RecruitingPhase 1Phase 2NCT05601726

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

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.


Sponsor

Advanced BioDesign

Enrollment

36 participants

Start Date

Nov 8, 2022

Study Type

INTERVENTIONAL

Conditions

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.


Eligibility

Min Age: 18 Years

Plain Language Summary

Simplified for easier understanding

This is a first-in-human study testing a new drug called ABD-3001 in adults with relapsed or refractory acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS) — both serious blood cancers that have not responded to other treatments. The study focuses on safety, how the drug behaves in the body, and early signs of whether it works. **You may be eligible if...** - You have AML that has come back or stopped responding after at least one standard treatment - You have high-risk or very high-risk MDS that is no longer responding to treatment and you are not eligible for other salvage therapies - Your general health and organ function meet the required levels **You may NOT be eligible if...** - You have active brain involvement from your blood cancer - You have had a bone marrow transplant recently and still have transplant-related complications - You have serious infections or other serious health conditions - You are pregnant or breastfeeding Talk to your doctor to see if this trial is right for you.

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

DRUGABD-3001

Each patient will receive a fixed 4 hours-intravenous infusion dose of ABD-3001 once or twice a week. For SAD : The first dose of the first cohort will receive an estimated infusion dose of 18 mg/m² at Day 1. Subsequent cohorts will be given the following doses: 54 mg/m², 135 mg/m², 270 mg/m², 405 mg/m², 540 mg/m².

DRUGABD-3001

For MAD : Based on all data gathered during the SAD including safety, PK and preliminary efficacy data, up to three doses were selected in accordance with the Safety review Committee (SRC). A dosage optimization analysis was performed at the end of the SAD cohort 6 using population pharmacokinetic modelling to set the optimal frequency of infusion per week for each selected dose to achieve sustained exposition throughout the treatment period. Based on this analysis, the Sponsor, in agreement with the SRC, defined 3 doses regimens that will be set up in parallel, with infusion of ABD-3001 once or twice a week during 3 cycles of 28 days.


Locations(3)

Hôpital de la Timone

Marseille, France, France

Hôpital Saint-Louis

Paris, France, France

Centre Hospitalier Lyon Sud

Pierre-Bénite, France, France

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NCT05601726


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