Experimental combo therapy for blood cancers shows early promise but trial halted
NCT ID NCT03940352
First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This early-stage trial tested two new drug combinations in 52 adults with acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS). One group received HDM201 plus MBG453, the other HDM201 plus venetoclax. The main goal was to check safety and find the right dose. The study was terminated early, so full results are not available.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- HDM201 combined with either MBG453 or venetoclax
- What this could lead to
- If successful, this could point toward new combination treatments for AML and high-risk MDS.
- What could go wrong
- This was a very early (phase 1) trial that was terminated, so results are limited. The combinations may cause serious side effects and may not work for everyone.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
-
52 people
The number who actually took part.
- Started
-
Jun 2019
- Finished
-
Aug 2024
- Lead sponsor
-
A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Main Inclusion Criteria: * Male or female patients ≥ 18 years of age at the date of ICF signature who present with one of the following: 1. Relapsed/refractory AML following ≥1 prior therapies (but ≤3 prior therapies) who have relapsed or exhibited refractory disease (primary failure) and are deemed by the Investigator not to be candidates for standard therapy, including re-induction with cytarabine or other established chemotherapy regimens for patients with AML (patients who are suitable for standard re-induction chemotherapy or hematopoietic stem cell transplantation and willing to receive it are excluded) 2. First line AML patient unfit for standard induction chemotherapy (includes both de novo and secondary AML), except in countries where approved therapies are available. Patients who are suitable for hematopoietic stem cell transplantation and willing to receive it are excluded. 3. High-risk MDS patient (high and very high-risk groups according to rIPSS) who have failed hypomethylating agent therapy. * ECOG performance status ≤ 1 * TP53wt tumor. At minimum exons 5, 6, 7 and 8 in the TP53 gene must be sequenced and determined to contain no mutations. The TP53 status must be obtained from a bone-marrow sample, collected no longer than 3 months before signing the main ICF. * Patient must be a candidate for serial bone marrow aspirate and/or biopsy according to the institutional guidelines and be willing to undergo a bone marrow aspirate and/or biopsy at screening, during and at the end of therapy on this study. Exceptions may be considered after documented discussion with Novartis. Main Exclusion Criteria: Patients eligible for this study must not meet any of the following criteria: * Prior combination treatment with compounds having the same mode of action: * mdm2 or mdm4 inhibitors combined with TIM-3 inhibitors (for patients enrolled in treatment arm1) * mdm2 or mdm4 inhibitors combined with Bcl-2 inhibitor (for patients enrolled in treatment arm2) * History of severe hypersensitivity reactions to any ingredient of study drug(s) and other monoclonal antibodies (mAbs) and/or their excipients. * Patients with acute promyelocytic leukemia with PML-RARA. * Allogeneic stem cell transplant (HSCT) within last 6 months and/or active GvHD requiring systemic immunosuppressive therapy. * GI disorders impacting absorption of oral HDM201 or venetoclax. * Evidence of active bleeding or bleeding diathesis or major coagulopathy (including familial). * Patients with active, known or suspected autoimmune disease (treatment arm 1 only). Other eligibility criteria apply.
Get updates
Get notified about this study
Sign up to get updates when this study changes or when new studies for Acute myeloid leukemia (AML) are added.
By submitting, you agree to our Terms of use
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
-
Duke University Medical Center .
Durham, North Carolina, 27710, United States
-
Novartis Investigative Site
Melbourne, Victoria, 3004, Australia
-
Novartis Investigative Site
Helsinki, FIN 00290, Finland
-
Novartis Investigative Site
Heidelberg, 69120, Germany
-
Novartis Investigative Site
Würzburg, 97080, Germany
-
Novartis Investigative Site
Milan, MI, 20132, Italy
-
Novartis Investigative Site
Roma, RM, 00161, Italy
-
Novartis Investigative Site
Singapore, 119228, Singapore
-
Novartis Investigative Site
Madrid, 28041, Spain
More trials for these conditions
Other studies related to the condition(s) this trial covers.
- Can engineered cells beat relapsed blood cancers?
- Can a new pill outsmart resistant leukemia?
- Can a smart order system ensure older AML patients get the support they need?
- Can a new pill boost the power of an existing leukemia treatment?
- Can a new drug combo outperform standard care for a tough blood cancer?
- Can donor immune cells fight leukemia relapse after transplant?