Clinical trial • Phase II/III • Oncology
Annamycin for Acute myeloid leukemia
Phase II/III trial of Annamycin for Acute myeloid leukemia.
Overview
- Trial Therapeutic Area
- Oncology
- Trial Disease
- Acute myeloid leukemia
- Trial Stage
- Phase II/III
- Drug Modality
- Small molecule
- Orphan Drug
- Yes
Key dates
- Initial CTIS Submission Date
- 20-12-2024
- First CTIS Authorization Date
- 30-04-2025
Trial design
Randomised, placebo (0.9% sodium chloride injection) administered as iv infusion for three consecutive days in combination with cytarabine injection 2.0 g/m2/day iv infusion for five consecutive days (comparator arm).-controlled, adaptive Phase II/III trial across 32 sites in Belgium, Czechia, France and others.
- Randomised
- Yes
- Comparator
- Placebo (0.9% Sodium Chloride Injection) administered as IV infusion for three consecutive days in combination with Cytarabine Injection 2.0 g/m2/day IV infusion for five consecutive days (comparator arm).
- Adaptive
- True, Part A uses an adaptive design: initial randomization 1:1:1 to three arms (placebo, 190 mg/m2/day L-Annamycin, 230 mg/m2/day L-Annamycin), with interim analyses to identify optimal dose and potential dropping of an arm and re-randomization/enrollment adjustments; planned blind breaks after interim assessments as described in protocol.
- Single Multiple Or Escalation Dose Combined
- Yes
- Target Sample Size
- 135
Eligibility
Recruits 135 No vulnerable populations selected (isVulnerablePopulationSelected: false). Participants must be adults (between 18 and 80 years) able to understand and sign the informed consent form (ICF). Consent is provided by the participant; no assent procedures for minors are described. Multiple country-specific ICF and SIS documents (including pregnancy-specific ICFs) are provided in several languages..
- Pregnancy Exclusion
- 9. Pregnant or breastfeeding
- Vulnerable Population
- No vulnerable populations selected (isVulnerablePopulationSelected: false). Participants must be adults (between 18 and 80 years) able to understand and sign the informed consent form (ICF). Consent is provided by the participant; no assent procedures for minors are described. Multiple country-specific ICF and SIS documents (including pregnancy-specific ICFs) are provided in several languages.
Inclusion criteria
- {"criterion_text":"- 1. Has a pathologically confirmed diagnosis of AML per the 2022 International Consensus Classification (ICC) (Arber et al. 2022) as adopted in the European LeukemiaNet (ELN) 2022 recommendations for the diagnosis and management of AML (Döhner et al. 2022). The tests and procedures used to establish the diagnosis of AML should be consistent with the ELN’s 2022 recommendations (i.e., Döhner et al. 2022 Table 4 and crossreferenced Table 5)\n- 10. For women of childbearing potential (WCBP): Must have a negative serum beta-human chorionic gonadotropin (ß-hCG) pregnancy test within 72 hours prior to the first randomized dose of study drug.\n- 11. For WCBP: Must agree to not donate ova and use a highly effective method of birth control from the time of informed consent through 6 months after their last randomized dose of study drug.\n- 12. For males with partners who are WCBP: Must agree to not donate sperm and use a highly effective method of birth control from the time of informed consent through 6 months after their last randomized dose of study drug.\n- 2. Has refractory/relapsed AML after having received only one prior line of therapy, as defined by the protocol\n- 3. Between 18 and 80 years of age (inclusive) at the time of signing the ICF\n- 4. Has received no chemotherapy, radiation, or major surgery within 2 weeks prior to the first randomized dose of study drug or has recovered from the toxic side effects of that therapy. Hydroxyurea to control white blood cell (WBC) count, supportive measures, and prophylaxes as required under the protocol will be allowed. Treatment of opportunistic or other infections with antibiotics, antifungals, and/or antiviral agents, including therapy for meningeal disease (i.e., intrathecal chemotherapy), per institutional standards of care will be allowed during this period, as long as the symptoms of infection have resolved by 1 week prior to the first dose of randomized study drug\n- 5. Has received no investigational therapy within 4 weeks prior to the first randomized dose of study drug\n- 6. Has an Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2 at screening.\n- 7. Has a life expectancy of greater than six weeks at screening\n- 8. Has adequate laboratory results at screening, as per protocol\n- 9. Can understand and sign the ICF, can communicate with the PI, and can understand and comply with the requirements of the protocol."}
