NCT00261053

Brief Summary

The purpose of this single-center study is to explore the efficacy, safety, tolerability and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor in the treatment of acute attacks in patients with hereditary angioedema.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
14

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Jun 2003

Geographic Reach
1 country

1 active site

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

Study Start

First participant enrolled

June 1, 2003

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

September 1, 2005

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

December 1, 2005

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

December 2, 2005

Completed
Last Updated

April 25, 2013

Status Verified

April 1, 2013

Enrollment Period

2.3 years

First QC Date

December 1, 2005

Last Update Submit

April 24, 2013

Conditions

Genetic Disorders

Outcome Measures

Primary Outcomes (1)

  • Primary outcomes: Relief of angioedema symptoms

Secondary Outcomes (1)

  • Secondary outcomes: Safety and tolerability; pharmacokinetics/pharmacodynamics

Study Arms (1)

1

OTHER

Open-label i.v. administration of 100 U/kg rhC1INH

Drug: i.v. recombinant human C1 inhibitor

Interventions

i.v. recombinant human C1 inhibitorDRUG
1

Eligibility Criteria

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

You may qualify if:

  • Clinical and laboratory diagnosis of HAE
  • Plasma level of functional C1INH of less than 50% of normal
  • Severe attack of abdominal, facial-oro-pharyngeal, genito-urinary and/or peripheral HAE.

You may not qualify if:

  • Acquired angioedema
  • Pregnancy or breastfeeding
  • Participation in another clinical study within prior 3 months

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

For information contact Sonja Visscher

Leiden, 2333 CN, Netherlands

Location

MeSH Terms

Conditions

Genetic Diseases, Inborn

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

  • Jan Nuijens, MD, PhD

    Pharming Technologies B.V.

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY

Study Record Dates

First Submitted

December 1, 2005

First Posted

December 2, 2005

Study Start

June 1, 2003

Primary Completion

September 1, 2005

Study Completion

December 1, 2005

Last Updated

April 25, 2013

Record last verified: 2013-04

Locations

NL(1)