Alpha1-Proteinase Inhibitor for Alpha-1 Antitrypsin Deficiency

A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)

Recruiting Phase 3 Interventional Study
Alpha 1 Antitrypsin Deficiency
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Pivotal Trial This treatment is in the last trial phase before FDA approval. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
18 – 80
Sex
Any
Trial phase
Phase 3
Study type
Interventional
Purpose
Treatment
Participants needed
40 (estimated)
Sponsor
Grifols Therapeutics LLC · Industry
Who this trial is looking for

This trial is looking for adults with Alpha-1 Antitrypsin Deficiency. Participants will receive either a weekly injection or a standard intravenous treatment of the same medication and help researchers understand the treatment's effectiveness and safety.

Are You a Good Fit for This Trial?

You may be able to join if

  • I have been diagnosed with Alpha1-Antitrypsin Deficiency
  • I have a low alpha1-PI serum level
  • I have a post-bronchodilator FEV1 of 25% to 80% predicted
  • I am willing to stop any current treatment for Alpha1-PI
  • I am willing to give consent to participate

You may not be able to join if

  • I have had a COPD exacerbation in the last 4 weeks
  • I have had lung or liver surgery in the past year
  • I have elevated liver enzymes at screening
  • I am pregnant or breastfeeding
  • I have a history of severe allergic reactions to similar treatments
  • I have smoked in the past 6 months
  • I have known Hepatitis B, C, or HIV infection
  • I have serious health issues that may put me at risk in this trial

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial
This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses of the same medicine given through a vein. Adults with Alpha-1 Antitrypsin Deficiency will take part. Participants will be randomly assigned to one of the treatment groups, and both the study doctors and participants will know whic…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Have a diagnosis of congenital AATD with an allelic combination of ZZ, SZ, Z(null), (null)(null), S(null), or "at-risk" alleles (patients with "at-risk" alleles must be individually evaluated for eligibility by the Medical Monitor). If the genotype has yet to be documented, a …
Contacts

Beatriz Garcia Castro

+(34) 670923669

beatriz.garcia@grifols.com

CONTACT

Lydia Martinez Garrido

+(34) 670929415

lydia.martinez@grifols.com

CONTACT