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

This study is currently recruiting participants.

Verified by Pharming Technologies B.V., January 2009

First Received: December 1, 2005 Last Updated: January 12, 2009 History of Changes

Sponsored by:	Pharming Technologies B.V.
Information provided by:	Pharming Technologies B.V.
ClinicalTrials.gov Identifier:	NCT00262301

Purpose

The purpose of this multi-center study is to demonstrate the efficacy and assess the safety and tolerability of recombinant human C1 inhibitor in the (re)treatment of multiple acute attacks in patients with hereditary angioedema.

Condition	Intervention	Phase
Genetic Disorders	Drug: recombinant human C1 inhibitor	Phase III

Genetics Home Reference related topics: hereditary angioedema

Drug Information available for: C1 esterase inhibitor

U.S. FDA Resources

Study Type:	Interventional
Study Design:	Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study
Official Title:	A Randomized, Placebo-Controlled, Double-Blind Phase III Study of the Efficacy and Safety of Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema

Further study details as provided by Pharming Technologies B.V.:

Primary Outcome Measures:

Time to the beginning of relief of angioedema symptoms based on VAS [ Time Frame: 1 acute HAE attack ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Time to complete resolution of angioedema symptoms based on VAS [ Time Frame: 1 acute HAE attack ] [ Designated as safety issue: No ]

Estimated Enrollment:	30
Study Start Date:	June 2004
Estimated Study Completion Date:	September 2009

Arms	Assigned Interventions
1 As per August 2007, (re)treatments are to take place in an open-label fashion, where approved. No patients are to be treated with placebo from that date.	Drug: recombinant human C1 inhibitor I.V. injection

Eligibility

Ages Eligible for Study:	16 Years and older
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

Clear clinical and laboratory diagnosis of HAE
Baseline plasma level of functional C1INH of less than 50% of normal
Evidence for exacerbation or development of a severe abdominal, facial-oro-pharyngeal, genito-urinary and/or peripheral HAE attack

Exclusion Criteria:

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

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00262301

Contacts

Contact: Sonja Visscher, MSc

hae-trials@pharming.com

Locations

Netherlands
For information on sites in Europe, please contact Pharming Technologies.	Recruiting
Leiden, Netherlands, 2333 CN
Contact: Sonja Visscher, MSc hae-trials@pharming.com

Sponsors and Collaborators

Pharming Technologies B.V.

Investigators

Study Chair:

Jan Nuijens, MD, PhD

Pharming Group N.V.

More Information

No publications provided

Responsible Party:	Pharming Technologies B.V. ( Jan Nuijens )
Study ID Numbers:	C1 1304-01
Study First Received:	December 1, 2005
Last Updated:	January 12, 2009
ClinicalTrials.gov Identifier:	NCT00262301 History of Changes
Health Authority:	Netherlands: Independent Ethics Committee

Study placed in the following topic categories:

Hereditary Angioedema
Hypersensitivity
Genetic Diseases, Inborn
Skin Diseases
Angioedema

Hypersensitivity, Immediate
Vascular Diseases
Urticaria
Angioedema, Hereditary

Additional relevant MeSH terms:

Hypersensitivity
Skin Diseases, Vascular
Genetic Diseases, Inborn
Immune System Diseases
Skin Diseases
Angioedema

Hypersensitivity, Immediate
Vascular Diseases
Urticaria
Cardiovascular Diseases
Angioedema, Hereditary

ClinicalTrials.gov processed this record on May 07, 2009