Study on Pegfilgrastim for Patients with Severe Chronic Neutropenia

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What is this study about?

This clinical trial is focused on studying a condition known as severe chronic neutropenia (SCN). This is a disorder where the body has a lower than normal number of a type of white blood cell called neutrophils, which are important for fighting off infections. The treatment being tested in this study is a medication called pegfilgrastim, which is marketed under the name Neulasta. Pegfilgrastim is a type of medicine known as a colony-stimulating factor, which helps the body produce more neutrophils.

The purpose of the study is to evaluate how effective pegfilgrastim is in maintaining a healthy level of neutrophils in patients with severe chronic neutropenia who are already receiving daily treatment with another medication called G-CSF and anti-infective therapy. The study will observe patients over a period of time to see if pegfilgrastim can help keep their neutrophil levels above a certain threshold, which is important for reducing the risk of infections.

Participants in the study will receive pegfilgrastim as a solution for injection, administered under the skin. The study will monitor the number of infections, the use of antibiotics, and any hospitalizations that occur during the study period. This information will help determine the effectiveness of pegfilgrastim in managing severe chronic neutropenia. The study is expected to continue until 2030, providing valuable insights into the long-term benefits of this treatment.

1 initial assessment

The trial begins with an initial assessment to confirm eligibility. This involves verifying that the absolute neutrophil count is less than 500/mmc based on three consecutive tests conducted at least one week apart over a minimum three-month period.

Eligibility is confirmed for patients already receiving daily treatment with G-CSF and anti-infective therapy.

2 medication administration

The medication used in this trial is Neulasta, which contains the active substance pegfilgrastim. It is administered as a solution for injection.

The injection is given subcutaneously, meaning it is injected under the skin.

The dosage is 6 mg per injection. The frequency and duration of administration are determined by the study protocol and the patient’s response.

3 monitoring and evaluation

Throughout the trial, the absolute neutrophil count is monitored to ensure it remains above 1000/mmc.

The primary endpoint is the median of the absolute neutrophil count from the second to the sixth month of the study.

Secondary endpoints include the number, type, and duration of infections, the number of antibiotic courses, and the number of hospitalizations during the same period.

4 follow-up

Regular follow-up appointments are scheduled to assess the patient’s health and response to the treatment.

These appointments include reviewing any side effects, infections, or hospitalizations that may occur during the trial.

5 completion

The trial is estimated to end on September 28, 2030.

Upon completion, the data collected will be analyzed to evaluate the efficacy of pegfilgrastim in maintaining the desired neutrophil levels and reducing infection-related complications.

Who Can Join the Study?

Patients of any age can participate.
Both males and females are eligible.
Patients must have either congenital (present from birth) or acquired (developed later) chronic neutropenia.
Chronic neutropenia is defined as having an absolute neutrophil count (a type of white blood cell) of less than 500 per cubic millimeter, based on three tests done at least one week apart over a minimum of three months.
Patients must already be receiving treatment with G-CSF (a medication given daily to help increase white blood cells).

Who Cannot Join the Study?

Patients who are not already being treated with G-CSF, which is a medication used to help the body make more white blood cells.
Patients who are not receiving anti-infective therapy, which is treatment to prevent or fight infections.
Patients who do not have severe chronic neutropenia, a condition where the body has a very low number of a type of white blood cell called neutrophils.
Patients who are outside the specified age range for the study.
Patients who are not part of the specified clinical trial groups.

Where you can join this trial?

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Verified Sites

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Other Sites

Site Name	City	Country	Status
IRCCS Istituto Giannina Gaslini	Genoa	Italy

Trial status

Country	Status	Recruitment Start
Italy	Not yet recruiting	29.09.2005

Trial locations

Investigated drugs:

Pegfilgrastim

Pegfilgrastim is a medication used in this trial to help increase the number of white blood cells, specifically neutrophils, in patients with severe chronic neutropenia. It is designed to maintain neutrophil levels above a certain threshold to help reduce the risk of infections. This medication is a long-acting form of a protein that stimulates the bone marrow to produce more neutrophils.

G-CSF (Granulocyte Colony-Stimulating Factor) is another medication used in the trial. It is administered daily to help increase the production of neutrophils in the bone marrow. This medication is crucial for patients with severe chronic neutropenia to help prevent infections by maintaining adequate levels of neutrophils in the blood.

Anti-infective therapy is also part of the treatment regimen in this trial. This therapy involves the use of medications that help prevent or treat infections. Patients with severe chronic neutropenia are at a higher risk of infections due to low neutrophil counts, so anti-infective therapy is important to protect them from potential infections.

Investigated diseases:

Neutropenia

Severe Chronic Neutropenia – Severe chronic neutropenia is a condition characterized by an abnormally low number of neutrophils, a type of white blood cell essential for fighting off infections. This disease can be congenital, cyclic, or idiopathic, meaning its cause is unknown. Individuals with this condition often experience frequent infections due to their compromised immune system. The progression of the disease involves persistent low neutrophil counts, which can lead to recurrent bacterial infections, particularly affecting the skin, respiratory tract, and other areas. Over time, the condition may cause complications due to repeated infections and inflammation. Management typically focuses on maintaining neutrophil levels to reduce infection risk.

Trial ID:

2024-519009-36-01

Protocol code:

CDF 711

Trial Phase:

Therapeutic exploratory (Phase II)

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Study on Pegfilgrastim for Patients with Severe Chronic Neutropenia

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