NCT00438815

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.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
113

participants targeted

Target at P25-P50 for phase_3

Timeline
Completed

Started Sep 2006

Typical duration for phase_3

Geographic Reach
1 country

30 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

Study Start

First participant enrolled

September 21, 2006

Completed
5 months until next milestone

First Submitted

Initial submission to the registry

February 21, 2007

Completed
1 day until next milestone

First Posted

Study publicly available on registry

February 22, 2007

Completed
2.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

March 31, 2009

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

March 31, 2009

Completed
1.2 years until next milestone

Results Posted

Study results publicly available

June 9, 2010

Completed
Last Updated

June 8, 2021

Status Verified

May 1, 2021

Enrollment Period

2.5 years

First QC Date

February 21, 2007

Results QC Date

March 31, 2010

Last Update Submit

May 19, 2021

Conditions

Keywords

Hereditary angioedemaC1 esterase inhibitor (human)

Outcome Measures

Primary Outcomes (2)

  • Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf

    Duration of the study (2.5 years)

  • Percent of HAE Attacks With Substantial Relief of the Defining Symptom

    Subjects were to assess their symptoms every 15 minutes up to 4 hours after the initial dose or until substantial relief of the defining symptom was achieved. The conservative analysis defined substantial relief as 3 consecutive assessments of improvement of the defining symptom; any attack that did not have 3 consecutive documented reports of improvement was considered a treatment failure. In the less conservative analysis, attacks also were considered to have responded if clinical improvement of the defining symptom occurred but data were incomplete due to cessation of symptom assessments.

    Within 4 hours after initial treatment

Secondary Outcomes (5)

  • Time to Beginning of Substantial Relief of the Defining Symptom

    Within 4 hours after initial treatment

  • Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments

    Within 4 hours after initial treatment

  • Antigenic C1 Inhibitor (C1INH) Serum Levels

    Pre-infusion to 1 hour post-infusion

  • Functional C1INH Serum Levels

    Pre-infusion to 1 hour post-infusion

  • Complement C4 Serum Levels

    Pre-infusion to 1 hour post-infusion

Study Arms (1)

Open-label C1INH-nf

EXPERIMENTAL

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.

Biological: C1 esterase inhibitor [human] (C1INH-nf)

Interventions

Open-label C1INH-nf

Eligibility Criteria

Age1 Year+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (30)

Allergy and Immunology Associates

Scottsdale, Arizona, 85251, United States

Location

Allergy and Asthma Clinic of Northwest Arkansas

Bentonville, Arkansas, 72712, United States

Location

UCLA-David Geffen School of Medicine

Los Angeles, California, 90095, United States

Location

University of California, San Diego

San Diego, California, 92093-0732, United States

Location

Allergy and Asthma Clinical Research, Inc

Walnut Creek, California, 94598, United States

Location

Allergy and Asthma Center

Fort Lauderdale, Florida, 33334, United States

Location

Orlando Regional Healthcare

Orlando, Florida, 32806, United States

Location

Family Allergy and Asthma Center

Atlanta, Georgia, 30342, United States

Location

Welborn Clinic Allergy and Immunology

Evansville, Indiana, 47713, United States

Location

Institute for Asthma and Allergy

Wheaton, Maryland, 20902, United States

Location

University of Massachusetts Medical School

Worcester, Massachusetts, 01655, United States

Location

Grand Traverse Allergy

Traverse City, Michigan, 49684, United States

Location

MeritCare Clinical Research

Bemidji, Minnesota, 56601, United States

Location

Nevada Access to Research and Education Society

Las Vegas, Nevada, 89102, United States

Location

UMDNJ Asthma and Allergy Research Center

Newark, New Jersey, 07103, United States

Location

Winthrop University Hospital

Mineola, New York, 11501, United States

Location

Mount Sinai School of Medicine

New York, New York, 10029, United States

Location

Montefiore Medical Center

The Bronx, New York, 10461, United States

Location

MeritCare Clinical Research

Fargo, North Dakota, 58122, United States

Location

Allergy & Asthma Centre of Dayton

Centerville, Ohio, 45458, United States

Location

Allergy Clinic of Tulsa

Tulsa, Oklahoma, 74133, United States

Location

Allergy Asthma and Dermatology Research Center

Lake Oswego, Oregon, 97035, United States

Location

Penn State University

Hershey, Pennsylvania, 17033, United States

Location

Allergy Partners of the Upstate

Greenville, South Carolina, 29615, United States

Location

AARA Research Center

Dallas, Texas, 75231, United States

Location

University of Texas Medical Branch

Galveston, Texas, 77555-1083, United States

Location

Baylor College of Medicine

Houston, Texas, 77030, United States

Location

Allergy and Asthma Research Center

San Antonio, Texas, 78229, United States

Location

Marycliff Allergy Specialists

Spokane, Washington, 99204, United States

Location

Cornerstone Healthcare

Parkersburg, West Virginia, 26101, United States

Location

Related Publications (5)

  • 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.

  • 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.

  • 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.

  • 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.

  • 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.

MeSH Terms

Conditions

Angioedemas, Hereditary

Interventions

Complement C1 Inhibitor Protein

Condition Hierarchy (Ancestors)

AngioedemaVascular DiseasesCardiovascular DiseasesHereditary Complement Deficiency DiseasesPrimary Immunodeficiency DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesUrticariaSkin 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
Study Director
Organization
Shire

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

February 21, 2007

First Posted

February 22, 2007

Study Start

September 21, 2006

Primary Completion

March 31, 2009

Study Completion

March 31, 2009

Last Updated

June 8, 2021

Results First Posted

June 9, 2010

Record last verified: 2021-05

Locations