~3 spots leftby Sep 2026

Ruxolitinib + Chemotherapy for Acute Lymphoblastic Leukemia

Wendy Stock, MD - UChicago Medicine
Overseen byWendy Stock, MD
Age: 18 - 65
Sex: Any
Travel: May Be Covered
Time Reimbursement: Varies
Trial Phase: Phase 1
Recruiting
Sponsor: University of Chicago
Must be taking: Intrathecal chemotherapy
Must not be taking: CYP3A4 inhibitors
Disqualifiers: Second malignancy, Allergic reactions, Uncontrolled illness, others
No Placebo Group
Breakthrough Therapy
Approved in 2 Jurisdictions

Trial Summary

What is the purpose of this trial?

This study will test if adding ruxolitinib to standard multi-drug chemotherapy regimen will be safe and tolerated in adolescents and young adults with newly diagnosed Ph-like acute lymphoblastic leukemia (ALL).

Will I have to stop taking my current medications?

The trial does not specify if you need to stop all current medications, but you cannot take any potent CYP3A4 inhibitors or inducers within 5 half-lives before starting the study drug. It's important to discuss your current medications with the study team to avoid any interactions.

What data supports the effectiveness of the drug Ruxolitinib for treating Acute Lymphoblastic Leukemia?

Research shows that Ruxolitinib, a drug that blocks certain proteins involved in cancer growth, has helped some patients with specific types of acute lymphoblastic leukemia (ALL) achieve remission, meaning their cancer symptoms improved or disappeared. It has been particularly effective in cases with certain genetic mutations that make the cancer more aggressive.12345

Is Ruxolitinib generally safe for humans?

Ruxolitinib has been associated with some side effects, including infections (like viral, fungal, and mycobacterial infections), skin issues, and blood clotting problems. There have also been reports of rare skin reactions and virus reactivations. However, major heart problems were not significantly increased in reports.25678

How does the drug Ruxolitinib combined with chemotherapy differ from other treatments for acute lymphoblastic leukemia?

Ruxolitinib, when combined with chemotherapy, offers a unique approach for treating acute lymphoblastic leukemia by targeting the JAK-STAT signaling pathway, which is often overactive in certain high-risk subtypes of the disease. This combination is particularly beneficial for patients with specific genetic mutations, such as JAK mutations, that make them less responsive to standard chemotherapy alone.145910

Research Team

Wendy Stock, MD - UChicago Medicine

Wendy Stock, MD

Principal Investigator

University of Chicago

Eligibility Criteria

This trial is for adolescents and young adults aged 18-40 with newly diagnosed Ph-like acute lymphoblastic leukemia (ALL). Participants must have completed a specific chemotherapy regimen, have normal organ function, and agree to use contraception. Excluded are those with other active cancers, certain heart or psychiatric conditions, pregnant or breastfeeding women, and individuals on strong CYP3A4 inhibitors.

Inclusion Criteria

I have been newly diagnosed with a specific type of leukemia (B-precursor ALL).
My cancer has a 'Ph-like' signature.
Agreement to use adequate contraception for women of child-bearing potential and men
See 7 more

Exclusion Criteria

I haven't taken strong CYP3A4 inhibitors recently.
Down Syndrome
My leukemia was Ph+ at diagnosis.
See 6 more

Trial Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Induction Therapy

Participants must have completed a 4-drug induction therapy regimen with intrathecal chemotherapy

4 weeks

Remission Consolidation Therapy

Participants receive remission consolidation therapy as part of the standard chemotherapy regimen

8 weeks

Interim Maintenance

Participants undergo interim maintenance therapy as part of the standard chemotherapy regimen

8 weeks

Delayed Intensification

Participants undergo delayed intensification therapy as part of the standard chemotherapy regimen

8 weeks

Maintenance Therapy

Participants receive maintenance therapy in 12-week courses/84-day cycles lasting 2-3 years

2-3 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

24 weeks

Treatment Details

Interventions

  • Ruxolitinib (Janus Kinase (JAK) Inhibitor)
Trial OverviewThe study tests the safety of adding ruxolitinib to a standard multi-drug chemotherapy regimen in treating Ph-like ALL. The goal is to determine if this combination improves outcomes for patients who fit the trial's criteria.
Participant Groups
1Treatment groups
Experimental Treatment
Group I: RuxolitinibExperimental Treatment13 Interventions
Participants will receive ruxolitinib in addition to standard chemotherapy. Standard Chemotherapy Consists of: * Remission consolidation therapy (lasting 8 weeks) * Interim Maintenance (lasting 8 weeks) * Delayed Intensification (lasting 8 weeks * Maintenance Therapy (12 week courses/84 day cycles lasting 2-3 years) Prior to study entry, patients must have completed a 4-drug induction therapy regimen with intrathecal chemotherapy (modified Berlin-Frankfurt-Münster (aBFM) regimen or equivalent) as per the institution standard of care.

