#1 Clinical Trials Search in Europe

Alx148

Clinical trials are investigating Alx148 in people with higher risk myelodysplastic syndrome, including patients who have not been treated before. These studies are looking at safety, tolerability, and how well the treatment works, especially when Alx148 is given with azacitidine.

Table of Contents

Trial overview

The main study in the data is NCT04417517, a Phase 1/2 study of Alx148 with azacitidine in people with higher risk myelodysplastic syndrome (MDS).[1] The study was designed to look first at safety and dose finding, and then at how well the treatment worked in patients with previously untreated higher risk MDS.[1]

Who was studied

This trial included adults with higher risk MDS, which means a form of bone marrow disease with a greater chance of serious progression.[1] The brief summary says the Phase 1 part included adult patients with relapsed or refractory MDS, as well as previously untreated higher risk MDS, while the Phase 2 part focused on previously untreated higher risk MDS with an IPSS-R score above 3.5.[1]

Relapsed means the disease came back after improvement, and refractory means it did not respond well to treatment.[1] Previously untreated means patients had not yet received treatment for their disease.[1]

What was tested

The study tested Alx148 in combination with azacitidine, and both drugs were given by intravenous use in the trial record.[1] The Phase 2 part compared the combination of Alx148 at the recommended Phase 2 dose with azacitidine alone.[1]

The trial was not only asking whether the treatment might help, but also whether it could be given safely in this patient group.[1] In simple terms, the study was trying to find the best balance between possible benefit and acceptable safety.[1]

Study phases and endpoints

Phase 1 focused on safety, tolerability, and finding the recommended Phase 2 dose, also called RP2D.[1] The dose escalation part looked for dose-limiting toxicities, which are side effects serious enough to limit treatment at a certain dose.[1]

The dose expansion part measured adverse events, meaning any medical problems that happened during treatment, and recorded their type, frequency, severity, timing, seriousness, and relationship to the study therapy.[1] These details help researchers understand how the treatment behaves in more patients.[1]

The Phase 2 part measured complete response rate (CRR), which is the share of patients who reached a complete response by the study’s response rules.[1] The study used modified IWG 2006 response criteria for MDS, and the final analysis was planned after the last patient finished 6 treatment cycles or was ready to be assessed for response.[1]

Trial status and size

The trial status is listed as Completed.[1] The enrollment was small, with 6 participants in the record provided.[1] Because this was an early-phase study, the main goal was to learn about safety and the right dose before larger studies could be done.[1]

Key patient terms

  • Interventional study: a trial where researchers give a treatment and then measure what happens.[1]
  • Intravenous use: medicine given through a vein.[1]
  • Response criteria: the rules used to decide whether the disease improved.[1]
  • Complete response: a strong improvement defined by the study’s rules.[1]
  • IPSS-R score: a risk score used to show how serious MDS is.[1]
Trial ID Phase Condition studied Status Enrollment
NCT04417517 Phase 1 Higher risk myelodysplastic syndrome (MDS), including previously untreated patients Completed 6

FAQ

  • What is being studied in the Alx148 trials? The trial is studying Alx148 together with azacitidine. It is looking at whether the combination is safe and whether it can improve responses in myelodysplastic syndrome.
  • Who can join the trial? The study includes adults with higher risk myelodysplastic syndrome. Some parts also included patients with relapsed or refractory disease, while the phase 2 part focused on previously untreated higher risk MDS.
  • What phase is the Alx148 study? This is a Phase 1/2 study. Phase 1 looks at safety, tolerability, and dose finding. The phase 2 part looks at how well the treatment works.
  • What condition is the trial for? The trial is for myelodysplastic syndrome, also called MDS. This is a group of blood disorders where the bone marrow does not make healthy blood cells normally.
  • What results are the researchers measuring? The study measures side effects, dose-limiting toxicities, and complete response rate. Complete response means the disease signs improve enough to meet the study’s response criteria.

Ongoing Clinical Trials on Alx148

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

    Not recruiting

    1 1 1
    Investigated diseases:
    Investigated drugs:
    Spain

Glossary

  • Myelodysplastic syndrome (MDS): A group of bone marrow disorders where blood cells do not develop normally, which can lead to low blood counts and other problems.
  • Higher risk: A term used for disease that is more likely to get worse or cause serious problems.
  • Previously untreated: The person has not yet received treatment for the disease.
  • Relapsed: The disease came back after a period of improvement.
  • Refractory: The disease did not respond well to treatment.
  • Phase 1: An early trial phase that mainly checks safety, tolerability, and dose.
  • Phase 2: A trial phase that mainly checks whether the treatment seems to work.
  • Dose escalation: A study step where researchers slowly increase the dose to find a suitable level.
  • Dose expansion: A study step where more people receive the chosen dose to learn more about safety and effects.
  • Adverse events: Medical problems or side effects that happen during a study.
  • Complete response rate (CRR): The percentage of patients whose disease meets the study definition of a complete response.
  • IPSS-R score: A risk score used to describe how serious myelodysplastic syndrome is.

References