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      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?
      No Placebo
      Highly Paid
      Stay on Current Meds
      Pivotal Trials (Near Approval)
      Breakthrough Medication

      INBRX-101 vs Zemaira for Emphysema

      Washington, District of Columbia
      Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Cancer, Heart Failure, Others
      Must Not Be Taking:A1PI, Immunoglobulins, Biologics, Others

      99 Participants Needed

      Fazirsiran for Alpha-1 Antitrypsin Deficiency

      Baltimore, Maryland
      This trial is testing fazirsiran, a drug that may reduce liver scarring and improve liver health, in patients with liver fibrosis. The drug works by decreasing harmful proteins and reducing inflammation in the liver.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Liver Decompensation, Chronic Liver Diseases, Others

      160 Participants Needed

      Fazirsiran for Alpha-1 Antitrypsin Deficiency

      Hershey, Pennsylvania
      The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of the AAT protein, called Z-AAT. Making an abnormal version of the AAT protein can result in liver disease as Z-AAT builds up in liver cells, which leads to liver problems such as liver scarring (fibrosis), continuing liver damage (cirrhosis), and eventually end stage liver disease. Fazirsiran is a medicine that reduces the creation of the Z-AAT protein and thus the build-up of this abnormal protein in the liver. People with this type of liver disease who already have mild liver scarring will take part in the study. They will be treated with fazirsiran or a placebo for about 2 years. This study will check the long-term safety of fazirsiran, whether participants tolerate the treatment and if there are any effects on liver scarring. A liver biopsy, a way of collecting a small tissue sample from the liver, will be taken twice during the study.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Liver Fibrosis, Chronic Liver Diseases, Malignancy, Others

      50 Participants Needed

      Alpha-1 MP for Emphysema Due to AATD

      Hershey, Pennsylvania
      This trial tests if Alpha-1 MP, given through an IV drip regularly, can improve lung health in patients with lung conditions. The effectiveness will be monitored using CT scans over a long period. Alpha-1 MP is related to a therapy that has been used to reduce severe lung issues in patients.
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Lung Transplant, Lung Surgery, Smoking, Others
      Must Not Be Taking:Systemic Steroids, Antibiotics

      345 Participants Needed

      SAR447537 for Emphysema

      Hershey, Pennsylvania
      Phase 2 open label extension study to evaluate SAR447537 (INBRX-101) in adults with AATD emphysema
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 2

      Key Eligibility Criteria

      Disqualifiers:Diabetes, Cancer, Heart Failure, Others
      Must Not Be Taking:A1PI, Immunoglobulins, Biologics, Others

      185 Participants Needed

      Fazirsiran for Alpha-1 Antitrypsin Deficiency

      New York, New York
      The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein. People who have taken part in previous fazirsiran studies (AROAAT2001 \[NCT03945292\] or AROAAT2002 \[NCT03946449\]) can continue to receive fazirsiran every 3 months as long as they participate in this study, the study is ongoing or until health authorities in their country approve fazirsiran to be publicly available. The study may also provide information on whether fazirsiran has a long-term effect in reducing liver fibrosis or slowing down the progression of liver fibrosis in people with liver disease due to the abnormal Z-AAT protein.
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 3

      Key Eligibility Criteria

      Disqualifiers:Chronic Liver Diseases, Major Surgery, Others
      Must Not Be Taking:Chronic Anticoagulants

      31 Participants Needed

      Gene Therapy for Alpha-1 Antitrypsin Deficiency

      New York, New York
      This is a study of gene therapy to treat alpha-1 antitrypsin deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of the AAT protein, which if safe and if efficacious, will protect the lung on a persistent basis. We hope to learn the safety/toxicity and initial evidence of efficacy of intravenous delivery of this gene therapy to alpha 1-antitrypsin (AAT) deficient individuals.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1

      Key Eligibility Criteria

      Disqualifiers:Immunodeficiency, Cardiovascular Disease, Cancer, Others
      Must Not Be Taking:Corticosteroids, Immunosuppressants, Beta-blockers, Others

      16 Participants Needed

      Alpha1-Proteinase Inhibitor for Alpha-1 Antitrypsin Deficiency

      Cleveland, Ohio
      This trial tests if Alpha-1 protein injections are safe and tolerable for people with Alpha1-Antitrypsin Deficiency. The treatment aims to protect their lungs by giving them extra Alpha-1 protein. Alpha-1 proteinase inhibitor, a human-derived blood product, has been used for over 20 years to treat individuals with Alpha1-Antitrypsin Deficiency.
      No Placebo Group

