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NCT00168103 Clinical Trial

Human C1 Esterase Inhibitor (C1-INH) in Subjects With Acute Abdominal or Facial Hereditary Angioedema (HAE) Attacks

Hereditary Angioedema Clinical Trial

Official title:
Human Pasteurized C1 Esterase Inhibitor Concentrate (CE1145) in Subjects With Congenital C1-INH Deficiency and Acute Abdominal or Facial HAE Attacks

Clinical Trial Details — Status: Completed

Administrative data
NCT number NCT00168103
Other study ID # CE1145_3001
Secondary ID 2004-001186-17
Status Completed
Phase Phase 2/Phase 3
First received September 12, 2005
Last updated March 11, 2015
Start date June 2005
Est. completion date December 2007

Study information
Verified date February 2011
Source CSL Behring
Contact n/a
Is FDA regulated No
Health authority United States: Food and Drug AdministrationAustralia: Department of Health and Ageing Therapeutic Goods AdministrationArgentina: Ministry of HealthBulgaria: Ministry of HealthCanada: Health CanadaCzech Republic: State Institute for Drug ControlHungary: National Institute of PharmacyIsrael: Ministry of HealthMacedonia: Ministry of HealthPoland: Office for Registration of Medicinal Products, Medical Devices and Biocidal ProductsRomania: National Medicines AgencyRussia: Ministry of Health of the Russian FederationSpain: Ministry of Health and ConsumptionSweden: Medical Products AgencyUnited Kingdom: Medicines and Healthcare Products Regulatory Agency
Study type Interventional

Clinical Trial Summary

HAE is a rare disorder characterized by functional C1 esterase inhibitor deficiency. If not treated adequately, the acute attacks of HAE can be life-threatening and may even result in fatalities, especially in case of swelling of the larynx. This clinical Phase 2/Phase 3 study was designed to provide clinically relevant data on dosing, efficacy and safety in subjects with HAE.

Description:

For each subject, only a single abdominal or facial attack was treated and evaluated. After receiving treatment, subjects were observed for a minimum of 4 hours, after which they could be discharged from the study center if they reported onset of symptom relief. Starting from 4 hours after treatment, subjects who reported insufficient or no symptom relief could receive a second dose of double-blind treatment (called "rescue medication") as follows: C1-INH 20 U/kg bw for subjects initially receiving placebo, C1-INH 10 U/kg bw for subjects initially receiving C1-INH 10 U/kg bw, and placebo for subjects initially receiving C1-INH 20 U/kg bw.

The study was defined to be successful if the primary outcome measure and at least one of the secondary outcome measures were met in the comparison between the C1-INH 20 U/kg bw group and the Placebo group.

Recruitment information / eligibility
Status Completed
Enrollment 126
Est. completion date December 2007
Est. primary completion date October 2007
Accepts healthy volunteers No
Gender Both
Age group 6 Years and older
Eligibility Key Inclusion Criteria:

- Documented congenital C1-INH deficiency

- Acute facial or abdominal HAE attack

Key Exclusion Criteria:

- Acquired angioedema

- Treatment with any other investigational drug within the last 30 days before study entry

- Treatment with any C1-INH concentrate within the previous 7 days

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Investigator), Primary Purpose: Treatment

Related Conditions & MeSH terms

Intervention

Biological:
• C1 Esterase Inhibitor
Single application of C1-INH administered intravenously by slow injection or infusion at a recommended rate of 4mL/min.
• Placebo
Single application of physiological saline solution equivalent to the volume calculated for subjects in the C1-INH 20 U/kg bw arm.

Locations
Country Name City State
Argentina Study Site Buenos Aires
Australia Study Site Westmead
Bulgaria Study Site Plovdiv
Bulgaria Study Site Sofia
Canada Study Site Edmonton
Canada Study Site Ottawa
Czech Republic Study Site Brno
Hungary Study Site Budapest
Israel Study Site Tel Hashomer
Macedonia, The Former Yugoslav R Study Site Skopje
Poland Study Site Grodzisk Mazowiecki
Poland Study Site Krakow
Romania Study Site Tirgu-Mures
Russian Federation Study Site 1 Moscow
Russian Federation Study Site 2 Moscow
Russian Federation Study Site 3 Moscow
Spain Study Site Madrid
Sweden Study Site Goeteborg
United Kingdom Study Site London
United States Study Site Atlanta Georgia
United States Study Site Bellingham Washington
United States Study Site Boston Massachusetts
United States Study Site Bronx New York
United States Study Site Chicago Illinois
United States Study Site Cincinnati Ohio
United States Study Site Dallas Texas
United States Study Site Eugene Oregon
United States Study Site Granada Hills California
United States Study Site Hershey Pennsylvania
United States Study Site Idaho Falls Idaho
United States Study Site Omaha Nebraska
United States Study Site Plymouth Minnesota
United States Study Site Rapid City South Dakota
United States Study Site Shreveport Louisiana
United States Study Site Tulsa Oklahoma
United States Study Site Weston Florida