Exclusion criteria
- {"criterion_text":"- 1. Has prior or current diagnosis of acute promyelocytic leukemia (APL) or MDS/AML (as defined in Döhner et al. 2022 Table 1).\n- 7. Has any condition that, in the opinion of the PI, places the subject at unacceptable risk if he/she were to participate in the study\n- 8. Has received prior treatment with L-asparaginase\n- 11. Has received a total cumulative prior anthracycline dose of > 300 mg/m2 (daunorubicin equivalent dose).\n- 9. Pregnant or breastfeeding\n- 12. Has relapsed or refractory AML with a FLT3 mutation, unless resides in a country where gilteritinib is not available\n- 10. Known hypersensitivity to anthracyclines, cytarabine, the excipients of L-Annamycin for Injection or Cytarabine Injection, or contrast media that may be used for the protocol specified GLS assessments\n- 2. Received prior mediastinal radiotherapy\n- 3. Has central nervous system involvement\n- 4. Has impaired cardiac function, as per protocol\n- 5. Has clinically relevant serious comorbid medical conditions including, but not limited to, active infection, chronic obstructive or chronic restrictive pulmonary disease, history of positive status for human immunodeficiency virus (virus detected in serum), hepatitis B, or hepatitis C with current serious symptoms or signs of underlying chronic infection, or psychiatric illness/social situations that would limit compliance with study requirements\n- 6. Has evidence of mucositis/stomatitis at screening or baseline, or has history of severe (≥Grade 3) mucositis/stomatitis from prior therapy"}
Endpoints
Primary endpoints
- {"endpoint_text":"- Rate of CR after one treatment cycle (35 days ± 14 days after initiation of randomized treatment)","definition_or_measurement_approach":"Complete remission (CR) rate assessed 35 days ± 14 days after initiation of randomized treatment"}
Secondary endpoints
- {"endpoint_text":"- DoR","definition_or_measurement_approach":"Duration of response (DoR)"}
- {"endpoint_text":"- OS","definition_or_measurement_approach":"Overall survival (OS)"}
- {"endpoint_text":"- Rate of subsequent transplantation (i.e., alloHSCT/autoHSCT) for subjects who achieve CR or CRh at 35 days ± 14 days after initiation of randomized treatment (CR + CRh)","definition_or_measurement_approach":"Rate of subsequent alloHSCT/autoHSCT among subjects achieving CR or CRh at Day 35 ± 14 days after initiation of randomized treatment"}
- {"endpoint_text":"- Pharmacokinetics of annamycin (Part A and Part B), annamycinol (Part A and Part B), and cytarabine (Part A).","definition_or_measurement_approach":"Plasma pharmacokinetic profiling of annamycin, annamycinol, and cytarabine per protocol-specified sampling in Parts A and B as applicable"}
- {"endpoint_text":"- Safety","definition_or_measurement_approach":"Safety assessments per protocol (adverse events, labs, vital signs, ECG etc.)"}
- {"endpoint_text":"- Tolerability","definition_or_measurement_approach":"Tolerability assessments per protocol (treatment discontinuations, dose modifications, adverse events)"}
Recruitment
- Planned Sample Size
- 135
- Recruitment Window Months
- 47
- Consent Approach
- Informed consent is obtained from each participant (must be able to understand and sign the ICF). Study uses Subject Information Sheets (SIS) and ICF documents; country- and language-specific versions are provided (documents available in English, French, Dutch/Netherlands (NL), Italian, Polish, Ukrainian and other country-specific translations). Pregnancy-specific ICFs are provided where applicable. No parental consent or assent provisions (study restricted to adults 18+).
