Study Summary
This trial will test a new treatment for AATD that is given as a single infusion, then once a week for 8 weeks. The goal is to see if it is safe and tolerated.
- Alpha-1 Antitrypsin Deficiency
Treatment Effectiveness
Phase-Based Effectiveness
Study Objectives
11 Primary · 0 Secondary · Reporting Duration: Treatment Period 1- Single-Dose Week 1; Treatment Period 2- Repeat-Dose Weeks 1 and 9
Trial Safety
Phase-Based Safety
Awards & Highlights
Trial Design
6 Treatment Groups
Cohort 2: Treatment Period 2 (Alpha-1 15%, 144 mg/kg)
1 of 6
Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)
1 of 6
Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibito...
1 of 6
Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg)
1 of 6
Cohort 2: Treatment Period 1 (Alpha-1 15%, 144 mg/kg)
1 of 6
Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor...
1 of 6
Experimental Treatment
16 Total Participants · 6 Treatment Groups
Primary Treatment: Alpha-1 15% · No Placebo Group · Phase 1 & 2
Trial Logistics
Trial Timeline
Who is running the clinical trial?
Eligibility Criteria
Age 18 - 75 · All Participants · 0 Total Inclusion Criteria
Mark “Yes” if the following statements are true for you:Frequently Asked Questions
What is the geographic spread for this research endeavor?
"Currently, this research trial is recruiting at 6 different medical centres including the University of Florida in Gainesville, The University of Miami in Miami, and Medical Univeristy of South Carolina - Children's Hospital in Charleston." - Anonymous Online Contributor
How many participants are contributing to this experiment?
"To meet the set criteria, 16 volunteers are needed for this study. Potential participants can partake in this trial at University of Florida or University of Miami, both located within the state of Florida." - Anonymous Online Contributor
Are seniors over 70 eligible to participate in this clinical trial?
"The eligibility criteria for this research requires participants to be between 18 and 70 years old." - Anonymous Online Contributor
What prior research has been done regarding Alpha-1 15%?
"Alpha-1 15% was initially researched in 2013 at Clinic Dr.G Curteanu Oradea. 25 studies have been finished since then, with 5 of those actively enrolling new participants - primarily from sites located in Gainesville, Florida." - Anonymous Online Contributor
Are there any restrictions that prevent me from participating in this experiment?
"This clinical experiment is attempting to register 16 individuals with alpha1-antitrypsin deficiency aged between 18 and 70. Candidates should possess a ZZ, SZ, Z(null), (null)(null), S(null) or "at-risk" alleles combination of AATD as well as be off any kind of Alpha1-PI treatment other than the IPs for this study at baseline. Furthermore, their post bronchodilator FEV1/FVC ratio must be lower than 70% while also being higher than 30%." - Anonymous Online Contributor
Is there currently an enrollment window open for this clinical investigation?
"Affirmative, according to clinicaltrials.gov this investigation is actively recruiting partakers. This trial was first advertised on August 13th 2021 and most recently revised on August 15th 2022; it seeks 16 participants across 6 locations." - Anonymous Online Contributor
Has there ever been a similar trial of this magnitude conducted before?
"Alpha-1 15% has been subjected to clinical research since 2013, beginning with a Grifols Therapeutics LLC sponsored trial of 339 participants. After achieving Phase 3 drug approval in the wake of that first study and 5 live trials across 34 cities and 17 countries are now being conducted." - Anonymous Online Contributor