Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks

NCT ID: NCT00438815

Last Updated: 2021-06-08

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 113 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-09-21
• Study Completion Date: 2009-03-31

Brief Summary

The study objective was to evaluate the safety and efficacy of repeat use of C1INH-nf for the treatment of acute HAE attacks.

Detailed Description

A total of 113 subjects were enrolled in the study. One-hundred-one (101) subjects received C1INH-nf for the treatment of 1 or more HAE attacks and were analyzed for efficacy. The study design also allowed for short-term prophylaxis with C1INH-nf prior to emergency or non-cosmetic surgical or dental procedures, and an additional 12 subjects received C1INH-nf only for this purpose. All 113 subjects were exposed to C1INH-nf and analyzed for safety.

Conditions

Hereditary Angioedema

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Open-label C1INH-nf

1,000 Units (U) of C1INH-nf administered intravenously. If there was no response to treatment 60 minutes after the first dose, a second 1,000 U dose could be administered.

Group Type: EXPERIMENTAL

C1 esterase inhibitor [human] (C1INH-nf)

Intervention Type: BIOLOGICAL

Interventions

C1 esterase inhibitor [human] (C1INH-nf)

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

This study was open to all subjects who:

• Completed participation in LEVP2005-1/A (NCT00289211) and were not participating in LEVP2005-1/B (NCT01005888), any time after the 3-day telephone follow-up
• Completed participation in LEVP2005-1/B any time after the final prophylactic therapy in Part B
• Were enrolled but not randomized in LEVP2005-1/A after Part A was closed
• Were excluded from LEVP2005-1 for any of the following reasons:

• Pregnancy or lactation
• Age less than 6 years
• Narcotic addiction
• Presence of anti-C1INH autoantibodies
• Were not enrolled in LEVP2005-1 after enrollment in LEVP2005-1 was closed, under the following circumstances:

• Had a diagnosis of HAE: evidence of a low C4 level plus either a low C1INH antigenic level or a low C1INH functional level, or
• Had a known HAE-causing C1INH mutation, or
• Had a diagnosis of HAE based on a strong family history of HAE as determined by the principal investigator

Exclusion Criteria

• History of allergic reaction to C1INH or other blood products
• Participated in any other investigational drug study within the past 30 days
• Received blood or a blood product in the past 60 days other than C1INH-nf

• Minimum Eligible Age: 1 Year
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Allergy and Immunology Associates

Scottsdale, Arizona, United States

Allergy and Asthma Clinic of Northwest Arkansas

Bentonville, Arkansas, United States

UCLA-David Geffen School of Medicine

Los Angeles, California, United States

University of California, San Diego

San Diego, California, United States

Allergy and Asthma Clinical Research, Inc

Walnut Creek, California, United States

Allergy and Asthma Center

Fort Lauderdale, Florida, United States

Orlando Regional Healthcare

Orlando, Florida, United States

Family Allergy and Asthma Center

Atlanta, Georgia, United States

Welborn Clinic Allergy and Immunology

Evansville, Indiana, United States

Institute for Asthma and Allergy

Wheaton, Maryland, United States

University of Massachusetts Medical School

Worcester, Massachusetts, United States

Grand Traverse Allergy

Traverse City, Michigan, United States

MeritCare Clinical Research

Bemidji, Minnesota, United States

Nevada Access to Research and Education Society

Las Vegas, Nevada, United States

UMDNJ Asthma and Allergy Research Center

Newark, New Jersey, United States

Winthrop University Hospital

Mineola, New York, United States

Mount Sinai School of Medicine

New York, New York, United States

Montefiore Medical Center

The Bronx, New York, United States

MeritCare Clinical Research

Fargo, North Dakota, United States

Allergy & Asthma Centre of Dayton

Centerville, Ohio, United States

Allergy Clinic of Tulsa

Tulsa, Oklahoma, United States

Allergy Asthma and Dermatology Research Center

Lake Oswego, Oregon, United States

Penn State University

Hershey, Pennsylvania, United States

Allergy Partners of the Upstate

Greenville, South Carolina, United States

AARA Research Center

Dallas, Texas, United States

University of Texas Medical Branch

Galveston, Texas, United States

Baylor College of Medicine

Houston, Texas, United States

Allergy and Asthma Research Center

San Antonio, Texas, United States

Marycliff Allergy Specialists

Spokane, Washington, United States

Cornerstone Healthcare

Parkersburg, West Virginia, United States

Countries

United States

References

Baker JW, Craig TJ, Riedl MA, Banerji A, Fitts D, Kalfus IN, Uknis ME. Nanofiltered C1 esterase inhibitor (human) for hereditary angioedema attacks in pregnant women. Allergy Asthma Proc. 2013 Mar-Apr;34(2):162-9. doi: 10.2500/aap.2013.34.3645.

Reference Type: DERIVED

PMID: 23484892 (View on PubMed)

Lumry W, Manning ME, Hurewitz DS, Davis-Lorton M, Fitts D, Kalfus IN, Uknis ME. Nanofiltered C1-esterase inhibitor for the acute management and prevention of hereditary angioedema attacks due to C1-inhibitor deficiency in children. J Pediatr. 2013 May;162(5):1017-22.e1-2. doi: 10.1016/j.jpeds.2012.11.030. Epub 2013 Jan 11.

Reference Type: DERIVED

PMID: 23312695 (View on PubMed)

Grant JA, White MV, Li HH, Fitts D, Kalfus IN, Uknis ME, Lumry WR. Preprocedural administration of nanofiltered C1 esterase inhibitor to prevent hereditary angioedema attacks. Allergy Asthma Proc. 2012 Jul-Aug;33(4):348-53. doi: 10.2500/aap.2012.33.3585.

Reference Type: DERIVED

PMID: 22856635 (View on PubMed)

Riedl MA, Hurewitz DS, Levy R, Busse PJ, Fitts D, Kalfus I. Nanofiltered C1 esterase inhibitor (human) for the treatment of acute attacks of hereditary angioedema: an open-label trial. Ann Allergy Asthma Immunol. 2012 Jan;108(1):49-53. doi: 10.1016/j.anai.2011.10.017. Epub 2011 Nov 21.

Reference Type: DERIVED

PMID: 22192966 (View on PubMed)

Zuraw BL, Busse PJ, White M, Jacobs J, Lumry W, Baker J, Craig T, Grant JA, Hurewitz D, Bielory L, Cartwright WE, Koleilat M, Ryan W, Schaefer O, Manning M, Patel P, Bernstein JA, Friedman RA, Wilkinson R, Tanner D, Kohler G, Gunther G, Levy R, McClellan J, Redhead J, Guss D, Heyman E, Blumenstein BA, Kalfus I, Frank MM. Nanofiltered C1 inhibitor concentrate for treatment of hereditary angioedema. N Engl J Med. 2010 Aug 5;363(6):513-22. doi: 10.1056/NEJMoa0805538.

Reference Type: DERIVED

PMID: 20818886 (View on PubMed)

Other Identifiers

LEVP2006-1

Identifier Type: -

Identifier Source: org_study_id