Hereditary Angioedema (HAE)Phase 1NCT02093923
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
A Phase 1 study testing DX-2930 in people with Hereditary Angioedema (HAE), run by Shire at 14 sites in 3 countries.
Trial summary
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hereditary Angioedema (HAE)
- What is being tested?
- DX-2930
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 38 participants
- Where
- 14 sitesin 3 countries
- Study dates
- 14 May 2014to 18 May 2015
- Phase
- Phase 1
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- At least 18 years of age at the time of screening
- Documented diagnosis of HAE (Type I or II)
- Experiencing ≥2 HAE attacks per year, with at least 1 attack in the past 6 months reported by the participant
- Willing and able to read, understand, and sign an informed consent form
- Females of childbearing potential must agree to be abstinent or else use acceptable forms of contraception throughout study
- Males with female partners of childbearing potential must agree to be abstinent or use a medically acceptable form of contraception throughout study
Who cannot join
- Exposure to an investigational drug or device within 90 days prior to study
- History of exposure within the past 5 years to a monoclonal antibody or recombinant protein bearing an Fc domain
- Concomitant diagnosis of another form of chronic angioedema
- Use of long-term prophylaxis for HAE within 90 days prior to study
- Use of C1-INH that exceeds a total of 30 days within the past 90 days prior to study; any use of C1-INH within 7 days prior to study
- Exposure to angiotensin-converting enzyme (ACE) inhibitors or any estrogen-containing medications with systemic absorption within 90 days prior to study
Show all 13Show fewer
- Exposure to androgens within 90 days prior to study
- Presence of an indwelling catheter
- Diagnosis of HIV
- Active liver disease or liver function test abnormalities
- History of substance abuse or dependence
- Pregnancy or breastfeeding
- Any condition that, in the opinion of the Investigator, may compromise their safety or compliance, preclude successful conduct of the study, or interfere with interpretation of the results
Trial locations and contacts
14 sites in 3 countries.
14 sites in 3 countries
United States12 sites
Massachusetts General Hospital Allergy Associates
Boston, Massachusetts, 02114
Institute for Asthma & Allergy, PC
Chevy Chase, Maryland, 20815
UC Physicians Company
Cincinnati, Ohio, 45267
AARA Research Center
Dallas, Texas, 75231
Penn State Hershey Medical Center
Hershey, Pennsylvania, 17033
Baker Allergy, Asthma and Dermatology Research Center
Lake Oswego, Oregon, 97035
Winthrop-University Hospital, Clinical Trials Center
Mineola, New York, 11501
Icahn School of Medicine at Mount Sinai - The Mount Sinai Medical Center
New York, New York, 10029
UC San Diego Health System - La Jolla
San Diego, California, 92122
Washington University School of Medicine
St Louis, Missouri, 63110
University of South Florida Asthma, Allergy or Immunology Clinical Research Unit
Tampa, Florida, 33613
Allergy & Asthma Clinical Research
Walnut Creek, California, 94598
About this study
Official title: A Phase 1b, Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Subjects
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Conditions
- Hereditary Angioedema (HAE)
- Being tested
- DX-2930, Placebo
- Keywords
- HAE
- 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-2930Drug
Participants will receive SC injection of DX-2930 (a recombinant, Chinese hamster ovary [CHO] cell expressed, fully human immunoglobulin IgG1, kappa light chain, monoclonal antibody) once and followed by the second dose after 2 week into the upper arm.
