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	<title>Alpha-1 antitrypsin deficiency &#8211; European Clinical Trials Information Network</title>
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	<title>Alpha-1 antitrypsin deficiency &#8211; European Clinical Trials Information Network</title>
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		<title>Safety and Efficacy of TSRA-196 (rnaivt9315 and rnacs24757) in Adults with Lung or Liver Disease due to Alpha‑1 Antitrypsin Deficiency</title>
		<link>https://clinicaltrials.eu/trial/phase-1-2-study-of-rnaivt9315-and-rnacs24757-in-adults-with-pizz-genotype-and-lung-and-or-liver-disease-from-severe-alpha-1-antitrypsin-deficiency/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 17 Jun 2026 04:03:06 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/phase-1-2-study-of-rnaivt9315-and-rnacs24757-in-adults-with-pizz-genotype-and-lung-and-or-liver-disease-from-severe-alpha-1-antitrypsin-deficiency/</guid>

					<description><![CDATA[People with the PiZZ genotype of Alpha-1 Antitrypsin Deficiency often have low levels of a protein that protects the lungs and liver, which can lead to breathing problems, similar to chronic lung disease, and to liver damage. The study uses an investigational medicine called TSRA-196, given as an intravenous (through the vein) infusion, designed to [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>People with the <b>PiZZ genotype</b> of <b>Alpha-1 Antitrypsin Deficiency</b> often have low levels of a protein that protects the lungs and liver, which can lead to breathing problems, similar to chronic lung disease, and to liver damage. The study uses an investigational medicine called <b>TSRA-196</b>, given as an intravenous (through the vein) infusion, designed to increase the amount of this protective protein in the blood.</p>
<p>The main goal of the study is to see whether a single dose of the medicine is safe and whether it can raise protein levels to a normal range, with some participants receiving a second dose later to assess longer‑term effects. Participants will receive the infusion, then attend regular visits over several months during which blood samples and simple health checks are performed.</p>
<p>Researchers will watch for any side effects, measure the amount of the protective protein in the blood, and test how well it works using a laboratory test called a <b>elastase inhibition assay</b>. They will also check lung function and liver health through routine exams and questionnaires to understand how the treatment impacts overall well‑being.</p>
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		<title>Study on the Safety of Self-Infusion Therapy with Human Alpha1-Proteinase Inhibitor for Patients with Severe Alpha-1 Antitrypsin Deficiency</title>
		<link>https://clinicaltrials.eu/trial/study-on-the-safety-of-self-infusion-therapy-with-human-alpha1-proteinase-inhibitor-for-patients-with-severe-alpha-1-antitrypsin-deficiency/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 15:02:52 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-the-safety-of-self-infusion-therapy-with-human-alpha1-proteinase-inhibitor-for-patients-with-severe-alpha-1-antitrypsin-deficiency/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Severe Alpha-1 Antitrypsin Deficiency (AATD). This is a genetic disorder that can lead to lung and liver problems. The study will use a treatment called Prolastin, which contains a substance known as human alpha1-proteinase inhibitor. This treatment is given as a solution through an [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <b>Severe Alpha-1 Antitrypsin Deficiency (AATD)</b>. This is a genetic disorder that can lead to lung and liver problems. The study will use a treatment called <b>Prolastin</b>, which contains a substance known as <b>human alpha1-proteinase inhibitor</b>. This treatment is given as a solution through an intravenous infusion, which means it is administered directly into the bloodstream through a vein.</p>
<p>The purpose of the study is to assess the safety of using <b>Prolastin</b> for self-infusion therapy at home over a period of 12 weeks. Participants will be trained to perform the infusions themselves or with the help of a caregiver. The study will monitor the occurrence of any adverse effects during the training and main study period. Additionally, it will evaluate various health parameters, such as lung function and patient satisfaction, to understand the impact of the treatment.</p>
<p>Throughout the study, participants will have regular visits to assess their health and the ease of using the treatment. The study aims to provide valuable information on how safe and effective it is for patients with <b>Severe Alpha-1 Antitrypsin Deficiency</b> to manage their condition with home-based self-infusion therapy using <b>Prolastin</b>.</p>
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		<title>Study on the Safety and Tolerability of WVE-006 for Patients with Alpha-1 Antitrypsin Deficiency (AATD) Pi*ZZ</title>
