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

Grifols

Status and phase: Completed; Phase 3

Conditions: Alpha 1-Antitrypsin Deficiency

Treatments: Drug: alpha-1 proteinase inhibitor (human)

Study type: Interventional

Funder types: Industry

Identifiers

NCT00301366

11815

Details and patient eligibility

About

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

Full 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.

Enrollment: 38 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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38 participants in 1 patient group

Alpha-1 Proteinase Inhibitor (Human), modified process

Experimental group

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.

Treatment:

Drug: alpha-1 proteinase inhibitor (human)