A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration (OPERA)

Pharming Healthcare

Status and phase

Completed

Phase 2

Conditions

Genetic Disorders

Hereditary Angioedema

Treatments

Drug: Recombinant Human C1 Inhibitor

Study type

Interventional

Funder types

Industry

Identifiers

NCT00851409

C1 1207

Details and patient eligibility

About

Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").

Enrollment

25 patients

Sex

All

Ages

18+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

Aged at least 18 years
Signed informed consent
Comfirmed diagnosis of HAE with baseline plasma level of functional C1INH activity of less than 50% of normal, and/or proven HAE ,mutation in C1INH gene.

Exclusion criteria

A history of anaphylaxis or severe allergy (i.e. requiring medication) to food, proteins and/or drugs.
A history of allergic reactions to C1INH products or rabbit protein.
Any reported SAE related to study drug administration (withdrawal criterium)
Elevated IgE against rabbit dander (>0.35 kU/L; ImmunoCap assay; Phadia)
A diagnosis of acquired C1INH deficiency.
Woman of child bearing potential, pregnancy or breast-feeding
previous treatment within the last 3 months with plasma-derived C1INH
Any clinically significant abnormality in the routine haematology, biochemistry and urinalysis
Any condition or treatment that in the opinion of the investigator might interfere with the evaluation of the study objectives.
Any changes since screening that would exclude subject based on above exclusion criteria.