A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
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ClinicalTrials.gov Identifier: NCT02093923 |
Recruitment Status :
Completed
First Posted : March 21, 2014
Results First Posted : August 1, 2016
Last Update Posted : May 27, 2021
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Condition or disease | Intervention/treatment | Phase |
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Hereditary Angioedema (HAE) | Drug: DX-2930 Drug: Placebo | Phase 1 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 38 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor) |
Primary Purpose: | Treatment |
Official Title: | A Phase 1b, Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Subjects |
Actual Study Start Date : | May 14, 2014 |
Actual Primary Completion Date : | May 18, 2015 |
Actual Study Completion Date : | May 18, 2015 |

Arm | Intervention/treatment |
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Experimental: DX-2930, Cohort 1
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.
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Drug: DX-2930
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. |
Experimental: DX-2930, Cohort 2
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.
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Drug: DX-2930
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. |
Experimental: DX-2930, Cohort 3
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.
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Drug: DX-2930
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. |
Experimental: DX-2930, Cohort 4
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.
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Drug: DX-2930
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. |
Placebo Comparator: Placebo
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.
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Drug: Placebo
Participants will receive matching placebo subcutaneously. |
- 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.
- Maximum Plasma Concentration (Cmax) [ Time Frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120 ]Pharmacokinetic (PK) parameter Cmax data were reported.
- Time to Maximum Plasma Concentration (Tmax) [ Time Frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120 ]PK parameter Tmax data were reported.
- 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 ]PK paramenter AUC data were reported.
- Apparent Clearance (CL/F) [ Time Frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120 ]PK parameter CL/F data were reported.
- Apparent Volume of Distribution (Vd/F) [ Time Frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120 ]PK parameter Vd/F data were reported.
- Terminal Elimination Half-Life (t1/2) [ Time Frame: Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120 ]PK parameter t(1/2) data were reported.
- HAE Attack Rate Per Week From Day 8 to Day 50 [ Time Frame: Day 8 to Day 50 ]Analysis of this outcome measure was based on participants in the 300 mg, 400 mg, and placebo dose groups with a historical baseline attack rate of at least 2 attacks over the last 3 months prior to enrollment. The result is based on General Estimating Equation (GEE) analysis of repeated counts per week during the pre-specified assessment period (Days 8 to 50; predicted to correspond to a period of notable drug exposure). Baseline HAE attack rate per week was a covariate, treatment group is a fixed effect, and participant was a random effect in the GEE model with independence working correlation structure.
- HAE Attacks Per Week From Day 8 to Day 92 [ Time Frame: Day 8 to Day 92 ]This endpoint analysis was based on participants in the 300 mg, 400 mg, and placebo dose groups with a historical baseline attack rate of at least 2 attacks over the last 3 months prior to enrollment. The result is based on General Estimating Equation (GEE) analysis of repeated counts per week during the prespecified assessment period (Day 8 to Day 92 predicted to correspond to a period of notable drug exposure). Baseline HAE attack rate per week is a covariate, treatment group is a fixed effect, and participant is a random effect in the GEE model with independence working correlation structure.
- HAE Attacks Per Week From Day 8 to Day 64 [ Time Frame: Day 8 to Day 64 ]This endpoint analysis was based on participants in the 300 mg, 400 mg, and placebo dose groups with a historical baseline attack rate of at least 2 attacks over the last 3 months prior to enrollment. The result is based on General Estimating Equation (GEE) analysis of repeated counts per week during the prespecified assessment period (Day 8 to Day 64 predicted to correspond to a period of notable drug exposure). Baseline HAE attack rate per week is a covariate, treatment group is a fixed effect, and participant is a random effect in the GEE model with independence working correlation structure.
- HAE Attack Rate Per Week From Day 1 to Day 50 [ Time Frame: Day 1 to Day 50 ]This endpoint analysis was based on participants in the 300 mg, 400 mg, and placebo dose groups with a historical baseline attack rate of at least 2 attacks over the last 3 months prior to enrollment. The result is based on General Estimating Equation (GEE) analysis of repeated counts per week during the prespecified assessment period (Day 1 to Day 50 predicted to correspond to a period of notable drug exposure). Baseline HAE attack rate per week is a covariate, treatment group is a fixed effect, and participant is a random effect in the GEE model with independence working correlation structure.

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Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- 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
Exclusion Criteria:
- 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
- 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

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02093923
United States, California | |
UC San Diego Health System - La Jolla | |
San Diego, California, United States, 92122 | |
Allergy & Asthma Clinical Research | |
Walnut Creek, California, United States, 94598 | |
United States, Florida | |
University of South Florida Asthma, Allergy or Immunology Clinical Research Unit | |
Tampa, Florida, United States, 33613 | |
United States, Maryland | |
Institute for Asthma & Allergy, PC | |
Chevy Chase, Maryland, United States, 20815 | |
United States, Massachusetts | |
Massachusetts General Hospital Allergy Associates | |
Boston, Massachusetts, United States, 02114 | |
United States, Missouri | |
Washington University School of Medicine | |
Saint Louis, Missouri, United States, 63110 | |
United States, New York | |
Winthrop-University Hospital, Clinical Trials Center | |
Mineola, New York, United States, 11501 | |
Icahn School of Medicine at Mount Sinai - The Mount Sinai Medical Center | |
New York, New York, United States, 10029 | |
United States, Ohio | |
UC Physicians Company | |
Cincinnati, Ohio, United States, 45267 | |
United States, Oregon | |
Baker Allergy, Asthma and Dermatology Research Center | |
Lake Oswego, Oregon, United States, 97035 | |
United States, Pennsylvania | |
Penn State Hershey Medical Center | |
Hershey, Pennsylvania, United States, 17033 | |
United States, Texas | |
AARA Research Center | |
Dallas, Texas, United States, 75231 | |
Italy | |
Ospedale L. Sacco | |
Milano, MI, Italy, 20157 | |
Jordan | |
Jordan University Hospital | |
Amman, Jordan, 11942 |
Study Director: | Study Director | Takeda |
Responsible Party: | Shire |
ClinicalTrials.gov Identifier: | NCT02093923 |
Other Study ID Numbers: |
DX-2930-02 |
First Posted: | March 21, 2014 Key Record Dates |
Results First Posted: | August 1, 2016 |
Last Update Posted: | May 27, 2021 |
Last Verified: | May 2021 |
HAE |
Angioedema Angioedemas, Hereditary Vascular Diseases Cardiovascular Diseases Urticaria Skin Diseases, Vascular Skin Diseases |
Hypersensitivity, Immediate Hypersensitivity Immune System Diseases Hereditary Complement Deficiency Diseases Primary Immunodeficiency Diseases Genetic Diseases, Inborn Immunologic Deficiency Syndromes |