A Randomized Study to Compare Post-transplant Cyclophosphamide, Sirolimus, Ruxolitinib and Post-transplant Cyclophosphamide, Sirolimus, Mycophenolate Mofetil to Prevent Graft Versus Host Disease

NCT06973668 | Recruiting Phase 2 DMC FDA Drug

• 2 other identifiers: 2024-1940NCI-2025-03239
• Study Type: interventional
• Target: 80
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this clinical research study is to compare the effects of these drug combinations (cyclophosphamide, sirolimus, and MMF vs cyclophosphamide, sirolimus, and ruxolitinib) on the prevention of GVHD after a stem cell transplant.

Conditions

AML

Outcome Measures

Primary Outcomes (1)

• Safety and Adverse Events (AEs)

  Incidence of Adverse Events, Graded According to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) Version (v) 5.0

  Through study completion; an average of 1 year

Study Arms (2)

01A:Treatment With Post-Transplant Cyclophosphamide (PTCy), Sirolimus and MMF

EXPERIMENTAL

15 mg/kg/dose (max: 1,000mg/dose) IV/PO three times daily

Drug: Mycophenolate Mofetil

01B:Treatment With Post-Transplant Cyclophosphamide (PTCy),Sirolimus and Ruxolitinib

EXPERIMENTAL

5 mg PO every 12 hours

Drug: Ruxolitinib

Interventions

Mycophenolate Mofetil DRUG

15 mg/kg/dose (max: 1,000mg/dose) IV/PO three times daily

01A:Treatment With Post-Transplant Cyclophosphamide (PTCy), Sirolimus and MMF

Ruxolitinib DRUG

5 mg PO every 12 hours

Also known as: Jakafi®

01B:Treatment With Post-Transplant Cyclophosphamide (PTCy),Sirolimus and Ruxolitinib

Eligibility Criteria

• Age: 65 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Older Adult (65+)

You may qualify if:

• Age ≥ 65 and \< 75 years are eligible if they have one of the following diseases.
• Acute Myeloid Leukemia
• Myelodysplastic syndrome
• Chronic myelomonocytic leukemia
• Available HLA-identical or haploidentical related donor or a 7/8 or 8/8 HLA matched unrelated donor.
• Peripheral blood stem cells as a graft source
• Subject must voluntarily sign an informed consent.
• Adequate organ function per local laboratory reference range as follows: - Aspartate transaminase (AST) and alanine transaminase (ALT) \< 3.0X ULN - Total Bilirubin \<1.5 x ULN (unless bilirubin rise is due to Gilbert's syndrome or of nonhepatic origin) - Subject must have adequate renal function as demonstrated by a creatinine clearance ≥ 40 mL/min/1.73 m2 (as reported in epic using 2021 CKD-EPI creatinine equation)
• DLCO corrected for Hgb, if applicable) ≥ 50% of predicted
• Ejection Fraction ≥ 50%
• Postmenopausal (no menses in greater than or equal to 12 consecutive months).
• History of hysterectomy or bilateral salpingo-oophorectomy.
• Ovarian failure (Follicle Stimulating Hormone and Estradiol in menopausal range, who have received Whole Pelvic Radiation Therapy).
• History of bilateral tubal ligation or another surgical sterilization procedure.
• Approved methods of birth control are as follows: Hormonal contraception (i.e. birth control pills, injection, implant, transdermal patch, vaginal ring), Intrauterine device (IUD), Tubal Ligation or hysterectomy, Subject/Partner post vasectomy, Implantable or injectable contraceptives, and condoms plus spermicide. Not engaging in sexual activity for the total duration of the trial and the drug washout period is an acceptable practice; however periodic abstinence, the rhythm method, and the withdrawal method are not acceptable methods of birth control. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately.

You may not qualify if:

• Subject is known to be positive for HIV.
• Subject has acute promyelocytic leukemia.
• Subject has known active CNS involvement with AML.
• Hematopoietic Cell Transplantation Comorbidity Index (HCT-CI) score of \>5
• Evidence of other clinically significant uncontrolled condition(s) including, but not limited to:
• Uncontrolled and/or active systemic infection (viral, bacterial or fungal)
• Chronic hepatitis B virus (HBV) or hepatitis C (HCV) requiring treatment. Note: subjects with serologic evidence of prior vaccination to HBV (i.e. hepatitis B surface (HBs) antigen negative-, anti-HBs antibody positive and anti-hepatitis B core (HBc) antibody negative) or subjects with positive anti-HBc antibody but negative Hep B DNA may participate.
• Cardiac history of CHF requiring treatment or Ejection Fraction \< 50% or unstable angina or MI within 1 year of study entry
• Major adverse cardiac events such as MI/stroke and pulmonary embolism (PE)/deep vein thrombosis (DVT) within 6 months. Recent history of Central line-associated DVT may be allowed after discussion with PI.
• Current and/or history of active TB
• White Blood Cell count \> 25 X 109 /L.
• Pregnant women are excluded from this study because the study agent has unknown potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother, breastfeeding should be discontinued if the mother is treated with the study agent. These potential risks may also apply to other agents used in this study.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead

Study Sites (1)

The University of Texas M. D. Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• MD Anderson Cancer Center Website

MeSH Terms

Interventions

Mycophenolic Acid; ruxolitinib

Intervention Hierarchy (Ancestors)

Caproates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Fatty Acids; Lipids

Study Officials

• Uday Popat, MBA,MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Uday Popat, MBA,MD

CONTACT

(713) 563-0812; upopat@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 6, 2025
• First Posted: May 15, 2025
• Study Start: July 22, 2025
• Primary Completion (Estimated): January 31, 2028
• Study Completion (Estimated): January 31, 2030
• Last Updated: May 20, 2026

Record last verified: 2026-05

Locations

US (1)