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

Grifols Therapeutics LLC38 enrolled

Overview

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

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 Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Alpha 1-Antitrypsin Deficiency

Interventions

alpha-1 proteinase inhibitor (human)DRUG

60 mg/kg weekly for 20 weeks

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (10)

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

Outcomes

Primary Outcomes

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.

Time frame: 24 weeks

Data from ClinicalTrials.gov

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