Alpha-1 15% for Alpha-1 Antitrypsin Deficiency

Medical University of South Carolina - Children's Hospital, Charleston, SC
Alpha-1 Antitrypsin DeficiencyAlpha-1 15% - Biological
Eligibility
18 - 75
All Sexes

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.

Eligible Conditions
  • Alpha-1 Antitrypsin Deficiency

Treatment Effectiveness

Phase-Based Effectiveness

1 of 3
Phase 1 & 2

Study Objectives

11 Primary · 0 Secondary · Reporting Duration: Treatment Period 1- Single-Dose Week 1; Treatment Period 2- Repeat-Dose Weeks 1 and 9

Week 1
Immunogenicity: Number of Participants With Alpha1-PI Antibodies
Up to 668 days
Change from Baseline in Forced Expiratory Volume in 1 Second (FEV1)
Change from Baseline in Forced Vital Capacity (FVC)
Number of Participants With AEs and SAEs Leading to Discontinuation
Number of Participants With Adverse Events (AEs)
Number of Participants With Chronic Obstructive Pulmonary Disease (COPD) Exacerbations
Number of Participants With Clinically Significant Abnormalities in Laboratory Parameters (Chemistry, Hematology, Urinalysis)
Number of Participants With Clinically Significant Abnormalities in Vital Signs (Heart Rate, Blood Pressure, Respiratory Rate, and Temperature)
Number of Participants With Infusion Site Reactions
Number of Participants With Serious Adverse Events (SAEs)
Number of Participants With Suspected Adverse Drug Reactions (ADRs)

Trial Safety

Phase-Based Safety

1 of 3

Awards & Highlights

No Placebo Group
All patients enrolled in this trial will receive the new treatment.
All Individual Drugs Already Approved
Therapies where all constituent drugs have already been approved are likely to have better-understood side effect profiles.

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

Cohort 2: Treatment Period 2 (Alpha-1 15%, 144 mg/kg)
Biological
Experimental Group · 1 Intervention: Alpha-1 15% · Intervention Types: Biological
Cohort 1: Treatment Period 1 (Alpha-1 15%, 72 mg/kg)
Biological
Experimental Group · 1 Intervention: Alpha-1 15% · Intervention Types: Biological
Cohort 1: Single-Dose Data Evaluation Period (Liquid Alpha 1-Proteinase Inhibitor 60 mg/kg)
Biological
Experimental Group · 1 Intervention: Liquid Alpha1-Proteinase Inhibitor (Human) · Intervention Types: Biological
Cohort 1: Treatment Period 2 (Alpha-1 15%, 72 mg/kg)
Biological
Experimental Group · 1 Intervention: Alpha-1 15% · Intervention Types: Biological
Cohort 2: Treatment Period 1 (Alpha-1 15%, 144 mg/kg)
Biological
Experimental Group · 1 Intervention: Alpha-1 15% · Intervention Types: Biological
Cohort 2: Single-Dose Data Evaluation Period (Liquid Alpha1-Proteinase Inhibitor 120 mg/kg)
Biological
Experimental Group · 1 Intervention: Liquid Alpha1-Proteinase Inhibitor (Human) · Intervention Types: Biological

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: treatment period 1- single-dose week 1; treatment period 2- repeat-dose weeks 1 and 9

Who is running the clinical trial?

Grifols Therapeutics LLCLead Sponsor
57 Previous Clinical Trials
5,746 Total Patients Enrolled

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer

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

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.