		<link>https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-wve-006-for-patients-with-alpha-1-antitrypsin-deficiency-aatd-pizz/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 15:02:09 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-the-safety-and-tolerability-of-wve-006-for-patients-with-alpha-1-antitrypsin-deficiency-aatd-pizz/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency (AATD). AATD is a genetic disorder that can lead to lung and liver problems. The study will use a treatment called WVE-006, which is a solution for injection. WVE-006 is a type of medication known as an oligonucleotide, which is a [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <b>Alpha-1 Antitrypsin Deficiency (AATD)</b>. AATD is a genetic disorder that can lead to lung and liver problems. The study will use a treatment called <b>WVE-006</b>, which is a solution for injection. WVE-006 is a type of medication known as an oligonucleotide, which is a small piece of genetic material designed to target specific genes.</p>
<p>The purpose of the study is to evaluate the safety and tolerability of WVE-006 in participants with a specific genetic form of AATD, known as <b>Pi*ZZ</b>. Participants will receive either single or multiple doses of WVE-006. The study will monitor how the body processes the medication and any potential side effects. Participants will receive the medication through a subcutaneous injection, which means it is injected under the skin.</p>
<p>Throughout the study, researchers will observe changes in the levels of a protein called M-AAT in the blood, which is related to the condition. The study will also measure how much of the medication is present in the blood over time. This research aims to gather important information about how WVE-006 works in people with AATD and to ensure it is safe for use. The study is expected to continue until the end of 2025.</p>
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		<title>Study on the Safety and Effectiveness of BEAM-302 with MR0005 and GR0015 for Adults with Alpha-1 Antitrypsin Deficiency-Related Lung or Liver Disease</title>
		<link>https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-beam-302-with-mr0005-and-gr0015-for-adults-with-alpha-1-antitrypsin-deficiency-related-lung-or-liver-disease/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 15:02:04 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-the-safety-and-effectiveness-of-beam-302-with-mr0005-and-gr0015-for-adults-with-alpha-1-antitrypsin-deficiency-related-lung-or-liver-disease/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency (AATD), which can lead to lung and liver diseases. The trial will explore a new treatment called BEAM-302, which is a type of gene therapy. Gene therapy involves using genetic material to treat or prevent disease. In this study, BEAM-302 is [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <i>Alpha-1 Antitrypsin Deficiency (AATD)</i>, which can lead to lung and liver diseases. The trial will explore a new treatment called <i>BEAM-302</i>, which is a type of gene therapy. Gene therapy involves using genetic material to treat or prevent disease. In this study, <i>BEAM-302</i> is administered as a solution for injection through an intravenous infusion, meaning it is delivered directly into the bloodstream.</p>
<p>The purpose of the study is to evaluate the safety and effectiveness of <i>BEAM-302</i> in adults with AATD-associated lung and/or liver disease. The study is divided into two phases. The first phase will explore different doses to determine the safest and most effective amount of the treatment. The second phase will expand on this by confirming the optimal dose and further assessing how well the treatment works. Participants will receive the treatment and be monitored for any side effects or changes in their condition.</p>
<p>Throughout the study, researchers will collect information on how the treatment affects the levels of certain proteins in the blood, specifically those related to <i>Alpha-1 Antitrypsin</i>. This will help determine the treatment&#8217;s impact on the disease. The study aims to provide valuable insights into the potential of <i>BEAM-302</i> as a treatment option for individuals with AATD-related health issues.</p>
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		<item>
		<title>Study on Long-Term Safety of INBRX-101 for Adults with Alpha-1 Antitrypsin Deficiency Emphysema</title>
		<link>https://clinicaltrials.eu/trial/study-on-long-term-safety-of-inbrx-101-for-adults-with-alpha-1-antitrypsin-deficiency-emphysema/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 14:59:23 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-long-term-safety-of-inbrx-101-for-adults-with-alpha-1-antitrypsin-deficiency-emphysema/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency (AATD) Emphysema. This is a genetic disorder that can lead to lung problems, including emphysema, which is a type of chronic lung disease. The trial will use a treatment called INBRX-101, which is a concentrate for solution for infusion. This treatment [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <i>Alpha-1 Antitrypsin Deficiency (AATD) Emphysema</i>. This is a genetic disorder that can lead to lung problems, including emphysema, which is a type of chronic lung disease. The trial will use a treatment called <i>INBRX-101</i>, which is a concentrate for solution for infusion. This treatment contains a special protein called <i>human alpha-1-proteinase inhibitor immunoglobulin G fusion protein, recombinant</i>, designed to help manage the symptoms of AATD emphysema.</p>
