Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Treatment of Acute Hereditary Angioedema (HAE) Attacks (CHANGE 2)

This study has been completed.

Sponsor:

Information provided by:

Shire

ClinicalTrials.gov Identifier:

NCT00438815

First received: February 21, 2007

Last updated: March 19, 2014

Last verified: March 2014

History of Changes

Results First Received: March 31, 2010

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study; Intervention Model: Single Group Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Hereditary Angioedema
Intervention:	Biological: C1 esterase inhibitor [human] (C1INH-nf)

Participant Flow

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Recruitment Details

Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
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Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
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Reporting Groups

	Description
Open-label 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.

Participant Flow: Overall Study

	Open-label C1INH-nf
STARTED	113
COMPLETED	43
NOT COMPLETED	70
Transferred to LEVP2006-4 (NCT00462709)	30
3-month follow-up no longer required	12
Noncompliance with protocol requirements	9
Transitioned to commercial C1INH-nf	6
Lost to Follow-up	6
Withdrawal by Subject	6
Death	1

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
Open-label 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.

Baseline Measures

	Open-label C1INH-nf
Number of Participants [units: participants]	113
Age [units: years] Mean ± Standard Deviation	34.5 ± 17.6
Gender [units: participants]
Female	75
Male	38

Outcome Measures

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1. Primary:

Number of Hereditary Angioedema (HAE) Attacks Treated With C1INH-nf [ Time Frame: Duration of the study (2.5 years) ]

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2. Primary:

Percent of HAE Attacks With Substantial Relief of the Defining Symptom [ Time Frame: Within 4 hours after initial treatment ]

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3. Secondary:

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

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4. Secondary:

Time to Beginning of Substantial Relief of the Defining Symptom for Subjects Who Received Multiple Treatments [ Time Frame: Within 4 hours after initial treatment ]

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5. Secondary:

Antigenic C1 Inhibitor (C1INH) Serum Levels [ Time Frame: Pre-infusion to 1 hour post-infusion ]

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6. Secondary:

Functional C1INH Serum Levels [ Time Frame: Pre-infusion to 1 hour post-infusion ]

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7. Secondary:

Complement C4 Serum Levels [ Time Frame: Pre-infusion to 1 hour 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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Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
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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:

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.

Responsible Party:	Chief Scientific Officer, ViroPharma
ClinicalTrials.gov Identifier:	NCT00438815 History of Changes
Other Study ID Numbers:	LEVP2006-1
Study First Received:	February 21, 2007
Results First Received:	March 31, 2010
Last Updated:	March 19, 2014
Health Authority:	United States: Food and Drug Administration

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