PlaceboDrug
Participants will receive matching placebo subcutaneously.
| Study group | Role | What they receive |
|---|---|---|
| DX-2930, Cohort 1 | Experimental | Participants will receive 30 milligram (mg) dose of DX-2930 subcutaneous (SC) injection once and followed by the second dose after 2 week into the upper arm. Receives: Drug: DX-2930 |
| DX-2930, Cohort 2 | Experimental | Participants will receive 100 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm. Receives: Drug: DX-2930 |
| DX-2930, Cohort 3 | Experimental | Participants will receive 300 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm. Receives: Drug: DX-2930 |
| DX-2930, Cohort 4 | Experimental | Participants will receive 400 mg dose of DX-2930 subcutaneous (SC) injection once and followed by the second dose after 2 week into the upper arm. Receives: Drug: DX-2930 |
| Placebo | Placebo comparator | Participants will receive placebo matched to 30, 100, 300 and 400 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Serious Adverse Events (SAE) and Treatment-Emergent Adverse Events (TEAE)
A SAE was any adverse experience occurring at any dose that resulted in any of the following outcomes: Death, Life-threatening experience, required inpatient hospitalization or prolongation of existing hospitalization. Resulted in persistent or significant disability or incapacity. Was a congenital anomaly or birth defect. Was considered to be an important medical event. An AE was considered treatment-emergent if the onset time was after administration of study drug through the Day 120 post-dose final follow-up visit or, in the event that onset time preceded study drug administration, the AE increased in severity during the 120-day post-dose follow-up period.
Measured: From Day 1 up to final follow-up (Day 123)
Secondary outcomes (6)
Maximum Plasma Concentration (Cmax)
Pharmacokinetic (PK) parameter Cmax data were reported.
Measured: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Time to Maximum Plasma Concentration (Tmax)
PK parameter Tmax data were reported.
Measured: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Area Under the Plasma Concentration-Time Curve (AUC)
PK paramenter AUC data were reported.
Measured: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Apparent Clearance (CL/F)
PK parameter CL/F data were reported.
Measured: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Apparent Volume of Distribution (Vd/F)
PK parameter Vd/F data were reported.
Measured: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Terminal Elimination Half-Life (t1/2)
PK parameter t(1/2) data were reported.
Measured: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Mar 2014 (estimated)
- Study start14 May 2014
- Primary completion18 May 2015
- Study completion18 May 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)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 1
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 38 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
- NCT02093923
- Sponsor's protocol ID
- DX-2930-02
- Status last verified
- May 2021
- First submitted
- 17 Mar 2014
- Last updated
- 27 May 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
Publications
Banerji A, Busse P, Shennak M, Lumry W, Davis-Lorton M, Wedner HJ, Jacobs J, Baker J, Bernstein JA, Lockey R, Li HH, Craig T, Cicardi M, Riedl M, Al-Ghazawi A, Soo C, Iarrobino R, Sexton DJ, TenHoor C, Kenniston JA, Faucette R, Still JG, Kushner H, Mensah R, Stevens C, Biedenkapp JC, Chyung Y, Adelman B. Inhibiting Plasma Kallikrein for Hereditary Angioedema Prophylaxis. N Engl J Med. 2017 Feb 23;376(8):717-728. doi: 10.1056/NEJMoa1605767.
PubMed 28225674
Study at a glance
From the registry record, last updated by the sponsor on 27 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1b, Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Subjects
- ClinicalTrials.gov ID
- NCT02093923
- Acronym / sponsor ID
- DX-2930-02
- Conditions
- Hereditary Angioedema (HAE)
- Keywords
- HAE
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 38 participants (actual)
- Lead sponsor
- ShireIndustry (pharma, biotech, devices)
- Study start
- 14 May 2014 (actual)
- Primary completion
- 18 May 2015 (actual)
- Study completion
- 18 May 2015 (actual)
- First posted
- 21 Mar 2014 (estimated)
- Results
- Posted 1 Aug 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| DX-2930, Cohort 1 | Experimental | Drug: DX-2930Participants will receive 30 milligram (mg) dose of DX-2930 subcutaneous (SC) injection once and followed by the second dose after 2 week into the upper arm. |
| DX-2930, Cohort 2 | Experimental | Drug: DX-2930Participants will receive 100 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm. |
| DX-2930, Cohort 3 | Experimental | Drug: DX-2930Participants will receive 300 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm. |
