NCT03654716

Phase 1 Study of the Dual MDM2/MDMX Inhibitor ALRN-6924 in Pediatric Cancer

Completed Phase 1 Last updated 14 February 2025

What this trial tests

Phase 1 trial testing ALRN-6924 in Leukemia in 21 participants. Completed in 17 July 2023.

Timeline

1 November 2018

Primary endpoint
17 July 2023

17 July 2023

Quick facts

Lead sponsor	Dana-Farber Cancer Institute
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	parallel
Masking	none
Primary purpose	treatment
Enrollment	21
Start date	1 November 2018
Primary completion	17 July 2023
Estimated completion	17 July 2023
Sites	4 locations across United States

Drugs / interventions tested

• ALRN-6924 — full drug profile →
• Cytarabine — full drug profile →

Conditions studied

• Leukemia — all drugs for Leukemia →
• Brain Tumor — all drugs for Brain Tumor →
• Solid Tumor — all drugs for Solid Tumor →
• Lymphoma — all drugs for Lymphoma →

Sponsor

Dana-Farber Cancer Institute

Who can join

Adults 1 to 21, any sex, with Leukemia or Brain Tumor. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

This research study is studying a novel drug called ALRN-6924 as a possible treatment for resistant (refractory) solid tumor, brain tumor, lymphoma or leukemia.

The drugs involved in this study are:

* ALRN-6924
* Cytarabine (for patients with leukemia only)

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Targeting p53 pathways: mechanisms, structures, and advances in therapy.
   Wang H, Guo M, Wei H, Chen Y. · · 2023 · cited 580× · PMID 36859359 · DOI 10.1038/s41392-023-01347-1
2. Drugging p53 in cancer: one protein, many targets.
   Hassin O, Oren M. · · 2023 · cited 496× · PMID 36216888 · DOI 10.1038/s41573-022-00571-8
3. Recent advances in targeting the "undruggable" proteins: from drug discovery to clinical trials.
   Xie X, Yu T, Li X, Zhang N, et al · · 2023 · cited 246× · PMID 37669923 · DOI 10.1038/s41392-023-01589-z
4. The Role of p53 Signaling in Colorectal Cancer.
   Liebl MC, Hofmann TG. · · 2021 · cited 231× · PMID 33924934 · DOI 10.3390/cancers13092125
5. Ubiquitination and deubiquitination in cancer: from mechanisms to novel therapeutic approaches.
   Liu F, Chen J, Li K, Li H, et al · · 2024 · cited 195× · PMID 39048965 · DOI 10.1186/s12943-024-02046-3
6. An expanded universe of cancer targets.
   Hahn WC, Bader JS, Braun TP, Califano A, et al · · 2021 · cited 160× · PMID 33667368 · DOI 10.1016/j.cell.2021.02.020
7. Dysregulated haematopoietic stem cell behaviour in myeloid leukaemogenesis.
   Yamashita M, Dellorusso PV, Olson OC, Passegué E. · · 2020 · cited 128× · PMID 32415283 · DOI 10.1038/s41568-020-0260-3
8. The BCL2 family: from apoptosis mechanisms to new advances in targeted therapy.
   Vogler M, Braun Y, Smith VM, Westhoff MA, et al · · 2025 · cited 104× · PMID 40113751 · DOI 10.1038/s41392-025-02176-0