelling, breathing, speech, and swallowing; the facial VAS instrument measure illness, pain and swelling; the Peripheral VAS measures swelling, pain, and use of extremity; and the Urogenital VAS measures illness, pain, swelling, nausea, and urination. Time to beginning of relief will also be calculated based on the Investigator Score (IS) and Treatment Effect Questionnaire (TEQ).

Secondary Endpoints
Time to Minimal Symptoms
24 hours
Time to Minimal Symptoms Based on Patient's VAS Scores; Time From the Start of the Infusion of Study Medication to the First Assessment Time at Which the Overall Severity VAS Reaches a Value of Less Than 20 mm for All Locations
The assessment of the angioedema signs by the VAS and TEQ will be performed just before start of infusion, and at T30m, T1h, T2h, T4h, T8h and T24h after study medication infusion all locations where VAS Scores were recorded.
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
rhC1INHEXPERIMENTAL -
Placebo (Saline)PLACEBO_COMPARATOR -
Recombinant Human C1 InhibitorEXPERIMENTALPatients presented to the clinic within 5 hours of onset received rhC1INH 50 U/kg body weight up to a maximum of 4200 U.
Interventions
NameTypeDescription
rhC1INHDRUGOne i.v. injection of rhC1INH at the dose of 50 U/kg, for patients up to 84 kg; one i.v. injection of rhC1INH at the dose of 4200U (2 vials) for patients of 84 kg body weight or greater.
Placebo (Saline)DRUGOne i.v. injection of saline (NaCl 0.9% w/v), equivalent in volume to the active treatment
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Eligibility Criteria
Age Range13 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites27

Inclusion Criteria: * Aged at least 13 years * Signed written informed consent * Clear clinical and laboratory diagnosis of HAE with baseline plasma level of functional C1INH of less than 50% of normal * Willingness and ability to comply with all protocol procedures * Clinical symptoms of an eligib...

Countries:United StatesBulgariaCanadaHungaryIsraelItalyNorth MacedoniaPolandRomaniaSerbiaSouth AfricaCzechiaGermanySlovakia
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