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

GLASSIA

Baxalta US Incorporated (formerly Baxter) 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 a study drug GLASSIA with a high particle number versus a low particle number, and how the immune system may react to the study 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 Alphas.

If you would like to learn more about this study please visit clinicaltrials.gov NCT02525861.

HYALURONIC ACID INHALATION STUDY

MatRx Therapeutics is sponsoring a Hyaluronic Acid Inhalation Study that will be focusing on treating the effects of Alpha-1 emphysema by using an inhaled therapy. The study plans to enroll adult Alphas between the ages of 18 and 80 with SZ, ZZ, SNull or ZNull genotypes diagnosed with 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.

SPARTA

Grifols Therapeutics is sponsoring a study to investigate a study drug called Alpha-1 MP to discover if it is safe and effective at slowing down the progression of lung damage in severely deficient Alphas. This study will look at two different doses of the study drug as well as a placebo over a 3 year period. Multiple medical centers around the United States are currently recruiting Alphas.

Please visit clinicaltrials.gov NCT01983241 for more information.

TARGETING PULMONARY PERFUSION IN ALPHA-1 ANTITRYPSIN DEFICIENCY

The Alpha-1 Foundation is sponsoring a research study at Columbia University in New York, NY. The study plans to enroll subjects 40 years and older with alpha-1 antitrypsin deficiency-associated lung disease, PiZZ phenotype. The study evaluates whether aspirin improves pulmonary blood flow in persons with alpha-1 antitrypsin deficiency.  

Please visit www.clinicaltrials.gov/ct2/show/NCT03008915 for more information.

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 yearlong study is hoping to recruit Alphas with a ZZ or SZ genotype between the ages of 14 and 80.

Please visit www.chp.edu/liverstudy for more information and other eligibility requirements.

You can view the study advertisement here


ALPHA-1 FOUNDATION LIVER STUDY

The Alpha-1 Foundation is funding a study to understand the natural history of liver disease in ZZ Alphas. This is a 5 year study that hopes to determine what causes liver disease and how liver disease progresses. There are 3 sites enrolling:

St Louis- Jackie Cerkoski: 314-577-5611 cerkoski@slu.edu
San Diego- Phirum Nguyen: 619-471-0774
Boston- Mark Dodge: 617-414-2968 mdodge1@bu.edu

View the study invitation here

Therapy

GLASSIA

Baxalta US Incorporated (formerly Baxter) 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 a study drug GLASSIA with a high particle number versus a low particle number, and how the immune system may react to the study 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 Alphas.

If you would like to learn more about this study please visit clinicaltrials.gov NCT02525861.

SPARTA

Grifols Therapeutics is sponsoring a study to investigate a study drug called Alpha-1 MP to discover if it is safe and effective at slowing down the progression of lung damage in severely deficient Alphas. This study will look at two different doses of the study drug as well as a placebo over a 3 year period. Multiple medical centers around the United States are currently recruiting Alphas.

Please visit clinicaltrials.gov NCT01983241 for more information. 

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