NCT00242385

Brief 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.

Trial Health

90
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
25

participants targeted

Target at P25-P50 for phase_1

Timeline
Completed

Started Dec 2005

Shorter than P25 for phase_1

Geographic Reach
2 countries

7 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

October 19, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

October 20, 2005

Completed
2 months until next milestone

Study Start

First participant enrolled

December 20, 2005

Completed
6 months until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 5, 2006

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

June 5, 2006

Completed
5.1 years until next milestone

Results Posted

Study results publicly available

July 20, 2011

Completed
Last Updated

May 13, 2021

Status Verified

April 1, 2021

Enrollment Period

6 months

First QC Date

October 19, 2005

Results QC Date

February 15, 2011

Last Update Submit

April 17, 2021

Conditions

Keywords

Severe congenital Alpha1-Proteinase Inhibitor (Alpha1-PI) deficiency

Outcome Measures

Primary Outcomes (1)

  • Area Under the Curve/Dose

    Area under the plasma alpha1-proteinase inhibitor (α1-PI) concentration versus time curve (AUC) calculated by linear trapezoidal method per dose.

    Pharmacokinetic evaluation: 30 minutes pre-infusion up to 35 days post-infusion

Secondary Outcomes (9)

  • Total Area Under the Curve Per Dose

    Pharmacokinetic evaluation: 30 minutes pre-infusion up to 35 days post-infusion

  • Systemic Clearance (CL)

    Pharmacokinetic evaluation: 30 minutes pre-infusion up to 35 days post-infusion

  • Mean Residence Time (MRT)

    Pharmacokinetic evaluation: 30 minutes pre-infusion up to 35 days post-infusion

  • Apparent Volume of Distribution at Steady State

    Pharmacokinetic evaluation: 30 minutes pre-infusion up to 35 days post-infusion

  • Terminal Half-life

    Pharmacokinetic evaluation: 30 minutes pre-infusion up to 35 days post-infusion

  • +4 more secondary outcomes

Study Arms (2)

ARALAST Fr. IV-1

EXPERIMENTAL

60 mg/kg

Biological: Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor

ARALAST

ACTIVE COMPARATOR

60mg/kg

Biological: Dose of 60 mg/kg alpha1-proteinase inhibitor

Interventions

Subjects meeting the eligibility criteria were randomized to receive either single dose ARALAST alpha1-proteinase inhibitor 60 mg/kg or single-dose ARALAST alpha1-proteinase inhibitor Fr. IV-1 60 mg/kg at 0.2 mL/kg/min during the first treatment period with crossover to the alternate study product during the second treatment period, with a minimum of 7 days between the two treatment periods.

ARALAST Fr. IV-1

Subjects meeting the eligibility criteria were randomized to receive either single dose ARALAST alpha1-proteinase inhibitor 60 mg/kg or single-dose ARALAST alpha1-proteinase inhibitor Fr. IV-1 60 mg/kg at 0.2 mL/kg/min during the first treatment period with crossover to the alternate study product during the second treatment period, with a minimum of 7 days between the two treatment periods.

ARALAST

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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 \< 8 Micromolar
  • Subject is of the genotype Pi\*Z/Z, Pi\*Z/Null, Pi\*Null/Null, Pi\*Malton/Z, or others, dependent on the approval by the Sponsor
  • If the subject is female or of childbearing potential, the subject has a negative urine test for pregnancy within 7 days prior to first study product administration and agrees to employ adequate birth control measures for the duration of the study
  • Laboratory results obtained at the screening visit, meeting the following criteria:
  • Serum alanine aminotransferase (ALT), aspartate aminotransferase (AST) \<= 2 times the upper limit of normal (ULN)
  • Serum total bilirubin \<= 2 times ULN
  • Proteinuria \< +2 on dipstick analysis
  • Serum creatinine \<= 1.5 times ULN
  • Absolute neutrophil count (ANC) \>= 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (7)

Unknown Facility

Adelaide, South Australia, Australia

Location

Unknown Facility

Woodville, South Australia, Australia

Location

Unknown Facility

Fitzroy, Victoria, Australia

Location

Unknown Facility

Nedlands, Western Australia, Australia

Location

Unknown Facility

Otahuhu, Auckland, New Zealand

Location

Unknown Facility

Christchurch, New Zealand

Location

Unknown Facility

Hamilton, New Zealand

Location

Related Publications (1)

  • Li Z, Franke RM, Morris DN, Yel L. Pharmacokinetics and Biochemical Efficacy of an alpha1-Proteinase Inhibitor (Aralast NP) in alpha1-Antitrypsin Deficiency: a Cross-Product Retrospective Comparability Analysis. Pulm Ther. 2022 Sep;8(3):311-326. doi: 10.1007/s41030-022-00199-4. Epub 2022 Aug 24.

MeSH Terms

Conditions

alpha 1-Antitrypsin Deficiency

Interventions

alpha 1-Antitrypsin

Condition Hierarchy (Ancestors)

Liver DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesSubcutaneous EmphysemaEmphysemaPathologic ProcessesPathological Conditions, Signs and Symptoms

Intervention Hierarchy (Ancestors)

GlycoproteinsGlycoconjugatesCarbohydratesSerpinsPeptidesAmino Acids, Peptides, and ProteinsAcute-Phase ProteinsBlood ProteinsProteinsAlpha-GlobulinsSerum GlobulinsGlobulins

Results Point of Contact

Title
Study Director
Organization
Shire

Study Officials

  • Study Director

    Takeda

    STUDY DIRECTOR

Publication Agreements

PI is Sponsor Employee
No
Restriction Type
OTHER
Restrictive Agreement
Yes

Study Design

Study Type
interventional
Phase
phase 1
Allocation
RANDOMIZED
Masking
DOUBLE
Purpose
TREATMENT
Intervention Model
CROSSOVER
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

October 19, 2005

First Posted

October 20, 2005

Study Start

December 20, 2005

Primary Completion

June 5, 2006

Study Completion

June 5, 2006

Last Updated

May 13, 2021

Results First Posted

July 20, 2011

Record last verified: 2021-04

Locations