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

• Status: Recruiting
• Trial end date: 2024-12-01
• Target enrollment: 0
• Participant gender: All

Summary

This First In Human (FIH) study is a prospective, open-label, multicenter, Phase 1 study, with a dose escalation design, followed by an optimized design. It will consist in a Single Ascending Dose (SAD) part and a Multiple Ascending Dose (MAD) part followed by a "Regimen optimization" part with an extension cohort.

• Phase: Phase 1
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Advanced BioDesign

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 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

• 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

• History of other malignancy in the last 12 months prior to study Visit 1

• Other active solid tumor

• Patients taking medications that are known to prolong the QT interval

• 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