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

ARALAST NP AND GLASSIA IN ALPHA-1 ANTITRYPSIN DEFICIENCY

Baxalta (now part of Shire) is sponsoring a study that aims to evaluate the safety and efficacy of weekly administration of Alpha-1 Proteinase Inhibitor (A1PI) augmentation therapy in subjects with alpha-1 antitrypsin deficiency and emphysema/chronic obstructive pulmonary disease (COPD). The study aims to determine whether ARALAST NP or GLASSIA given at two doses (one approved dose of 60 mg/kg weekly and one higher than approved dose of 120 mg/kg weekly) helps to slow down the rate of change in lung tissue that takes place over time in people with Alpha-1, emphysema, and COPD. Eligible participants will be randomly assigned to receive one of five treatment regimens, one of which is a placebo. Participants must also be at least 18 years of age with a documented genotype of PiZZ, PiZ/Null, PiMalton/Z, PiNull/Null, or other rare genotype. Multiple centers around Australia, USA, and Canada are currently recruiting.

Please visit https://clinicaltrials.gov/ct2/show/NCT02722304 for more information.

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.

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.

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.

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.

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.


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.

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

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

Therapy

ARALAST NP AND GLASSIA IN ALPHA-1 ANTITRYPSIN DEFICIENCY

Baxalta (now part of Shire) is sponsoring a study that aims to evaluate the safety and efficacy of weekly administration of Alpha-1 Proteinase Inhibitor (A1PI) augmentation therapy in subjects with alpha-1 antitrypsin deficiency and emphysema/chronic obstructive pulmonary disease (COPD). The study aims to determine whether ARALAST NP or GLASSIA given at two doses (one approved dose of 60 mg/kg weekly and one higher than approved dose of 120 mg/kg weekly) helps to slow down the rate of change in lung tissue that takes place over time in people with Alpha-1, emphysema, and COPD. Eligible participants will be randomly assigned to receive one of five treatment regimens, one of which is a placebo. Participants must also be at least 18 years of age with a documented genotype of PiZZ, PiZ/Null, PiMalton/Z, PiNull/Null, or other rare genotype. Multiple centers around Australia, USA, and Canada are currently recruiting.

Please visit https://clinicaltrials.gov/ct2/show/NCT02722304 for more information.

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.


Gene Therapy

ADVM-043 GENE THERAPY

Adverum Biotechnologies, Inc is sponsoring a study to investigate the safety and efficacy of ADVM-043 gene therapy treatment. Gene therapy can be defined as replacing a missing or defective gene with a normal or functioning gene. The ADVM-043 study will use a virus called adeno-associated virus (AAV) to carry a functional copy of the alpha-1 antitrypsin gene into the cells of patients with alpha-1 antitrypsin deficiency. Eligible participants must be at least 18 years of age with a genotype of ZZ or Z/Null. Participation will last approximately 15 months and subjects will be enrolled at multiple medical centers throughout the United States.

Please visit https://clinicaltrials.gov/ct2/show/NCT02168686 for more information.