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Leukemia, Myeloid, AcutePhase 1/2NCT05319587

Study of Liposomal Annamycin in Combination With Cytarabine for the Treatment of Subjects With Acute Myeloid Leukemia (AML)

A Phase 1/2 study testing Liposomal Annamycin and Cytarabine in people with Leukemia, Myeloid, Acute, run by Moleculin Biotech, Inc. at 9 sites in 2 countries.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

This is a Phase 1/2, multicenter, open-label, dose-escalation study that will determine the MTD and RP2D of L-Annamycin in combination with cytarabine for the treatment of subjects with AML. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Leukemia, Myeloid, Acute
What is being tested?
Liposomal Annamycin and Cytarabine
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
22 participants
Where
9 sitesin 2 countries
Study dates
29 Sep 2022to 10 Aug 2023
Phase
Phase 1/2
Are there results?
None posted in the registry

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

  • The subject has a pathologically confirmed diagnosis of AML by World Health Organization classification. This must be in the form of either a bone marrow aspirate or biopsy or a CBC that demonstrates >5% myeloblasts.
  • The subject has AML and has not received prior therapy or is refractory to or relapsed after induction therapy. To be defined as relapse, there must be >5% blasts in the bone marrow.
  • For the expansion phase only (i.e., after the MTD/RP2D is established), subjects must be treated with L-Annamycin as first- second- or third-line therapy (i.e., subjects will not have received more than two prior therapies).
  • The subject is age ≥18 years at the time of signing informed consent.
  • The subject has received no chemotherapy, radiation, or major surgery within 2 weeks prior to first dose of study drug and/or has recovered from the toxic side effects of that therapy unless treatment is indicated as a result of progressive disease, such as hydroxyurea.
  • The subject has received no investigational therapy within 4 weeks of the first dose of study drug.
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  • The subject has an Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2.
  • The subject has adequate laboratory results including the following:
  • Bilirubin ≤2 times the upper limit of normal unless due to Gilbert Syndrome or leukemic infiltration of the liver.
  • Serum glutamic-oxaloacetic transaminase (SGOT), serum glutamic-pyruvic transaminase (SGPT), and alkaline phosphatase <2.5 times the upper limit of normal unless due to organ involvement.
  • Adequate renal function with creatinine levels ≤2 times the upper limit of normal.
  • The subject can understand and sign the informed consent document, can communicate with the Investigator, and can understand and comply with the requirements of the protocol.
  • Women of childbearing potential must have a negative serum or urine beta-human chorionic gonadotropin test within 72 hours prior to first dose of study drug to rule out pregnancy.
  • All men and women must agree to practice effective contraception during the entire study period and after discontinuing study drug, unless documentation of infertility exists.
  • Sexually active, fertile women must use 2 effective forms of contraception (abstinence, intrauterine device, oral contraceptive, or double barrier device) from the time of informed consent until at least 6 months after discontinuing study drug.
  • Sexually active men and their sexual partners must use effective contraceptive methods from the time of subject informed consent until at least 3 months after discontinuing study drug.

