The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

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Alpha-1 antitrypsin deficiency
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Brief Title

The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency

Official Title

Multi-center, Open-label Trial to Evaluate the Safety and Tolerability of Alpha-1 MP in Subjects With Alpha-1-antitrypsin (AAT) Deficiency

Brief Summary

      The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in
      adult Alpha1-antitrypsin deficient patients.

Detailed Description

      The objective of this clinical trial (STAMP: Safety and Tolerability of Alpha-1 Modified
      Process) is to study the safety and tolerability of Alpha-1 MP in adult Alpha 1-antitrypsin
      deficient subjects as reported over 20 weeks of therapy. The primary objective is to describe
      the nature and frequency of treatment-emergent adverse events with "treatment-emergent"
      defined as any adverse event occurring after the start of the first study drug infusion.

Study Phase

Phase 3

Study Type

Interventional

Primary Outcome

Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.

Condition

Alpha 1-Antitrypsin Deficiency

Intervention

alpha-1 proteinase inhibitor (human)

Study Arms / Comparison Groups

 Alpha-1 Proteinase Inhibitor (Human), modified process
Description:  Study the safety and tolerability of weekly infusions of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1 MP, 60 mg/kg) over 20 weeks of therapy in adult Alpha-1 antitrypsin deficient subjects.

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

38

Start Date

June 2006

Completion Date

March 2007

Primary Completion Date

March 2007

Eligibility Criteria

        Inclusion Criteria:

          -  Documented diagnosis of congenital Alpha1-antitrypsin deficiency

          -  Documented forced expiratory volume in 1 second (FEV1 ) between 20% - 80% of predicted
             value within last 6 months.

          -  Signed written informed consent prior to initiation of any study related procedures.

        Exclusion Criteria:

          -  Females who are pregnant, breast feeding, or if of child-bearing potential, unwilling
             to practice adequate contraception throughout the study

          -  Use of systemic steroids within the 2 weeks prior to receiving study treatment (this
             does not include the use of inhaled steroids used on a routine or as needed basis).

          -  Subjects who have had exacerbations of their disease within one month of trial entry

Gender

All

Ages

18 Years - N/A

Accepts Healthy Volunteers

No

Contacts

Kim Hanna, MSc, ,

Location Countries

United Kingdom

Location Countries

United Kingdom

Administrative Informations

NCT ID

NCT00301366

Organization ID

11815

Responsible Party

Sponsor

Study Sponsor

Grifols Therapeutics LLC

Study Sponsor

Kim Hanna, MSc, Study Director, Grifols Therapeutics LLC

Verification Date

July 2014