Clinical trial NCT02663687: Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
Official title: A Randomized, Double-blind, Placebo-controlled, Ascending Dose, Phase 1 Study to Assess the Safety, Tolerability, and Pharmacokinetics of Single Intravenous and Subcutaneous Doses of Recombinant Human C1 Esterase Inhibitor in Healthy Adult Subjects
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
Conditions: Hereditary Angioedema (HAE)
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2016-01-26
Enrollment: 48
Sex: ALL
Minimum age (years): 18
Maximum age (years): 50
Standard ages: ADULT
Accepts healthy volunteers: yes
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: 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).


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Participants With Treatment-emergent Adverse Events (TEAEs ) Including Serious Adverse Events (SAEs)
timeFrame: From the start of study treatment up to 28 days after the last dose of the study treatment (up to 56 days)
Secondary outcomes:
measure: Maximum Observed Plasma Concentration (Cmax) of SHP623 Occurring at Time of Maximum Observed Concentration During a Dosing Interval (Tmax)
timeFrame: 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.
measure: Time of Maximum Plasma Concentration (Tmax) of SHP623 Sampled During a Dosing Interval
timeFrame: 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.
measure: Terminal Half-life (t1/2) of SHP623
timeFrame: 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.
measure: Area Under the Plasma Concentration Curve From Time Zero to Infinity (AUC 0-inf) of SHP623
timeFrame: 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.
measure: Area Under the Plasma Concentration Curve From Time Zero to 168 Hours Postdose (AUC 0-168) of SHP623
timeFrame: 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.
measure: Area Under the Curve From the Time of Dosing to the Last Measurable Concentration (AUClast) of SHP623
timeFrame: 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.
measure: Total Body Clearance (CL) for Intravascular (IV) Administration of SHP623
timeFrame: 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.
measure: Volume of Distribution Associated With the Terminal Slope (Vz) Following Intravenous (IV) Administration of SHP623
timeFrame: 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.
measure: Total Body Clearance for Extravascular Administration (CL/F) of SHP623 for Subcutaneous (SC) Administration
timeFrame: 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.
measure: Volume of Distribution Influenced by Fraction of Dose Absorbed (Vz/F) Following Extravascular Administration of SHP623
timeFrame: 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.
Other outcomes:
Locations:
city: Miami
country: United States
facility: Clinical Pharmacology of Miami
state: Florida
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: