RecruitingPHASE1, PHASE2INTERVENTIONAL

First-in-human Study Aiming to Characterize the Safety, Tolerability, Pharmacokinetic and Preliminary Signs of Activity of ABD-3001 in Refractory or Relapsed AML and High Risk MDS Adult Patients

This research study is looking at a new drug called ABD-3001. It's for adults who have acute myeloid leukaemia (AML) or a type of myelodysplastic syndrome (MDS) that is high-risk or hasn't improved with other treatments. The main goals are to find out if ABD-3001 is safe, what the best dose is, and how the body handles the medicine. The study will also look for any early signs that the medicine might be working against these conditions. This is an early-stage study, meaning it's one of the first times this drug is being tested in people. It will involve giving different doses to see what is both safe and effective.

At a glance

Status

Recruiting

Phase

PHASE1, PHASE2

Sponsor

Advanced BioDesign

Enrolment target

Start

08 Nov 2022

Estimated completion

01 Dec 2026

Acute Myeloid Leukemia, Adult Myelodysplastic Syndromes

What is this study about?

This study is a pioneering effort to test a new medicine, ABD-3001, in people for the first time. It's specifically designed for adults who have acute myeloid leukaemia (AML) or a type of myelodysplastic syndrome (MDS). These are serious blood and bone marrow cancers. Many patients have already tried other treatments, but their condition hasn't improved or has returned. This study aims to offer a new potential treatment option.

The research will happen in two main stages. The first stage will carefully test different single doses of ABD-3001 to see how safe they are and how the body copes with the medicine. This helps determine a safe range for the drug. Once a safe single dose range is found, the study will move to the second stage, where participants will receive multiple doses. This helps researchers understand how the body responds to repeated doses over time.

Ultimately, the goal is to find a dose and treatment schedule for ABD-3001 that is not only safe but also shows early signs of being effective in treating these challenging conditions. This type of early study is crucial for determining if new medicines have the potential to eventually become widely available treatments.

Key takeaways

This is an early-stage study (Phase 1/2) testing a brand new drug called ABD-3001.
It's for adults with specific types of leukaemia (AML) or bone marrow conditions (MDS) that haven't responded well to other treatments.
The study aims to check if the new drug is safe, find the right dose, and look for early signs it might work.
Participation involves regular visits, assessments, and getting the study drug through an IV.
You can stop participating at any time.
The study is about investigating a potential new option for patients with limited treatment choices.

Who may be eligible?

This study is looking for adults aged 18 or older who have acute myeloid leukaemia (AML) or high-risk myelodysplastic syndrome (MDS). Generally, you would be considered if your AML or MDS has not responded to previous treatments, or if it has come back. It's also for those who might not be able to have certain other treatments like a stem cell transplant.

There are also some important reasons why someone might not be able to join. For example, if you have certain types of AML that can be treated with specific targeted therapies, or if the cancer has spread to your brain or spinal cord. You also can't have serious active infections, certain other illnesses, or have had other treatments for cancer very recently. Women would also need to have a negative pregnancy test to participate.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you an adult (18 years or older)?
Have you been diagnosed with Acute Myeloid Leukaemia (AML) or high-risk Myelodysplastic Syndrome (MDS)?
Has your condition not responded well to previous treatments or has it come back?
Are you able to have a pregnancy test (if applicable)?
Are you generally well enough to participate in a clinical study, apart from your condition?

Answer every question to see your result.

What does participation involve?

If you decide to take part in this study, you will receive the new medicine, ABD-3001, given into a vein (intravenously). This will happen as a single dose initially, and if you continue, as multiple doses over time. The study involves regular visits to the hospital or clinic for assessments. During these visits, the study team will take blood samples, check your vital signs, and monitor you closely for any side effects. There might also be other tests like scans. The total duration of your participation will depend on how you respond to the treatment and the stage of the study you are in, but it will involve ongoing check-ups for a period after your last dose of medication.

Potential risks and benefits

Taking part in this study might offer a potential benefit: access to a new medicine that could help your condition if other treatments haven't worked. However, like all new medicines, ABD-3001 could have side effects, which might range from mild to serious. The research team will monitor you very closely to manage any side effects. It’s important to remember that there's no guarantee the medicine will help you, and there's always a chance of risks. You have the right to withdraw from the study at any time, for any reason, without it affecting your usual medical care.

Locations (3)

Hôpital de la Timone
Verified postcode
Marseille, France· Recruiting
Hôpital Saint-Louis
Verified postcode
Paris, France· Recruiting
Centre Hospitalier Lyon Sud
Verified postcode
Pierre-Bénite, France· Recruiting

Common questions

What is AML and MDS?

AML (Acute Myeloid Leukaemia) and MDS (Myelodysplastic Syndromes) are types of cancer that affect the bone marrow and blood cells. In these conditions, the body doesn't produce healthy blood cells properly.

What does 'first-in-human study' mean?

This means it's one of the very first times this new drug, ABD-3001, is being tested in people. Before this, it would have been tested in laboratories and on animals to understand its safety.

What is ABD-3001?

ABD-3001 is the name of the new experimental medicine being tested in this study. Researchers are hoping it could be a future treatment for AML and MDS.

Will I definitely get the new medicine?

Yes, if you join this study, you will receive the active study drug ABD-3001. This is not a study where some people get a dummy treatment (placebo).

What does 'relapsed/refractory' mean?

This means your condition has either come back after previous treatment, or it hasn't responded to the treatments you've had so far.

How to find out more

Guillaume MARTIN

guillaume.martin@a-biodesign.com +33 (4) 82 53 89 62 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.