Current Alpha-1 Studies

Volunteer
The mission of the Alpha -1 Foundation is to find a cure for Alpha-1 and to improve the lives of people affected by Alpha-1 worldwide. Being a volunteer for research makes you a partner in this mission statement and helps lead to the discovery of improvements and new methods of health care for Alpha-1. Research studies are designed to answer questions about disease prevention, detection, and treatment. There are many different types of research studies. There are clinical trials, studies that test new treatments or medical devices. There are also studies that look at health or behavior by conducting interviews or surveys.

The Alpha-1 Research Registry was created in 1997 to bring together researchers and the Alpha-1 community, and to promote the development of improved treatments and a cure for Alpha-1 through research. By joining the Alpha-1 Research Registry you can aid in this endeavor. Below you can learn more about some of the studies for which the registry is actively recruiting participants.

Another great resource for Alpha-1 studies is the Clinical Trials home page, a service of the U.S. National Institutes of Health. You can search for clinical trials involving Alpha-1 by going to the Clinical Trials home page and typing Alpha-1 into the search box on the home page or at the top of any page on the Clinical Trials site. Studies that most closely match your search terms will be listed first.

Lung

ALVELESTAT FOR THE TREATMENT OF ALPHA-1

The National Institutes of Health(NIH)/National Center for Advancing Translational Science (NCATS) and Mereo Biopharma are sponsoring an ongoing study that seeks to test the safety and efficacy of the pill Alvelestat, and investigational drug treatment. This study will test how Alvelestat affects lung damage related to alpha-1 antitrypsin deficiency. Eligible participants must be 25-80 years of age with a genotype of SZ, ZZ, SNull, or ZNull. Participants receiving augmentation therapy must be willing to stop for at least 12 weeks before treatment. Those eligible to participate will receive either Alvelestat or placebo for 12 weeks. Multiple centers in the US are recruiting.

Please visit https://clinicaltrials.gov/ct2/show/NCT03679598 for more information.

COPD EXACERBATION STUDY

Columbia University in New York, NY is seeking to recruit adults aged 35-80 with alpha-1 antitrypsin deficiency (PiZZ, PiZNull, PiSZ, etc) or alpha-1 antitrypsin carrier state (PiMZ, PiMNull, etc.) AND physician diagnosed COPD for a study examining the levels of enzymes that damage the lung before, during, and after recovery from COPD exacerbations (flare of worse lung symptoms). They believe that there are enzymes that are elevated in the setting of COPD exacerbations, and that some of these enzymes might be inactivated by alpha-1 antitrypsin.

GLASSIA

Shire-Baxalta HealthCare Corporation is sponsoring an augmentation therapy research study. This study is being conducted to see if there are any differences in the symptoms and side effects experienced when receiving study drug GLASSIA with a high particle number versus a low particle number, and how the immune system may react to the drug. GLASSIA’s ability to increase the Alpha-1 Proteinase Inhibitor (A1PI) levels in the lungs of patients with alpha-1 antitrypsin deficiency (AATD) will also be examined. All augmentation therapy drugs have extra particles that are usually removed during infusion by a filter. Some batches have more particles (high end) than other batches (low end). This study will compare the two batches on side effects when a 5 micron in-line filter is used. Multiple medical centers around the United States are currently recruiting severely deficient adult Alphas.

Please visit https://clinicaltrials.gov/ct2/show/NCT02525861?term=GLASSIA for more information.

GRIFOLS SELF-INFUSION TRAINING STUDY

Grifols, Inc has developed a training program and training materials that may be used to prepare patients with Alpha-1 Antitrypsin Deficiency to perform their own self-infusions. This will be a 'simulated use' study. This means that no drug or other product will be infused into your body. Instead, you will be provided with a dummy system that will simulate a vein in your arm or an implanted port in your chest, depending on how you currently receive your infusions. You will attend up to 6 one-on-one training sessions with a professional infusion nurse over the course of up to 4 weeks. The study is open to Adult Alpha-1 patients and their caregivers, and expects to enroll 30-40 participants across 4 sites: Tampa, FL; Boston, MA; Indianapolis,IN; and Pittsburgh, PA.

Researchers from Core Human Factors will be coordinating the study. To find out more:

Contact: Lauren at Core Recruiting

Call: (267) 705-6071

Email: join@corerecruiting.com

Web: https://www.corerecruiting.com/

HYALURONIC ACID INHALATION STUDY

MatRX Therapeutics is sponsoring a research study that will be focusing on treating the effects of Alpha-1 emphysema with an inhaled hyaluronic acid therapy. The study plans to enroll adult Alphas between the ages of 18 and 80 with SZ, ZZ, SNull, or ZNull genotypes with diagnosed emphysema. The goal is to determine if the hyaluronic acid inhalation therapy improves markers of lung destruction in individuals receiving drug versus placebo.

Please visit https://clinicaltrials.gov/ct2/show/NCT03114020 for more information.

HYPERPOLARIZED XENON GAS MRI FOR ASSESSING PULMONARY FUNCTION

Duke University in Durham, NC is seeking to recruit those at risk for developing pulmonary disease, and those with known pulmonary disease to examine whether magnetic resonance imaging (MRI) using inhaled hyperpolarized (HP) 129Xenon gas can provide useful images of the functioning of the lung. Study doctors anticipate these images will provide more specific information about lung disease than standard lung function tests. Duke is seeking adults aged 18-80 with alpha-1 genotype of MZ who have a smoking history less than 5 pack-years and have normal lung function.

