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

NCT ID: NCT01005888

Last Updated: 2021-06-11

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

26 participants

Study Classification

INTERVENTIONAL

Study Start Date

2005-03-14

Study Completion Date

2007-08-22

Brief Summary

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The study objective was to determine the safety and efficacy of C1INH-nf for the prevention of acute HAE attacks.

Detailed Description

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Subjects were given diary cards and instructed to document all HAE attacks on a daily basis. Subjects evaluated their symptoms over the previous 24 hours, noting the severity and duration of swelling at each of 5 locations (abdominal, genitourinary, facial, respiratory \[including laryngeal\], and/or extremity).

The study design also allowed for administration of open-label C1INH-nf (1,000 U of C1INH-nf administered IV \[repeated after 60 minutes, if necessary\] for treatment of laryngeal angioedema or if deemed necessary by the investigator; 1,000 U of C1INH-nf administered IV \[single dose\] prior to emergency surgical procedures).

A total of 26 subjects were enrolled in the study. One subject received open-label C1INH-nf but withdrew prior to randomization. Another subject was randomized but withdrew prior to receiving study drug. Twenty-four (24) subjects were randomized and treated with blinded study drug. In total, 25 subjects received at least 1 dose of study drug and were analyzed for safety; all 25 subjects were exposed to C1INH-nf and 23 subjects were exposed to placebo.

Conditions

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Hereditary Angioedema

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

Primary Study Purpose

PREVENTION

Blinding Strategy

DOUBLE

Participants Investigators

Study Groups

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C1INH-nf First, then Placebo

1,000 Units (U) of C1INH-nf administered intravenously (IV) every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by matching placebo (saline) administered IV every 3 to 4 days for 12 weeks.

Group Type EXPERIMENTAL

C1 esterase inhibitor [human] (C1INH-nf)

Intervention Type BIOLOGICAL

Placebo (saline)

Intervention Type DRUG

Placebo First, then C1INH-nf

Matching placebo (saline) administered IV every 3 to 4 days (approximately twice weekly) for 12 weeks, followed by 1,000 U of C1INH-nf administered IV every 3 to 4 days for 12 weeks.

Group Type EXPERIMENTAL

C1 esterase inhibitor [human] (C1INH-nf)

Intervention Type BIOLOGICAL

Placebo (saline)

Intervention Type DRUG

Interventions

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C1 esterase inhibitor [human] (C1INH-nf)

Intervention Type BIOLOGICAL

Placebo (saline)

Intervention Type DRUG

Eligibility Criteria

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Inclusion Criteria

* Documented HAE
* Normal C1q level
* Relatively frequent angioedema attacks (at least 2 per month on average)

Exclusion Criteria

* Low C1q level
* B-cell malignancy
* Presence of anti-C1INH autoantibody
* History of allergic reaction to C1INH or other blood products
* Narcotic addiction
* Current participation in any other investigational drug study or within the past 30 days
* Participation in a C1 esterase inhibitor trial, or received blood or a blood product in the past 90 days
* Pregnancy or lactation
* Any clinically significant medical condition, such as renal failure, that in the opinion of the investigator would interfere with the subject's ability to participate in the study
Minimum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

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Allergy and Immunology Associates

Scottsdale, Arizona, United States

Site Status

University of California, San Diego

San Diego, California, United States

Site Status

Allergy and Asthma Clinical Research, Inc

Walnut Creek, California, United States

Site Status

Atlanta Allergy and Asthma Clinic

Suwanee, Georgia, United States

Site Status

Hawaii Pacific Health Research Institute

Honolulu, Hawaii, United States

Site Status

Welborn Clinic Allergy and Immunology

Evansville, Indiana, United States

Site Status

Lake Charles Memorial Hospital

Lake Charles, Louisiana, United States

Site Status

Institute for Asthma and Allergy

Wheaton, Maryland, United States

Site Status

Libby Clinic

Libby, Montana, United States

Site Status

Mount Sinai School of Medicine

New York, New York, United States

Site Status

Nationwide Childrens Hospital Clinical Research

Columbus, Ohio, United States

Site Status

Allergy Clinic of Tulsa

Tulsa, Oklahoma, United States

Site Status

Allergy Asthma and Dermatology Research Center

Lake Oswego, Oregon, United States

Site Status

AARA Research Center

Dallas, Texas, United States

Site Status

Tyler County Hospital

Woodville, Texas, United States

Site Status

St. Joseph's Hospital/Cornerstone Healthcare

Parkersburg, West Virginia, United States

Site Status

Countries

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United States

References

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Beard N, Frese M, Smertina E, Mere P, Katelaris C, Mills K. Interventions for the long-term prevention of hereditary angioedema attacks. Cochrane Database Syst Rev. 2022 Nov 3;11(11):CD013403. doi: 10.1002/14651858.CD013403.pub2.

Reference Type DERIVED
PMID: 36326435 (View on PubMed)

Lumry WR, Miller DP, Newcomer S, Fitts D, Dayno J. Quality of life in patients with hereditary angioedema receiving therapy for routine prevention of attacks. Allergy Asthma Proc. 2014 Sep-Oct;35(5):371-6. doi: 10.2500/aap.2014.35.3783.

Reference Type DERIVED
PMID: 25295804 (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)

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

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LEVP2005-1/Part B

Identifier Type: -

Identifier Source: org_study_id

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