      Trial Details

      Trial Status:Active Not Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Lung Or Liver Transplant, Severe Disease, Smoking, Others
      Must Not Be Taking:Systemic Steroids, Antibiotics

      16 Participants Needed

      Alpha-1 MP Safety for Alpha-1 Antitrypsin Deficiency

      Wilmington, North Carolina
      This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60 mg/kg/week in subjects with alpha1-antitrypsin deficiency (AATD).
      No Placebo Group
      Pivotal Trial (Near Approval)

      Trial Details

      Trial Status:Enrolling By Invitation
      Trial Phase:Phase 3
      Age:20 - 72

      Key Eligibility Criteria

      Disqualifiers:Congestive Heart Failure, Liver Cirrhosis, Pulmonary Malignancy, Smoking, Others

      290 Participants Needed

      WVE-006 for Alpha-1 Antitrypsin Deficiency

      Toronto, Ontario
      The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose (SAD) and Period 2 multiple ascending doses (MAD), respectively.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1, 2

      Key Eligibility Criteria

      Disqualifiers:Multiple Drug Allergies, Recent Infections, Alcohol Use, Others
      Must Not Be Taking:AAT Augmentation Therapy

      24 Participants Needed

      BMN 349 for Alpha-1 Antitrypsin Deficiency

      Cincinnati, Ohio
      The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH. Primary outcome measures include incidence of any adverse events (including serious adverse events, dose limit toxicities, and adverse events of special interest), incidence of any laboratory test abnormalities, incidence of lung function test abnormalities and 12-lead ECG parameters. Participants will receive a single dose of either BMN 349 or placebo and then monitored for safety and tolerability.

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1

      Key Eligibility Criteria

      Disqualifiers:INR > 1.2, ALT/AST > 125, Pneumonia, Others
      Must Not Be Taking:AAT Augmentation Therapy

      12 Participants Needed

      Nebulized KB408 for Alpha-1 Antitrypsin Deficiency

      Charleston, South Carolina
      The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1 antitrypsin deficiency (AATD) via nebulization. This study is designed to evaluate safety and pharmacodynamics of KB408 in adults with AATD with a PI\*ZZ or PI\*ZNull genotype. Three planned dose levels of KB408 will be evaluated in single dose escalation cohorts. Repeat dosing will be evaluated at the mid dose level. Subjects taking intravenous AAT augmentation therapy are not required to wash out from IV AAT in the low and mid dose cohorts. In the repeat dose and the high dose cohorts, subjects must wash out from IV AAT for at least 10 days, as applicable.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 1

      Key Eligibility Criteria

      Disqualifiers:Pulmonary Impairment, Smoking, Liver Dysfunction, Others

      15 Participants Needed

      Pneumococcal Vaccine for Alpha-1 Antitrypsin Deficiency

      Birmingham, Alabama
      The goal of this study is to learn whether patients who have a genetic mutation in the genes that cause alpha 1 antitrypsin deficiency also have genetic variation in nearby genes that can increase risk for reduced immune function and respiratory infections. To investigate this hypothesis, we will compare immune responses to the 20-valent pneumococcal conjugate vaccine (PCV20, Pfizer) between participants who have one abnormal copy of the SERPINA1 gene and either no COPD exacerbations, vs those with 2 or more COPD exacerbations in the past year.
      No Placebo Group

      Trial Details

      Trial Status:Recruiting
      Trial Phase:Phase 4
      Age:19+

      Key Eligibility Criteria

      Disqualifiers:Pneumococcal Vaccine Allergy, Others

      30 Participants Needed

      Why Other Patients Applied

      "My orthopedist recommended a half replacement of my right knee. I have had both hips replaced. Currently have arthritis in knee, shoulder, and thumb. I want to avoid surgery, and I'm open-minded about trying a trial before using surgery as a last resort."

      HZ
      Arthritis PatientAge: 78

      "I changed my diet in 2020 and I’ve lost 95 pounds from my highest weight (283). I am 5’3”, female, and now 188. I still have a 33 BMI. I've been doing research on alternative approaches to continue my progress, which brought me here to consider clinical trials."

      WR
      Obesity PatientAge: 58

      "I've been struggling with ADHD and anxiety since I was 9 years old. I'm currently 30. I really don't like how numb the medications make me feel. And especially now, that I've lost my grandma and my aunt 8 days apart, my anxiety has been even worse. So I'm trying to find something new."

