Not recruitingRare disease

Study of ALX148 and Azacitidine for Untreated Higher Risk Myelodysplastic Syndrome Patients

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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 Myelodysplastic Syndrome (MDS), which is a type of blood disorder where the bone marrow does not produce enough healthy blood cells. The study is specifically looking at patients with a higher risk of this condition who have not yet received treatment. The trial will test a combination of two treatments: ALX148, a protein-based medication, and Azacitidine, a chemical-based medication. Both treatments are administered through an injection into the vein.

The purpose of the study is to evaluate the safety and effectiveness of the combination of ALX148 and Azacitidine in treating patients with higher-risk MDS. The study is divided into two phases. In the first phase, researchers will determine the best dose of ALX148 to use with Azacitidine. In the second phase, they will compare the effects of the combination treatment to Azacitidine alone. Participants will receive the study treatments and be monitored for their response to the treatment and any side effects they may experience.

Throughout the study, participants will have regular visits to the clinic for treatment and monitoring. The study aims to understand how well the combination of ALX148 and Azacitidine works in improving the condition of patients with higher-risk MDS and to gather information on any potential side effects. The trial will help determine if this combination can be a more effective treatment option for patients with this type of blood disorder.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, eligibility is confirmed based on specific criteria, including age, medical history, and current health status.
A signed informed consent document is required, indicating understanding of the study's aspects.
Step 2
Initial assessment
An initial assessment is conducted to evaluate health status, including blood tests and other necessary examinations.
This assessment ensures that participation in the study is safe and appropriate.
Step 3
Treatment phase 1
The first phase involves receiving ALX148 and azacitidine through intravenous injections.
The goal is to evaluate the safety and tolerability of the combination treatment.
Dosage and frequency are determined by the study protocol, and adjustments may be made based on individual response.
Step 4
Treatment phase 2
In the second phase, the effect of ALX148 at the recommended dose is assessed in combination with azacitidine.
The focus is on the complete response rate, which measures the effectiveness of the treatment.
Step 5
Monitoring and follow-up
Regular monitoring is conducted to track health status and any side effects.
Follow-up visits include laboratory tests and assessments to ensure safety and evaluate treatment progress.
Step 6
End of study
The study concludes after the final analysis, which occurs after completing six cycles of treatment or when the patient is considered evaluable for response.
Results are analyzed to determine the overall effectiveness and safety of the treatment.

Who can join the trial?

16 criteria

Adult patients with a confirmed diagnosis of **higher risk myelodysplastic syndrome (MDS)**, which is a type of blood disorder, or **relapsed/refractory MDS**, meaning the disease has returned or is not responding to treatment.
For patients with relapsed/refractory MDS, previous treatment with **hypomethylating agents** (a type of drug used to treat MDS) is allowed.
Patients with previously untreated higher risk MDS must not have had prior treatment with hypomethylating agents or **cytotoxic chemotherapy** (a type of cancer treatment that kills cells) for MDS.
Patients must be suitable candidates for treatment with a single drug called **AZA**.
Female patients who can have children must have a negative pregnancy test before starting the study.
Male and female patients who can have children must agree to use effective birth control during the study and for at least 120 days after the last dose of treatment.
Patients must sign a consent form indicating they understand the study details before participating.
Patients must be willing and able to attend scheduled visits and follow the treatment plan, including tests and procedures.
For certain parts of the study, patients must have less than 20% **myeloblasts** (a type of immature blood cell) in their bone marrow.
Patients must have adequate kidney function, with a creatinine clearance (a measure of kidney function) of at least 30 mL/min.
Patients must have adequate liver function, with specific limits on liver enzymes and bilirubin levels.
Patients must have a **white blood cell (WBC)** count of less than 20,000 per microliter. Use of a drug called **hydroxyurea** to control WBC is allowed before starting the study treatment.
The **QTcF interval** (a measure of heart rhythm) must be 480 milliseconds or less.
Patients must be 18 years or older, as MDS is very rare in children.
Patients must have an **ECOG Performance Status** of 0, 1, or 2, which indicates their level of daily functioning.
Any side effects from previous treatments must have returned to their original severity or be mild, except for those not considered a safety risk by the study doctor.

Who cannot join the trial?

7 criteria

Patients who have already received treatment for their **higher risk myelodysplastic syndrome (MDS)** cannot participate. **Myelodysplastic syndrome (MDS)** is a group of disorders caused by poorly formed or dysfunctional blood cells.
Patients with a **lower risk** of MDS, as determined by a specific scoring system, are not eligible. This scoring system helps to assess the severity of the condition.
Patients who are not adults are excluded. This means only individuals who are 18 years or older can participate.
Patients who are not able to provide informed consent, which means they cannot understand the study and agree to participate, are not eligible.
Patients who are pregnant or breastfeeding cannot take part in the study.
Patients with other serious health conditions that might interfere with the study or increase risk are excluded.
Patients who are participating in another clinical trial at the same time are not eligible.

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Verified sites

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Hospital Universitario De Salamanca
Salamanca, Spain
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Hospital Universitario Y Politecnico La Fe
Valencia, Spain

Trial locations

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Investigated drugs

ALX148
is an investigational medication being studied for its potential to help treat patients with higher risk myelodysplastic syndrome (MDS). It is being tested to see if it can be safely combined with another medication to improve treatment outcomes. The goal is to determine the best dose that can be used in combination with other treatments.
Azacitidine
is a medication used to treat certain types of blood disorders, including higher risk myelodysplastic syndrome (MDS). It works by helping to restore normal growth and function to blood cells. In this study, it is being used in combination with ALX148 to see if the combination can improve treatment results for patients with MDS.

What is already known about the treatment

ALX148
ALX148 is administered intravenously and is currently being studied in clinical trials for its potential use in treating higher-risk myelodysplastic syndrome (MDS) in patients who have not yet received treatment. It is in the investigational stage, with ongoing research to determine its safety and effectiveness. The main therapeutic indication is for patients with higher-risk MDS. ALX148 works by targeting specific proteins on cancer cells, helping the immune system recognize and destroy these cells. It is classified as an immune checkpoint inhibitor.
Azacitidine
Azacitidine is administered via injection, either subcutaneously or intravenously, and is an established medication used in the treatment of myelodysplastic syndromes (MDS). It is well-documented in medical literature and is approved for use in patients with higher-risk MDS. Azacitidine works by incorporating into the DNA of cancer cells, disrupting their growth and leading to cell death. It is classified as a nucleoside metabolic inhibitor.

Investigated diseases

Myelodysplastic Syndrome (MDS) – Myelodysplastic Syndrome is a group of disorders caused by poorly formed or dysfunctional blood cells. It occurs when something goes wrong in the bone marrow, where blood cells are produced. In MDS, the bone marrow produces immature cells that do not develop into healthy blood cells, leading to a shortage of one or more types of blood cells. This can result in symptoms such as fatigue, infections, or easy bruising and bleeding. Over time, MDS can progress to more severe forms of blood disorders, including acute myeloid leukemia. The progression and symptoms can vary widely among individuals, depending on the specific type and severity of the syndrome.