Gainesville, FLNCT06512454Now EnrollingIRB Ready

Alpha1-Antitrypsin Deficiency Clinical Trial in Gainesville, FL

Access cutting-edge alpha1-antitrypsin deficiency treatment through this clinical trial at a research site in Gainesville. Study-provided care at no cost to qualified participants.

Sponsored by Takeda

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Expert Care in Gainesville

Access alpha1-antitrypsin deficiency specialists at no cost

IRB Approved

This study follows strict safety protocols and ethical guidelines

No-Cost Care

All study-related alpha1-antitrypsin deficiency treatment provided free

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Why Participate?

  • No-Cost Study Care

  • Local to Gainesville

    Convenient for FL residents

  • Cutting-Edge Treatment

    Access to innovative therapies

  • Expert Medical Care

    Close monitoring by specialists

  • Possible Compensation*

    For time and travel

*Compensation varies by study. Confirm details with coordinator.

Simple Process

  1. 1Submit this form
  2. 2Phone screening
  3. 3Visit Gainesville site if eligible
  4. 4Begin participation

About This Alpha1-Antitrypsin Deficiency Study in Gainesville

The main aim of this study is to learn about liver problems caused by the lack of alpha-1 antitrypsin (called Alpha-1 Antitrypsin Deficiency or AATD) in adults when not treated (this is called the natural history of a condition) over 5 years. Other aims are to learn what can predict the AATD-liver condition starting and getting better or worse, describe how this condition is currently being diagnosed and watched in normal hospital care, and describe how the AATD also affects and adult's lung function. Data in this study will be collected to include medical history of a participant, including the date AATD was first identified and/or the date on which the first AATD-related liver or lung problems were diagnosed. At study start and then every year until study end, participants will be asked to completed questionnaires (called patient-reported outcomes or PRO).

Sponsor: Takeda

Who Can Participate

Inclusion Criteria

Participants who meet all the following criteria will be included in the study. Cohorts 1 and 2:
Willing to provide written informed consent or currently enrolled in an ongoing participating AATD patient registry that does not require reconsenting to participate in the study.
\>=18 years of age at enrollment in this study.
Participants with documented diagnosis of AATD, meeting the following criteria:
Cohort 1 (AATD-Pi\*ZZ genotype/phenotype). • Pi\*ZZ genotype as documented from rapid genetic assay, sequencing, or polymerase chain reaction (PCR), or Pi\*ZZ phenotype as documented from iso-electric focusing (IEF) electrophoresis.
Cohort 2 (AATD-Pi\*SZ genotype/phenotype with liver disease manifestation).
Pi\*SZ genotype as documented from rapid genetic assay, sequencing, or PCR, or Pi\*SZ phenotype as documented from IEF electrophoresis, and
Moderate-advanced or severe liver disease manifestation as defined by either liver biopsy or surrogate laboratory or imaging measures, as determined through:
Lab and imaging measures to define liver disease manifestation

Exclusion Criteria

Participants who meet any following criteria will be excluded from the study.
Documented AATD genotype/phenotype other than Pi\*ZZ or Pi\*SZ.
History of liver transplant.
No results for either biopsies, magnetic resonance elastography (MRE), fibro scan (vibration controlled transient elastography \[VCTE\]), or Aspartate aminotransferase to platelet ratio index (APRI) in the 24 months prior to the index/enrollment date and has none of these tests ordered during the index period.
Participants who had previously been treated or in an active participation in an interventional trial studying liver or lung disease.
Treatment with liver directed AATD investigational therapy as part of a compassionate use request.

Not sure if you qualify? Submit your interest and a study coordinator will help determine your eligibility.

Frequently Asked Questions

Q:Is this study available in Gainesville?

Yes, this clinical trial (NCT06512454) has an active research site in Gainesville, FL that is currently enrolling participants.

Q:Is it safe to participate?

Clinical trials follow strict safety guidelines and ethical standards. This study has been reviewed and approved, and participants are closely monitored by medical professionals. You can withdraw at any time.

Q:Will I be compensated?

Many clinical trials offer compensation for your time and travel expenses. Specific compensation details will be discussed during the screening process. All study-related medical care is provided at no cost.

Q:Can I leave the trial if I change my mind?

Absolutely. Participation is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty.

Still have questions? Our study coordinators are here to help.

Alpha1-Antitrypsin Deficiency Treatment Options in Gainesville, FL

If you're searching for alpha1-antitrypsin deficiency treatment options in Gainesville, FL, this clinical trial (NCT06512454) may be an excellent opportunity. Clinical trials provide access to cutting-edge treatments that aren't yet available to the general public, often at no cost to participants.

Our Gainesville research site is actively enrolling participants for this clinical trial. You'll receive care from experienced alpha1-antitrypsin deficiency specialists who are at the forefront of medical research. All study-related care, including examinations, treatments, and monitoring, is provided at no cost to qualified participants.

Looking for more options? Browse all alpha1-antitrypsin deficiency clinical trials near you to find additional studies recruiting in your area.

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