| DX-2930, Cohort 4 | Experimental | Drug: DX-2930Participants will receive 400 mg dose of DX-2930 subcutaneous (SC) injection once and followed by the second dose after 2 week into the upper arm. |
| Placebo | Placebo comparator | Drug: PlaceboParticipants will receive placebo matched to 30, 100, 300 and 400 mg dose of DX-2930 SC injection once and followed by the second dose after 2 week into the upper arm. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- At least 18 years of age at the time of screening
- Documented diagnosis of HAE (Type I or II)
- Experiencing ≥2 HAE attacks per year, with at least 1 attack in the past 6 months reported by the participant
- Willing and able to read, understand, and sign an informed consent form
- Females of childbearing potential must agree to be abstinent or else use acceptable forms of contraception throughout study
Key exclusion criteria (13 listed)
- Exposure to an investigational drug or device within 90 days prior to study
- History of exposure within the past 5 years to a monoclonal antibody or recombinant protein bearing an Fc domain
- Concomitant diagnosis of another form of chronic angioedema
- Use of long-term prophylaxis for HAE within 90 days prior to study
- Use of C1-INH that exceeds a total of 30 days within the past 90 days prior to study; any use of C1-INH within 7 days prior to study
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-2930Participants will receive SC injection of DX-2930 (a recombinant, Chinese hamster ovary [CHO] cell expressed, fully human immunoglobulin IgG1, kappa light chain, monoclonal antibody) once and followed by the second dose after 2 week into the upper arm. | Drug | DX-2930, Cohort 1, DX-2930, Cohort 2, DX-2930, Cohort 3, DX-2930, Cohort 4 |
| PlaceboParticipants will receive matching placebo subcutaneously. | Drug | Placebo |
Primary outcome
Number of Participants With Serious Adverse Events (SAE) and Treatment-Emergent Adverse Events (TEAE)
Time frame: From Day 1 up to final follow-up (Day 123)
A SAE was any adverse experience occurring at any dose that resulted in any of the following outcomes: Death, Life-threatening experience, required inpatient hospitalization or prolongation of existing hospitalization. Resulted in persistent or significant disability or incapacity. Was a congenital anomaly or birth defect. Was considered to be an important medical event. An AE was considered treatment-emergent if the onset time was after administration of study drug through the Day 120 post-dose final follow-up visit or, in the event that onset time preceded study drug administration, the AE increased in severity during the 120-day post-dose follow-up period.
6 secondary outcomes
Maximum Plasma Concentration (Cmax)
Time frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Time to Maximum Plasma Concentration (Tmax)
Time frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Area Under the Plasma Concentration-Time Curve (AUC)
Time frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Apparent Clearance (CL/F)
Time frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Apparent Volume of Distribution (Vd/F)
Time frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Terminal Elimination Half-Life (t1/2)
Time frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 May 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 28225674Banerji A, Busse P, Shennak M, Lumry W, Davis-Lorton M, Wedner HJ, Jacobs J, Baker J, Bernstein JA, Lockey R, Li HH, Craig T, Cicardi M, Riedl M, Al-Ghazawi A, Soo C, Iarrobino R, Sexton DJ, TenHoor C, Kenniston JA, Faucette R, Still JG, Kushner H, Mensah R, Stevens C, Biedenkapp JC, Chyung Y, Adelman B. Inhibiting Plasma Kallikrein for Hereditary Angioedema Prophylaxis. N Engl J Med. 2017 Feb 23;376(8):717-728. doi: 10.1056/NEJMoa1605767.
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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
14 sites in 3 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing DX-2930 in people with Hereditary Angioedema (HAE), run by Shire at 14 sites in 3 countries.
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 6 inclusion and 13 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 27 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States, Italy, Jordan. 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. 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 phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 1 Aug 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Number of Participants With Serious Adverse Events (SAE) and Treatment-Emergent Adverse Events (TEAE) (time frame: From Day 1 up to final follow-up (Day 123)). 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 NCT02093923, which also lists 1 linked publication.
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 27 May 2021.