Clinical trial · Interventional
A Study of RO5503781 as a Single Agent or in Combination With Cytarabine in Participants With Acute Myelogenous Leukemia
A Multi-Center, Open-Label, Phase 1/1b Study of Escalating Doses of RO5503781 Administered Orally as 1) a Single Agent, 2) In Combination With Cytarabine, or 3) With Cytarabine and Anthracycline and 4) Assessing PK and Safety of New Optimized Formulation of RO5503781 With Cytarabine in Patients With Acute Myelogenous Leukemia (AML)
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
This Phase 1/1b, open-label study will evaluate the safety and pharmacokinetics of escalating doses of RO5503781 as a single agent or in combination with cytarabine in participants with acute myelogenous leukemia. In Part 1, RO5503781 will be administered in escalating doses as a single agent, and in Part 2, RO5503781 will be administered in combination with cytarabine. An optional Part 3 in which RO5503781 will be administered with cytarabine and anthracycline may be considered . In Part 4, the safety and pharmacokinetic profile of an optimized formulation of RO5503781 in combination with cytarabine will be assessed.
Conditions
Conditions (1)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Myelogenous Leukemia, Acute | Acute Myeloid Leukemia | ALIAS | 0.90 |
Interventions
Interventions (5)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Cytarabine | Drug | Cytarabine | ALIAS |
| Daunorubicin | Drug | Daunorubicin | ALIAS |
| Idarubicin | Drug | Idarubicin | ALIAS |
| RO5503781 MBP | Drug | — | UNRESOLVED |
| RO5503781 SDP | Drug | — | UNRESOLVED |
Design
Arms and outcomes
Arms (4)
- type
- EXPERIMENTAL
- label
- Part 1: RO5503781
- description
- Participants will receive RO5503781 alone in escalating doses on Days 1 to 5 of each 28-day cycle until disease progression or unacceptable toxicity.
- interventionNames
- Drug: RO5503781 MBP
- type
- EXPERIMENTAL
- label
- Part 2: RO5503781 + Cytarabine
- description
- Participants will receive RO5503781 in escalating doses on Days 1 to 5 and cytarabine on Days 1 to 6 of each 28-day cycle until disease progression or unacceptable toxicity.
- interventionNames
- Drug: RO5503781 MBP
- Drug: Cytarabine
- type
- EXPERIMENTAL
- label
- Part3:RO5503781+Cytarabine+Anthracycline
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Documented/confirmed acute myelogenous leukemia (AML), except for acute promyelocytic leukemia * Eastern Cooperative Oncology Group (ECOG) performance status 0 to 2 in Part 1 and Part 2, participants enrolled in the extension/tail portion, Part 3 and Part 4 must have an ECOG performance status of 0 or 1 * All non-hematological adverse events of any prior chemotherapy, surgery or radiotherapy must have resolved to National Cancer Institute-Common Terminology Criteria for Adverse Events (NCI-CTCAE) Grade less than or equal to (\</=) 2 prior to starting therapy * Adequate hepatic and renal function * Willing to submit the blood sampling and bone marrow sampling required by protocol Additional inclusion criteria for Parts 1-4 may apply. Exclusion Criteria: * Participants receiving any other investigational or commercial agents or therapies administered with the intention to treat their malignancy within 14 days of first receipt of study drug, with the exception of hydroxyurea as defined in protocol * History of allergic or toxic reactions attributed to cytarabine (Part 2) or history of allergic reactions to components of the formulated product * Current evidence of central nervous system (CNS) leukemia * Participants with severe and/or uncontrolled medical conditions or other conditions that could affect their participation in the study * Participants with evidence of electrolyte imbalance greater than or equal to (\>/=) Grade 2 which cannot be corrected prior to study initiation * Pregnant or breastfeeding women * Human immunodeficiency virus (HIV) - positive participants receiving anti-retroviral therapy * Participants who refuse to potentially receive blood products and/or have a hypersensitivity to blood products Additional exclusion criteria for Parts 1-4 may apply.
References
Publications (1)
- DERIVEDYee K, Papayannidis C, Vey N, Dickinson MJ, Kelly KR, Assouline S, Kasner M, Seiter K, Drummond MW, Yoon SS, Lee JH, Blotner S, Jukofsky L, Pierceall WE, Zhi J, Simon S, Higgins B, Nichols G, Monnet A, Muehlbauer S, Ott M, Chen LC, Martinelli G. Murine double minute 2 inhibition alone or with cytarabine in acute myeloid leukemia: Results from an idasanutlin phase 1/1b study small star, filled. Leuk Res. 2021 Jan;100:106489. doi: 10.1016/j.leukres.2020.106489. Epub 2020 Dec 1. PMID 33302031