Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE)

This study has been completed.

Study NCT00262080 Information provided by Dyax Corp.

First Received on December 5, 2005. Last Updated on April 9, 2010 History of Changes

Results First Received: December 30, 2009

Study Type:	Interventional
Study Design:	Allocation: Randomized; Endpoint Classification: Efficacy Study; Intervention Model: Parallel Assignment; Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Hereditary Angioedema (HAE)
Interventions:	Drug: ecallantide Drug: Phosphate Buffer Saline (PBS),

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
Patients were screened in advance of presenting with an HAE attack but were randomized only upon attack.

Reporting Groups

	Description
Ecallantide / Ecallantide	Patients treated with ecallantide in the double-blind part and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Placebo / Ecallantide	Patients treated with placebo in the double-blind part and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Ecallantide (Repeat Dose Only)	Patients not treated in the double-blind part but treated with ecallantide in the repeat-dosing part.

Participant Flow for 2 periods

Period 1: Double Blind Treatment Period

	Ecallantide / Ecallantide	Placebo / Ecallantide
STARTED	36 ^[1]	36 ^[2]
COMPLETED	35	36
NOT COMPLETED	1	0
Lost to Follow-up	1	0

[1]	One patient was randomized to receive ecallantide but was treated with placebo.
[2]	One patient was randomized to receive placebo but was treated with ecallantide.

Period 2: Repeat Dose Treatment Period

	Ecallantide / Ecallantide	Placebo / Ecallantide	Ecallantide (Repeat Dose Only)
STARTED	22 ^[1]	26 ^[1]	19 ^[2]
COMPLETED	18 ^[3]	25	14
NOT COMPLETED	4	1	5
Adverse Event	1	0	0
Withdrawal by Subject	0	1	1
Lost to Follow-up	2	0	2
Decision to discontinue the study	0	0	2
enrolled in EDEMA4	1	0	0

[1]	Not all patients treated in the double-blind part came for treatment in the repeat-dose part.
[2]	This arm represents new patients, not treated in the double-blind part of the study.
[3]	Some patients participated in only the double-blind or the open label phase.

Baseline Characteristics

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Reporting Groups

	Description
Ecallantide / Ecallantide	Patients treated with ecallantide in the double-blind part (ITT as treated) and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Placebo / Ecallantide	Patients treated with placebo in the double-blind part (ITT as treated) and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.
Ecallantide (Repeat-Dosing Part Only)	Patients not treated in the double-blind part but treated with ecallantide in the repeat-dosing part. One patient was omitted from analysis in the intent-to-treat and per-protocol populations due to the loss of the data for the 4-hour post-dose assessments during treatment episode 1.

Description

Ecallantide / Ecallantide

Patients treated with ecallantide in the double-blind part (ITT as treated) and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.

Placebo / Ecallantide

Patients treated with placebo in the double-blind part (ITT as treated) and eligible for treatment with ecallantide in the repeat-dosing part of the study once their Follow-up Visit 1 (Day 7) in the double-blind part was completed.

Ecallantide (Repeat-Dosing Part Only)

Patients not treated in the double-blind part but treated with ecallantide in the repeat-dosing part.

One patient was omitted from analysis in the intent-to-treat and per-protocol populations due to the loss of the data for the 4-hour post-dose assessments during treatment episode 1.

Baseline Measures

	Ecallantide / Ecallantide	Placebo / Ecallantide	Ecallantide (Repeat-Dosing Part Only)	Total
Number of Participants [units: participants]	36	36	18	90
Age [units: years] Mean ± Standard Deviation	37.1 ± 14.3	33.6 ± 14.6	35.0 ± 14.6	34.7 ± 14.7
Gender [units: participants]
Female	23	24	11	58
Male	13	12	7	32

Outcome Measures

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1. Primary:

Treatment Outcome Score at 4 Hours Post-Dose [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ]

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2. Secondary:

Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose [ Time Frame: baseline, 4 hours post-dose (DOUBLE-BLIND PART) ]

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3. Secondary:

Time to Significant Improvement in Overall Response [ Time Frame: 4 hours post-dose (DOUBLE-BLIND PART) ]

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4. Other Pre-specified:

Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) [ Time Frame: Baseline ]

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5. Other Pre-specified:

Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes [ Time Frame: 4 hours post-dose (REPEAT-DOSING PART) ]

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6. Other Pre-specified:

Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes [ Time Frame: baseline, 4 hours post-dose (REPEAT-DOSING PART) ]

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7. Other Pre-specified:

Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes [ Time Frame: 4 hours post-dose (REPEAT-DOSING PART) ]

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Serious Adverse Events

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Other Adverse Events

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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: Dyax agreements vary, but Dyax will not prohibit any investigator from publishing. Dyax reviews publications prior to public release and can request deferral by up to 60 days beyond the proposed publication date if necessary to preserve its intellectual property. Dyax can request changes to publications to remove non-study-related confidential information. Dyax can also request deferral of single-center publications until after disclosure of the clinical trial’s primary publication.

Limitations and Caveats

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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Results Point of Contact:

Name/Title: Pat T Horn
Organization: Dyax Corp.
phone: 617 250 5948
e-mail: phorn@dyax.com

No publications provided by Dyax Corp.

Publications automatically indexed to this study:

Riedl M, Campion M, Horn PT, Pullman WE. Response time for ecallantide treatment of acute hereditary angioedema attacks. Ann Allergy Asthma Immunol. 2010 Dec;105(6):430-436.e2. Epub 2010 Oct 25.

Cicardi M, Levy RJ, McNeil DL, Li HH, Sheffer AL, Campion M, Horn PT, Pullman WE. Ecallantide for the treatment of acute attacks in hereditary angioedema. N Engl J Med. 2010 Aug 5;363(6):523-31.

Responsible Party:	Bill Pullman, MD, PhD, Executive Vice President, Chief Development Officer, Dyax Corp.
ClinicalTrials.gov Identifier:	NCT00262080 History of Changes
Other Study ID Numbers:	EDEMA3 (DX-88/14)
Study First Received:	December 5, 2005
Results First Received:	December 30, 2009
Last Updated:	April 9, 2010
Health Authority:	United States: Food and Drug Administration