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Phase 1 N=25 Randomized Double-blind Treatment

Pharmacokinetic Study of ARALAST (Human Alpha1- PI)

Alpha 1-Antitrypsin Deficiency

Enrolled (actual)
25
Serious AEs
0.0%
Results posted
Jul 2011
Primary outcome: Primary: Area Under the Curve/Dose — 0.0822; 0.0920 days*kg/mL

Study Design & Population

Study type
Interventional
Phase
Phase 1
Interventions
Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor (Biological); Dose of 60 mg/kg alpha1-proteinase inhibitor (Biological)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
Baxalta now part of Shire
Primary completion
Jun 2006

Outcome Measures

OutcomeResultp-value
PRIMARY
Area Under the Curve/Dose
0.0822; 0.0920
SECONDARY
Total Area Under the Curve Per Dose
0.0825; 0.0928
SECONDARY
Systemic Clearance (CL)
951; 856
SECONDARY
Mean Residence Time (MRT)
6.8; 6.9
SECONDARY
Apparent Volume of Distribution at Steady State
5606; 5405
SECONDARY
Terminal Half-life
4.1; 4.2
SECONDARY
Maximum Plasma Concentration (Cmax)
1.7; 1.7
SECONDARY
Time to Maximum α1-PI Concentration Post-infusion (Tmax)
0.00; 0.00
SECONDARY
Incremental Recovery
0.0259; 0.0258
SECONDARY
Adverse Events (AEs)
0; 0; 43; 45; 16; 12

Summary

The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor (α1-PI). This pharmacokinetic study will be a randomized controlled clinical trial with a cross-over design. Twenty-four subjects will be enrolled into the study. Overall study duration will be approximately 6-8 months.

Eligibility Criteria

Inclusion Criteria

  • The subject or subject´s legally authorized representative has provided written informed consent
  • Subject is 18 years of age or older
  • Subject has a documented, endogenous plasma Alpha1-PI level = 1500 cells/mm3
  • Hemoglobin >= 10.0 g/dL
  • Platelet count >= 10^5/mm3
  • If the subject is treated with any respiratory medications, including inhaled bronchodilators and inhaled or oral corticosteroids, the subjects´ medication doses were unchanged for at least 14 days prior to first study product administration
  • Nonsmoker for a minimum of 3 months prior to first study product administration

Exclusion Criteria

  • The subject has received any Alpha1-PI augmentation therapy (including Aralast and investigational Alpha1-PIs, by any route including intravenous and inhaled) within 42 days prior to first study product administration
  • The subject has received an investigational drug or device within 1 month prior to first study product administration, or the subject is currently receiving an investigational drug
  • The subject has a known selective immunoglobulin A (IgA) deficiency (IgA level < 15 mg/dL) and/or antibody to IgA
  • The subject has a pulmonary exacerbation or had a pulmonary exacerbation in the past 14 days prior to first study product administration
  • The subject is pregnant or lactating, or intends to become pregnant during the course of the study
  • The subject has a clinically significant medical, psychiatric, or cognitive illness, or recreational drug/alcohol use that, in the opinion of the investigator, would affect subject safety or compliance
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT00242385). 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.

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