Aralast alpha1-proteinase Inhibitor Surveillance Study

Brief Title

Aralast alpha1-proteinase Inhibitor Surveillance Study

Official Title

ARALAST alpha1-proteinase Inhibitor (α1-PI) Surveillance Study

Brief Summary

      The primary objectives of this Phase 4, open label, prospective U.S. surveillance study are
      to evaluate the health outcomes of Alpha 1-Antitrypsin (AAT)-deficient subjects who are
      initiating treatment with ARALAST on patient-related outcomes (PRO), i.e., health-related
      quality of life (HRQoL), healthcare resource utilization (HCRU), and various laboratory
      analyses to evaluate the safety of long-term administration of ARALAST.

      Up to 120 subjects will be enrolled and assessed for HRQoL and HCRU at baseline and every
      6-months thereafter, for 2 years. A subset of subjects will be enrolled into the blood draw
      portion of the study, which will also include assessments of antibodies to ARALAST, and
      chemistry panel. Subjects will be treated according to the prescribing (attending)
      physician's instructions based on the prescribing information given in the ARALAST package
      insert.

Study Phase

Phase 4

Study Type

Interventional

Primary Outcome

HRQoL 'Physical Functioning (PF)' From Baseline to ≤6 Months

Secondary Outcome

 Healthcare Resource Utilization (HCRU) 'Frequency of Emergency Room (ER) Visits'

Condition

Alpha1-antitrypsin Deficiency

Intervention

ARALAST Alpha1-Proteinase Inhibitor

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information

Recruitment Status

Drug

Estimated Enrollment

Start Date

June 9, 2006

Completion Date

May 1, 2009

Primary Completion Date

December 1, 2008

Eligibility Criteria

        Inclusion Criteria:

          -  Male or female 18 years of age or older

          -  Diagnosis of AAT deficiency associated emphysema

          -  Active prescription for augmentation therapy with ARALAST

          -  On service with Coram (a speciality pharmacy provider)

          -  Signed and dated informed consent

        Exclusion Criteria:

          -  Clinically significant medical (other than COPD), psychiatric, or cognitive illness
             that, in the opinion of Coram or the sponsor or the investigator, may compromise
             subject safety or compliance (such as end stage renal or hepatic or heart disease, or
             metastatic cancer or any difficulty in communicating over the telephone lines)

          -  Previous treatment with ARALAST (i.e. subjects who had previously received and then
             discontinued ARALAST augmentation therapy and are now restarting ARALAST will be
             excluded from the study)

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Study Director, ,

Location Countries

United States

Location Countries

United States

Administrative Informations

NCT ID

NCT00313144

Organization ID

Responsible Party

Sponsor

Study Sponsor

Baxalta now part of Shire

Study Sponsor

Study Director, Study Director, Takeda

Verification Date

May 2021