- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT01832896
Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
A Multicenter, Open-Label Study to Assess the Tolerability and Safety of a Single, Subcutaneous Administration of Ecallantide in Children and Adolescents With Hereditary Angioedema
Study Overview
Status
Conditions
Intervention / Treatment
Detailed Description
This pilot study is an open-label, non-randomized, single-arm study to evaluate the tolerability and safety of a single SC administration of ecallantide in up to approximately 10 pediatric subjects with HAE during an initial acute attack. The study is planned to enroll subjects 2 through 15 years of age who present with an acute cutaneous, abdominal, or laryngeal HAE attack. No more than 3 study sites will be included until a goal of 10 patients is achieved.
After treatment for an initial attack, one additional open label treatment with ecallantide will be offered to subjects contingent upon having been treated previously and presenting with a subsequent acute cutaneous, abdominal, or laryngeal attack of HAE at least 7 days after initial treatment. Open-label treatment for a second HAE attack will continue until 10 patients have been treated for an initial attack. Safety evaluations will be performed at each subsequent ecallantide-treated attack as for the initial treated attack
Study Type
Phase
- Phase 2
Contacts and Locations
Study Contact
- Name: Mark A Davis-Lorton, MD
- Phone Number: 516-663-2097
- Email: mdavislorton@winthrop.org
Study Locations
-
-
New York
-
Mineola, New York, United States, 11501
- Winthrop-University Hosptial Clinical Trials Center
-
-
Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Description
Inclusion Criteria:
- 2 through 15 years of age, inclusive (ie, from the second birthday through the day prior to the sixteenth birthday) at the time of the subject's first attack.
- Documented diagnosis of HAE type I or II. Diagnosis must be confirmed by a documented immunogenic (below the lower limit of normal) and/or functional (< 50% of normal levels) C1- Inhibitor deficiency. Diagnosis may be on the basis of historic data or by diagnostic testing conducted at the time of screening.
- Informed consent (and subject assent as appropriate) signed by the subject's parent(s) or legal guardian(s).
Exclusion Criteria:
- History of an adverse reaction (AE) to Ecallantide in the past
- Diagnosis of angioedema other than HAE
- Participation in another clinical study during the 30 days prior to treatment
- Any known factor/disease that might interfere with the treatment compliance, study conduct, or result interpretation
- Congenital or acquired cardiac anomalies that interfere significantly with cardiac function.
- Treatment with angiotensin converting enzyme (ACE) inhibitors within 7 days prior to treatment.
- Use of hormonal contraception within the 90 days prior to treatment for females of childbearing potential
- The subject is pregnant or breastfeeding
Study Plan
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Ecallantide
Study Medication, Dose, and Mode of Administration: Single dose of ecallantide subcutaneous dosing:
|
For acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema
Time Frame: 28 days
|
Time to symptom relief. Time to minimal symptoms. An improvement assessment will also be performed at each of the following timepoints: pre -treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit. Incidence of need for rescue medication. Incidence of worsening despite use of ecallantide. |
28 days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Number of Partcipants with Adverse Events
Time Frame: 28 Days
|
Study Partcipants will be monitored for adverse events, changes in laboratory values, physical exam, vital sign changes and ECG changes.
Vital signs, including body temperature, heart rate and sitting blood pressure, will be assessed at screening, Pre-treatment, every 30 minutes for the first 2 hours and then hourly through discharge and at the 28 day clinic visit.