Please visit https://clinicaltrials.gov/ct2/show/NCT01280994 for more information.

LUNG IMAGING STUDY

Columbia University in New York, NY is seeking to recruit adults aged 45-80 with alpha-1 antitrypsin deficiency (PiZZ, PiZNull, PiSZ, etc) or alpha-1 antitrypsin carrier state (PiMZ, PiMNull, etc.) for a lung imaging study. This technology, Annexin V Targeted SPECT-CT imaging, will screen for minor changes in the lung to improve disease prognosis in Alpha-1. Individuals can be enrolled with moderate to severe COPD, or without COPD. Columbia University would like to study all deficient and carrier individuals, including those with liver disease and without known disease manifestations.

Please visit https://clinicaltrials.gov/ct2/show/NCT02978144?term=wagener for more information.

O2VERLAP COPD AND OBSTRUCTIVE SLEEP APNEA (OSA) STUDY

The COPD Foundation, Inc. and American Sleep Apnea Association are interested in trying to improve the quality of life and care for individuals living with both COPD and OSA. O2VERLAP Study Investigators are seeking adults aged 40 and older who have been diagnosed with both COPD and OSA and have a prescription for continuous positive airway pressure (CPAP) to see if they would benefit from an online, educational curriculum, coupled with access to peer support and remote CPAP adherence monitoring data. The curriculum and peer coaching is meant to provide participants the information and tools they need to be more compliant in using their CPAP device.

Please visit https://clinicaltrials.gov/ct2/show/NCT03446768 for more information.

To join the study and reserve your seat in the virtual classroom, go to https://www.o2verlap.org/portal?REFER=Misc19.

SPARTA

Grifols Therapeutics is sponsoring a research study to investigate whether a study drug called Alpha-1 MP is safe and effective in slowing down the progression of lung damage in patients with alpha-1 antitrypsin deficiency. This study will look at two different doses of the study drug, as well as a placebo over a 3 year period. The study is enrolling severely deficient adults between the ages of 18-70 at multiple medical centers around the United States, as well as in 15 other countries.

Please visit https://clinicaltrials.gov/ct2/show/study/NCT01983241?term=SPARTA&rank=4&show_locs=Y#locn for more information.

VX-814 INVESTIGATIONAL ORAL MEDICATION STUDY

Vertex Pharmaceuticals is evaluating an investigational medication called VX-814. "Investigational" means that VX-814 is not yet approved for use by the Food and Drug Administration (FDA) in the United States. This treatment is oral and is intended to replace intravenous augmentation therapy. Vertex has developed a family of protein correctors that address the underlying cause of AATD by facilitating proper folding of the AAT protein. Vertex's therapy is designed to restore the body's ability to produce its own alpha-1 antitrypsin.

Male ZZ's between 18-65 and females between 45-65 may be eligible to join. Individuals who enroll can expect participation to last approximately 10 weeks. Participants will receive either VX-814 (75% chance) or placebo (25% chance).

To find out more, please telephone the Covance CRU recruitment team: 469-914-8330 quoting 8396-754.

Liver

THE ALPHA-1 CARBAMAZEPINE STUDY

This study is sponsored by the National Institutes of Health (NIH) and is investigating whether carbamazepine (CBZ), a drug which has been used safely for many years for seizures and depression, can reduce the severity of liver disease that occurs in Alpha-1 patients. This year-long study is hoping to recruit Alphas with a ZZ or SZ genotype between the ages of 14 and 80.

Please visit https://clinicaltrials.gov/ct2/show/NCT01379469 for more information.

THE ALPHA-1 FOUNDATION LIVER STUDY

This Alpha-1 Foundation is funding a study to understand the natural history of liver disease in Adult ZZ Alphas. The Known Severe Liver Disease group is still enrolling.

There are 3 U.S. study centers:

St. Louis – Jackie Cerkoski: 314-577-5611 cerkoski@slu.edu

San Diego – Phirum Nguyen: 619-471-0774 psnguyen@ucsd.edu

Boston – Mark Dodge: 617-414-2968 mdodge1@bu.edu

Please visit https://clinicaltrials.gov/ct2/show/NCT02014415 for more information.



Therapy

GLASSIA

Shire-Baxalta HealthCare Corporation is sponsoring an augmentation therapy research study. This study is being conducted to see if there are any differences in the symptoms and side effects experienced when receiving study drug GLASSIA with a high particle number versus a low particle number, and how the immune system may react to the drug. GLASSIA’s ability to increase the Alpha-1 Proteinase Inhibitor (A1PI) levels in the lungs of patients with alpha-1 antitrypsin deficiency (AATD) will also be examined. All augmentation therapy drugs have extra particles that are usually removed during infusion by a filter. Some batches have more particles (high end) than other batches (low end). This study will compare the two batches on side effects when a 5 micron in-line filter is used. Multiple medical centers around the United States are currently recruiting severely deficient adult Alphas.

Please visit https://clinicaltrials.gov/ct2/show/NCT02525861?term=GLASSIA for more information.

SPARTA

Grifols Therapeutics is sponsoring a research study to investigate whether a study drug called Alpha-1 MP is safe and effective in slowing down the progression of lung damage in patients with alpha-1 antitrypsin deficiency. This study will look at two different doses of the study drug, as well as a placebo over a 3 year period. The study is enrolling severely deficient adults between the ages of 18-70 at multiple medical centers around the United States, as well as in 15 other countries.

Please visit https://clinicaltrials.gov/ct2/show/study/NCT01983241?term=SPARTA&rank=4&show_locs=Y#locn for more information.