Find a Clinic Near You

Who Is Running the Clinical Trial?

University of Chicago

Lead Sponsor

Trials
1,086
Recruited
844,000+
Pete Salzmann profile image

Pete Salzmann

University of Chicago

Chief Executive Officer since 2018

MD from University of Chicago’s Pritzker School of Medicine, MBA from Stanford University’s Graduate School of Business

Anh Nguyen profile image

Anh Nguyen

University of Chicago

Chief Medical Officer

MD from Rutgers New Jersey Medical School, MBA from University of Chicago

Incyte Corporation

Industry Sponsor

Trials
408
Recruited
66,800+
Steven Stein profile image

Steven Stein

Incyte Corporation

Chief Medical Officer since 2015

MD from University of Witwatersrand

Hervé Hoppenot profile image

Hervé Hoppenot

Incyte Corporation

Chief Executive Officer since 2014

MBA from ESSEC Business School

Findings from Research

The study demonstrates that targeting the IL-7R pathway with the JAK1/2 inhibitor ruxolitinib effectively induces cell death in T cell acute lymphoblastic leukemia (T-ALL) models, leading to reduced leukemia burden and extended survival in mice.
Combining ruxolitinib with the BCL-2 inhibitor venetoclax shows promise for enhanced therapeutic efficacy, suggesting a potential new treatment strategy for T-ALL that minimizes the toxicity associated with traditional chemotherapy.
Inhibiting Janus Kinase 1 and BCL-2 to treat T cell acute lymphoblastic leukemia with IL7-Rα mutations.Senkevitch, E., Li, W., Hixon, JA., et al.[2019]
In a study of 8 pediatric B-ALL cases, the JAK inhibitor ruxolitinib significantly reduced peripheral and splenic blast counts, especially in samples with JAK-activating mutations, indicating its potential efficacy in treating high-risk Ph-like acute lymphoblastic leukemia.
The mTOR inhibitor rapamycin effectively controlled leukemia burden in all samples and significantly prolonged survival in xenograft models, suggesting it could be a valuable treatment option for this aggressive leukemia subtype.
Targeting JAK1/2 and mTOR in murine xenograft models of Ph-like acute lymphoblastic leukemia.Maude, SL., Tasian, SK., Vincent, T., et al.[2022]
Ruxolitinib, a treatment for myelofibrosis, has been associated with a case of EBV reactivation leading to a severe lymphoproliferative disorder in a 57-year-old female patient, highlighting a potential risk of opportunistic infections.
This case emphasizes the need for close monitoring of patients on Ruxolitinib for signs of viral reactivations and opportunistic infections, as they may pose serious health risks.
Case-report: EBV driven lymphoproliferative disorder associated with Ruxolitinib.Pálmason, R., Lindén, O., Richter, J.[2022]

References

Efficacy of ruxolitinib in acute lymphoblastic leukemia: A systematic review. [2022]
Targeting BCR-ABL and JAK2 in Ph+ ALL. [2021]
Inhibiting Janus Kinase 1 and BCL-2 to treat T cell acute lymphoblastic leukemia with IL7-Rα mutations. [2019]
Ruxolitinib is effective in the treatment of a patient with refractory T-ALL. [2022]
Targeting JAK1/2 and mTOR in murine xenograft models of Ph-like acute lymphoblastic leukemia. [2022]
Case-report: EBV driven lymphoproliferative disorder associated with Ruxolitinib. [2022]
Adverse events associated with JAK inhibitors in 126,815 reports from the WHO pharmacovigilance database. [2023]
Erythematous skin lesions with necrotic centers on lower extremities due to the use of ruxolitinib for primary myelofibrosis. [2021]
Efficacy and safety of ruxolitinib and hydroxyurea combination in patients with hyperproliferative myelofibrosis. [2021]
10.United Statespubmed.ncbi.nlm.nih.gov
CXCR4 allows T cell acute lymphoblastic leukemia to escape from JAK1/2 and BCL2 inhibition through CNS infiltration. [2022]