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

NCT ID: NCT00301366

Last Updated: 2014-08-19

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE3

Total Enrollment

38 participants

Study Classification

INTERVENTIONAL

Study Start Date

2006-06-30

Study Completion Date

2007-03-31

Brief Summary

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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

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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.

Conditions

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Alpha 1-Antitrypsin Deficiency

Study Design

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Allocation Method

NON_RANDOMIZED

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Alpha-1 Proteinase Inhibitor (Human), modified process

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.

Group Type EXPERIMENTAL

alpha-1 proteinase inhibitor (human)

Intervention Type DRUG

60 mg/kg weekly for 20 weeks

Interventions

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alpha-1 proteinase inhibitor (human)

60 mg/kg weekly for 20 weeks

Intervention Type DRUG

Other Intervention Names

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Prolastin Alpha-1 antitrypsin (AAT) BAY x 5747 BAY 10-5233 TAL-05-00007

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Grifols Therapeutics LLC

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Kim Hanna, MSc

Role: STUDY_DIRECTOR

Grifols Therapeutics LLC

Locations

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National Jewish Medical and Research Center

Denver, Colorado, United States

Site Status

University of Florida College of Medicine

Gainesville, Florida, United States

Site Status

University of Miami School of Medicine

Miami, Florida, United States

Site Status

St Lukes-Roosevelt Hospital Center, New York

New York, New York, United States

Site Status

Cleveland Clinic Foundation

Cleveland, Ohio, United States

Site Status

Temple University Hospital

Philadelphia, Pennsylvania, United States

Site Status

Medical University of South Carolina

Charleston, South Carolina, United States

Site Status

University of Texas Health Center at Tyler

Tyler, Texas, United States

Site Status

University of Cambridge - Cambridge Institute for Medical Research

Cambridge, England, United Kingdom

Site Status

University Teaching Hospital of Edinburgh

Edinburgh, Scotland, United Kingdom

Site Status

Countries

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United States United Kingdom

References

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Wewers MD, Casolaro MA, Sellers SE, Swayze SC, McPhaul KM, Wittes JT, Crystal RG. Replacement therapy for alpha 1-antitrypsin deficiency associated with emphysema. N Engl J Med. 1987 Apr 23;316(17):1055-62. doi: 10.1056/NEJM198704233161704.

Reference Type BACKGROUND
PMID: 3494198 (View on PubMed)

Gadek JE, Klein HG, Holland PV, Crystal RG. Replacement therapy of alpha 1-antitrypsin deficiency. Reversal of protease-antiprotease imbalance within the alveolar structures of PiZ subjects. J Clin Invest. 1981 Nov;68(5):1158-65. doi: 10.1172/jci110360.

Reference Type BACKGROUND
PMID: 7028785 (View on PubMed)

Gadek JE, Fells GA, Zimmerman RL, Rennard SI, Crystal RG. Antielastases of the human alveolar structures. Implications for the protease-antiprotease theory of emphysema. J Clin Invest. 1981 Oct;68(4):889-98. doi: 10.1172/jci110344.

Reference Type BACKGROUND
PMID: 6169740 (View on PubMed)

Related Links

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http://www.alphaone.org

The "Alpha-1 Foundation", dedicated to providing the leadership and resources that will result in increased research, improved health, worldwide detection and a cure for Alpha-1 Antitrypsin Deficiency

http://www.alphanet.org

AlphaNet, Inc devoted to improving the lives of individuals with Alpha-1 antitrypsin deficiency through comprehensive disease management services, clinical research administration, and consultative services

Other Identifiers

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11815

Identifier Type: -

Identifier Source: org_study_id

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