Geography
- Total Number Of Sites
- 32
- Total Number Of Participants
- 177
Belgium
- Earliest CTIS Part Ii Submission Date
- 04-04-2025
- Latest Decision Or Authorization Date
- 23-02-2026
- Processing Time Days
- 325
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Institut Jules Bordet
- Department Name
- Hematology
- Contact Person Name
- Hanne Massa
- Contact Person Email
- hanne.massa@hubruxelles.be
Czechia
- Earliest CTIS Part Ii Submission Date
- 11-04-2025
- Latest Decision Or Authorization Date
- 24-02-2026
- Processing Time Days
- 319
- Number Of Sites
- 1
- Number Of Participants
- 10
Sites
- Site Name
- Fakultni Nemocnice Hradec Kralove
- Department Name
- IV. interní hematologická klinika
- Contact Person Name
- Benjamín Víšek
- Contact Person Email
- benjamin.visek@fnhk.cz
France
- Earliest CTIS Part Ii Submission Date
- 26-02-2025
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 366
- Number Of Sites
- 4
- Number Of Participants
- 20
Sites
- Site Name
- Hôpital Saint-Louis
- Department Name
- Hematology
- Contact Person Name
- Emmanuel RAFFOUX
- Contact Person Email
- Emmanuel.Raffoux@aphp.fr
- Site Name
- Institut De Cancerologie Strasbourg Europe
- Department Name
- Hematology
- Contact Person Name
- Célestine SIMAND
- Contact Person Email
- c.simand@icans.eu
- Site Name
- CHU Angers
- Department Name
- Hematology
- Contact Person Name
- Mathilde HUNAULT
- Contact Person Email
- MaHunault@chu-angers.fr
- Site Name
- Hôpital Michallon (CHU Grenoble)
- Department Name
- Hematology
- Contact Person Name
- Martin CARRE
- Contact Person Email
- MCarre1@chu-grenoble.fr
Germany
- Earliest CTIS Part Ii Submission Date
- 15-04-2025
- Latest Decision Or Authorization Date
- 23-02-2026
- Processing Time Days
- 314
- Number Of Sites
- 2
- Number Of Participants
- 15
Sites
- Site Name
- Universitaet Rostock
- Department Name
- Medizinische Klinik III für Hämatologie, Onkologie und Palliativmedizin
- Contact Person Name
- Christian Junghanß
- Contact Person Email
- christian.junghanss@med.uni-rostock.de
- Site Name
- Staedtisches Klinikum Braunschweig gGmbH
- Department Name
- Medizinische Klinik III, Hämatologie und Onkologie
- Contact Person Name
- Jürgen Krauter
- Contact Person Email
- j.krauter@skbs.de
Italy
- Earliest CTIS Part Ii Submission Date
- 04-04-2025
- Latest Decision Or Authorization Date
- 24-02-2026
- Processing Time Days
- 326
- Number Of Sites
- 7
- Number Of Participants
- 30
Sites
- Site Name
- Azienda Ospedaliero Universitaria Careggi
- Department Name
- SOD Ematologia
- Contact Person Name
- Matteo Piccini
- Contact Person Email
- piccinim@aou-careggi.toscana.it
- Site Name
- Istituto Romagnolo Per Lo Studio Dei Tumori Dino Amadori IRST S.r.l.
- Department Name
- Hematology
- Contact Person Name
- Irene Zacheo
- Contact Person Email
- Irene.zacheo@irst.emr.it
- Site Name
- Azienda Ospedaliero-Universitaria Di Bologna IRCCS Istituto Di Ricerca E Di Cura A Carattere Scientifico
- Department Name
- UOC Ematologia
- Contact Person Name
- Cristina Papayannidis
- Contact Person Email
- cristina.papayannidis@unibo.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- UOC Ematologia e Trapianti di CSE
- Contact Person Name
- Simona Sica
- Contact Person Email
- simona.sica@unicatt.it
- Site Name
- Azienda Unita Sanitaria Locale Della Romagna
- Department Name
- Onco-Ematologia
- Contact Person Name
- Giovanni Marconi
- Contact Person Email
- giovanni.marconi@unibo.it
- Site Name
- Humanitas Mirasole S.p.A.
