RecruitingPhase 2ACTRN12622000410752

Advancing treatment therapies in Myelodysplasia

MDS05: Advancing therapies in Myelodysplasia: . A multi-domain platform trial for patients with myelodysplasia investigating new treatments - The Master Protocol

Sponsor

Australasian Leukaemia and Lymphoma Group (ALLG)

Enrollment

1,000 participants

Start Date

Jun 30, 2023

Study Type

Interventional

Conditions

Myelodysplasia

Summary

Traditionally, combination therapy has not proven to be effective in Myelodysplasia (MDS) given the increased toxicity of drugs used in combination with standard therapies. The aim of this master platform trial is to test a range of novel treatments aimed at targeting MDS to find safe and effective drug combinations. You may be eligible to participate in this study if you are aged 16 or older (depending on the study, this may be limited to 18 and older) and you have a diagnosis of either MDS or acute myeloid leukemia (AML) with less than 30% blasts. If you choose to enrol in this trial you will undergo a screening process where blood and bone marrow samples are taken prior to starting any treatments. The study doctors will assess your samples for specific MDS markers and will then prescribe a combination treatment that may be effective. Participants who do not respond to treatment or whose disease worsens may be removed from treatment and be reassessed for the next best treatment option to receive. During treatment participants will be assessed regularly which will include physical exams, blood tests, ECG (heart monitoring), bone marrow biopsies, toxicities to the treatment, quality of life. After treatment, disease will continue to be monitored and if participants have MDS progression or morphological relapse they will be assessed for the next best treatment option. It is hoped that the results of this trial will help us to determine which drug combinations are safe (minimal side effects/toxicity) and effective for treating MDS.

Eligibility

Sex: Both males and femalesMin Age: 16 Yearss

Plain Language Summary

Simplified for easier understanding

Myelodysplastic syndrome (MDS) is a group of blood cancers where the bone marrow does not produce healthy blood cells properly. The standard treatment — a drug called azacitidine — helps many patients but does not work for everyone, and combinations of drugs have historically been too toxic. New targeted therapies have changed the landscape, and this trial is designed to find safe and effective drug combinations. This Australian master platform trial takes a flexible approach: patients are screened and then matched to a drug combination based on their specific disease characteristics. Rather than testing one combination at a time, the trial can run multiple experimental arms simultaneously and adapt as results come in. Participants are monitored closely with regular blood tests, bone marrow biopsies, and quality-of-life questionnaires. You may be eligible if you are 16 years of age or older (or 18 for some sub-studies) and have a confirmed diagnosis of MDS or acute myeloid leukaemia with fewer than 30% blasts in the bone marrow. People with other active cancers requiring treatment, HIV, viral hepatitis, or prior bone marrow transplant for MDS or AML are generally not eligible.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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Interventions

The MYDAS-T study is a multi-domain platform trial. This master protocol will govern the development and management of an adaptive clinical trial platform that will incorporate novel therapeutic combinations for medium and high risk patients with MDS. The main objective is to assess safety and tolerability of these new treatment combinations for this in need patient population. The overall durations of the treatment domains will range from 5-10 years depending on the treatment combination being trialed the required follow up period length. The therapies trialed in this domain will be new and established investigational medicinal products that may range from chemotherapy to biologic target therapies. Patients will be monitored at their standard of care clinic visits and associated tests eg bone marrow aspiration, blood tests, physical examinations. Depending on the number of domains open, patients may be stratified or randomised to a domain based on strata such as MDS risk and age. The allocation will be dependent on the number of domains available at the time of patient entry.

Locations(1)

New Zealand

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