Hereditary Angioedema
Conditions
Brief summary
The investigators propose a study to evaluate the safety, local tolerability, convenience, and efficacy of self-administered Icatibant for the treatment of acute attacks of hereditary angioedema. The investigators believe that self administration with Icatibant for treatment of an acute attack of angioedema will not change the time to complete or near complete resolution of symptoms compared to treatment with Icatibant in a medical facility.
Interventions
DRUGIcatibant
30 mg subcutaneous dose of Icatibant
Sponsors
Shire Human Genetic Therapies, Inc.
Massachusetts General Hospital
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
1. Males and females at least 18 years of age at the time of informed consent 2. Documented diagnosis of hereditary angioedema Type I or II based on ALL of the following criteria: * Family and/or medical history * Characteristic attack manifestations, recurrent attacks * Historical low C4, normal C1q and either low C1-INH or low C1INH function 3. Women of childbearing potential must use consistently and correctly a highly effective, adequate method of birth control (failure rate less than 1% per year), sexual abstinence or have a vasectomised partner during the duration of the study. Hormonal contraception can be continued if verified by a physician that it doesn't affect the course of hereditary angioedema attacks. 4. Mental and physical condition allowing patients to complete baseline assessment, to self-administer Icatibant and to follow other study procedures. 5. Ability to provide signed written informed consent after all aspects of the study have been explained and discussed with the patient.
Exclusion criteria
1. Participation in a clinical therapeutic trial of another investigational medicinal product within the past month (except a previous Icatibant study). 2. Diagnosis of angioedema other than Type I or Type II hereditary angioedema. 3. Evidence of symptomatic coronary artery disease based on medical history, in particular, unstable angina pectoris or severe coronary heart disease. 4. Congestive heart failure (NYHA Class 3 and 4). 5. Stroke within the past 6 months. 6. Treatment with angiotensin converting enzyme inhibitor. 7. Pregnancy and/or breast-feeding. 8. In the opinion of the investigator: mental condition rendering the patient unable to understand the nature, scope and possible consequences of the study. 9. In the opinion of the investigator: unlikely to comply with the protocol, for example, uncooperative attitude, inability to return for follow-up visits, or unlikely to complete the study for any reason. 10. In the opinion of the investigator: inability to complete the patient diary, manage study medication or self-administration of an injection.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Time to Complete or Near Complete Resolution From Onset of Symptoms | Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours | Time of onset of HAE attack, time icatibant was administered, and time to complete relief of symptoms were recorded in minutes. Time to complete relief of symptoms was defined as time from onset of symptoms to complete or near complete resolution as reported by the patient. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percent Change in VAS Scores | Percent Change in VAS Score from Baseline to 4 Hours | Baseline, 4 hours VAS scale ranges from 0-100 with 0 being the lowest severity and 100 being the highest severity |
Countries
United States
Participant flow
Participants by arm
| Arm | Count |
|---|---|
| Icatibant Icatibant: 30 mg subcutaneous dose of Icatibant | 19 |
| Total | 19 |
Baseline characteristics
| Characteristic | Icatibant |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 2 Participants |
| Age, Categorical Between 18 and 65 years | 17 Participants |
| Age, Continuous | 41 years |
| Region of Enrollment United States | 19 participants |
| Sex: Female, Male Female | 14 Participants |
| Sex: Female, Male Male | 5 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 4 / 78 |
| serious Total, serious adverse events | 0 / 78 |
Outcome results
Primary
Time to Complete or Near Complete Resolution From Onset of Symptoms
Time of onset of HAE attack, time icatibant was administered, and time to complete relief of symptoms were recorded in minutes. Time to complete relief of symptoms was defined as time from onset of symptoms to complete or near complete resolution as reported by the patient.
Time frame: Time to complete or near complete resolution of symptoms as reported by the patient, an expected average of 8-10 hours
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Icatibant Treatment With Health Care Provider | Time to Complete or Near Complete Resolution From Onset of Symptoms | 735 minutes |
| Icatibant Treatment by Self Administration | Time to Complete or Near Complete Resolution From Onset of Symptoms | 377 minutes |
Secondary
Percent Change in VAS Scores
Baseline, 4 hours VAS scale ranges from 0-100 with 0 being the lowest severity and 100 being the highest severity
Time frame: Percent Change in VAS Score from Baseline to 4 Hours
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| Icatibant Treatment With Health Care Provider | Percent Change in VAS Scores | -97 percent change |
| Icatibant Treatment by Self Administration | Percent Change in VAS Scores | -96 percent change |