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Acute Myeloid Leukemia, AdultPhase 1/2ODYSSEYNCT05601726

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

A Phase 1/2 study testing ABD-3001 in people with Acute Myeloid Leukemia, Adult and Myelodysplastic Syndromes, run by Advanced BioDesign at 3 sites in France.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial 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. Read more about this study

Who is it for?
Ages 18 years and older
What is being tested?
ABD-3001
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
36 participants
Where
3 sitesFrance
Study dates
8 Nov 2022to Dec 2026
Phase
Phase 1/2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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

Who cannot join

  • 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
Show all 17Show fewer
  • 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.

Trial locations and contacts

3 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Guillaume MARTIN contact

+33 (4) 82 53 89 62

guillaume.martin@a-biodesign.com

Laurent BASSET contact

+33 (0)4 28 29 46 78

laurent.basset@a-biodesign.com

France3 sites, 3 recruiting

  • Hôpital de la Timone

    Marseille, France, 13005

    Régis COSTELLO, regis.costello@ap-hm.fr

    Recruiting

  • Hôpital Saint-Louis

    Paris, France, 75475

    Lina BENAJIBA, lina.benajiba@aphp.fr

    Recruiting

  • Centre Hospitalier Lyon Sud

    Pierre-Bénite, France, 69495

    Maël HEIBLIG, mael.heiblig@chu-lyon.fr

    Recruiting

Other trials in France

About this study

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.

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.

Read the detailed description

This FIH study combines, in patients with primary refractory or relapsed AML patients and in patients with high risk MDS a Single Ascending Dose (SAD) part (Part A) and a Multiple Ascending Dose (MAD) part (Part B).

The objective of the SAD phase is to explore a wide range of dose administered as a single and fixed 4-hours intravenous infusion in order to select a dose and a dosing frequency (determined using pharmacokinetic and pharmacodynamics parameters).

The objective of the MAD is to elucidate the pharmacokinetic (PK) and pharmacodynamics (PD) of multiple doses of ABD-3001. The dose levels and dosing intervals (i.e., time between consecutive doses) will be selected as those that are predicted to be safe from the SAD. Dose levels and dosing frequency will be derived from data obtained during the SAD.

Being tested
ABD-3001
Keywords
Acute Myeloid Leukemia, Adult, Myelodysplastic syndromes, First in Human, ABD-3001
Medical subject headings
Leukemia, Myeloid, Acute, Myelodysplastic Syndromes

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ABD-3001Drug

    Also called DIMATE

    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.

  • ABD-3001Drug

    Also called DIMATE

    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.

Study groups in NCT05601726
Study groupRoleWhat they receive
Single Administration Dose (SAD) of ABD-3001ExperimentalDose escalation of 6 doses level using a 3+3 design.
Receives: Drug: ABD-3001
Multiple Administration Dose (MAD) of ABD-3001Experimental3 doses regimens in parallel during 3 cycles of 28 days
Receives: Drug: ABD-3001

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. SAD : Estimation of the Maximum Tolerated Dose (MTD) of ABD-3001 as well as the doses and frequencies to be explored during the MAD

    The primary endpoint is to assess the recommended dose range to be tested during the MAD by evaluation of incidence of patients who experienced Dose Limiting Toxicities (DLTs). according to CTCAE v5.0.

    The recommendation for doses to be tested during the MAD will be determined using all available data, which will include Dose Limiting Toxicities (DLTs), MTD if it has been reached, and other toxicities, signs of anti-leukemic activity, pharmacokinetic and pharmacodynamic characteristics.

    Measured: 7 days for SAD part

  2. MAD : To evaluate the safety, tolerability and to define a recommended dose range for further trials.

    To assess the impact of ABD-3001 on safety and on its tolerability by evaluating:

    1. Incidence of Adverse Events as characterized by type, frequency, severity (as graded by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0 Nov. 27, 2017)), timing, seriousness, and relationship to ABD-3001 either observed by the investigator during physical examination, or reported spontaneously by the patient,
    2. Incidence of laboratory abnormalities as characterized by type, frequency, severity (as graded by NCI CTCAE v5.0 Nov. 27, 2017), and timing.

    Measured: 3 months for MAD part.