      FF
      ADHD PatientAge: 31

      "I have dealt with voice and vocal fold issues related to paralysis for over 12 years. This problem has negatively impacted virtually every facet of my life. I am an otherwise healthy 48 year old married father of 3 living. My youngest daughter is 12 and has never heard my real voice. I am now having breathing issues related to the paralysis as well as trouble swallowing some liquids. In my research I have seen some recent trials focused on helping people like me."

      AG
      Paralysis PatientAge: 50

      "I was diagnosed with stage 4 pancreatic cancer three months ago, metastatic to my liver, and I have been receiving and responding well to chemotherapy. My blood work revealed that my tumor markers have gone from 2600 in the beginning to 173 as of now, even with the delay in treatment, they are not going up. CT Scans reveal they have been shrinking as well. However, chemo is seriously deteriorating my body. I have 4 more treatments to go in this 12 treatment cycle. I am just interested in learning about my other options, if any are available to me."

      ID
      Pancreatic Cancer PatientAge: 40
      Match to a Alpha-1 Antitrypsin Deficiency Trial

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      Why We Started Power

      We started Power when my dad was diagnosed with multiple myeloma, and I struggled to help him access the latest immunotherapy. Hopefully Power makes it simpler for you to explore promising new treatments, during what is probably a difficult time.

      Bask
      Bask GillCEO at Power
      Learn More About Trials
      How Do Clinical Trials Work?Are Clinical Trials Safe?What Can I Expect During a Clinical Trial?

      Frequently Asked Questions

      13 Alpha-1 Antitrypsin Deficiency Trials Near You

      Power is an online platform that helps thousands of Alpha-1 Antitrypsin Deficiency patients discover FDA-reviewed trials every day. Every trial we feature meets safety and ethical standards, giving patients an easy way to discover promising new treatments in the research stage.

      Learn More About Power
      How much do Alpha-1 Antitrypsin Deficiency clinical trials pay?

      Each trial will compensate patients a different amount, but $50-100 for each visit is a fairly common range for Phase 2–4 trials (Phase 1 trials often pay substantially more). Further, most trials will cover the costs of a travel to-and-from the clinic.

      How do Alpha-1 Antitrypsin Deficiency clinical trials work?

      After a researcher reviews your profile, they may choose to invite you in to a screening appointment, where they'll determine if you meet 100% of the eligibility requirements. If you do, you'll be sorted into one of the treatment groups, and receive your study drug. For some trials, there is a chance you'll receive a placebo. Across Alpha-1 Antitrypsin Deficiency trials 30% of clinical trials have a placebo. Typically, you'll be required to check-in with the clinic every month or so. The average trial length for Alpha-1 Antitrypsin Deficiency is 12 months.

      How do I participate in a study as a "healthy volunteer"?

      Not all studies recruit healthy volunteers: usually, Phase 1 studies do. Participating as a healthy volunteer means you will go to a research facility several times over a few days or weeks to receive a dose of either the test treatment or a "placebo," which is a harmless substance that helps researchers compare results. You will have routine tests during these visits, and you'll be compensated for your time and travel, with the number of appointments and details varying by study.

      What does the "phase" of a clinical trial mean?

      The phase of a trial reveals what stage the drug is in to get approval for a specific condition. Phase 1 trials are the trials to collect safety data in humans. Phase 2 trials are those where the drug has some data showing safety in humans, but where further human data is needed on drug effectiveness. Phase 3 trials are in the final step before approval. The drug already has data showing both safety and effectiveness. As a general rule, Phase 3 trials are more promising than Phase 2, and Phase 2 trials are more promising than phase 1.

      Do I need to be insured to participate in a Alpha-1 Antitrypsin Deficiency medical study?

      Clinical trials are almost always free to participants, and so do not require insurance. The only exception here are trials focused on cancer, because only a small part of the typical treatment plan is actually experimental. For these cancer trials, participants typically need insurance to cover all the non-experimental components.

      What are the newest Alpha-1 Antitrypsin Deficiency clinical trials?

      Most recently, we added Pneumococcal Vaccine for Alpha-1 Antitrypsin Deficiency, Gene Therapy for Alpha-1 Antitrypsin Deficiency and BMN 349 for Alpha-1 Antitrypsin Deficiency to the Power online platform.