- Department Name
- Ematologia
- Contact Person Name
- Matteo Giovanni Della Porta
- Contact Person Email
- matteo.della_porta@hunimed.eu
- Site Name
- Azienda Ospedaliero Universitaria Careggi (additional listed site entries aggregated)
Lithuania
- Earliest CTIS Part Ii Submission Date
- 09-04-2025
- Latest Decision Or Authorization Date
- 30-03-2026
- Processing Time Days
- 355
- Number Of Sites
- 1
- Number Of Participants
- 7
Sites
- Site Name
- Hospital of Lithuanian University of Health Sciences Kauno Klinikos
- Department Name
- Oncology and Hematology
- Contact Person Name
- Domas Vaitiekus
- Contact Person Email
- rastine@kaunoklinikos.lt
Romania
- Earliest CTIS Part Ii Submission Date
- 30-01-2025
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 393
- Number Of Sites
- 4
- Number Of Participants
- 15
Sites
- Site Name
- Institute Of Oncology Prof. Dr. Ion Chiricuta Cluj-Napoca
- Department Name
- Haematology
- Contact Person Name
- Ciprian Tomuleasa
- Contact Person Email
- ionut.tomuleasa.ext@arensia-em.com
- Site Name
- Coltea Clinical Hospital
- Department Name
- Oncology
- Contact Person Name
- Gabriela Borsaru
- Contact Person Email
- gabriex2001@yahoo.it
- Site Name
- Spitalul Clinic Municipal De Urgenta Timisoara
- Department Name
- Haematology
- Contact Person Name
- Ioana Ionita
- Contact Person Email
- mdioanaionita@yahoo.com
- Site Name
- Spitalul Clinic Municipal Filantropia Craiova
- Department Name
- Haematology
- Contact Person Name
- Luminita Ocroteala
- Contact Person Email
- diaconu_luminita@yahoo.com
Spain
- Earliest CTIS Part Ii Submission Date
- 08-04-2025
- Latest Decision Or Authorization Date
- 27-02-2026
- Processing Time Days
- 325
- Number Of Sites
- 5
- Number Of Participants
- 30
Sites
- Site Name
- Hospital Universitario Central De Asturias
- Department Name
- Hematology
- Contact Person Name
- Teresa Bernal del Castillo
- Contact Person Email
- bernalmaria@uniovi.es
- Site Name
- Institut Catala D'oncologia
- Department Name
- Hematology
- Contact Person Name
- Daniel Esteban Corredera
- Contact Person Email
- danielesteban@iconcologia.net
- Site Name
- MD Anderson Cancer Center (Spain)
- Department Name
- Hematology
- Contact Person Name
- Adolfo de la Fuente Burguera
- Contact Person Email
- afuente@mdanderson.es
- Site Name
- Hospital Universitario Y Politecnico La Fe
- Department Name
- Hematology
- Contact Person Name
- Pau Montesinos Fernandez
- Contact Person Email
- montesinos_pau@gva.es
- Site Name
- Hospital Universitario Ramon Y Cajal
- Department Name
- Hematology
- Contact Person Name
- Pilar Herrera Puente
- Contact Person Email
- pherrera.hrc@salud.madrid.org
Poland
- Earliest CTIS Part Ii Submission Date
- 11-04-2025
- Latest Decision Or Authorization Date
- 24-02-2026
- Processing Time Days
- 319
- Number Of Sites
- 7
- Number Of Participants
- 40
Sites
- Site Name
- Instytut Hematologii I Transfuzjologii
- Department Name
- Klinika Hematologii
- Contact Person Name
- Ewa Lech-Marańda
- Contact Person Email
- emaranda@ihit.waw.pl
- Site Name
- Pratia Hematologia Sp. z o.o.
