Phase 2
N=4
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency
Alpha1-Antitrypsin Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02870309 ↗Enrolled (actual)
4
Serious AEs
0.0%
Results posted
Feb 2021
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Alpha-1 MP (Biological)
- Age
- Adult, Older Adult · 20+ yrs
- Sex
- All
- Sponsor
- Grifols Therapeutics LLC
- Primary completion
- Mar 2017
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
3 | — |
| PRIMARY Number of Participants With Adverse Drug Reaction (ADRs) |
1 | — |
| PRIMARY Number of Participants With Serious Adverse Events (SAEs) |
— | — |
| PRIMARY Number of Discontinuations Due to Adverse Events (AEs) or Serious Adverse Events (SAEs) |
— | — |
| PRIMARY Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations |
— | — |
| SECONDARY Trough Level of Total alpha1-PI for Weekly IV Infusions of 60 mg/kg Alpha-1 MP |
11.450; 55.567; 55.425; 56.900 | — |
Summary
This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of Alpha-1 MP (alpha1-proteinase inhibitor (human), modified process) for 8 weeks.
Eligibility Criteria
Inclusion Criteria
- Participants aged ≥20 years at the time of providing informed consent.
- Participants with clinically apparent pulmonary emphysema diagnosed by Computed Tomography (CT) scan.
- AATD participants with documented serum alpha1-PI levels of 5 mg/day within 4 weeks before the Week 1 (baseline) visit (Note: inhaled steroids are not regarded as systemic steroids).
- Participants who have used any systemic or aerosolized antibiotic drug for the treatment of COPD exacerbation within 4 weeks before the Week 1(baseline) visit.
- Participants with a previous or current diagnosis of selective, severe Immunoglobulin A (IgA) deficiency.
- Participants who are mentally challenged and cannot independently give consent.
- Participants who have difficulty in adhering to the protocol or its procedures in the opinion of the investigator.
- Participants who have medical conditions that may confound the results of this clinical trial or may endanger other participants during the participation in this clinical trial in the opinion of the investigator.
Data sourced from ClinicalTrials.gov (NCT02870309). 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.