Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema (HAE) Subjects

This study has been completed.

Sponsor:

Information provided by (Responsible Party):

Shire

ClinicalTrials.gov Identifier:

NCT02093923

First received: March 17, 2014

Last updated: June 20, 2016

Last verified: June 2016

History of Changes

Results First Received: April 20, 2016

Study Type:	Interventional
Study Design:	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition:	Hereditary Angioedema
Interventions:	Drug: DX-2930 Drug: Placebo

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
For this study, patients were recruited in United States, Italy, and Jordan between May 2014 and May 2015.

Pre-Assignment Details

Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
A total of 38 subjects were enrolled but one subject was a screen failure after randomization and only 37 subjects were treated and analyzed for safety.

Reporting Groups

	Description
DX-2930, Dose Level 1	30 mg of DX-2930 administered twice, two weeks apart
DX-2930, Dose Level 2	100 mg of DX-2930 administered twice, two weeks apart
DX-2930, Dose Level 3	300 mg of DX-2930 administered twice, two weeks apart
DX-2930, Dose Level 4	400 mg of DX-2930 administered twice, two weeks apart
Placebo	Placebo administered twice, two weeks apart

Participant Flow: Overall Study

	DX-2930, Dose Level 1	DX-2930, Dose Level 2	DX-2930, Dose Level 3	DX-2930, Dose Level 4	Placebo
STARTED	4	4	5	12	13
Total Treated	4	4	5	11	13
Treated - 1 Dose Only:	0	0	0	1	0
Treated - 2 Doses:	4	4	5	10	13
COMPLETED	4	4	5	10	13
NOT COMPLETED	0	0	0	2	0
Lost to Follow-up	0	0	0	1	0
Screen failure after randomization	0	0	0	1	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
DX-2930, Dose Level 1	30 mg of DX-2930 administered twice, two weeks apart
DX-2930, Dose Level 2	100 mg of DX-2930 administered twice, two weeks apart
DX-2930, Dose Level 3	300 mg of DX-2930 administered twice, two weeks apart
DX-2930, Dose Level 4	400 mg of DX-2930 administered twice, two weeks apart
Placebo	Placebo administered twice, two weeks apart
Total	Total of all reporting groups

Baseline Measures

	DX-2930, Dose Level 1	DX-2930, Dose Level 2	DX-2930, Dose Level 3	DX-2930, Dose Level 4	Placebo	Total
Overall Participants Analyzed [Units: Participants]	4	4	5	11	13	37

Age, Customized [Units: Participants]
<18 years	0	0	0	0	0	0
Between 18 and 65 years	4	4	5	10	12	35
>65 years	0	0	0	1	1	2

Gender [Units: Participants]
Female	3	3	1	9	7	23
Male	1	1	4	2	6	14

Outcome Measures

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1. Primary:

Number of Patients With Treatment-Emergent Adverse Events (TEAE) [ Time Frame: through 4 months ]

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2. Primary:

Proportion of Patients With Treatment-Emergent Adverse Events (TEAE) [ Time Frame: through 4 months ]

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3. Primary:

Number of Patients With Serious Adverse Events (SAEs) [ Time Frame: through 4 months ]

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4. Primary:

Proportion of Patients With Serious Adverse Events [ Time Frame: through 4 months ]

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5. Secondary:

Maximum Plasma Concentration (Cmax) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]

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6. Secondary:

Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]

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7. Secondary:

Area Under the Plasma Concentration-time Curve (AUC) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]

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8. Secondary:

Apparent Clearance (CL/F) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]

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9. Secondary:

Apparent Volume of Distribution (Vd/F) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]

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10. Secondary:

Terminal Elimination Half-life (t1/2) [ Time Frame: Pharmacokinetic samples were drawn on Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120. ]

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11. Other Pre-specified:

HAE Attack Rate Per Week [ Time Frame: Baseline, Day 8 to Day 50 ]

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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: 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.

Results Point of Contact:

Name/Title: Carolyn Soo
Organization: Dyax Corp.
phone: +1-617-250-5874
e-mail: csoo@dyax.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Banerji A, Busse P, Shennak M, Lumry W, Davis-Lorton M, Wedner HJ, Jacobs J, Baker J, Bernstein JA, Lockey R, Li HH, Craig T, Cicardi M, Riedl M, Al-Ghazawi A, Soo C, Iarrobino R, Sexton DJ, TenHoor C, Kenniston JA, Faucette R, Still JG, Kushner H, Mensah R, Stevens C, Biedenkapp JC, Chyung Y, Adelman B. Inhibiting Plasma Kallikrein for Hereditary Angioedema Prophylaxis. N Engl J Med. 2017 Feb 23;376(8):717-728. doi: 10.1056/NEJMoa1605767.

Responsible Party:	Shire
ClinicalTrials.gov Identifier:	NCT02093923 History of Changes
Other Study ID Numbers:	DX-2930-02
Study First Received:	March 17, 2014
Results First Received:	April 20, 2016
Last Updated:	June 20, 2016

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