Experimental combo therapy for blood cancers shows early promise but trial halted

NCT ID NCT03940352

What the study statuses mean

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Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early This study
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

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.

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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

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