Ruxolitinib + Allogeneic Stem Cell Transplantation in AML

NCT03286530 | Active Not Recruiting Phase 2 DMC FDA Drug

• 1 other identifier: 17-273
• Study Type: interventional
• Target: 64
• Locations: 1 country
• Sites: 6

Brief Summary

This research study is studying a drug that may help decrease the chances of relapse after Allogeneic Stem Cell transplantation for Acute Myeloid Leukemia. The name of the study drug involved in this study is: • Ruxolitinib

Conditions

Acute Myeloid Leukemia; Acute Myeloid Leukemia in Remission; Allogeneic Stem Cell Transplantation

Keywords

Acute Myeloid Leukemia; Acute Myeloid Leukemia in Remission; Allogenic Stem Cell Transplantation

Outcome Measures

Primary Outcomes (1)

• 1-year GVHD/relapse free survival rate (GRFS rate)

  The number of participants surviving after one year that have not experienced graft-versus-host disease (GVHD) or graft relapse (GRFS rate).

  1 Year

Secondary Outcomes (5)

• Progression Free Survival

  Until disease progression or death from any cause, approximately 5 years

• Overall Survival

  Until death, approximately 5 years

• Cumulative incidence of drug related toxicities

  2 Years

• Time to Relapse

  2 Years

• Time to treatment-related mortality (TRM)

  2 Years

Study Arms (1)

Ruxolitinib

EXPERIMENTAL

Following a standard of care allogeneic stem cell transplantation, participants will be started on Ruxolitinib. Ruxolitinib is administered orally 2 times per day at a fixed dose. Each study treatment cycle lasts 28 days. Up to 24 cycles.

Drug: Ruxolitinib

Interventions

Ruxolitinib DRUG

Patients who fulfill eligibility criteria will be entered into the trial to receive Ruxolitinib. After the screening procedures confirm participation in the research study. The participant will be given a drug diary. The participant will be asked to document information in the drug diary about the study treatment.

Also known as: Jakafi

Ruxolitinib

Eligibility Criteria

• Age: 60 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants must have pathologically confirmed AML in CR1 as defined by:
• Bone marrow biopsy with \< 5% blasts
• No clusters or collections of blast cells
• No extramedullary leukemia
• Absolute neutrophil count ≥ 1000/µL (achieved post-induction at some point)
• Please note that full platelet recovery is not necessary, and thus, patients achieving CRp are eligible.
• Or participants have pathologically confirmed MDS as defined by:
• Bone marrow biopsy with \<10% blasts
• Patients receiving MDS-directed therapy must be off treatment for \> 2 weeks prior to start of conditioning.
• Participants must be designated to undergo reduced intensity allogeneic peripheral blood hematopoietic stem cell transplantation (HCT). Consent will be obtained prior to admission for HCT. The following HCT conditions must be planned:
• Donors must be 8/8 HLA-matched (at the allele level) as defined by matching at HLA-A, -B, -DR and -C who pass institutional standard to serve as a peripheral blood stem cell donor
• Donor grafts must be G-CSF mobilized peripheral blood stem cells with dose and apheresis logistics at the discretion of institutional standard
• Conditioning therapy will be one of the following 3 options:
• Fludarabine / Melphalan where fludarabine is ≥ 90 mg/m2 IV total dose and melphalan is 100-140 mg/m2 IV total dose. Exact logistics of administration are at the discretion of institutional standard.
• Fludarabine / Busulfan where fludarabine is ≥ 90 mg/m2 IV total dose and busulfan = 6.4 mg/kg IV total dose. Exact logistics of administration are at the discretion of institutional standard.

You may not qualify if:

• Have had a prior allogeneic HSCT.
• Patients without normal organ function defined as follows:
• AST (SGOT), ALT (SGPT) and Alkaline Phosphatase \>3 × institutional Upper Limit of Normal (ULN)
• Direct bilirubin \>2.0 mg/dL
• Adequate renal function as defined by calculated creatinine clearance ≤ 40 mL/min (Cockcroft-Gault formula)
• Have a history of other malignancy(ies) unless:
• They have been disease-free for at least 5 years and are deemed by the treating investigator to be at low risk for recurrence of that malignancy,
• \--- or
• The only cancer they have had is cervical cancer in situ, or basal cell or squamous cell carcinoma of the skin
• Have a chronic or active infection that requires systemic antibiotics, antifungal or antiviral treatment.
• Have current or a history of congestive heart failure New York Heart Association (NYHA) class 3 or 4, or any history of documented diastolic or systolic dysfunction (LVEF \< 40%, as measured by MUGA scan or echocardiogram)
• Have an uncontrolled intercurrent illness including, but not limited to, ongoing infection, symptomatic congestive heart failure, unstable angina pectoris, cardiac arrhythmia, or psychiatric illness/social situations that would limit compliance with study requirements.
• Have active uncontrolled infection. An active uncontrolled infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs, or radiographic findings attributable to infection. Persisting fever without signs or symptoms will not be interpreted as an active uncontrolled infection.
• Be HIV-positive
• Have a systemic infection requiring IV antibiotic therapy, nor any other severe infection

Sponsors & Collaborators

• Massachusetts General Hospital: lead
• Vanderbilt University: collaborator
• Ohio State University: collaborator
• Medical College of Wisconsin: collaborator
• Washington University School of Medicine: collaborator

Study Sites (6)

Beth Israel Deaconess Medical Center

Boston, Massachusetts, 02115, United States

Location

Massachusetts General Hospital

Boston, Massachusetts, 02115, United States

Location

Washington University

St Louis, Missouri, 63130, United States

Location

The Ohio State University

Columbus, Ohio, 43210, United States

Location

Vanderbilt University

Nashville, Tennessee, 37235, United States

Location

Medical College of Wisconsin

Wauwatosa, Wisconsin, 53226, United States

Location

Related Publications (1)

• DeFilipp Z, Kim HT, Knight LW, O'Connor SM, Dhaver SE, White M, Dholaria B, Schroeder MA, Vasu S, Abedin S, Chung J, El-Jawahri A, Frigault MJ, McAfee S, Newcomb RA, O'Donnell PV, Spitzer TR, Chen YB, Hobbs GS. Low rates of chronic graft-versus-host disease with ruxolitinib maintenance following allogeneic HCT. Blood. 2025 May 15;145(20):2312-2316. doi: 10.1182/blood.2024028005.

  PMID: 40106768 DERIVED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

ruxolitinib

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Study Officials

• Gabriell Hobbs, MD

  Massachusetts General Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Prinicipal Investigator

Study Record Dates

• First Submitted: August 9, 2017
• First Posted: September 18, 2017
• Study Start: November 3, 2017
• Primary Completion (Estimated): December 1, 2026
• Study Completion (Estimated): December 1, 2026
• Last Updated: March 27, 2026

Record last verified: 2026-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (6)