Alpha 1-Antitrypsin Deficiency
Conditions
Keywords
alpha 1-Antitrypsin Deficiency, alpha 1-Antitrypsin, pulmonary emphysema
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.
Interventions
60 mg/kg weekly for 20 weeks
Sponsors
Grifols Therapeutics LLC
Study design
Allocation
NON_RANDOMIZED
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. | 24 weeks | 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. |
Countries
United Kingdom, United States
Participant flow
Recruitment details
First-subject-first-dose was 12 June 2006, last-subject-last-visit was 20 March 2007. The study was performed in 10 clinical sites, 8 sites in the United States and 2 sites in the United Kingdom.
Participants by arm
| Arm | Count |
|---|---|
| Entered Study | 38 |
| Total | 38 |
Withdrawals & dropouts
| Period | Reason | FG000 |
|---|---|---|
| Overall Study | Adverse Event | 1 |
Baseline characteristics
| Characteristic | Entered Study |
|---|---|
| Age, Categorical <=18 years | 0 Participants |
| Age, Categorical >=65 years | 6 Participants |
| Age, Categorical Between 18 and 65 years | 32 Participants |
| Age, Continuous | 53.4 years STANDARD_DEVIATION 8.63 |
| Region of Enrollment United Kingdom | 3 participants |
| Region of Enrollment United States | 35 participants |
| Sex: Female, Male Female | 15 Participants |
| Sex: Female, Male Male | 23 Participants |
Adverse events
| Event type | EG000 affected / at risk |
|---|---|
| deaths Total, all-cause mortality | — / — |
| other Total, other adverse events | 26 / 38 |
| serious Total, serious adverse events | 2 / 38 |
Outcome results
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.
Time frame: 24 weeks
Population: 38 subjects received study medication and one subject discontinued from the study due to an AE. Therefore, 37 subjects completed the study. 18 subjects were naive ((i.e., never having received previous Alpha-1 protease inhibitor augmentation therapy) and 19 subjects were non-naive.
| Arm | Measure | Group | Value (NUMBER) |
|---|---|---|---|
| Alpha-1 MP Treatment Group | Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. | Number of Participants Analyzed | 38 Participants |
| Alpha-1 MP Treatment Group | Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. | Subjects experiencing TEAEs | 26 Participants |
| Alpha-1 MP Treatment Group | Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. | Subjects with TEAEs <24 hrs. of A-1 MP infusion | 17 Participants |
| Alpha-1 MP Treatment Group | Treatment-emergent Adverse Events (TEAEs) Defined as Any Adverse Event (AE) Occurring During or After the Start of the First Study Drug Infusion. | Alpha-1 MP subjects with ≥1 drug-related TEAE | 5 Participants |