Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects

Shire48 enrolled

Overview

This trial is looking to gain information about the safety and tolerability of an investigational treatment (SHP623) in healthy adult volunteers. This study will also collect pharmacokinetic data (how the body absorbs and breaks down the study drug).

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

DOUBLE

Enrollment

Conditions

Hereditary Angioedema (HAE)

Interventions

Recombinant human C1 esterase inhibitorDRUG

Subjects will receive escalating doses I-IV as both IV and SC injections

PlaceboDRUG

Subjects will receive matching placebo

SHP623DRUG

SHP623

Eligibility

Sex: ALLMin age: 18 YearsMax age: 50 YearsHealthy volunteers:

Medical Language ↔ Plain English

Inclusion Criteria: 1. Must be considered healthy. Healthy status is defined by absence of evidence of any active or chronic disease 2. Male, or non-pregnant, non-lactating female, who agrees to comply with any applicable contraceptive requirements of the protocol, or females of non-child-bearing potential. 3. Body mass index between 18.0 and 30.0 kg/m2 inclusive with a body weight \>50 kg (110 lbs.). This inclusion criterion will be assessed only at the first screening visit. 4. Hemoglobin ≥12.0g/ld. Exclusion Criteria: 1. Have a history of allergic reaction to C1 INH products (e.g. C1 Inhibitor \[Human\], Berinert \[C1 Estrace Inhibitor (Human)\] and C1 estrace \[recombinant\] 2. Known history of alcohol or other substance abuse within the last year. 3. Donation of blood or blood products within 60 days prior to receiving investigational product. 4. Current use of any medication except hormonal replacement therapy, hormonal contraceptives and occasional use of any over-the-counter non-steroidal anti-inflammatory drug (NSAID) or acetaminophen. 5. Have a history of hypercoagulability or other predisposition to thrombotic events.

Locations (1)

Clinical Pharmacology of Miami

Miami, Florida, United States

Outcomes

Primary Outcomes

Number of Participants With Treatment-emergent Adverse Events (TEAEs ) Including Serious Adverse Events (SAEs)

An adverse event (AE) was defined as any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that did not necessarily have a causal relationship with this treatment. An AE was considered to be a TEAE in a specific treatment period of the study if the date and time of onset were after investigational product administration in that period and if it occurred less than equals to (\<=) Day 28 and was both not present at the start of that period and was not a chronic condition that was part of the participant's medical history, or it was present at the start of that period or as part of the participant's medical history but the severity or frequency increased during that period \<= Day 28. An SAE was defined as any untoward medical occurrence (whether considered to be related to investigational product or not) that at any dose.

Time frame: From the start of study treatment up to 28 days after the last dose of the study treatment (up to 56 days)

Secondary Outcomes

Maximum Observed Plasma Concentration (Cmax) of SHP623 Occurring at Time of Maximum Observed Concentration During a Dosing Interval (Tmax)

Cmax of SHP623 recombinant human C1 esterase inhibitor (rC1 INH) antigen at Tmax was calculated based on observed concentration-versus-time data. Cmax at Tmax of SHP623 for both treatment period 1 (IV) and treatment period 2 (SC) was presented in the categories for each dosing group.

Time frame: Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

Time of Maximum Plasma Concentration (Tmax) of SHP623 Sampled During a Dosing Interval

Tmax of SHP623 (rC1 INH) antigen was calculated based on observed concentration-versus-time data. Tmax of SHP623 for both treatment period 1 (IV) and treatment period 2 (SC) was presented in the categories for each dosing group.

Time frame: Pre-dose, 0, 0.25, 0.5, 1, 2, 3, 4, 6, 8, 12, 24, 36, 48, 72, 96, 120, 144, 168, 216, 312, 648 hours post-dose.

Data from ClinicalTrials.gov

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