<p>The purpose of the study is to evaluate the long-term safety and tolerability of <i>INBRX-101</i> in adults with AATD emphysema. Participants in the study will receive the treatment through an infusion, which is a method of delivering medication directly into the bloodstream. The study will monitor participants over a period to observe any side effects and to assess how well the treatment works in managing the condition.</p>
<p>Throughout the study, researchers will keep track of any adverse events, which are any unwanted effects that occur during the treatment. They will also look at changes in lung density using a method called <i>computed tomography (CT)</i>, which is a type of imaging that helps visualize the lungs. Additionally, the study will examine how the body processes the treatment and whether any antibodies, which are proteins made by the immune system, develop against <i>INBRX-101</i>. The study aims to provide valuable information on the safety and effectiveness of this treatment for people with AATD emphysema.</p>
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		<title>Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency Liver Disease and Mild Fibrosis</title>
		<link>https://clinicaltrials.eu/trial/study-on-fazirsiran-for-patients-with-alpha-1-antitrypsin-deficiency-liver-disease-and-mild-fibrosis/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 14:30:39 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-fazirsiran-for-patients-with-alpha-1-antitrypsin-deficiency-liver-disease-and-mild-fibrosis/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency-Associated Liver Disease, which is a genetic disorder that can lead to liver damage. The study is investigating a treatment called Fazirsiran, also known by its code name TAK-999. Fazirsiran is a type of medication designed to target specific genes in the [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <i>Alpha-1 Antitrypsin Deficiency-Associated Liver Disease</i>, which is a genetic disorder that can lead to liver damage. The study is investigating a treatment called <i>Fazirsiran</i>, also known by its code name <i>TAK-999</i>. Fazirsiran is a type of medication designed to target specific genes in the liver to help reduce the damage caused by this disease. The trial will compare the effects of Fazirsiran with a placebo to understand its safety and effectiveness, particularly in people with mild liver scarring, also known as fibrosis.</p>
<p>The main goal of the study is to evaluate the long-term safety of Fazirsiran, with a special focus on lung health, as the condition can also affect the lungs. Participants in the study will receive either Fazirsiran or a placebo through injections. The study will monitor various health indicators, including lung function and liver health, over a period of time to see how the treatment affects these areas. Participants will undergo regular check-ups and tests to track any changes in their condition.</p>
<p>This study is important for understanding how Fazirsiran can help people with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease and to ensure that it is safe for long-term use. By participating in this trial, researchers hope to gather valuable information that could lead to better treatment options for those affected by this genetic disorder.</p>
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		<item>
		<title>Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor for Adults with Alpha-1 Antitrypsin Deficiency Emphysema</title>
		<link>https://clinicaltrials.eu/trial/study-comparing-inbrx-101-and-human-alpha1-proteinase-inhibitor-for-adults-with-alpha-1-antitrypsin-deficiency-emphysema/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 14:29:58 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-comparing-inbrx-101-and-human-alpha1-proteinase-inhibitor-for-adults-with-alpha-1-antitrypsin-deficiency-emphysema/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency (AATD) Emphysema. This is a genetic disorder that can lead to lung problems, including emphysema, which is a type of chronic lung disease. The study is testing a new treatment called INBRX-101, which is a concentrate for solution for infusion. This [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <i>Alpha-1 Antitrypsin Deficiency (AATD) Emphysema</i>. This is a genetic disorder that can lead to lung problems, including emphysema, which is a type of chronic lung disease. The study is testing a new treatment called <i>INBRX-101</i>, which is a concentrate for solution for infusion. This treatment is being compared to an existing therapy known as <i>plasma-derived Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy</i>. The goal of the study is to evaluate how well INBRX-101 works in maintaining certain protein levels in the blood, which are important for lung health, over a period of up to 32 weeks.</p>
<p>Participants in the study will receive either the new treatment, INBRX-101, or the existing A1PI therapy. The study is designed to be double-blind, meaning neither the participants nor the researchers will know who is receiving which treatment. This helps ensure that the results are unbiased. The study will also include a placebo group, which will receive a solution that does not contain the active treatment. Throughout the study, participants will have regular check-ups to monitor their health and the effects of the treatment.</p>
