NCT03515200

Treatment With Combination Chemotherapy for Relapsed or Refractory Acute Lymphoblastic Leukemia

Terminated Phase 1 Last updated 1 September 2020

What this trial tests

Phase 1 trial testing Palbociclib Oral Capsule in Acute Lymphoblastic Leukemia, in Relapse in 12 participants. Terminated before completion.

Timeline

20 April 2018

Primary endpoint
29 July 2020

29 July 2020

Quick facts

Lead sponsor	St. Jude Children's Research Hospital
Phase	Phase 1
Status	Terminated
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	20 April 2018
Primary completion	29 July 2020
Estimated completion	29 July 2020
Sites	1 location across United States

Drugs / interventions tested

• Palbociclib Oral Capsule
• Intrathecal Triple Therapy
• Dexamethasone (dexamethasone) — full drug profile →
• Bortezomib (bortezomib) — full drug profile →
• Dasatinib (dasatinib) — full drug profile →
• Doxorubicin (doxorubicin) — full drug profile →
• Ruxolitinib

Conditions studied

• Acute Lymphoblastic Leukemia, in Relapse — all drugs for Acute Lymphoblastic Leukemia, in Relapse →
• Acute Lymphoblastic Leukemia With Failed Remission — all drugs for Acute Lymphoblastic Leukemia With Failed Remission →

Sponsor

St. Jude Children's Research Hospital

Who can join

Under 21, any sex, with Acute Lymphoblastic Leukemia, in Relapse or Acute Lymphoblastic Leukemia With Failed Remission. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

Leukemia cells grow and divide fast and out of control. In normal cells, certain proteins called CDK4 and CDK6 control cell growth. The study drug called palbociclib works by blocking the CDK4 and CDK6 proteins. Palbociclib has been shown to kill leukemia cells in the laboratory and in animal studies. Palbociclib will be added to other chemotherapy drugs, such as dexamethasone, that are known to be effective in treating childhood ALL.

This study will be done in two parts: Part 1: Dose Escalation and Part 2: Dose Expansion. The goal of Part 1 of the study is to find the highest tolerable combination of palbociclib and chemotherapy that the investigators can give to patients with leukemia. Once those doses are determined, the investigators will enroll patients on Part 2: Dose Expansion. This phase will enroll additional patients that receive the highest tolerated dose of palbociclib as determined in part 1, in order to better understand the side effects and how effective this treatment approach is.

With this research study, the investigators hope to meet the following goals:

* To find the highest tolerable dose of palbociclib in combination with chemotherapy that can be given without causing severe side effects;
* To learn what kind of side effects palbociclib in combination with chemotherapy may have; and
* To learn more about the biology effects of palbociclib on the cells in the participant's body.

Up to 40 children, adolescents and young adults will participate in both parts of this study at St. Jude only.

Publications & conference data

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

1. The New Therapeutic Strategies in Pediatric T-Cell Acute Lymphoblastic Leukemia.
   Lato MW, Przysucha A, Grosman S, Zawitkowska J, et al · · 2021 · cited 59× · PMID 33925883 · DOI 10.3390/ijms22094502
2. Strategies to Overcome Resistance Mechanisms in T-Cell Acute Lymphoblastic Leukemia.
   Follini E, Marchesini M, Roti G. · · 2019 · cited 52× · PMID 31226848 · DOI 10.3390/ijms20123021
3. The Current Genomic and Molecular Landscape of Philadelphia-like Acute Lymphoblastic Leukemia.
   Shiraz P, Payne KJ, Muffly L. · · 2020 · cited 33× · PMID 32235787 · DOI 10.3390/ijms21062193
4. Molecular Approaches to Treating Pediatric Leukemias.
   Kuhlen M, Klusmann JH, Hoell JI. · · 2019 · cited 33× · PMID 31555628 · DOI 10.3389/fped.2019.00368
5. Precision Medicine in Pediatric Oncology.
   Vo KT, Parsons DW, Seibel NL. · · 2020 · cited 23× · PMID 31757314 · DOI 10.1016/j.soc.2019.08.005
6. The Yin and Yang-Like Clinical Implications of the <i>CDKN2A/ARF/CDKN2B</i> Gene Cluster in Acute Lymphoblastic Leukemia.
   González-Gil C, Ribera J, Ribera JM, Genescà E. · · 2021 · cited 20× · PMID 33435487 · DOI 10.3390/genes12010079
7. Targeting Leukemia-Initiating Cells and Leukemic Niches: The Next Therapy Station for T-Cell Acute Lymphoblastic Leukemia?
   Zhang Z, Yang K, Zhang H. · · 2022 · cited 8× · PMID 36428753 · DOI 10.3390/cancers14225655
8. Promising Drugs Targeting Specific Mechanisms of Deregulation in T Cell Lineage Acute Lymphoblastic Leukemia.
   Thomas X. · · 2025 · cited 1× · PMID 40249557 · DOI 10.1007/s40487-025-00339-1