Secondary outcomes (15)

  1. SAD : To evaluate the overall safety and tolerability profile of ABD-3001

    To assess the impact of ABD-3001 on safety and on its tolerability by evaluating:

    1. Incidence of Adverse Events as characterized by type, frequency, severity (as graded by National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0 Nov. 27, 2017)), timing, seriousness, and relationship to ABD-3001 either observed by the investigator during physical examination, or reported spontaneously by the patient,
    2. Incidence of laboratory abnormalities as characterized by type, frequency, severity (as graded by NCI CTCAE v5.0 Nov. 27, 2017), and timing,

    Measured: 7 days for SAD part

  2. SAD: To assess the pharmacokinetic (PK) parameters of ABD-3001

    Maximum concentration (Cmax) of DIMATE and metabolites (CEY1410/H11)

    Measured: 7 days for SAD part

  3. SAD: To assess the pharmacokinetic (PK) parameters of ABD-3001

    Time to maximum concentration (Tmax)

    Measured: 7 days for SAD part

  4. SAD: To assess the pharmacokinetic (PK) parameters of ABD-3001

    Area Under the plasma Concentration versus time curve (AUC)

    Measured: 7 days for SAD part

  5. SAD: To assess the pharmacokinetic (PK) parameters of ABD-3001

    Elimination half-life.

    Measured: 7 days for SAD part

  6. MAD : 1. To assess the pharmacokinetic (PK) parameter of multiple administration of ABD-3001.

    PK parameter assessment: maximum concentration (Cmax) of DIMATE and metabolites (CEY1410/H11)

    Measured: 3 months for MAD part.

  7. MAD : 2. To assess the pharmacokinetic (PK) parameter of multiple administration of ABD-3001.

    PK parameter assessment: time to maximum concentration (Tmax)

    Measured: 3 months for MAD part.

  8. MAD : 3. To assess the pharmacokinetic (PK) parameter of multiple administration of ABD-3001.

    PK parameter assessment: Area Under the plasma Concentration versus time curve (AUC)

    Measured: 3 months for MAD part.

  9. MAD : 4. To assess the pharmacokinetic (PK) parameter of multiple administration of ABD-3001.

    PK parameter assessment: elimination half-life.

    Measured: 3 months for MAD part.

  10. MAD : 1. To assess the pharmacodynamic (PD) parameter of multiple administration of ABD-3001.

    PD parameter assessment: ALDH activity measurement in red blood cells

    Measured: 3 months for MAD part.

  11. MAD : 2. To assess the pharmacodynamic (PD) parameter of multiple administration of ABD-3001.

    PD parameter assessment: JUNK protein phosphorylation in plasma.

    Measured: 3 months for MAD part.

  12. MAD : To evaluate the anti-leukemic activity as assessed by overall response rate (ORR: complete response [CR] + complete response with incomplete hematopoiesis [CRi] + morphologic leukemia-free state [MFLS] + partial response [PR])

    To assess the potential preliminary evidence of anti-leukemia activity at each cycle using response criteria in AML without MRD assessment as defined by ELN 2022

    Measured: 3 months for MAD part.

  13. MAD: To evaluate the duration of ORR.

    To assess duration of ORR.

    Measured: 3 months for MAD part.

  14. MAD: To evaluate Event free survival (EFS) and overall survival (OS) rates at 3-months and 6-months after the end of treatment

    To assess the Event Free Survival and the Overall Survival as defined by ELN2022 until 6-months after end of treatment.

    Measured: 3 months for MAD part.

  15. MAD: To assess the PRO-QoL during the treatment.

    To assess the impact of ABD-3001 on quality of life according QLQ-C30 questionnaire at D1 of each cycle and at the end of treatment.

    Measured: 3 months for MAD part.

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted1 Nov 2022
  2. Study start8 Nov 2022
  3. Primary completionJun 2026 (estimated)
  4. Study completionDec 2026 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1/2
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Sequential
Model description
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.
Masking
None (open label)
Enrolment
36 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05601726
Sponsor's protocol ID
ABD3001CLIN1
Acronym
ODYSSEY
Status last verified
Jan 2025
First submitted
17 Oct 2022
Last updated
20 Jan 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Publications

  • Venton G, Colle J, Tichadou A, Quessada J, Baier C, Labiad Y, Perez M, De Lassus L, Loosveld M, Arnoux I, Abbou N, Ceylan I, Martin G, Costello R. Reactive oxygen species and aldehyde dehydrogenase 1A as prognosis and theragnostic biomarker in acute myeloid leukaemia patients. J Cell Mol Med. 2024 Oct;28(19):e70011. doi: 10.1111/jcmm.70011.

    PubMed 39392121

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing ABD-3001 in people with Acute Myeloid Leukemia, Adult and Myelodysplastic Syndromes, run by Advanced BioDesign at 3 sites in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 17 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 20 Jan 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 3 sites in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Guillaume MARTIN (+33 (4) 82 53 89 62, guillaume.martin@a-biodesign.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

SAD : Estimation of the Maximum Tolerated Dose (MTD) of ABD-3001 as well as the doses and frequencies to be explored during the MAD (time frame: 7 days for SAD part). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT05601726, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Jan 2025.

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