Trial Outcomes & Findings for Efficacy and Safety Study of DX-88 to Treat Acute Attacks of Hereditary Angioedema (HAE) (NCT NCT00262080)

NCT ID: NCT00262080

Last Updated: 2021-06-11

Results Overview

Treatment Outcome Score (TOS) is a validated, comprehensive measure of symptom response to treatment. At 4 hours , patient assessment of response characterized by their change from baseline in symptom severity and collected by anatomic site of attack involvement, was recorded on a categorical scale (significant improvement \[100\] to significant worsening \[-100\]). The response at each anatomic site was weighted by baseline severity and then the weighted scores across all involved sites were averaged to calculate the TOS. Clinically meaningful improvement was indicated by a TOS of 30 or higher.

Recruitment status

COMPLETED

Study phase

PHASE3

Target enrollment

91 participants

Primary outcome timeframe

4 hours post-dose (DOUBLE-BLIND PART)

Results posted on

2021-06-11

Participant Flow

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

Participant milestones

Participant milestones
Measure	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.
Double Blind Treatment Period STARTED	36	36	0
Double Blind Treatment Period COMPLETED	35	36	0
Double Blind Treatment Period NOT COMPLETED	1	0	0
Repeat Dose Treatment Period STARTED	22	26	19
Repeat Dose Treatment Period COMPLETED	18	25	14
Repeat Dose Treatment Period NOT COMPLETED	4	1	5

Reasons for withdrawal

Reasons for withdrawal
Measure	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.
Double Blind Treatment Period Lost to Follow-up	1	0	0
Repeat Dose Treatment Period Adverse Event	1	0	0
Repeat Dose Treatment Period Withdrawal by Subject	0	1	1
Repeat Dose Treatment Period Lost to Follow-up	2	0	2
Repeat Dose Treatment Period Decision to discontinue the study	0	0	2
Repeat Dose Treatment Period enrolled in EDEMA4	1	0	0

Baseline Characteristics

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

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Ecallantide / Ecallantide n=36 Participants 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 n=36 Participants 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) n=18 Participants 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.	Total n=90 Participants Total of all reporting groups
Age, Continuous	37.1 years STANDARD_DEVIATION 14.3 • n=5 Participants	33.6 years STANDARD_DEVIATION 14.6 • n=7 Participants	35.0 years STANDARD_DEVIATION 14.6 • n=5 Participants	34.7 years STANDARD_DEVIATION 14.7 • n=4 Participants
Sex: Female, Male Female	23 Participants n=5 Participants	24 Participants n=7 Participants	11 Participants n=5 Participants	58 Participants n=4 Participants
Sex: Female, Male Male	13 Participants n=5 Participants	12 Participants n=7 Participants	7 Participants n=5 Participants	32 Participants n=4 Participants

PRIMARY outcome

Timeframe: 4 hours post-dose (DOUBLE-BLIND PART)

Population: ITT as treated: two patients randomized on the same day at the same study center were administered treatment opposite to their randomized treatment assignment. Data were analyzed based on actual treatment received. Imputation was used to account for emerging symptoms and medical intervention. Best possible score = 100; worst possible score = -100.

Outcome measures

Outcome measures
Measure	Ecallantide n=36 Participants Patients treated with ecallantide in the double-blind part.	Placebo n=36 Participants Patients treated with placebo in the double-blind part.
Treatment Outcome Score at 4 Hours Post-Dose	49.5 units on a scale Standard Deviation 59.43 • Interval 50.0 to 100.0	18.5 units on a scale Standard Deviation 67.78 • Interval 75.0 to 100.0

SECONDARY outcome

Timeframe: baseline, 4 hours post-dose (DOUBLE-BLIND PART)

Population: ITT as treated: two patients randomized on the same day at the same center were administered treatment opposite to their randomized treatment assignments. Data were analyzed based on their actual treatment received. Imputation was used to account for emerging symptoms and medical intervention. Best possible score = 0.0; worst possible score = 3.0.

Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement (minimally important difference) was indicated by a reduction in the score of 0.30 or more.

