Aralast alpha1-proteinase Inhibitor Surveillance Study

NCT ID: NCT00313144

Last Updated: 2021-05-26

Basic Information

• Recruitment Status: TERMINATED
• Clinical Phase: PHASE4
• Total Enrollment: 127 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2006-06-09
• Study Completion Date: 2009-05-01

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.

Conditions

Alpha1-antitrypsin Deficiency

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Interventions

ARALAST Alpha1-Proteinase Inhibitor

Weekly ARALAST infusions for 2 years, dose and mode of administration as prescribed by the physician

Intervention Type: DRUG

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)

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Baxalta now part of Shire

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

Takeda

Locations

Adupa Rao, MD

San Marino, California, United States

Countries

United States

Other Identifiers

450501

Identifier Type: -

Identifier Source: org_study_id