Hereditary Angioedema Types I and IIPhase 2NCT01832896
Study to Assess the Tolerability and Safety of Ecallantide in Children and Adolescents With Hereditary Angioedema
A Phase 2 study testing Ecallantide subcutaneous dosing in people with Hereditary Angioedema Types I and II, run by NYU Langone Health at 1 site in United States.
Trial summary
The objective of this study is to evaluate the safety and tolerability of a single Subcutaneous (SC) dose of Ecallantide in children and adolescents with Hereditary Angioedema (HAE). Read more about this study
- Who is it for?
- Ages 2 to 16 years
- Conditions the study is for
- Hereditary Angioedema Types I and II
- What is being tested?
- Ecallantide subcutaneous dosing
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- Aug 2013to Dec 2017
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 2 to 16 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
United States1 site
Winthrop-University Hosptial Clinical Trials Center
Mineola, New York, 11501
About this study
Official title: 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
The objective of this study is to evaluate the safety and tolerability of a single Subcutaneous (SC) dose of Ecallantide in children and adolescents with Hereditary Angioedema (HAE).
Read the 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
- Conditions
- Hereditary Angioedema Types I and II
- Being tested
- Ecallantide subcutaneous dosing
- Keywords
- Hereditary Angioedema, Children, Adolescents, Ecallantide
- Medical subject headings
- Hereditary Angioedema Types I and II, Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Ecallantide subcutaneous dosingDrug
For acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered.
| Study group | Role | What they receive |
|---|---|---|
| Ecallantide | Experimental | Study Medication, Dose, and Mode of Administration:
Single dose of ecallantide subcutaneous dosing:
* Age less than 10: Weight <25 Kg: 10mg subcutaneously at one site; 25-50kg: 20mg subcutaneously, 10mg per site for 2 separate sites; >50 kg 30mg subcutaneously, 10mg per site for 3 separate sites. Dosing will not exceed 30mg.
* Age greater than 10: 10mg per site for 3 separate sites. Dosing will not exceed 30mg. Receives: Drug: Ecallantide subcutaneous dosing |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema
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.
Measured: 28 days
Secondary outcomes (1)
Number of Partcipants with Adverse Events
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.
Measured: 28 Days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted16 Apr 2013 (estimated)
- Study startAug 2013
- Primary completionJul 2017
- Study completionDec 2017
Why it stopped: PI indicated departure from institution, inability to complete study.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema Types I and IIStudies of the same condition, open and under way.
- More trials run by NYU Langone HealthThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- NYU Langone Health
- Sponsor type
- University, hospital or other
- Collaborators
- Dyax Corp.
- Responsible party
- Sponsor
- Investigators
- Mark A Davis-Lorton, MD, Principal investigator, Winthrop University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01832896
- Sponsor's protocol ID
- DX-88/IST-5
- Status last verified
- Jan 2017
- First submitted
- 4 Apr 2013
- Last updated
- 31 Mar 2023
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Mar 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT01832896
- Acronym / sponsor ID
- DX-88/IST-5
- Conditions
- Hereditary Angioedema Types I and II
- Keywords
- Hereditary Angioedema, Children, Adolescents, Ecallantide
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: PI indicated departure from institution, inability to complete study.
- Enrolment
- 0 participants (actual)
- Lead sponsor
- NYU Langone HealthUniversity, hospital or other
- Collaborators
- Dyax Corp.
- Study start
- Aug 2013
- Primary completion
- Jul 2017 (actual)
- Study completion
- Dec 2017 (actual)
- First posted
- 16 Apr 2013 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The objective of this study is to evaluate the safety and tolerability of a single Subcutaneous (SC) dose of Ecallantide in children and adolescents with Hereditary Angioedema (HAE).
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Ecallantide | Experimental | Drug: Ecallantide subcutaneous dosingStudy Medication, Dose, and Mode of Administration: Single dose of ecallantide subcutaneous dosing: * Age less than 10: Weight <25 Kg: 10mg subcutaneously at one site; 25-50kg: 20mg subcutaneously, 10mg per site for 2 separate sites; >50 kg 30mg subcutaneously, 10mg per site for 3 separate sites. Dosing will not exceed 30mg. * Age greater than 10: 10mg per site for 3 separate sites. Dosing will not exceed 30mg. |
Who may qualify
- Ages
- 2 to 16 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- 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).
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Ecallantide subcutaneous dosingFor acute attacks of Hereditary Angioedema in children and adolescents, Ecallantide will be administered. | Drug | Ecallantide |
Primary outcome
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.
1 secondary outcome
Number of Partcipants with Adverse Events
Time frame: 28 Days
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Mar 2023.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Mark A Davis-Lorton, MD principal investigator
Winthrop University Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Ecallantide subcutaneous dosing in people with Hereditary Angioedema Types I and II, run by NYU Langone Health at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 31 Mar 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurement of time to symptomatic improvement from acute attacks of hereditary angioedema (time frame: 28 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Mar 2023.