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

Alpha 1-Antitrypsin Deficiency Clinical Trial

— STAMP  

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00301366
• Other study ID #: 11815
• Status: Completed
• Phase: Phase 3
• First received: March 8, 2006
• Last updated: July 30, 2014
• Start date: June 2006
• Est. completion date: March 2007

Study information

• Verified date: July 2014
• Source: Grifols Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationNetherlands: Medicines Evaluation Board (MEB)United Kingdom: Medicines and Healthcare Products Regulatory Agency
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: March 2007
• Est. primary completion date: March 2007
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Allocation: Non-Randomized, Endpoint Classification: Safety Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Alpha 1-Antitrypsin Deficiency

Intervention

Drug:
• alpha-1 proteinase inhibitor (human)
60 mg/kg weekly for 20 weeks

Locations

Country	Name	City	State
United Kingdom	University of Cambridge - Cambridge Institute for Medical Research	Cambridge	England
United Kingdom	University Teaching Hospital of Edinburgh	Edinburgh	Scotland
United States	Medical University of South Carolina	Charleston	South Carolina
United States	Cleveland Clinic Foundation	Cleveland	Ohio
United States	National Jewish Medical and Research Center	Denver	Colorado
United States	University of Florida College of Medicine	Gainesville	Florida
United States	University of Miami School of Medicine	Miami	Florida
United States	St Lukes-Roosevelt Hospital Center, New York	New York	New York
United States	Temple University Hospital	Philadelphia	Pennsylvania
United States	University of Texas Health Center at Tyler	Tyler	Texas

Sponsors (1)

Lead Sponsor	Collaborator
Grifols Therapeutics Inc.

Countries where clinical trial is conducted

United States,  United Kingdom, 

References & Publications (3)

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. — View Citation

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. — View Citation

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.	An adverse event is any untoward medical occurrence in a subject or clinical investigation subject administered with a pharmaceutical product. The adverse event does not necessarily have to have a causal relationship with this treatment. An adverse event can therefore be any unfavorable and unintended sign (including an abnormal laboratory finding), symptom, or disease temporally associated with the use of an investigational product, whether or not considered related to the medicinal product.	24 weeks	Yes

External Links

•   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
•   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