Hereditary Angioedema (HAE)Phase 2/3NCT01253382
Study to Evaluate Ecallantide in Paediatric Patients With Acute Attacks of Hereditary Angioedema
A Phase 2/3 study testing ecallantide in people with Hereditary Angioedema (HAE), run by Shire.
Trial summary
The primary objective of this study is: assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE The secondary objectives are: evaluate the pharmacokinetic profile of ecallantide in paediatric patients treated for acute attacks of HAE assess the efficacy of ecallantide in… Read more about this study
- Who is it for?
- Ages 2 to 17 years
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- ecallantide
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 0 participants
- Where
- No sites listed yet
- Study dates
- 1 Jun 2012to 1 Apr 2015
- Phase
- Phase 2/3
- 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 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- Male or female from 2 years of age and prepubertal
- Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-1NH activity below the lower limit of normal or up to 15% above the lower limit of the normal range
- Present at the site with moderate to severe signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack. Spontaneous resolution of the attack must not have begun before the administration of study drug.
- Must have signed informed consent by parent or caregiver.
Who cannot join
- <2 years of age or have reached puberty
- Received treatment with ecallantide within previous 72 hours
- Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit
- Pharyngeal/laryngeal symptoms
- Mild attacks including mild edema of the extremities and mild abdominal attacks
- Are unable or unwilling to give informed consent (parent or caregiver)
- Any other condition that, in the opinion of the investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
About this study
Official title: A 3-Part Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Subcutaneous Ecallantide in Prepubertal Paediatric Patients Experiencing Acute Attacks of Hereditary Angioedema (HAE)
The primary objective of this study is:
- assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE
The secondary objectives are:
- evaluate the pharmacokinetic profile of ecallantide in paediatric patients treated for acute attacks of HAE
- assess the efficacy of ecallantide in paediatric patients treated for moderate to severe acute attacks of HAE
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- ecallantide
- Medical subject headings
- Angioedemas, Hereditary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ecallantideBiological
10 - 30mg subcutaneous injection.
| Study group | Role | What they receive |
|---|---|---|
| ecallantide | Active comparator | Not described Receives: Biological: ecallantide |
| placebo | Placebo comparator | phosphate buffered saline Receives: Biological: ecallantide |
What is being measured
The primary outcome is the main question the study is designed to answer.
safety and tolerability of ecallantide
Measured: 4 years
Secondary outcomes (2)
evaluate the PK profile of ecallantide
Measured: 4 years
assess the efficacy of ecallantide
Measured: 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Dec 2010 (estimated)
- Study start1 Jun 2012
- Primary completion1 Apr 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hereditary Angioedema (HAE)Studies of the same condition, open and under way.
- 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 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Takeda
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01253382
- Sponsor's protocol ID
- DX-88/26
- Other IDs
- 2010-022716-39
- Status last verified
- May 2021
- First submitted
- 2 Dec 2010
- Last updated
- 18 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 3-Part Study to Evaluate the Pharmacokinetics, Safety, and Efficacy of Subcutaneous Ecallantide in Prepubertal Paediatric Patients Experiencing Acute Attacks of Hereditary Angioedema (HAE)
- ClinicalTrials.gov ID
- NCT01253382
- Acronym / sponsor ID
- DX-88/26
- Conditions
- Hereditary Angioedema (HAE)
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Withdrawn
- Enrolment
- 0 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 1 Jun 2012 (actual)
- Primary completion
- 1 Apr 2015 (estimated)
- First posted
- 3 Dec 2010 (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 primary objective of this study is:
- assess the safety and tolerability of ecallantide in paediatric patients for acute attacks of HAE
The secondary objectives are:
- evaluate the pharmacokinetic profile of ecallantide in paediatric patients treated for acute attacks of HAE
- assess the efficacy of ecallantide in paediatric patients treated for moderate to severe acute attacks of HAE
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Single group
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| ecallantide | Active comparator | Biological: ecallantide |
| placebo | Placebo comparator | Biological: ecallantidephosphate buffered saline |
Who may qualify
- Ages
- 2 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (4 listed)
- Male or female from 2 years of age and prepubertal
- Physician diagnosis and history of HAE (Type I or II) including laboratory results showing C1-1NH activity below the lower limit of normal or up to 15% above the lower limit of the normal range
- Present at the site with moderate to severe signs and symptoms of an acute attack of HAE within 8 hours of recognition of the onset of the attack. Spontaneous resolution of the attack must not have begun before the administration of study drug.
- Must have signed informed consent by parent or caregiver.
Key exclusion criteria (7 listed)
- <2 years of age or have reached puberty
- Received treatment with ecallantide within previous 72 hours
- Received an investigational drug or device, other than ecallantide, within 30 days prior to the screening visit
- Pharyngeal/laryngeal symptoms
- Mild attacks including mild edema of the extremities and mild abdominal attacks
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 |
|---|---|---|
| ecallantide10 - 30mg subcutaneous injection. | Biological | ecallantide, placebo |
Primary outcome
safety and tolerability of ecallantide
Time frame: 4 years
2 secondary outcomes
evaluate the PK profile of ecallantide
Time frame: 4 years
assess the efficacy of ecallantide
Time frame: 4 years
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 18 May 2021.
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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.
Study Director study director
Takeda
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing ecallantide in people with Hereditary Angioedema (HAE), run by Shire.
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 4 inclusion and 7 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 18 May 2021. Status can change between updates, so confirm with the study contact before making plans.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined 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.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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?
safety and tolerability of ecallantide (time frame: 4 years). 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 18 May 2021.