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

Shire

Status and phase: Completed; Phase 1

Conditions: Hereditary Angioedema (HAE)

Treatments: Drug: DX-2930; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT02093923

DX-2930-02

Details and patient eligibility

About

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.

Enrollment: 38 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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38 participants in 5 patient groups, including a placebo group

DX-2930, Cohort 1

Experimental group

Description:

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.

Treatment:

Drug: DX-2930

DX-2930, Cohort 2

Experimental group

Description:

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.

Treatment:

Drug: DX-2930

DX-2930, Cohort 3

Experimental group

Description:

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.

Treatment:

Drug: DX-2930

DX-2930, Cohort 4

Experimental group

Description:

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.

Treatment:

Drug: DX-2930

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Drug: Placebo