Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

NCT00225147 | Completed Phase 2 Results DMC

• 1 other identifier: C1 1205-01
• Study Type: interventional
• Target: 77
• Locations: 1 country
• Sites: 1

Brief Summary

Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks. Funding Source - FDA OOPD

Conditions

Hereditary Angioedema; Angioneurotic Edema

Outcome Measures

Primary Outcomes (1)

• Time to Beginning of Relief of Symptoms

  The time to beginning of relief of symptoms at the location that showed the first visual analogue scale ("VAS") score decrease of at least 20 mm from baseline score with persistence to the next timepoint, assessment timepoints were taken on pre-scheduled time-points after study drug administration: baseline (0 minutes), 15 minutes, 30 minutes, 1 hour, 2 hours, 4 hours, 8 hours, 12 hours, 16 hours, 24 hours and 48 hours. Time to beginning of relief has been calculated as median time, by using the exact timepoints on which each assessment was performed.

  up to 48 hours after study drug administration

Secondary Outcomes (1)

• Time to Minimal Symptoms

  up to 48 hours after study drug administration

Study Arms (3)

100 IU/kg rhC1INH

EXPERIMENTAL

100 IU/kg Recombinant human C1 inhibitor

Drug: Recombinant Human C1 Inhibitor

50 IU/kg rhC1INH

EXPERIMENTAL

50 IU/kg Recombinant human C1 inhibitor

Drug: Recombinant Human C1 Inhibitor

Saline

PLACEBO COMPARATOR

Drug: placebo

Interventions

Recombinant Human C1 Inhibitor DRUG

IV

Also known as: "rhC1INH", Ruconest, conestat alfa

100 IU/kg rhC1INH; 50 IU/kg rhC1INH

placebo DRUG

saline solution

Also known as: saline, physiological salt solution

Saline

Eligibility Criteria

• Age: 12 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Clear clinical and laboratory diagnosis of HAE
• Plasma level of functional C1INH of less than 50% of normal
• Acute abdominal, urogenital, peripheral, and/or oro-facial/pharyngeal/laryngeal HAE attack

You may not qualify if:

• Acquired angioedema
• Pregnancy or breastfeeding
• Treatment with any investigational drug within prior 30 days
• Body weight \>120 kg

Sponsors & Collaborators

• Pharming Technologies B.V.: lead

Study Sites (1)

For information on sites please contact Pharming Medical Affairs Department

Leiden, 2300 AL, Netherlands

Location

Related Publications (3)

• Zuraw B, Cicardi M, Levy RJ, Nuijens JH, Relan A, Visscher S, Haase G, Kaufman L, Hack CE. Recombinant human C1-inhibitor for the treatment of acute angioedema attacks in patients with hereditary angioedema. J Allergy Clin Immunol. 2010 Oct;126(4):821-827.e14. doi: 10.1016/j.jaci.2010.07.021.

  PMID: 20920772 RESULT

• Bernstein JA, Relan A, Harper JR, Riedl M. Sustained response of recombinant human C1 esterase inhibitor for acute treatment of hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2017 Apr;118(4):452-455. doi: 10.1016/j.anai.2017.01.029. Epub 2017 Mar 9.

  PMID: 28284978 DERIVED

• Riedl MA, Levy RJ, Suez D, Lockey RF, Baker JW, Relan A, Zuraw BL. Efficacy and safety of recombinant C1 inhibitor for the treatment of hereditary angioedema attacks: a North American open-label study. Ann Allergy Asthma Immunol. 2013 Apr;110(4):295-9. doi: 10.1016/j.anai.2013.02.007. Epub 2013 Mar 6.

  PMID: 23535096 DERIVED

MeSH Terms

Conditions

Angioedemas, Hereditary; Angioedema

Interventions

Complement C1 Inhibitor Protein; conestat alfa; Sodium Chloride

Condition Hierarchy (Ancestors)

Vascular Diseases; Cardiovascular Diseases; Hereditary Complement Deficiency Diseases; Primary Immunodeficiency Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Urticaria; Skin Diseases, Vascular; Skin Diseases; Skin and Connective Tissue Diseases; Hypersensitivity, Immediate; Hypersensitivity; Immune System Diseases; Immunologic Deficiency Syndromes

Intervention Hierarchy (Ancestors)

Glycoproteins; Glycoconjugates; Carbohydrates; Complement C1 Inactivator Proteins; Serpins; Peptides; Amino Acids, Peptides, and Proteins; Complement Inactivator Proteins; Complement System Proteins; Immunoproteins; Blood Proteins; Proteins; Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Results Point of Contact

• Title: Medical Affairs Department
• Organization: Pharming Technologies BV

medicalinfo@pharming.com

+31715247400

Study Officials

• Anurag Relan, MD

  Pharming Group N.V.

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 20, 2005
• First Posted: September 23, 2005
• Study Start: July 1, 2005
• Primary Completion: October 1, 2009
• Study Completion: January 1, 2010
• Last Updated: February 22, 2013
• Results First Posted: August 31, 2012

Record last verified: 2013-02

Locations

NL (1)