Who cannot join

  • The subject was diagnosed with acute promyelocytic leukemia.
  • The subject is receiving concomitant therapy that includes other chemotherapy that is or may be active against AML except for agents such as hydroxyurea, just to control the WBC count until chemotherapy or prophylaxis and/or treatment of opportunistic or other infection with antibiotics, antifungals, and/or antiviral agents, including therapy for meningeal disease (i.e., intrathecal chemotherapy), supportive measures, and medications as per standard of care up to Day 1 of L-Annamycin administration.
  • The subject received prior mediastinal radiotherapy.
  • The subject has central nervous system involvement.
  • The subject has any condition that, in the opinion of the Investigator, places the subject at unacceptable risk if he/she were to participate in the study.
  • The subject has an LVEF <50%, valvular heart disease, or severe hypertension. Cardiac subjects with a New York Heart Association classification of 3 or 4 will be excluded. (Cardiology consultation should be requested if any question arises about cardiac function). This also includes subjects with baseline QT/QTc interval >480 msec, a history of additional risk factors for torsade des pointes (e.g., heart failure, hypokalemia, family history of Long QT Syndrome), and use of concomitant medications that significantly prolong the QT/QTc interval.
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  • The subject has clinically relevant serious comorbid medical conditions including, but not limited to, active infection, recent (less than or equal to 6 months) myocardial infarction, unstable angina, symptomatic congestive heart failure, uncontrolled hypertension, uncontrolled cardiac arrhythmias, chronic obstructive or chronic restrictive pulmonary disease, known positive status for human immunodeficiency virus and/or active hepatitis B or C, cirrhosis, or psychiatric illness/social situations that would limit compliance with study requirements.
  • Subjects with a documented COVID diagnosis within 14 days of screening must have a documented negative PCR test and remain asymptomatic for 14 days from that test result before starting study medication.
  • The subject is pregnant, lactating, or not using adequate contraception.
  • The subject has a known allergy to anthracyclines and/or hypersensitivity to cytarabine, its excipients, or to any contrast media needed for imaging required per protocol.
  • The subject has any evidence of mucositis/stomatitis at the time of study entry or previous history of severe (≥Grade 3) mucositis from prior therapy.
  • The subject is required to use moderate or strong inhibitors and inducers of Cytochrome P450 family of enzymes (CYP) and transporters that cannot be held for 3 days prior to Day 1 and during treatment days

Trial locations and contacts

9 sites in 2 countries.

9 sites in 2 countries

Poland6 sites

  • Apteka Szpitalna

    Gdansk

  • SP ZOZ MsWiA z Warminsko-Mazurksim Centrum Onkologii w Olsztynie Oddzial Kliniczny Hematologii

    Olsztyn, Warmian-Masurian Voivodeship, 10-228

  • Szpital Kliniczny Przemienienia Pańskiego Uniwersytetu Medycznego im. Karola Marcinkowskiego

    Poznan

  • Samodzielny Publiczny Szpital Kliniczny nr 1 im. Prof. Tadeusza Sokołowskiego w Szczecinie, Klinika Hematologii z Oddziałem Transplantacji Szpiku, Szczecin

    Szczecin

  • Instytut Hematologii i Transfuzjologii, Klinika Hematologii, Warsaw

    Warsaw

  • Uniwersytecki Szpital Kliniczny Klinika Hematologii Nowotworow

    Wroclaw, Dolnośląskie Województwo, 50-367

Other trials in Poland

Italy3 sites

  • IRCCS Azienda Ospedaliero-Universitaria di Bologna

    Bologna, 40138

  • Istituto Scientifico Romagolo per lo studio dei umori "Dino Amadori" (IRST) - IRCCS

    Meldola, FC, 47014

  • Fondazione Policlinico Universitario A. Gemelli IRCCS

    Roma

Other trials in Italy

About this study

Official title: Phase 1/2 Study of Liposomal Annamycin in Combination With Cytarabine for the Treatment of Subjects With Acute Myeloid Leukemia (AML)

This is a Phase 1/2, multicenter, open-label, dose-escalation study that will determine the MTD and RP2D of L-Annamycin in combination with cytarabine for the treatment of subjects with AML.

Read the detailed description

During Phase 1b, dose escalation will proceed according to a standard 3+3 design. An initial group of 3 subjects will be treated with an intravenous infusion of 190-mg/m2 L-Annamycin (Dose Level 1) for 3 consecutive days. Dose escalation will take place on the basis of safety assessments in sequential cohorts of 3 subjects each. Provided that there are no DLT's in the initial cohort of subjects treated at the 190 mg/m2/day dose level, the next cohort of subjects will receive the next highest L-Annamycin dose incremented by 40 mg/m2. In the absence of DLTs, dose escalation by 40 mg/m2 will continue in subsequent cohorts until an MTD is reached.

The dose and schedule of cytarabine during Cycle 1 (2.0 g/m2/day for 5 consecutive days) will remain constant for all cohorts. Enrolled subjects will receive cytarabine at a dose of 2.0 g/m2/day via intravenous infusion over 4 hours for 5 consecutive days (total dose cytarabine = 10.0 g/m2; starting on the first day of L-Annamycin treatment).