- Department Name
- Pratia Onkologia
- Contact Person Name
- Sebastian Grosicki
- Contact Person Email
- kontakt.onkologia.katowice@pratia.com
- Site Name
- Uniwersytecki Szpital Kliniczny W Bialymstoku
- Department Name
- Klinika Hematologii, Ch. Wewn. i Angiologii z Pododdziałem Transplantacji Komórek Krwiotwórczych
- Contact Person Name
- Jarosław Piszcz
- Contact Person Email
- jaroslaw.piszcz@umb.edu.pl
- Site Name
- Uniwersytecki Szpital Kliniczny W Poznaniu
- Department Name
- Oddział Hematologii i Transplantacji Szpiku
- Contact Person Name
- Lidia Gil
- Contact Person Email
- lidia.gil@usk.poznan.pl
- Site Name
- Wojewodzki Szpital Zespolony Im.L.Rydygiera W Toruniu
- Department Name
- Oddział Hematologii
- Contact Person Name
- Dominik Chraniuk
- Contact Person Email
- dominik.chraniuk@wszz.torun.pl
- Site Name
- Uniwersytecki Szpital Kliniczny Nr 1 Im. Prof. Tadeusza Sokolowskiego Pum W Szczecinie
- Department Name
- Klinika Hematologii i Transplantologii
- Contact Person Name
- Bogusław Machaliński
- Contact Person Email
- boguslaw.machalinski@pum.edu.pl
- Site Name
- Szpital Kliniczny Ministerstwa Spraw Wewnetrznych I Administracji Z Warminsko-Mazurskim Centrum Onkologii W Olsztynie
- Department Name
- Oddział Kliniczny Hematologii i Chorób Wewnętrznych z Ośrodkiem Transplantacji Szpiku
- Contact Person Name
- Janusz Hałka
- Contact Person Email
- janusz.halka@poliklinika.net
Sponsor
Primary sponsor
- Full Name
- Moleculin Biotech Inc.
- Organisation Type
- Pharmaceutical company
- Country Of Registered Address
- United States
Contract research organisations
- Name
- Catalyst Clinical Research LLC
- Responsibilities
- sponsorDuties codes: 1,12,5,6
Third parties
- {"country":"United States","full_name":"Catalyst Clinical Research LLC","duties_or_roles":"sponsorDuties codes: 1,12,5,6","organisation_type":"Laboratory/Research/Testing facility"}
Investigational products
- Investigational Product Name
- Liposomal Annamycin
- Active Substance
- Annamycin
- Modality
- Small molecule
- Routes Of Administration
- IV infusion
- Route
- IV infusion
- Orphan Designation
- Yes
- Starting Dose
- 190 mg/m2/day (one of two compared doses)
- Dose Levels
- 190 mg/m2/day; 230 mg/m2/day
- Frequency
- Three consecutive days
- Maximum Dose
- 230 mg/m2/day (max daily); max total 1380 mg/m2
- Dose Escalation Increase
- 190 mg/m2/day and 230 mg/m2/day
- Investigational Product Name
- Cytarabine
- Active Substance
- Cytarabine
- Modality
- Small molecule
- Routes Of Administration
- IV infusion
- Route
- IV infusion
- Starting Dose
- 2.0 g/m2/day
- Dose Levels
- 2.0 g/m2/day
- Frequency
- Daily for five consecutive days
- Maximum Dose
- 2 g/m2/day (daily); max total 20 gm/m2 per protocol fields
- Investigational Product Name
- 0.9% Sodium Chloride Injection
- Modality
- Other
- Routes Of Administration
- IV infusion (placebo/diluent)
- Route
- IV infusion
- Frequency
- Three consecutive days (placebo matched to L-Annamycin schedule)
- Combination Treatment
- Yes
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