Study on Long-Term Safety of INBRX-101 for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

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What is this study about?

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 contains a special protein called human alpha-1-proteinase inhibitor immunoglobulin G fusion protein, recombinant, designed to help manage the symptoms of AATD emphysema.

The purpose of the study is to evaluate the long-term safety and tolerability of INBRX-101 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.

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 computed tomography (CT), 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 INBRX-101. The study aims to provide valuable information on the safety and effectiveness of this treatment for people with AATD emphysema.

1 joining the study

Upon joining the study, eligibility is confirmed based on criteria such as age, diagnosis of alpha-1 antitrypsin deficiency (AATD), and current non-smoking status.

2 initial assessment

An initial assessment is conducted to establish baseline health status. This includes lung function tests to measure forced expiratory volume (FEV1) and other relevant health checks.

3 treatment administration

The treatment involves receiving INBRX-101, a concentrate for solution for infusion, administered at a dosage of 50mg/ml.

The medication is given through an infusion, which is a method of delivering the drug directly into the bloodstream over a set period.

4 ongoing monitoring

Regular monitoring is conducted to evaluate the long-term safety and tolerability of INBRX-101. This includes tracking any adverse events and changes in lung density using CT scans.

Blood tests are performed to assess the serum pharmacokinetic (PK) profile of INBRX-101 and to check for the presence of anti-drug antibodies.

5 end of treatment evaluation

At the end of the treatment period, a comprehensive evaluation is conducted to assess the overall impact of the treatment on lung function and any potential side effects experienced during the study.

Who Can Join the Study?

  • Must be a male or female between 18 and 80 years old at the time of screening.
  • Must have a diagnosis of Alpha-1 Antitrypsin Deficiency (AATD), which is a genetic condition that can affect the lungs.
  • Must show evidence of emphysema caused by AATD. Emphysema is a lung condition that makes it hard to breathe.
  • Must have a FEV1 (Forced Expiratory Volume in one second) that is 30% or more of what is predicted for a person of the same age, gender, and size. This is a measure of lung function.
  • For new patients in Cohort 1, the FEV1/FVC (Forced Vital Capacity) ratio must be less than 0.7 after using a bronchodilator, which is a medication that helps open the airways in the lungs.
  • Must be a current non-smoker, meaning you do not smoke cigarettes or use other tobacco products.

Who Cannot Join the Study?

  • Patients who have a different lung condition other than Alpha-1 Antitrypsin Deficiency (AATD) Emphysema cannot participate. This is a specific lung disease caused by a lack of a protein that protects the lungs.
  • Patients who are not within the specified age range for the study cannot participate. The study is looking for participants within certain age groups.
  • Patients who are not able to follow the study procedures or attend the required visits cannot participate.
  • Patients who have a medical condition that might interfere with the study or make it unsafe for them to participate cannot join the study.
  • Patients who are pregnant or breastfeeding cannot participate in the study.
  • Patients who are currently participating in another clinical trial cannot join this study.
  • Patients who have had a recent major surgery or are planning to have surgery during the study period cannot participate.
  • Patients who have a history of drug or alcohol abuse that might interfere with the study cannot participate.
  • Patients who have a known allergy to the study medication or its ingredients cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
Hospital Clinico San Carlos Madrid Spain

Other Sites

Site Name City Country Status
Sygehus Lillebaelt Vejle Sygehus Vejle Denmark
Universidade De Santiago De Compostela Santiago De Compostela Spain
Samodzielny Publiczny Zaklad Opieki Zdrowotnej Szpital Uniwersytecki W Krakowie Cracow Poland
Gentofte Hospital Hellerup Denmark
Queen Silvia Childrens Hospital – Sahlgrenska University Hospital – Vaestra Goetalandsregionen Gothenburg Sweden
Beaumont Hospital Dublin Ireland
Hrfaualc Ugiszjgjntlyd Msrshub Dx Vhyftxksjq Santander Spain
Njrkomci Iopbhhqwm Oc Tmlvsuauphau Avw Lxnw Dmlkdpuw Warsaw Poland

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Denmark Denmark
Not recruiting
28.09.2024
Ireland Ireland
Not recruiting
28.09.2024
Poland Poland
Not recruiting
28.09.2024
Spain Spain
Not recruiting
28.09.2024
Sweden Sweden
Not recruiting
28.09.2024

Trial locations

INBRX-101 is a medication being studied for its long-term safety and effectiveness in treating adults with Alpha-1 Antitrypsin Deficiency (AATD) emphysema. This condition is a genetic disorder that can lead to lung disease. The trial aims to understand how well patients tolerate this medication over an extended period.

Alpha-1 Antitrypsin Deficiency (AATD) Emphysema – This is a genetic disorder that affects the lungs and liver, primarily leading to lung disease. It occurs due to a deficiency of the protein alpha-1 antitrypsin, which protects the lungs from damage. Without enough of this protein, the lungs can become damaged, leading to emphysema, a condition where the air sacs in the lungs are gradually destroyed. Individuals with this condition may experience shortness of breath, wheezing, and a chronic cough. Over time, the lung function continues to decline, making breathing increasingly difficult. The progression of the disease can vary, with some individuals experiencing more rapid deterioration than others.

Trial ID:
2023-508137-14-00
Protocol code:
INBRX101-01-202
NCT ID:
NCT05897424
Trial Phase:
Therapeutic exploratory (Phase II)

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