If no subject experiences a dose-limiting toxicity (DLT), based on National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5, which constitutes a study drug-related irreversible or not medically manageable Grade 3 or higher toxicity during the initial treatment through Day 28, the subsequent group of 3 subjects will receive the next higher L-Annamycin dose. However, if 1 of the 3 initial subjects experiences a DLT, the cohort of subjects at that dose level will be expanded to 6. If at least 2 of the 6 subjects experience a DLT, this will be considered a non-tolerated dose and the next 3 subjects will be treated at a lower dose. The dose of L-Annamycin will be de-escalated by 20- mg/m2/day. As such, if at least 2 out of 6 subjects receiving 230 mg/m2/day experience a DLT, the next 3 subjects will receive L-Annamycin at a dose of 210 mg/m2/day. If 1 of the 3 initial subjects experiences a DLT, the cohort of subjects will be expanded to 6 subjects. If at least 2 of the 6 subjects experience a DLT, this will be considered a toxic dose and the MTD will be the previously proven safe dose level (i.e., the next lowest dose level at which no DLTs occurred in a cohort of 3 subjects or at which fewer than 2 subjects in a cohort of 6 experienced a DLT).

In the expansion phase, up to 21 subjects will be enrolled at the MTD/RP2D to better define toxicity and evaluate efficacy.

Being tested
Liposomal Annamycin, Cytarabine
Medical subject headings
Leukemia, Myeloid, Acute

What participants receive

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

  • Liposomal AnnamycinDrug

    2-hour intravenous infusion liposomal annamycin daily for 3 consecutive days followed by 18 days off study drug (i.e., one treatment cycle = 21 days).

  • CytarabineDrug

    Administered during cycle 1 at a dose of 2.0 g/m2/day by 4 hours IV infusion for 5 consecutive days and this dose will remain constant for all cohorts, including the expansion phase.

Study groups in NCT05319587
Study groupRoleWhat they receive
Liposomal annamycinExperimentalNot described
Receives: Drug: Liposomal Annamycin; Drug: Cytarabine

What is being measured

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

  1. Evaluate the safety and identify the MTD/RP2D of L-Annamycin in combination with a standard regimen of cytarabine (Ara-C, cytosine arabinoside)

    The number of patients who experience dose-limiting toxicities (DLT) will be captured at each dose level of LAnnamycin in order to determine the MTD/RP2D

    Measured: Day 1 through Day 28

Secondary outcomes (2)

  1. Pharmacokinetics - Area under the plasma concentration

    Area under the plasma concentration - time curve (AUC) of annamycin and its metabolite, annamycinol, and of cytarabine, when administered.

    Measured: Pre-dose and at 0.25, 0.5, 1, 2, 4, 8,12 and 24 hours after the start of liposomal annamycin infusion on Day 1 and Day 3

  2. Anti-leukemic activity

    Determined by acute myeloid leukemia (AML) response rate based on the International Working Group (IWG) Response Criteria in AML (Cheson, 2003). Leukemia response rate will be evaluated by the investigator at the end of each L-Annamycin cycle based on bone marrow aspirate and peripheral blood evaluations.

    Measured: 15-35 Days after the start of therapy

Timeline

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

  1. First posted8 Apr 2022
  2. Study start29 Sep 2022
  3. Primary completion2 Aug 2023
  4. Study completion10 Aug 2023

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
Not applicable
Intervention model
Sequential
Masking
None (open label)
Enrolment
22 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05319587
Sponsor's protocol ID
MB-106
Other IDs
2020-005493-10
Status last verified
Sep 2025
First submitted
22 Feb 2022
Last updated
30 Sep 2025
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing Liposomal Annamycin and Cytarabine in people with Leukemia, Myeloid, Acute, run by Moleculin Biotech, Inc. at 9 sites in 2 countries.

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 16 inclusion and 12 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: finished, as of the sponsor's last update on 30 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in Poland, Italy. 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?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

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

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Evaluate the safety and identify the MTD/RP2D of L-Annamycin in combination with a standard regimen of cytarabine (Ara-C, cytosine arabinoside) (time frame: Day 1 through Day 28). 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 NCT05319587.

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 30 Sep 2025.

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