<p>The study aims to gather information on the safety and effectiveness of INBRX-101 compared to the current standard treatment. It will also look at how the body processes the new treatment and whether it causes any immune reactions. By the end of the study, researchers hope to better understand if INBRX-101 can be a beneficial treatment option for people with AATD emphysema.</p>
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		<title>Study on Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease and Fibrosis</title>
		<link>https://clinicaltrials.eu/trial/study-on-fazirsiran-for-patients-with-alpha-1-antitrypsin-deficiency-related-liver-disease-and-fibrosis/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 14:24:23 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-fazirsiran-for-patients-with-alpha-1-antitrypsin-deficiency-related-liver-disease-and-fibrosis/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. This disease is linked to an abnormal version of a protein called alpha-1 antitrypsin, which can lead to liver damage and scarring, also known as fibrosis. The study is testing a treatment called Fazirsiran, which is a solution for [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <b>Alpha-1 Antitrypsin Deficiency-Associated Liver Disease</b>. This disease is linked to an abnormal version of a protein called alpha-1 antitrypsin, which can lead to liver damage and scarring, also known as fibrosis. The study is testing a treatment called <b>Fazirsiran</b>, which is a solution for injection. Fazirsiran is also known by its code name <b>TAK-999</b> and is designed to target specific liver cells to help reduce the effects of this disease.</p>
<p>The purpose of the study is to evaluate how effective Fazirsiran is compared to a placebo in improving liver health in people with this condition. Participants in the study will receive either Fazirsiran or a placebo through subcutaneous injections, which means the injection is given under the skin. The study will take place over a period of time, with regular check-ups to monitor the health of the liver and the overall safety of the treatment.</p>
<p>Throughout the study, participants will undergo various assessments to track changes in their liver condition. These assessments will help determine if Fazirsiran can reduce liver scarring and improve liver function. The study aims to provide valuable information on whether this treatment can be a beneficial option for people with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease.</p>
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		<item>
		<title>Study on Long-Term Safety of Fazirsiran for Patients with Alpha-1 Antitrypsin Deficiency-Related Liver Disease</title>
		<link>https://clinicaltrials.eu/trial/study-on-long-term-safety-of-fazirsiran-for-patients-with-alpha-1-antitrypsin-deficiency-related-liver-disease/</link>
		
		<dc:creator><![CDATA[]]></dc:creator>
		<pubDate>Wed, 29 Apr 2026 14:23:59 +0000</pubDate>
				<guid isPermaLink="false">https://clinicaltrials.eu/trial/study-on-long-term-safety-of-fazirsiran-for-patients-with-alpha-1-antitrypsin-deficiency-related-liver-disease/</guid>

					<description><![CDATA[This clinical trial is focused on studying a condition known as Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. This is a genetic disorder that can lead to liver problems due to a lack of a protein called alpha-1 antitrypsin, which is important for protecting the liver. The treatment being studied is a medication called Fazirsiran, also known [&#8230;]]]></description>
										<content:encoded><![CDATA[<p>This clinical trial is focused on studying a condition known as <i>Alpha-1 Antitrypsin Deficiency-Associated Liver Disease</i>. This is a genetic disorder that can lead to liver problems due to a lack of a protein called alpha-1 antitrypsin, which is important for protecting the liver. The treatment being studied is a medication called <i>Fazirsiran</i>, also known by its code name <i>TAK-999</i>. Fazirsiran is a solution for injection that is designed to target specific liver cells to help manage this condition.</p>
<p>The purpose of this study is to evaluate the long-term safety and effectiveness of Fazirsiran in people with Alpha-1 Antitrypsin Deficiency-Associated Liver Disease. Participants in this study will receive Fazirsiran through subcutaneous injections, which means the medication is injected under the skin. The study will monitor participants over a period of time to observe any changes in their health and to ensure the treatment is safe and well-tolerated.</p>
<p>Throughout the study, participants will have regular check-ups to assess their liver health and overall well-being. This includes monitoring vital signs, conducting laboratory tests, and evaluating liver function. The study aims to provide valuable information on how Fazirsiran can help manage liver disease associated with Alpha-1 Antitrypsin Deficiency over an extended period.</p>
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