RELMLL: A Phase II Study of Bortezomib and Vorinostat in Patients with Refractory or Relapsed MLL Rearranged Hematologic Malignancies


Leukemia / Lymphoma

Phase I/II

Diseases Treated:

Relapsed or refractory leukemia / lymphoma

Eligibility Overview:

This clinical trial has both inclusion and exclusion criteria. See Eligibility Requirements in the full trial for specifics.


This study will enroll participants who have Acute Lymphoblastic Leukemia (ALL), Biphenotypic/Mixed Lineage Leukemia, or Acute Myeloid Leukemia (AML) that has either come back (relapsed) or did not go into remission with treatment (relapsed).  The leukemia the participants in this study have been diagnosed with has cells that have a mutation (mistake) in a gene called Mixed Lineage Leukemia (MLL). MLL is a type of childhood cancer in which a piece of chromosome 11 has broken off and re-attached itself to another chromosome (called translocation).  Children with this type of leukemia have a higher risk of relapse and do not respond well to current leukemia therapies. The usual treatment for relapsed or refractory leukemia is combination chemotherapy followed by stem cell transplant (SCT). In this study the doctors want to test the good and bad effects of the study drugs called bortezomib and vorinostat when given in addition to the combination chemotherapy commonly used to treat relapsed or refractory leukemia. Bortezomib and vorinostat have been approved by the US Food and Drug Administration (FDA) to treat cancer in adults, but they have not been approved for treating children with leukemia.


  • To find out if adding bortezomib and vorinostat to a commonly used chemotherapy regimen will be effective in treating relapsed or refractory MLL leukemia.
  • To gather information about side effects of the combination therapy.
  • To learn more about the biology and genetics of MLL leukemia by studying blood and bone marrow samples from patients on the study in research laboratories at St. Jude.
  • To find better ways to measure minimal residual disease (MRD) in patients with leukemia.

Inclusion criteria: (among others)

  • Participant is less than or equal to twenty-one (21) years of age (eligible until twenty-second (22) birthday).
  • Participant has a hematologic malignancy that is positive for MLLr as determined by fluorescent in situ hybridization (FISH) or RT-PCR and disease meets at least one of the following criteria: relapsed after, or is refractory to chemotherapy, relapsed after hematopoietic stem cell transplantation (HSCT), relapsed or refractory secondary leukemia.
  • Participant relapsed while receiving standard ALL maintenance chemotherapy (no waiting period required) or relapsed on therapy other than standard ALL maintenance therapy and has fully recovered from the acute toxic effects of all prior chemotherapy, immunotherapy, or radiotherapy prior to entering this study.
  • Participant has been at least 14 days since the completion of cytotoxic therapy with the exception of hydroxyurea, low dose cytarabine, and intrathecal chemotherapy which is permitted up to twenty-four (24) hours prior to the start of protocol therapy. Participant has aggressive disease that is in the peripheral blood and rising. Participant relapsed while receiving standard ALL maintenance chemotherapy (no waiting period required).  
  • Participant is at least seven (7) days since the completion of therapy with a biologic agent or donor lymphocyte infusions (DLI). For agents that have known adverse events occurring beyond seven (7) days after administration, this period must be extended beyond the time during which adverse events are known to occur.
  • If the patient has relapsed after a stem cell transplant, greater than or equal to two (2) months must have elapsed since their transplant.

Exclusion criteria

  • Participant has an uncontrolled infection (infections controlled on concurrent anti-microbial agents are acceptable, and anti-microbial prophylaxes per institutional guidelines are acceptable).
  • Participant is known to have human immunodeficiency virus (HIV) infection.
  • Participant has any significant concurrent disease, illness or psychiatric disorder that would compromise safety or compliance, study participation, follow-up or interpretation of study research.
  • Participant has Fanconi anemia, Kostmann syndrome, Shwachman syndrome or other inherited bone marrow failure syndrome.
  • In the opinion of the investigator, the participant may not be able to comply with the safety monitoring requirements of the study.

For the current eligibility status of this clinical study, referring physicians must contact St. Jude Children's Research Hospital at 1-866-2ST-JUDE (1-866-278-5833).


Tanja Gruber, MD, PhD

St. Jude Children’s Research Hospital
262 Danny Thomas Place
Memphis, TN 38105  USA
Voice: 1-888-226-4343 or 901-595-4055
24-Hour Emergency Access Pager: 1-800-349-4334

The above information is intended to provide only a basic description about a research protocol that may be currently active at St. Jude. The details made available here may not be the most up-to-date information on protocols used by St. Jude. To receive full details about a protocol and its status and or use at St. Jude, a physician must contact St. Jude directly.