A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants

Phase 1

Completed

Brief 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.

Recruitment & Eligibility

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

Arm && Interventions

Group	Intervention	Description
DX-2930, Cohort 1	DX-2930	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.
DX-2930, Cohort 3	DX-2930	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.
Placebo	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.
DX-2930, Cohort 2	DX-2930	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.
DX-2930, Cohort 4	DX-2930	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.

Primary Outcome Measures

Name	Time	Method
Number of Participants With Serious Adverse Events (SAE) and Treatment-Emergent Adverse Events (TEAE)	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.

Secondary Outcome Measures

Name	Time	Method
Apparent Clearance (CL/F)	Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120	PK parameter CL/F data were reported.
Terminal Elimination Half-Life (t1/2)	Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120	PK parameter t(1/2) data were reported.
Time to Maximum Plasma Concentration (Tmax)	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)	Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120	PK paramenter AUC data were reported.
Apparent Volume of Distribution (Vd/F)	Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120	PK parameter Vd/F data were reported.
Maximum Plasma Concentration (Cmax)	Days 1, 2, 4, 8, 15, 16, 18, 22, 29, 36, 50, 64, 92, and 120	Pharmacokinetic (PK) parameter Cmax data were reported.

Locations (14): Penn State Hershey Medical Center
🇺🇸
Hershey, Pennsylvania, United States
Massachusetts General Hospital Allergy Associates
🇺🇸
Boston, Massachusetts, United States
UC San Diego Health System - La Jolla
🇺🇸
San Diego, California, United States
UC Physicians Company
🇺🇸
Cincinnati, Ohio, United States
Allergy & Asthma Clinical Research
🇺🇸
Walnut Creek, California, United States
University of South Florida Asthma, Allergy or Immunology Clinical Research Unit
🇺🇸
Tampa, Florida, United States
Institute for Asthma & Allergy, PC
🇺🇸
Chevy Chase, Maryland, United States
Winthrop-University Hospital, Clinical Trials Center
🇺🇸
Mineola, New York, United States
Baker Allergy, Asthma and Dermatology Research Center
🇺🇸
Lake Oswego, Oregon, United States
Icahn School of Medicine at Mount Sinai - The Mount Sinai Medical Center
🇺🇸
New York, New York, United States
AARA Research Center
🇺🇸
Dallas, Texas, United States
Ospedale L. Sacco
🇮🇹
Milano, MI, Italy
Jordan University Hospital
🇯🇴
Amman, Jordan
Washington University School of Medicine
🇺🇸
Saint Louis, Missouri, United States