CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Shire

ClinicalTrials.gov Identifier:

NCT01095510

First received: March 24, 2010

Last updated: July 14, 2015

Last verified: June 2014

History of Changes

Results First Received: June 26, 2014

Study Type:	Interventional
Study Design:	Allocation: Non-Randomized; Intervention Model: Parallel Assignment; Masking: Open Label; Primary Purpose: Treatment
Condition:	Hereditary Angioedema
Intervention:	Biological: CINRYZE

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
Participants were not enrolled in the lower body weight category (10-25 kilograms [kg]) 1000 Units (U) dose group despite substantial recruitment efforts.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
Of 12 participants screened, 9 participants were enrolled and treated. The reason for 3 participants were screen failures as they did not meet the inclusion criteria.

Reporting Groups

	Description
500 U CINRYZE (10-25 kg Body Weight)	Single intravenous (IV) dose of 500 U CINRYZE
1000 U CINRYZE (10-25 kg Body Weight)	Single IV dose of 1000 U CINRYZE
1000 U CINRYZE (>25 kg Body Weight)	Single IV dose of 1000 U CINRYZE
1500 U CINRYZE (>25 kg Body Weight)	Single IV dose of 1500 U CINRYZE

Participant Flow: Overall Study

	500 U CINRYZE (10-25 kg Body Weight)	1000 U CINRYZE (>25 kg Body Weight)	1500 U CINRYZE (>25 kg Body Weight)
STARTED	3	3	3
COMPLETED	3	3	3
NOT COMPLETED	0	0	0

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
500 U CINRYZE (10-25 kg Body Weight)	Single IV dose of 500 U CINRYZE
1000 U CINRYZE (>25 kg Body Weight)	Single IV dose of 1000 U CINRYZE
1500 U CINRYZE (>25 kg Body Weight)	Single IV dose of 1500 U CINRYZE
Total	Total of all reporting groups

Baseline Measures

	500 U CINRYZE (10-25 kg Body Weight)	1000 U CINRYZE (>25 kg Body Weight)	1500 U CINRYZE (>25 kg Body Weight)	Total
Overall Participants Analyzed [Units: Participants]	3	3	3	9

Age [Units: Years] Median (Full Range)	7 (6 to 9)	9 (7 to 9)	10 (8 to 11)	9 (6 to 11)

Gender [Units: Participants]
Female	3	3	2	8
Male	0	0	1	1

Region of Enrollment [Units: Participants]
United States	3	3	3	9

Outcome Measures

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1. Primary:

Presence of Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]

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2. Secondary:

Time to Unequivocal Beginning of Relief of the Defining Attack Symptom [ Time Frame: Within 4 hours following treatment ]

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3. Secondary:

Time to Complete Resolution of the Attack [ Time Frame: Within 1 week following treatment ]

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4. Secondary:

Change in C1 Inhibitor (C1 INH) Antigen and Functional C1 INH Concentrations [ Time Frame: Pre-dose, 2, 4, 8 hours post dose on Day 1; Day 2, 3, 5, 8 ]

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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
Change in C1 INH antigen and functional C1 INH concentrations endpoint was not analyzed as no participants agreed to additional and optional blood sampling. As a result, no PK parameters were calculated for this study.

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 Trial 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 60 days so Sponsor can file for patents or take other action to protect its patent rights.

Results Point of Contact:

Name/Title: Jennifer Schranz, MD
Organization: Shire
phone: 484-595-8940
e-mail: jschranz@shire.com

Responsible Party:	Shire
ClinicalTrials.gov Identifier:	NCT01095510 History of Changes
Other Study ID Numbers:	0624-203 2011-000369-11 ( EudraCT Number )
Study First Received:	March 24, 2010
Results First Received:	June 26, 2014
Last Updated:	July 14, 2015

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