Phase 2
N=25
A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration
Genetic Disorders · Hereditary Angioedema
Bottom Line
View on ClinicalTrials.gov: NCT00851409 ↗Enrolled (actual)
25
Serious AEs
8.0%
Results posted
Jan 2013
Primary outcome: Primary: HAE Attacks/Week — 0.9; 0.4 attacks/week
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Recombinant Human C1 Inhibitor (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Pharming Technologies B.V.
- Primary completion
- Mar 2010
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY HAE Attacks/Week |
0.9; 0.4 | — |
| SECONDARY The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters. |
1.06; 1.02; 1.00 | — |
Summary
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").
Eligibility Criteria
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.
Data sourced from ClinicalTrials.gov (NCT00851409). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.