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

NCT ID: NCT00225147

Last Updated: 2013-02-22

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2/PHASE3
• Total Enrollment: 77 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2005-07-31
• Study Completion Date: 2010-01-31

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

Detailed Description

A prospectively planned interim analysis will be performed on the double-blind data.

Conditions

Hereditary Angioedema; Angioneurotic Edema

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

100 IU/kg rhC1INH

100 IU/kg Recombinant human C1 inhibitor

Group Type: EXPERIMENTAL

Recombinant Human C1 Inhibitor

Intervention Type: DRUG

IV

50 IU/kg rhC1INH

50 IU/kg Recombinant human C1 inhibitor

Group Type: EXPERIMENTAL

Recombinant Human C1 Inhibitor

Intervention Type: DRUG

IV

Saline

Group Type: PLACEBO_COMPARATOR

placebo

Intervention Type: DRUG

saline solution

Interventions

Recombinant Human C1 Inhibitor

IV

Intervention Type: DRUG

placebo

saline solution

Intervention Type: DRUG

Other Intervention Names

"rhC1INH"; Ruconest; conestat alfa; saline; physiological salt solution

Eligibility Criteria

Inclusion Criteria

• 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

Exclusion Criteria

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

• Minimum Eligible Age: 12 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Pharming Technologies B.V.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Anurag Relan, MD

Role: STUDY_DIRECTOR

Pharming Group N.V.

Locations

For information on sites please contact Pharming Medical Affairs Department

Leiden, , Netherlands

Countries

Netherlands

References

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.

Reference Type: RESULT

PMID: 20920772 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 28284978 (View on PubMed)

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.

Reference Type: DERIVED

PMID: 23535096 (View on PubMed)

Other Identifiers

C1 1205-01

Identifier Type: -

Identifier Source: org_study_id