NCT00851409

Brief 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").

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2009

Shorter than P25 for phase_2

Geographic Reach
2 countries

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

February 25, 2009

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 26, 2009

Completed
3 months until next milestone

Study Start

First participant enrolled

June 1, 2009

Completed
9 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 1, 2010

Completed
1 month until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2010

Completed
2.8 years until next milestone

Results Posted

Study results publicly available

January 31, 2013

Completed
Last Updated

June 26, 2018

Status Verified

April 1, 2018

Enrollment Period

9 months

First QC Date

February 25, 2009

Results QC Date

November 9, 2012

Last Update Submit

April 3, 2018

Conditions

Genetic DisordersHereditary Angioedema

Outcome Measures

Primary Outcomes (1)

  • HAE Attacks/Week

    Prior to the treatment period, patients enrolled in the study, were asked about the amount of "HAE" attacks in the past 2 years, (calculated to attacks/week), this number is defined as "Historical". During the treatment period, patients received a dose of 50 IU/kg of "rhC1INH" administered by slow "IV" injection over 4 to 5 minutes, once a week during an eight week period. The amount of attacks during this period is defined as "Prophylaxis" (calculated to attacks/week).

    8 weeks

Secondary Outcomes (1)

  • The Evaluation of Pharmacokinetic/ Pharmacodynamic ("PK/PD")Parameters.

    8 weeks

Study Arms (1)

Recombinant Human C1 Inhibitor

OTHER

Weekly administration of 50 IU/kg Recombinant Human C1 Inhibitor

Drug: Recombinant Human C1 Inhibitor

Interventions

Recombinant Human C1 InhibitorDRUG

50 IU/kg "rhC1INH", "IV" injection over 4 to 5 minutes, once weekly over an 8-week treatment period.

Also known as: "rhC1INH", Ruconest
Recombinant Human C1 Inhibitor

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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.

You may not qualify if:

  • 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.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

For information on sites, please contact Pharming Technologies

Leiden, Netherlands

Location

Emergency County Hospital, Internal Medicin Clinica, Allergology-Immunology Department

Târgu Mureş, 541103, Romania

Location

MeSH Terms

Conditions

Genetic Diseases, InbornAngioedemas, Hereditary

Interventions

Complement C1 Inhibitor Proteinconestat alfa

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and AbnormalitiesAngioedemaVascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesUrticariaSkin Diseases, VascularSkin DiseasesSkin and Connective Tissue DiseasesHypersensitivity, ImmediateHypersensitivityImmune System DiseasesImmunologic Deficiency Syndromes

Intervention Hierarchy (Ancestors)

GlycoproteinsGlycoconjugatesCarbohydratesComplement C1 Inactivator ProteinsSerpinsPeptidesAmino Acids, Peptides, and ProteinsComplement Inactivator ProteinsComplement System ProteinsImmunoproteinsBlood ProteinsProteins

Results Point of Contact

Title
Medical Affairs Department
Organization
Pharming Technologies BV
medicalinfo@pharming.com
+31715247400

Study Officials

  • Bruno Giannetti, MD

    COO Pharming

    STUDY CHAIR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 25, 2009

First Posted

February 26, 2009

Study Start

June 1, 2009

Primary Completion

March 1, 2010

Study Completion

April 1, 2010

Last Updated

June 26, 2018

Results First Posted

January 31, 2013

Record last verified: 2018-04

Locations

NL(1)RO(1)