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Proteinase Inhibitor
Alpha1-Proteinase Inhibitor for Alpha-1 Antitrypsin Deficiency
Phase 1 & 2
Recruiting
Research Sponsored by Grifols Therapeutics LLC
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial Must have
Have a diagnosis of congenital Alpha1-antitrypsin deficiency (AATD) with specific allelic combinations
Subjects may be naïve to Alpha1-PI augmentation therapy or may be currently receiving it, with willingness to discontinue treatment for at least 25 days prior to the Week 1 (Baseline) Visit
Timeline
Screening 3 weeks
Treatment Varies
Follow Up treatment period 1- single-dose week 1; treatment period 2- repeat-dose weeks 1 and 9
Awards & highlights
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.
Who is the study for?
This trial is for individuals with Alpha1-Antitrypsin Deficiency (AATD) who have low serum AAT levels and specific lung function measurements. Participants can be new or current on Alpha1-PI therapy but must stop it before the study starts. Pregnant women, those not using effective contraception, people with severe allergies to plasma-derived treatments, recent lung surgery patients, or those with certain health conditions are excluded.Check my eligibility
What is being tested?
The study tests two doses of a medication called Alpha-1 15%, given as a subcutaneous infusion in participants with AATD. The first dose is administered once and then weekly over eight weeks to evaluate safety and how the body processes the drug.See study design
What are the potential side effects?
While specific side effects aren't listed here, common ones for similar treatments may include injection site reactions, possible allergic responses especially in those sensitive to plasma products, headaches, fatigue, and flu-like symptoms.
Eligibility Criteria
Inclusion Criteria
You may be eligible if you check “Yes” for the criteria belowSelect...
I have been diagnosed with Alpha1-antitrypsin deficiency.
Select...
I am willing to stop my Alpha1-PI treatment for 25 days before the study starts.
Select...
My alpha-1 antitrypsin levels are below the required threshold.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ treatment period 1- single-dose week 1; treatment period 2- repeat-dose weeks 1 and 9
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~treatment period 1- single-dose week 1; treatment period 2- repeat-dose weeks 1 and 9
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
Change from Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Change from Baseline in Forced Vital Capacity (FVC)
Immunogenicity: Number of Participants With Alpha1-PI Antibodies
+8 moreTrial Design
6Treatment groups
Experimental Treatment
Group I: Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1 and single-dose data evaluation phase, participants in Cohort 2 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 180 mg/kg, for 8 weekly SC infusions.
Group II: Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg)Experimental Treatment1 Intervention
Participants will receive Alpha-1 15% 180 mg/kg, single weekly SC infusion in treatment-period 1 (Single-Dose) at Week 1.
Group III: Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1, participants in Cohort 2 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation phase. During the single-dose data evaluation phase, Liquid Alpha1-PI 120 mg/kg, weekly IV Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose.
Group IV: Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1 and single-dose data evaluation period, participants in Cohort 1 will enter treatment period 2 (Repeat-Dose) and will receive Alpha-1 15% 72 mg/kg, for 8 weekly SC infusions.
Group V: Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)Experimental Treatment1 Intervention
Participants will receive Alpha-1 15% 72 mg/kg, single weekly subcutaneous (SC) infusion in treatment-period 1 (Single-Dose) at Week 1.
Group VI: Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg)Experimental Treatment1 Intervention
Following treatment period 1, participants in Cohort 1 will enter 21 days of washout/serial pharmacokinetic (PK) phase and then the single-dose data evaluation period. During the single-dose data evaluation phase, Liquid Alpha1- Proteinase Inhibitor (PI) 60 mg/kg, weekly intravenous (IV) Infusions will be administered from intravenous-dose Week 1 (single-dose Week 5) for up to Week 78, with the last IV dose given 1 week prior to the first repeat Alpha-1 15% SC dose.
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Who is running the clinical trial?
Grifols Therapeutics LLCLead Sponsor
57 Previous Clinical Trials
5,792 Total Patients Enrolled
Media Library
Eligibility Criteria:
This trial includes the following eligibility criteria:- I am not pregnant, breastfeeding, and if able to bear children, I agree to use effective birth control during the study.You have a severe deficiency in Immunoglobulin A (IgA).I have been diagnosed with Alpha1-antitrypsin deficiency.I haven't increased my steroid dose above 5 mg/day prednisone in the last 4 weeks.You have a serious other illness, a history of a severe lung reaction, or an ongoing lung condition.I am willing to stop my Alpha1-PI treatment for 25 days before the study starts.You have had a serious allergic reaction to a specific type of medication made from human blood.I have used antibiotics for COPD flare-ups in the last 4 weeks.I have had a serious worsening of my COPD in the last 4 weeks.You have smoked or used nicotine products in the past 6 months.I have not had lung surgery in the last 2 years, except for a biopsy.My alpha-1 antitrypsin levels are below the required threshold.I have had a lung or liver transplant.Your breathing test results at the Screening Visit need to meet specific requirements.
Research Study Groups:
This trial has the following groups:- Group 1: Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg)
- Group 2: Cohort 2: Treatment Period 2 (Alpha-1 15%, 180 mg/kg)
- Group 3: Cohort 2: Treatment Period 1 (Alpha-1 15%, 180 mg/kg)
- Group 4: Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)
- Group 5: Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg)
- Group 6: Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg)
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.
Frequently Asked Questions
These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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%."
Answered by AI
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."
Answered by AI
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."
Answered by AI
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