Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor 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 study is testing a new treatment called INBRX-101, which is a concentrate for solution for infusion. This treatment is being compared to an existing therapy known as plasma-derived Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy. 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.

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.

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.

1 joining the study

Upon joining the study, the patient will be informed about the trial’s purpose and procedures. The patient will be required to provide informed consent, acknowledging understanding of the study and agreeing to participate.

2 initial assessment

The patient will undergo an initial assessment to confirm eligibility. This includes verifying age, diagnosis of Alpha-1 Antitrypsin Deficiency (AATD), evidence of emphysema, and lung function tests to ensure FEV1 is between 30% and 80% of the predicted value. The patient must also be a non-smoker.

3 randomization

The patient will be randomly assigned to receive either INBRX-101 or a plasma-derived Alpha1-Proteinase Inhibitor (A1PI) therapy. This process ensures that each participant has an equal chance of receiving either treatment.

4 treatment administration

The patient will receive the assigned treatment through an infusion. INBRX-101 is administered as a concentrate for solution for infusion at a concentration of 50mg/ml. The alternative treatment, Respreeza, is a powder and solvent for solution for infusion. The frequency and dosage will be determined by the study protocol and medical staff.

5 treatment period

The treatment period will last up to 32 weeks. During this time, the patient will receive regular infusions and attend scheduled visits for monitoring and assessment of treatment effects.

6 monitoring and assessments

Throughout the study, the patient will undergo regular monitoring to assess the pharmacodynamic effects on serum functional AAT levels, safety, and any adverse events. Blood samples may be taken to measure the concentration of AAT and to check for any immune responses to the treatment.

7 completion of study

Upon completion of the treatment period, the patient will have a final assessment to evaluate the overall effects of the treatment. This includes measuring changes in AAT levels and reviewing any side effects experienced during the study.

Who Can Join the Study?

Must be a male or female between 18 and 80 years old.
Must have a diagnosis of Alpha-1 Antitrypsin Deficiency (AATD), which is a genetic condition affecting the lungs.
Must show signs of emphysema caused by AATD. Emphysema is a lung condition that makes it hard to breathe.
Must have a FEV1 (Forced Expiratory Volume in 1 second) between 30% and 80% of what is predicted for a healthy person. FEV1 is a measure of how much air you can forcefully exhale in one second.
Must be a current non-smoker, meaning you do not smoke cigarettes at the time of the study.

Who Cannot Join the Study?

Patients who have a known allergy or severe reaction to the study medication or its ingredients cannot participate.
Individuals with a history of certain lung diseases, other than Alpha-1 Antitrypsin Deficiency (AATD) Emphysema, are not eligible.
Participants who have had a recent lung infection or illness that affects breathing may be excluded.
People who are currently taking medications that could interfere with the study treatment are not allowed to join.
Patients with a history of drug or alcohol abuse may be excluded from the study.
Individuals with certain medical conditions that could affect the study results or their safety might not be eligible.
Pregnant or breastfeeding women cannot participate in the study.
Participants who are unable to follow the study procedures or attend scheduled visits may be excluded.

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
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
Htmpxkzs Ucvasknbxaytw Mflwykp Di Vkuqrsqmyu	Santander	Spain
Netojtch Igspwbuvx Oa Tneyfzlfbgxt Axr Ltag Dlnqhtwf	Warsaw	Poland

Trial status

Country	Status	Recruitment Start
Denmark	Not recruiting	01.03.2024
Ireland	Not recruiting	01.03.2024
Poland	Not recruiting	01.03.2024
Spain	Not recruiting	01.03.2024
Sweden	Not recruiting	01.03.2024

Trial locations

Investigated drugs:

INBRX-101 is an investigational medication being studied for its effects on patients with a condition called Alpha-1 Antitrypsin Deficiency (AATD) emphysema. This medication is designed to help increase the levels of a protein called functional AAT in the blood, which may help protect the lungs from damage.

Alpha1-Proteinase Inhibitor (A1PI) Augmentation Therapy is a treatment used for patients with AATD emphysema. It involves giving patients additional A1PI, a protein that helps protect the lungs from damage. This therapy aims to maintain adequate levels of A1PI in the blood to help prevent further lung damage.

Investigated diseases:

Emphysema

Alpha-1 Antitrypsin Deficiency (AATD) Emphysema – This is a genetic disorder that affects the lungs and liver, primarily leading to emphysema, a type of chronic obstructive pulmonary disease (COPD). It occurs due to a deficiency of the protein alpha-1 antitrypsin, which protects the lungs from damage caused by enzyme activity. Without enough of this protein, the lungs are more susceptible to damage from environmental factors like smoke and pollution. Over time, this can lead to the destruction of lung tissue, causing breathing difficulties and reduced lung function. The progression of the disease can vary, with some individuals experiencing rapid decline while others have a slower progression. Symptoms often include shortness of breath, wheezing, and chronic cough.

Trial ID:

2023-508084-76-00

Protocol code:

INBRX101-01-201

NCT ID:

NCT05856331

Trial Phase:

Therapeutic exploratory (Phase II)

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Study Comparing INBRX-101 and Human Alpha1-Proteinase Inhibitor for Adults with Alpha-1 Antitrypsin Deficiency Emphysema

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?