Recruiting
Early Phase 1

Cord Blood Cells with Ruxolitinib

Sponsor:

M.D. Anderson Cancer Center

Code:

NCT04744116

Conditions

Hematopoietic and Lymphoid Cell Neoplasm

Steroid Refractory Graft Versus Host Disease

Eligibility Criteria

Sex: All

Age: 12 - 70+

Healthy Volunteers: Not accepted

Interventions

Cellular Therapy

Ruxolitinib

Study Details

Brief summary:

This early phase I trial is to find out the effect of adding cord blood tissue-derived mesenchymal stromal cells (cb-MSCs) to ruxolitinib in treating patients with acute graft versus host disease that does not respond to steroid therapy (steroid-refractory). Ruxolitinib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. cb-MSCs are a type of tissue helper cell that can be removed from donated umbilical cord blood tissue and grown into many different cell types that can be used to treat cancer and other disease, such as graft versus host disease. This trial aims to learn if adding cb-MSCs to ruxolitinib may help control steroid-refractory acute graft versus host disease.

Conditions

Hematopoietic and Lymphoid Cell Neoplasm

Steroid Refractory Graft Versus Host Disease

Study ID

NCT04744116

Start date

Feb 17, 2021

Status verified date

Aug, 2026

Completion date

Mar 31, 2032

Anticipated

Primary completion date

Mar 31, 2032

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 70+

Healthy Volunteers: Not accepted

Inclusion Criteria:

1. Participants between the ages of 12 years and 80 years (inclusive).
2. Steroid refractory grades II-IV acute GVHD of the Lower GI tract or Liver (including those developing these manifestations after previous acute GVHD of skin) secondary to allogeneic HCT or donor lymphocyte infusion. (Grading, see Appendix I) GVHD with: No improvement after treatment with methylprednisolone at ≥ 2.0 mg/kg/day or equivalent for minimum 7 days, or progressive symptoms after minimum 3 days, or a flare in acute GVHD while on systemic steroids. