Phase 2
N=30
Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency
Emphysema · Alpha 1-antitrypsin Deficiency (AATD)
Bottom Line
View on ClinicalTrials.gov: NCT01213043 ↗Enrolled (actual)
30
Serious AEs
0.0%
Results posted
May 2013
Primary outcome: Primary: Subjects With Treatment-Emergent Adverse Events (TEAEs) — 23; 18 participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Prolastin-C, 60 mg/kg (Biological); Prolastin-C, 120 mg/kg (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Grifols Therapeutics LLC
- Primary completion
- Jan 2012
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Subjects With Treatment-Emergent Adverse Events (TEAEs) |
23; 18 | — |
| PRIMARY Subjects With Drug-Related TEAE(s) |
3; 1 | — |
| PRIMARY Subjects With Treatment-Emergent Serious Adverse Events (SAEs) |
0; 0 | — |
| PRIMARY Subjects Withdrawn Due to an AE(s) |
0; 0 | — |
| PRIMARY Subjects With Treatment-Emergent Pulmonary Exacerbation(s) |
7; 5 | — |
| PRIMARY Subjects With Severe TEAE(s) or Pulmonary Exacerbation(s) |
0; 0 | — |
| PRIMARY Number of TEAEs |
69; 43 | — |
| PRIMARY Number of Drug-related TEAEs |
5; 1 | — |
| PRIMARY Number of Treatment-Emergent Pulmonary Exacerbations |
9; 6 | — |
| SECONDARY AUC0-7days |
203.6; 344.8 | <0.0001 sig |
| SECONDARY Mean Trough |
17.3; 27.7 | — |
Summary
This is a study to assess the safety and pharmacokinetics of weekly infusions of 120 mg/kg of Prolastin-C (alpha1-proteinase inhibitor [alpha1-PI] [Human]), compared to weekly infusions of 60 mg/kg of Prolastin-C in patients with alpha 1-antitrypsin deficiency (AATD).
Eligibility Criteria
Inclusion Criteria
- Be between 18 and 70 years of age
- Have a documented diagnosis of congenital AATD
- Have a post-bronchodilator Forced Expired Volume in 1 second (FEV1) of ≥30% and <80% and FEV1/forced vital capacity (FVC) <70%
- If receiving alpha-1 PI augmentation therapy, be willing to discontinue the treatment for the duration of the study
Exclusion Criteria
- Had a moderate or severe pulmonary exacerbation during the 4 weeks before the study
- History of lung or liver transplant
- Any lung surgery during the past 2 years
- Confirmed liver cirrhosis
- Elevated liver enzymes
- Severe concurrent disease
- Females who are pregnant or breast-feeding or unwilling to practice effective contraception during the study
- Infection with hepatitis A, B, or C, human immunodeficiency or parvovirus B19
- Smoking during the past 6 months
- Use of systemic steroids within 4 weeks of the study
- Use of antibiotics for an exacerbation within 4 weeks of the study
Data sourced from ClinicalTrials.gov (NCT01213043). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.