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Phase 1 Completed N=48 Randomized Double-blind Treatment

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

Hereditary Angioedema (HAE)
Source: ClinicalTrials.gov NCT02663687 ↗
Enrolled (actual)
48
Serious AEs
0.0%
Results posted
Sep 2018
Primary outcomePrimary: Number of Participants With Treatment-emergent Adverse Events (TEAEs ) Including Serious Adverse Events (SAEs) — 8; 4; 3; 8 Participants

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).

Outcome Measures

OutcomeResultp-value
PRIMARY
Number of Participants With Treatment-emergent Adverse Events (TEAEs ) Including Serious Adverse Events (SAEs)
8; 4; 3; 8; 4; 0
SECONDARY
Maximum Observed Plasma Concentration (Cmax) of SHP623 Occurring at Time of Maximum Observed Concentration During a Dosing Interval (Tmax)
47.911; 130.367; 160.556; 307.000; 4.248; 15.694
SECONDARY
Time of Maximum Plasma Concentration (Tmax) of SHP623 Sampled During a Dosing Interval
0.250; 0.250; 0.250; 0.500; 72.000; 36.000
SECONDARY
Terminal Half-life (t1/2) of SHP623
28.8; 40.2; 38.2; 38.9; 51.8; 49.8
SECONDARY
Area Under the Plasma Concentration Curve From Time Zero to Infinity (AUC 0-inf) of SHP623
1114.535; 2941.364; 4278.273; 8282.120; 594.741; 1653.875
SECONDARY
Area Under the Plasma Concentration Curve From Time Zero to 168 Hours Postdose (AUC 0-168) of SHP623
1069.451; 2823.169; 4083.493; 7893.069; 407.936; 1414.762
SECONDARY
Area Under the Curve From the Time of Dosing to the Last Measurable Concentration (AUClast) of SHP623
1054.798; 2868.736; 4174.135; 8168.337; 401.030; 1518.124
SECONDARY
Total Body Clearance (CL) for Intravascular (IV) Administration of SHP623
0.961; 0.688; 0.718; 0.614
SECONDARY
Volume of Distribution Associated With the Terminal Slope (Vz) Following Intravenous (IV) Administration of SHP623
42.600; 37.284; 40.794; 35.684
SECONDARY
Total Body Clearance for Extravascular Administration (CL/F) of SHP623 for Subcutaneous (SC) Administration
1.723; 1.250; 1.399; 1.254
SECONDARY
Volume of Distribution Influenced by Fraction of Dose Absorbed (Vz/F) Following Extravascular Administration of SHP623
133.585; 94.861; 121.511; 94.463

Eligibility Criteria

Inclusion Criteria

  • Must be considered healthy. Healthy status is defined by absence of evidence of any active or chronic disease
  • 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.
  • 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.
  • Hemoglobin ≥12.0g/ld.

Exclusion Criteria

  • Have a history of allergic reaction to C1 INH products (e.g. C1 Inhibitor [Human], Berinert [C1 Estrace Inhibitor (Human)] and C1 estrace [recombinant]
  • Known history of alcohol or other substance abuse within the last year.
  • Donation of blood or blood products within 60 days prior to receiving investigational product.
  • 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.
  • Have a history of hypercoagulability or other predisposition to thrombotic events.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02663687). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.

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