Hereditary Angioedema (HAE)Phase 2NCT01826916
EDEMA2: Evaluation of DX-88's Effect in Mitigating Angioedema
A Phase 2 study testing DX-88 (ecallantide) in people with Hereditary Angioedema (HAE), run by Shire.
Trial summary
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema. Read more about this study
- Who is it for?
- Ages 10 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- DX-88 (ecallantide)
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 77 participants
- Where
- No sites listed yet
- Study dates
- 1 Nov 2003to 1 Jan 2006
- 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 10 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- 10 years of age or older
- Documented diagnosis of HAE (Type I or II)
- Patient reported to a study site no later than 4 hours following patient recognition of the onset of the attack
- Willing and able to give informed consent
Who cannot join
- Patients with a serious intercurrent illness or serious active infection
- Patient with serum creatinine greater than 110% the upper limit of normal or liver transaminases 2 times the upper limit of normal
- Receipt of an investigational drug or device, within 30 days prior to study treatment
- Pregnancy or breastfeeding
- Diagnosis of acquired angioedema (AAE)
- Patients who had not completed their Day-7 follow-up procedures for a previously treated attack
About this study
Official title: EDEMA2: An Open-Label Study to Assess the Efficacy and Tolerability of Repeated Doses of DX-88 (Recombinant Plasma Kallikrein Inhibitor) in Patients With Hereditary Angioedema
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- DX-88 (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.
DX-88 (ecallantide)Drug
solution for injection 10 mg/mL
| Study group | Role | What they receive |
|---|---|---|
| 5mg/m2 DX-88 IV | Experimental | 5mg/m2 DX-88 (ecallantide)administered intravenously Receives: Drug: DX-88 (ecallantide) |
| 10mg/m2 DX-88 IV | Experimental | 10mg/m2 DX-88(ecallantide)administered intravenously Receives: Drug: DX-88 (ecallantide) |
| 20mg/m2 DX-88 IV | Experimental | 20mg/m2 DX-88 (ecallantide) administered intravenously Receives: Drug: DX-88 (ecallantide) |
| 30 mg DX-88 SC | Experimental | 30mg DX-88(ecallantide)administered subcutaneously Receives: Drug: DX-88 (ecallantide) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of attacks treated with successful outcome
successful outcome is defined as attack resolution begun within 4 hrs after treatment and maintained for 24 hours
Measured: 24 hours
Proportion of attacks with a partial response
partial response is defined as an initial response to dosing followed by a relapse within 4 to 24 hours
Measured: 24 hours
Secondary outcomes (2)
Proportion of patients who respond to a 2nd dose of ecallantide after an initial, partial response
Measured: 24 hours
Time to resolution onset of each acute attack, as determined by patient report
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Apr 2013 (estimated)
- Study start1 Nov 2003
- Primary completion1 Dec 2005
- Study completion1 Jan 2006
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- 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
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 77 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
- NCT01826916
- Sponsor's protocol ID
- DX-88/5
- Other IDs
- EDEMA2
- Status last verified
- May 2021
- First submitted
- 16 Feb 2012
- Last updated
- 18 May 2021
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Rubinstein E, Stolz LE, Sheffer AL, Stevens C, Bousvaros A. Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency. BMC Gastroenterol. 2014 Apr 9;14:71. doi: 10.1186/1471-230X-14-71.
PubMed 24712435MacGinnitie AJ, Davis-Lorton M, Stolz LE, Tachdjian R. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490-7. doi: 10.1542/peds.2013-0646. Epub 2013 Jul 22.
PubMed 23878046
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
- EDEMA2: An Open-Label Study to Assess the Efficacy and Tolerability of Repeated Doses of DX-88 (Recombinant Plasma Kallikrein Inhibitor) in Patients With Hereditary Angioedema
- ClinicalTrials.gov ID
- NCT01826916
- Acronym / sponsor ID
- DX-88/5
- Conditions
- Hereditary Angioedema (HAE)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 77 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 1 Nov 2003 (actual)
- Primary completion
- 1 Dec 2005 (actual)
- Study completion
- 1 Jan 2006 (actual)
- First posted
- 9 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.
EDEMA2 is an open-label, Phase 2 dose-ranging study designed to assess the safety and efficacy of repeated dosing of DX-88 (recombinant plasma kallikrein inhibitor) in Patients with Hereditary Angioedema.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 4
| Arm | Type | Receives |
|---|---|---|
| 5mg/m2 DX-88 IV | Experimental | Drug: DX-88 (ecallantide)5mg/m2 DX-88 (ecallantide)administered intravenously |
| 10mg/m2 DX-88 IV | Experimental | Drug: DX-88 (ecallantide)10mg/m2 DX-88(ecallantide)administered intravenously |
| 20mg/m2 DX-88 IV | Experimental | Drug: DX-88 (ecallantide)20mg/m2 DX-88 (ecallantide) administered intravenously |
| 30 mg DX-88 SC | Experimental | Drug: DX-88 (ecallantide)30mg DX-88(ecallantide)administered subcutaneously |
Who may qualify
- Ages
- 10 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- 10 years of age or older
- Documented diagnosis of HAE (Type I or II)
- Patient reported to a study site no later than 4 hours following patient recognition of the onset of the attack
- Willing and able to give informed consent
Key exclusion criteria (6 listed)
- Patients with a serious intercurrent illness or serious active infection
- Patient with serum creatinine greater than 110% the upper limit of normal or liver transaminases 2 times the upper limit of normal
- Receipt of an investigational drug or device, within 30 days prior to study treatment
- Pregnancy or breastfeeding
- Diagnosis of acquired angioedema (AAE)
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 |
|---|---|---|
| DX-88 (ecallantide)solution for injection 10 mg/mL | Drug | 10mg/m2 DX-88 IV, 20mg/m2 DX-88 IV, 30 mg DX-88 SC, 5mg/m2 DX-88 IV |
Primary outcome
Proportion of attacks treated with successful outcome
Time frame: 24 hours
successful outcome is defined as attack resolution begun within 4 hrs after treatment and maintained for 24 hours
Proportion of attacks with a partial response
Time frame: 24 hours
partial response is defined as an initial response to dosing followed by a relapse within 4 to 24 hours
2 secondary outcomes
Proportion of patients who respond to a 2nd dose of ecallantide after an initial, partial response
Time frame: 24 hours
Time to resolution onset of each acute attack, as determined by patient report
Time frame: 24 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 May 2021.
- Derived from this study: PubMed 24712435Rubinstein E, Stolz LE, Sheffer AL, Stevens C, Bousvaros A. Abdominal attacks and treatment in hereditary angioedema with C1-inhibitor deficiency. BMC Gastroenterol. 2014 Apr 9;14:71. doi: 10.1186/1471-230X-14-71.
- Derived from this study: PubMed 23878046MacGinnitie AJ, Davis-Lorton M, Stolz LE, Tachdjian R. Use of ecallantide in pediatric hereditary angioedema. Pediatrics. 2013 Aug;132(2):e490-7. doi: 10.1542/peds.2013-0646. Epub 2013 Jul 22.
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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 study testing DX-88 (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 6 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: finished, 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. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Proportion of attacks treated with successful outcome (time frame: 24 hours). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01826916, which also lists 2 linked publications.
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.