Clinical trial NCT00301366: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Alpha 1-Antitrypsin Deficiency
Keywords: alpha 1-Antitrypsin Deficiency
alpha 1-Antitrypsin
pulmonary emphysema
Lead sponsor: Grifols Therapeutics LLC
Sponsor class: INDUSTRY
First posted: 2006-03-10
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion.
timeFrame: 24 weeks
Secondary outcomes:
Other outcomes:
Locations:
city: Denver
country: United States
facility: National Jewish Medical and Research Center
state: Colorado
city: Gainesville
country: United States
facility: University of Florida College of Medicine
state: Florida
city: Miami
country: United States
facility: University of Miami School of Medicine
state: Florida
city: New York
country: United States
facility: St Lukes-Roosevelt Hospital Center, New York
state: New York
city: Cleveland
country: United States
facility: Cleveland Clinic Foundation
state: Ohio
city: Philadelphia
country: United States
facility: Temple University Hospital
state: Pennsylvania
city: Charleston
country: United States
facility: Medical University of South Carolina
state: South Carolina
city: Tyler
country: United States
facility: University of Texas Health Center at Tyler
state: Texas
city: Cambridge
country: United Kingdom
facility: University of Cambridge - Cambridge Institute for Medical Research
state: England
city: Edinburgh
country: United Kingdom
facility: University Teaching Hospital of Edinburgh
state: Scotland
Study officials:
affiliation: Grifols Therapeutics LLC
name: Kim Hanna, MSc
role: STUDY_DIRECTOR
References:
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. doi: 10.1056/NEJM198704233161704.
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. doi: 10.1172/jci110360.
citation: 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.