Phase 2
N=4
Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)
Alpha1-Antitrypsin Deficiency
Bottom Line
View on ClinicalTrials.gov: NCT02870348 ↗Enrolled (actual)
4
Serious AEs
50.0%
Results posted
Mar 2022
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 4 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
- Feb 2021
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
4 | — |
| PRIMARY Number of Participants With Adverse Drug Reaction (ADRs) |
2 | — |
| PRIMARY Number of Participants With Serious Adverse Events (SAEs) |
2 | — |
| PRIMARY Number of Participants With Discontinuations Due to Adverse Events (AEs) or Serious Adverse Events (SAEs) |
— | — |
| PRIMARY Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations |
— | — |
| PRIMARY Number of Participants With Clinically Significant Findings in Vital Signs |
— | — |
| PRIMARY Number of Participants With Clinically Significant Findings in Clinical Laboratory Parameters |
— | — |
| PRIMARY Number of Participants With Clinically Significant Findings in Pulmonary Function Tests (PFTs) |
— | — |
| PRIMARY Trough Levels of Alpha1- Proteinase Inhibitor |
55.734 | — |
Summary
This is a multi-center, open-label study to evaluate the long-term safety of weekly intravenous (IV) infusions of 60 mg/kg alpha1-PI (human), modified process (Alpha-1 MP) in adult participants with Alpha1 Antitrypsin Deficiency (AATD) in Japan who have completed Study GTI1401 (NCT02870309).
Eligibility Criteria
Inclusion Criteria
- Subjects who complete participation in Study GTI1401 (i.e., have completed the study through the Week 9 Visit).
- Subjects who will and are able to provide written informed consent.
Exclusion Criteria
- Subjects with newly diagnosed severe concomitant disease including, but not limited to, congestive heart failure and liver cirrhosis.
- Subjects with newly diagnosed malignant tumor (including malignant melanoma; however, other forms of skin cancer are allowed).
- Female subjects who are pregnant, breastfeeding or, if of child-bearing potential, unwilling to practice a highly effective method of contraception (oral, injectable or implanted hormonal methods of contraception, placement of an intrauterine device (IUD) or intrauterine system (IUS), condom or occlusive cap with spermicidal foam/gel/film/cream/suppository, male sterilization, or abstinence) throughout the study or male subjects who have a partner who is of child-bearing potential and is unwilling to practice a highly effective method of contraception throughout the study.
- Subjects with clinical signs and symptoms of active hepatitis A virus (HAV), hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV) viral infection at the Week 9 Visit of Study GTI1401 and viral infection is further confirmed by testing.
- Subjects with current evidence of smoking or has a positive urine cotinine test at the Week 9 Visit in Study GTI1401 that is due to smoking.
- Subjects who currently participate in a study of another investigational product (other than Alpha-1 MP).
- Subjects who have difficulty in adhering to the protocol or its procedures, in the opinion of the investigator.
- Subjects who have medical conditions that may confound the results of this clinical trial or may endanger these subjects during their participation in this clinical trial in the opinion of the investigator.
Data sourced from ClinicalTrials.gov (NCT02870348). 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.