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i.v. recombinant human C1 inhibitor

Phase 3

Genetic Disorders | Small molecule | Rare Disease |Pharming Group N.V.|Trials Updated: Mar 29, 2024

i.v. recombinant human C1 inhibitor development status

Highest phase Phase 3 (NCT01188564)
Phase scored for PHARPhase 2
Registered trials 6 across 2 sponsors since Dec 2005

i.v. recombinant human C1 inhibitor target and mechanism

ModalitySmall molecule

Also known as rhC1INH

Success Probability

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Market & Valuation

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Trial Design

UNCONTROLLEDDMC
Total Trials4
Total Enrollment160

FDA Designations

No designations recorded

i.v. recombinant human C1 inhibitor clinical trials

i.v. recombinant human C1 inhibitor · 4 trials · 2 indications

Phase 3 1Phase 2 3
NCT01188564Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE AttacksHereditary Angioedema
COMPLETED75 Analytics
PHASE3COMPLETED
Efficacy, Safety and Immunogenicity Study of Recombinant Human C1 Inhibitor for the Treatment of Acute HAE Attacks
Hereditary AngioedemaUnlock trial analytics

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).

Primary outcomes: Relief of angioedema symptoms
24 hours

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.
Secondary outcomes: Safety and tolerability; pharmacokinetics/pharmacodynamics
90 days
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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.
Recombinant Human C1INHEXPERIMENTAL -
1OTHEROpen-label i.v. administration of 100 U/kg rhC1INH

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
i.v. recombinant human C1 inhibitorDRUG -
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Eligibility Criteria

Age Range13 Years to 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 AfricaCzechiaGermanySlovakiaNetherlands
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Frequently asked questions about i.v. recombinant human C1 inhibitor

What is i.v. recombinant human C1 inhibitor used for?

I.v. recombinant human C1 inhibitor is an investigational therapy being studied for the treatment of acute attacks in patients with hereditary angioedema, a genetic disorder. It is administered intravenously and is currently in Phase 2 clinical development.

Who makes i.v. recombinant human C1 inhibitor?

I.v. recombinant human C1 inhibitor is being developed by Pharming Group N.V., a biopharmaceutical company listed on the stock exchange under the ticker symbol PHAR.

What phase is i.v. recombinant human C1 inhibitor in?

I.v. recombinant human C1 inhibitor is in Phase 2 clinical development. It is an investigational drug and has not been approved by regulatory authorities. Two Phase 2 trials have been completed, but the drug remains in clinical development.

What clinical trials is i.v. recombinant human C1 inhibitor in?

I.v. recombinant human C1 inhibitor has been studied in two completed Phase 2 clinical trials: NCT00261053 and NCT00262288. Both trials evaluated the drug for the treatment of acute attacks in patients with hereditary angioedema and were conducted in the Netherlands.

Is i.v. recombinant human C1 inhibitor the same as recombinant human C1 esterase inhibitor?

I.v. recombinant human C1 inhibitor is a recombinant form of the human C1 inhibitor protein. It is designed to replace the deficient or dysfunctional C1 inhibitor in patients with hereditary angioedema, thereby helping to control acute attacks of the condition.