Outcome measures

Outcome measures
Measure	Ecallantide n=36 Participants Patients treated with ecallantide in the double-blind part.	Placebo n=36 Participants Patients treated with placebo in the double-blind part.
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose MSCS Score at baseline	2.17 units on a scale Standard Deviation 0.51	2.24 units on a scale Standard Deviation 0.55
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose MSCS Score at 4 hours post-dose	1.26 units on a scale Standard Deviation 0.96	1.75 units on a scale Standard Deviation 0.90
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Change from baseline in MSCS at 4 hours post-dose	-0.91 units on a scale Standard Deviation 1.10	-0.48 units on a scale Standard Deviation 0.68

SECONDARY outcome

Timeframe: 4 hours post-dose (DOUBLE-BLIND PART)

Population: ITT as treated. Patients not reporting significant improvement before 4 hours were censored at 4 hours. Patients receiving additional HAE therapy within 4 hours were censored at the time of the medical intervention. The time to significant improvement is not provided in this display as the median time for placebo was not reached by 4 hours.

The overall response assessment is a patient-reported assessment of global response to therapy. Patients are asked to perform an overall response assessment at regular intervals, relative to baseline. Patients were asked "overall how are you feeling" compared to how they felt before study drug. Answer options were "a lot worse", "a little worse", "same", "a little better" or "a lot better or resolved". Significant improvement was the first time that the patient responded to the assessment as "a little better or resolved".

Outcome measures

Outcome measures
Measure	Ecallantide n=36 Participants Patients treated with ecallantide in the double-blind part.	Placebo n=36 Participants Patients treated with placebo in the double-blind part.
Time to Significant Improvement in Overall Response Patients with Censored Data	17 participant	25 participant
Time to Significant Improvement in Overall Response Patients with Significant Improvement	19 participant	11 participant

OTHER_PRE_SPECIFIED outcome

Timeframe: Baseline

Patient-reported severity of symptom complexes at baseline, by symptom complex and treatment group. Patients were to have at least one symptom complex that was moderate or severe. Patients could present with multiple symptom complexes, some of which could be mild. Mild=noticeable but do not impact daily living activities; Moderate=treatment or intervention is highly desirable and activities of daily living are impacted; Severe=require treatment or intervention due to inability to perform activities of daily living. The results are for number of patients with symptom complexes including mild, moderate and severe, provided the patients have at least one symptom complex that was moderate or severe

Outcome measures

Outcome measures
Measure	Ecallantide n=36 Participants Patients treated with ecallantide in the double-blind part.	Placebo n=36 Participants Patients treated with placebo in the double-blind part.
Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) Genital/Buttocks	2 participants	4 participants
Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) Internal Head/Neck Symptoms	9 participants	4 participants
Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) Stomach/GI	19 participants	22 participants
Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) External Head/Neck	5 participants	8 participants
Number of Patients With Symptom Complexes of Treated Attack at Baseline(DOUBLE-BLIND PART) Cutaneous	21 participants	14 participants

OTHER_PRE_SPECIFIED outcome

Timeframe: 4 hours post-dose (REPEAT-DOSING PART)

Population: Treatment episode 1 contains data only from those participants who were new patients in the repeat-dosing part. Treatment episode 2 and beyond contain data pooled from patients treated in the double-blind part (ecallantide or placebo) and the repeat-dosing part. Data imputation was used to account for emerging symptoms and medical intervention.

Outcome measures

Outcome measures
Measure	Ecallantide n=51 Participants Patients treated with ecallantide in the double-blind part.	Placebo Patients treated with placebo in the double-blind part.
Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes Episode 1 (n =18)	71.3 units on a scale Standard Deviation 28.85	—
Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes Episode 2 (n = 51)	73.3 units on a scale Standard Deviation 44.9	—
Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes Episode 3 (n = 30)	81.9 units on a scale Standard Deviation 28.52	—
Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes Episode 4 (n = 21)	81.2 units on a scale Standard Deviation 24.53	—
Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes Episode 5 (n = 11)	48.5 units on a scale Standard Deviation 68.5	—
Treatment Outcome Score at 4 Hours Post-Dose Over Multiple Treatment Episodes Episode 6 (n = 9)	60.4 units on a scale Standard Deviation 49.26	—

OTHER_PRE_SPECIFIED outcome

Timeframe: baseline, 4 hours post-dose (REPEAT-DOSING PART)

The Mean Symptom Complex Severity (MSCS) score is a validated, comprehensive point-in-time measure of symptom severity. At baseline and 4 hours, patients rated the severity on a categorical scale (0 = normal, 1 = mild, 2 = moderate, 3 = severe) for symptoms at each affected anatomical location. Ratings were averaged to obtain the MSCS score. A decrease in MSCS score reflected an improvement in symptoms; clinically meaningful improvement was indicated by a reduction in the score of 0.30 or more.

