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Study on the Safety of Self-Infusion Therapy with Human Alpha1-Proteinase Inhibitor for Patients with Severe Alpha-1 Antitrypsin Deficiency

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What is this trial about?

A plain-language summary of the goals, design and what participants do

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 intravenous infusion, which means it is administered directly into the bloodstream through a vein.

The purpose of the study is to assess the safety of using Prolastin 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.

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 Severe Alpha-1 Antitrypsin Deficiency to manage their condition with home-based self-infusion therapy using Prolastin.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial training
Receive training on how to perform self-infusion of the medication Prolastin at home. This training ensures understanding of the procedure and safety measures.
The training includes learning how to prepare the solution for infusion and how to administer it through an intravenous infusion.
Step 2
Self-infusion therapy
Begin a 12-week period of self-infusion therapy with Prolastin. The medication is administered through an intravenous infusion.
The dosage of Prolastin is determined based on the specific product used: 1000 mg, 4000 mg, or 5000 mg. The frequency and duration of each infusion are specified during the training.
Step 3
Monitoring and assessment
Throughout the study, complete questionnaires and assessments to monitor health and treatment effects. This includes the COPD Assessment Test (CAT) and other lung function tests.
Regularly report any side effects or adverse events experienced during the therapy.
Step 4
End-of-study evaluation
At the end of the 12-week period, participate in a final evaluation to assess the overall safety and effectiveness of the therapy.
This evaluation includes a review of lung function parameters and patient satisfaction with the treatment process.

Who can join the trial?

9 criteria

Must have signed a written informed consent before any study-related activities begin. This means you agree to participate and understand what the study involves.
Must be at least 18 years old.
Must be able to understand and read German well enough to comprehend patient documents, like consent forms and instructions. If writing is needed, a caregiver can help.
Must have a documented diagnosis of severe Alpha-1 Antitrypsin Deficiency (AATD). This is a genetic condition that affects the lungs and liver.
Must have been treated with Prolastin® for at least 3 months and have shown good tolerance to the treatment.
Must be able and willing to learn how to perform self-infusion at home, or have a caregiver who can do it. This means you or your caregiver can give the treatment without a healthcare professional present, after training. If you need a caregiver, they must be available. If you do it yourself, someone must be available to monitor you during each infusion.
Must have access to and be able to use a device for phone and video calls, and have internet access to complete questionnaires.
Must have suitable veins for venepuncture, which means drawing blood or inserting a needle into a vein, as determined by the study doctor.
Must have a life expectancy of more than 3 years.

Who cannot join the trial?

10 criteria

Patients who have a known allergy or severe reaction to the study medication, Prolastin®.
Patients who have a history of severe allergic reactions, known as **anaphylaxis**, to any medication.
Patients who are currently participating in another clinical trial or have participated in one within the last 30 days.
Patients who have a medical condition that the study doctors believe would make it unsafe for them to participate.
Patients who are pregnant or breastfeeding.
Patients who have a history of drug or alcohol abuse within the past year.
Patients who have a serious illness or infection that is not well controlled.
Patients who have received a blood transfusion or blood products within the last 30 days.
Patients who have a history of certain lung diseases, such as **emphysema**, that are not related to Alpha-1 Antitrypsin Deficiency.
Patients who have a history of liver disease that is not related to Alpha-1 Antitrypsin Deficiency.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Asklepios Kliniken Hamburg GmbH
Verified
Hamburg, Germany
Goethe University Frankfurt
Verified
Frankfurt, Germany
Thoraxklinik Heidelberg gGmbH
Responsive
Heidelberg, Germany

Trial locations

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Investigated drugs

Prolastin is a medication used in this clinical trial to help patients with severe alpha-1-antitrypsin deficiency (AATD). It is a therapy that involves infusing the medication at home by the patients themselves. The main goal of the trial is to ensure that this self-infusion method is safe for the patients. Prolastin works by providing the body with the alpha-1-antitrypsin protein that is lacking in these patients, which helps protect the lungs from damage.

Human Alpha1-Proteinase Inhibitor

What is already known about the treatment

Prolastin – Prolastin is administered through intravenous infusion, typically performed at home by the patient themselves. It is currently being studied in clinical trials to evaluate its safety for self-infusion in individuals with severe Alpha-1 Antitrypsin Deficiency (AATD). The main therapeutic use of Prolastin is to supplement the deficient protein in patients with AATD, helping to protect the lungs from damage. At the molecular level, Prolastin works by providing the body with alpha-1 antitrypsin, a protein that inhibits enzymes that can damage lung tissue. It is classified as a proteinase inhibitor in pharmacology.

Investigated diseases

Severe Alpha-1 Antitrypsin Deficiency (AATD) – This is a genetic disorder that affects the lungs and liver, primarily due to a deficiency of the protein alpha-1 antitrypsin. This protein is responsible for protecting the lungs from damage caused by enzyme activity. In individuals with severe AATD, the lack of this protein leads to the gradual destruction of lung tissue, often resulting in conditions like emphysema. The disease progresses as lung function declines, causing symptoms such as shortness of breath, wheezing, and chronic cough. Over time, individuals may experience increased difficulty in breathing and reduced ability to perform physical activities. The condition is considered rare and can vary in severity among those affected.