Evaluating ABD-3001 for patients with difficult-to-treat blood cancers
Official title: First-In-Human, Open Label, Dose Escalation Study to Evaluate Safety, PK and PD of ABD-3001 As Monotherapy in Relapsed/Refractory Acute Myeloid Leukemia or High/Very-high Risk Myelodysplastic Syndromes Patients, Ineligible for Intensive or New Generation Targeted Therapy.
Run by Advanced BioDesign · NCT05601726 · Data updated 2026-11-12
This study is testing a new treatment called ABD-3001 to see if it can help adults with hard-to-treat blood cancers like Acute Myeloid Leukemia and high-risk Myelodysplastic Syndromes feel better and stay safe during the process.
Quick facts
| Phase | Phase1; Phase2 |
|---|---|
| Study type | Interventional |
| Ages | 18 Years and up |
| Sex | All |
| Enrollment | 36 (estimated) |
| Sponsor | Advanced BioDesign Industry-sponsored |
| Locations | 3 sites (Marseille, France and 2 other locations) |
| Status | Recruiting |
| Started | November 8, 2022 |
| Main results expected | June 2026 |
| Study ends | December 2026 |
| Registry record updated | January 20, 2025 |
| Trial ID | NCT05601726 on ClinicalTrials.gov |
Is this trial a fit for you?
✓Likely a good fit
Ideal candidates include adults with relapsed or refractory AML or high-risk MDS who are not eligible for salvage treatments.
✕Probably not a fit
Patients with specific karyotype abnormalities in AML or those with active central nervous system leukemia may not benefit from this study.
A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.
What this trial studies
This first-in-human study investigates the safety, tolerability, and preliminary effectiveness of ABD-3001 in adults with refractory or relapsed Acute Myeloid Leukemia (AML) and high-risk Myelodysplastic Syndromes (MDS). The study employs a dose escalation design, consisting of a Single Ascending Dose (SAD) phase to determine the optimal dosing and a Multiple Ascending Dose (MAD) phase to further assess pharmacokinetics and pharmacodynamics. Participants will receive ABD-3001 via intravenous infusion, with careful monitoring of their response and safety throughout the trial.
Why it matters
Potential benefit: If successful, this study could provide a new treatment option for patients with limited alternatives for refractory AML and high-risk MDS.
How similar studies have gone: While this approach is novel for ABD-3001, similar studies targeting refractory blood cancers have shown promising results in the past.
Who can join: the full rules
Show full inclusion / exclusion criteria
Inclusion Criteria: * Patients with relapsed/refractory Acute Myeloid Leukemia (AML) after failing at least one therapy regimen and a salvage treatment or are not eligible for salvage treatment regimens including targeted therapy * Patients with relapsed/refractory Myelodysplastic syndrome (MDS) ineligible for salvage treatment who are diagnosed high-risk and very high-risk using Revised International Prognostic Scoring System (IPSS-R) prognostic risk categorization * Patients not eligible to alloSCT * Negative blood or serum/urine pregnancy test Exclusion Criteria: * Patients with acute myeloid leukemia (AML) with Inv(16) MYH11-CBF-Beta or t(8;21) AML-ETO RUNX1-RUNX1 or (PML/RARA) karyotype abnormalities and eligible to targeted therapies * Participants with clinical symptoms suggestive of active central nervous system (CNS) leukemia or known CNS leukemia * Ongoing immunosuppressive treatment * Hematopoietic stem cell transplantation (HSCT) performed within 3 months prior to study Visit 1 * Active infection requiring intravenous anti-infectious treatment during the screening period * Life-threatening illnesses other than the studied one, uncontrolled medical conditions or organ system dysfunction which, in the investigator's opinion, could compromise the patient's safety or interfere with the patient's ability to comply with the study activities * Anti-tumor therapy within 14 days of study Visit 1 * Prior participation in an interventional investigational clinical study (drug or medical device) within 21 days of study Visit 1 * Radiotherapy within 28 days prior to study Visit 1 * Current history of seropositivity to human immunodeficiency virus (HIV) or infection with active hepatitis C virus (HCV) or active hepatitis B virus (HBV) or active SARS-CoV-2 (Covid-19) or Syphilis, or Cytomegalovirus (CMV), or Epstein-Barr virus (EBV), or Human T-Lymphotropic Virus (HTLV1) * History of other malignancy in the last 12 months prior to study Visit 1 * Other active solid tumor * Subjects with New York Heart Association (NYHA) Class III or IV congestive heart failure or Left Ventricular Ejection Fraction (LVEF) \<50% attested by echocardiogram (ECHO) or multi-gated acquisition (MUGA) scan within 28 days of C1D1 prior to study Visit 1 (Day 1, start of study therapy) * Subjects with a history of myocardial infarction within the last 3 months prior to study Visit 1 (Day 1, start of study therapy) * Subjects with heart-rate corrected QT (QTc) interval ≥450 ms or other factors that increase the risk of QT prolongation or arrhythmic events * Major surgery within 4 weeks prior to study Visit 1 (Day 1, start of study therapy) * Any condition deemed by the investigator to be likely to interfere with a subject's ability to participate in the clinical trial MAD specific exclusion criteria: Patients who have been part of SAD and have experienced a DLT.
Where it's running
-
Hôpital de la Timone
Marseille, France, France Recruiting -
Hôpital Saint-Louis
Paris, France, France Recruiting -
Centre Hospitalier Lyon Sud
Pierre-Bénite, France, France Recruiting
Study contacts
- Study coordinator: Guillaume MARTIN
- Email: guillaume.martin@a-biodesign.com
- Phone: +33 (4) 82 53 89 62
Similar studies
Other studies for the same condition or treatment. Recruiting studies are listed first.
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How to take part
- Review the eligibility criteria above with your treating physician.
- Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
- Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.