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

This study has been completed.

Sponsor:

Information provided by:

Shire

ClinicalTrials.gov Identifier:

NCT00289211

First received: February 7, 2006

Last updated: March 19, 2014

Last verified: March 2014

History of Changes

Results First Received: March 17, 2010

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Safety/Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Investigator); Primary Purpose: Treatment
Condition:	Hereditary Angioedema
Interventions:	Biological: C1 esterase inhibitor [human] (C1INH-nf) Drug: Placebo (saline)

Participant Flow

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Recruitment Details

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Pre-Assignment Details

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Reporting Groups

	Description
C1INH-nf	1,000 Units (U) of C1 esterase inhibitor (C1INH-nf) administered intravenously (IV). If there was no response to treatment 60 minutes after the first dose, a second 1,000 U dose could be administered.
Placebo	Matching placebo (saline) administered IV. If there was no response to treatment 60 minutes after the first dose, a second placebo (saline) dose could be administered.
Open-label C1INH-nf Only	Twelve subjects were never randomized but received open-label C1INH-nf for treatment of laryngeal angioedema and/or prior to emergency surgical procedures. These subjects were analyzed for safety only.

Participant Flow: Overall Study

	C1INH-nf	Placebo	Open-label C1INH-nf Only
STARTED	36	35	12
COMPLETED	36	34	2
NOT COMPLETED	0	1	10

Baseline Characteristics

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Population Description

Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
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Reporting Groups

	Description
C1INH-nf	1,000 U of C1INH-nf administered IV. If there was no response to treatment 60 minutes after the first dose, a second 1,000 U dose could be administered.
Placebo	Matching placebo (saline) administered IV. If there was no response to treatment 60 minutes after the first dose, a second placebo (saline) dose could be administered.
Open-label C1INH-nf Only	Twelve subjects were never randomized but received open-label C1INH-nf for treatment of laryngeal angioedema and/or prior to emergency surgical procedures. These subjects were analyzed for safety only.
Total	Total of all reporting groups

Baseline Measures

	C1INH-nf	Placebo	Open-label C1INH-nf Only	Total
Number of Participants [units: participants]	36	35	12	83
Age [units: years] Mean ± Standard Deviation	36.8 ± 17.68	37.0 ± 13.76	36.3 ± 19.42	36.8 ± 16.20
Gender [units: participants]
Female	27	28	6	61
Male	9	7	6	22

Outcome Measures

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1. Primary:

Time to Beginning of Substantial Relief of the Defining Symptom [ Time Frame: Within 4 hours after initial treatment ]

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2. Secondary:

Number of Subjects With Beginning of Substantial Relief of the Defining Symptom [ Time Frame: Within 4 hours after initial treatment ]

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3. Secondary:

Time to Complete Resolution of the HAE Attack [ Time Frame: 72 hours ]

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4. Secondary:

Antigenic C1 Inhibitor (C1INH) Serum Levels [ Time Frame: Pre-infusion to 1-, 2-, 4-, and 12 hours post-infusion ]

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5. Secondary:

Functional C1INH Serum Levels [ Time Frame: Pre-infusion to 1-, 2-, 4-, and 12 hours post-infusion ]

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6. Secondary:

Complement C4 Serum Levels [ Time Frame: Pre-infusion to 1-, 2-, 4-, and 12 hours post-infusion ]

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Serious Adverse Events

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Other Adverse Events

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Limitations and Caveats

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More Information

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Certain Agreements:

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

The agreement is:

	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
	Other disclosure agreement that restricts the right of the PI to discuss or publish trial results after the trial is completed. Restriction Description: Clinical Study Agreement. Most restrictive provision - PI will not publish results until after first of: multicenter publication is published or 24 months from study end. Thereafter, PI may publish his results. PI must provide copy of proposed publication to sponsor for pre-review. If sponsor requests, PI must delete sponsor confidential information before publication and/or delay publication for 90 days so sponsor can file for patents or take other action to protect its patent rights.

Results Point of Contact:

Name/Title: Chief Scientific Officer
Organization: ViroPharma
phone: 610-458-7300

No publications provided by Shire

Publications automatically indexed to this study:

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.

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.

Responsible Party:	Chief Scientific Officer, ViroPharma
ClinicalTrials.gov Identifier:	NCT00289211 History of Changes
Other Study ID Numbers:	LEVP2005-1/Part A
Study First Received:	February 7, 2006
Results First Received:	March 17, 2010
Last Updated:	March 19, 2014
Health Authority:	United States: Food and Drug Administration

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