|
28 Days
|
Collaborators and Investigators
Sponsor
Collaborators
Investigators
- Principal Investigator: Mark A Davis-Lorton, MD, Winthrop University Hospital
Study record dates
Study Major Dates
Study Start
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Estimate)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Cardiovascular Diseases
- Vascular Diseases
- Skin Diseases
- Immunologic Deficiency Syndromes
- Immune System Diseases
- Hypersensitivity, Immediate
- Genetic Diseases, Inborn
- Skin Diseases, Vascular
- Hypersensitivity
- Urticaria
- Hereditary Complement Deficiency Diseases
- Primary Immunodeficiency Diseases
- Angioedema
- Angioedemas, Hereditary
- Hereditary Angioedema Types I and II
- Physiological Effects of Drugs
- Peripheral Nervous System Agents
- Analgesics
- Sensory System Agents
- Anti-Inflammatory Agents, Non-Steroidal
- Analgesics, Non-Narcotic
- Anti-Inflammatory Agents
- Antirheumatic Agents
- Ecallantide
Other Study ID Numbers
- DX-88/IST-5
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Hereditary Angioedema Types I and II
-
KalVista Pharmaceuticals, Ltd.TerminatedAngioedema, Hereditary, Types I and IIUnited States, Czechia, Germany, Hungary, Italy, North Macedonia, United Kingdom, Australia, Bulgaria, Canada, France, New Zealand, Puerto Rico
-
Pharvaris Netherlands B.V.Active, not recruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Poland, Germany, Austria, Bulgaria, Canada, Ireland, Italy, United Kingdom
-
Pharvaris Netherlands B.V.CompletedHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsBulgaria, United States, Spain, Israel, Germany, Canada, Czechia, France, Hungary, Italy, Netherlands, Poland, United Kingdom
-
CENTOGENE GmbH RostockCompletedHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | C1 Esterase Inhibitor Deficiency | HAE | Angio Edema | C4 Deficiency | Hereditary Angioedema Type IIITurkey, Armenia, Georgia, India, Peru, Poland, Romania
-
HAE Global Registry FoundationRecruitingHereditary Angioedema Type I and IIItaly
-
Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States
-
Pharvaris Netherlands B.V.RecruitingHereditary Angioedema | Hereditary Angioedema Type I | Hereditary Angioedema Type II | Hereditary Angioedema Types I and II | Hereditary Angioedema Attack | Hereditary Angioedema With C1 Esterase Inhibitor Deficiency | Hereditary Angioedema - Type 1 | Hereditary Angioedema - Type 2 | C1 Esterase Inhibitor... and other conditionsUnited States, Bulgaria, Czechia, Hungary, Spain, France, Germany, Poland, Canada, Israel
-
KalVista Pharmaceuticals, Ltd.CompletedHereditary AngioedemaUnited States, Austria, Czechia, Germany, Hungary, Italy, Netherlands, North Macedonia, Poland, United Kingdom
-
Prothya BiosolutionsCompletedHereditary Angioedema Type I | Angioneurotic EdemaNetherlands
-
CSL BehringParexelCompletedHereditary Angioedema Types I and IIUnited States, Germany
Clinical Trials on Ecallantide subcutaneous dosing
-
Mural Oncology, IncRecruitingCutaneous Melanoma | Mucosal MelanomaUnited States, Australia, Canada, Italy, Korea, Republic of, Spain, Taiwan, United Kingdom
-
ShireTerminatedACE Inhibitor Induced AngioedemaUnited States
-
Novartis PharmaceuticalsActive, not recruitingStudy of Efficacy, Safety, and Tolerability of LNA043 in Patients With Knee Osteoarthritis (ONWARDS)OsteoarthritisTaiwan, Spain, United Kingdom, United States, Argentina, Australia, Denmark, India, China, Czechia, Canada, Japan, Poland, Mexico, Estonia
-
Wake Forest University Health SciencesTerminatedSepsis | Obesity | Septic ShockUnited States
-
BioMarin PharmaceuticalActive, not recruitingAchondroplasiaUnited States, Australia, Japan, United Kingdom
-
Spectrum Pharmaceuticals, IncTerminated
-
AstraZenecaActive, not recruitingPrimary Aldosteronism | HyperaldosteronismUnited States
-
AstraZenecaCompletedHealthy VolunteersUnited States
-
University of Illinois at Urbana-ChampaignNot yet recruitingDietary Carbohydrate | Para-AthletesUnited States
-
Rush University Medical CenterWithdrawnChronic Low-back Pain | Peripheral Neuropathy | CRPS