Outcome measures

Outcome measures
Measure	Ecallantide n=51 Participants Patients treated with ecallantide in the double-blind part.	Placebo Patients treated with placebo in the double-blind part.
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes Episode 1 (n = 17)	-1.16 units on a scale Standard Deviation 0.87	—
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes Episode 2 (n = 51)	-1.12 units on a scale Standard Deviation 0.90	—
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes Episode 3 (n = 30)	-1.31 units on a scale Standard Deviation 0.87	—
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes Episode 4 (n = 21)	-1.38 units on a scale Standard Deviation 0.79	—
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes Episode 5 (n = 11)	-0.89 units on a scale Standard Deviation 0.72	—
Change From Baseline in Mean Symptom Complex Severity (MSCS) Score at 4 Hours Post-dose Over Multiple Treatment Episodes Episode 6 (n = 9)	-0.87 units on a scale Standard Deviation 0.75	—

OTHER_PRE_SPECIFIED outcome

Timeframe: 4 hours post-dose (REPEAT-DOSING PART)

Population: Patients not reporting significant improvement before 4 hours were censored at 4 hours. Patients receiving additional HAE therapy within 4 hours were censored at the time of the medical intervention. The time to significant improvement is not provided in this display as the interquartile range (IQR) was not reached by 4 hours for most episodes.

The overall response assessment is a patient-reported assessment of global response to therapy. Patients are asked to perform an overall response assessment at regular intervals, relative to baseline (ie,immediately before treatment) using the following 5-category scale from significant improvement (Score = 100)to significant worsening (Score = -100)

Outcome measures

Outcome measures
Measure	Ecallantide n=51 Participants Patients treated with ecallantide in the double-blind part.	Placebo Patients treated with placebo in the double-blind part.
Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes Episode 1 (n=18)	10 participants	—
Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes Episode 2 (n=51)	37 participants	—
Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes Episode 3 (n=30)	24 participants	—
Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes Episode 4 (n=21)	14 participants	—
Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes Episode 5 (n=11)	6 participants	—
Time to Significant Improvement in Overall Response Over Multiple Treatment Episodes Episode 6 (n=9)	5 participants	—

Adverse Events

Double Blind - Ecallantide

Serious events: 3 serious events

Other events: 13 other events

Deaths: 0 deaths

Double-Blind Placebo

Serious events: 2 serious events

Other events: 7 other events

Deaths: 0 deaths

Repeat-Dosing Ecallantide

Serious events: 7 serious events

Other events: 22 other events

Deaths: 0 deaths

Serious adverse events

Other adverse events

Other adverse events
Measure	Double Blind - Ecallantide n=36 participants at risk Patients treated with ecallantide in the double-blind part only.	Double-Blind Placebo n=36 participants at risk Patients treated with placebo in the double-blind part only.	Repeat-Dosing Ecallantide n=67 participants at risk All patients treated with ecallantide in the repeat-dosing part, regardless of whether they were treated previously in the double-blind part or not. All adverse events reported in all repeat-dosing episodes are included. Patients reporting more than 1 AE with the same preferred term are counted only once for that preferred term.
Gastrointestinal disorders Diarrhoea	8.3% 3/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	4.5% 3/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Nervous system disorders Headache	11.1% 4/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	5.6% 2/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	14.9% 10/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Congenital, familial and genetic disorders Hereditary Angioedema	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	5.6% 2/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	6.0% 4/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Respiratory, thoracic and mediastinal disorders Nasal Congestion	5.6% 2/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	0.00% 0/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Infections and infestations Nasopharyngitis	5.6% 2/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	2.8% 1/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	6.0% 4/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Gastrointestinal disorders Nausea	2.8% 1/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	9.0% 6/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Investigations Prothrombin Time Prolonged	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	5.6% 2/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	1.5% 1/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
General disorders Pyrexia	8.3% 3/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	1.5% 1/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Cardiac disorders Tachycardia	5.6% 2/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	2.8% 1/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	0.00% 0/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).
Respiratory, thoracic and mediastinal disorders Upper Respiratory Tract Infection	0.00% 0/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	2.8% 1/36 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).	7.5% 5/67 • Adverse events were reported from enrollment (Study Day 1) through the conclusion of the Follow-up Visit 2 (Study Days 23-37) of each episode. Additionally, AEs suspected to be related to study procedures were collected from the time of informed consent through enrollment and from Follow-up Visit 2 through Follow-up Visit 3 (Study Days 83-97).

Additional Information

Study Director

Shire

Phone: +1 866 842 5335

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee 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.
Publication restrictions are in place

Restriction type: OTHER