Sponsors (1)
Lead Sponsor Collaborator
CSL Behring

Countries where clinical trial is conducted

United States,  Argentina,  Australia,  Bulgaria,  Canada,  Czech Republic,  Hungary,  Israel,  Macedonia, The Former Yugoslav Republic of,  Poland,  Romania,  Russian Federation,  Spain,  Sweden,  United Kingdom, 

References & Publications (2)

Bernstein JA, Ritchie B, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz DS, Obtulowicz K, Reshef A, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Keinecke HO, Craig TJ. Hereditary angioedema: Validation of the end point time to onset of relief by correlation with symptom intensity. Allergy Asthma Proc. 2011 Jan-Feb;32(1):36-42. doi: 10.2500/aap.2011.32.3404. — View Citation

Craig TJ, Levy RJ, Wasserman RL, Bewtra AK, Hurewitz D, Obtulowicz K, Reshef A, Ritchie B, Moldovan D, Shirov T, Grivcheva-Panovska V, Kiessling PC, Keinecke HO, Bernstein JA. Efficacy of human C1 esterase inhibitor concentrate compared with placebo in ac — View Citation

Outcome
Type Measure Description Time frame Safety issue
Other Time to Complete Resolution of All HAE Symptoms, Including Pain Complete resolution of symptoms was determined by subject self-assessment. Up to 24 h after start of study treatment No
Other Number of Subjects Receiving Rescue Study Medication Within 4 h after start of study treatment No
Primary Time to Start of Relief of Symptoms From HAE Attack The start of symptom relief was determined by subject self-assessment. Time to start of symptom relief was set to 24 hours if the subject received rescue medication (blinded study medication, narcotic analgesics, antiemetics, open-label C1-INH, or fresh frozen plasma) at any time point after the start of study treatment but before start of relief. Up to 24 h after start of study treatment No
Secondary Number of Subjects With Worsened Intensity of Clinical HAE Symptoms Includes any worsening of intensity of at least 1 of the HAE symptoms present at baseline. Routinely checked symptoms included pain, nausea, vomiting, cramps, and diarrhea. Baseline and between 2 and 4 h after start of study treatment No
Secondary Number of Vomiting Episodes Within 4 h after start of study treatment No
See also
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Completed NCT00438815 - Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks Phase 3
Completed NCT00748202 - Berinert P Study of Subcutaneous Versus Intravenous Administration Phase 3
Completed NCT01426763 - A Study to Evaluate the Safety and Pharmacology of Subcutaneous Administration of CINRYZE With Recombinant Human Hyaluronidase Phase 2
Terminated NCT04091113 - Hereditary Angioedema Kininogen Assay
Completed NCT00432510 - Pharmacokinetics of C1 Esterase Inhibitor in Hereditary Angioedema Subjects Phase 1
Completed NCT03712228 - A Study to Investigate CSL312 in Subjects With Hereditary Angioedema (HAE) Phase 2
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Completed NCT02303626 - 12-Week Safety and Efficacy Study of BCX4161 as an Oral Prophylaxis Against HAE Attacks Phase 2/Phase 3
Not yet recruiting NCT02159430 - Hereditary AngioEdema, Neurobiology and Psychopathology N/A
Completed NCT01984788 - Safety and Efficacy of Oral BCX4161 as a Prophylactic Treatment for HAE Phase 2
Completed NCT04888650 - Assessment of the State of Health, Quality of Life and Expectations of Patients With Hereditary Angioedema
Completed NCT02448264 - First-in-Human Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of BCX7353 in Healthy Western and Japanese Volunteers Phase 1
Completed NCT05118958 - Phase 1 Crossover Study in Healthy Subjects to Evaluate the PK Profile of KVD824 Following Single and Multiple Doses of Modified Release (MR) Formulations Phase 1
Completed NCT06414252 - Social Evaluated Cold Pressor Test in Hereditary Angioedema Patients
Active, not recruiting NCT04739059 - Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema Attacks Phase 3
Completed NCT02819102 - An Open-label Drug-Drug Interaction Study to Evaluate the Effect of BCX7353 on Cytochrome P450 Enzyme Activity Using Probe Substrates Phase 1

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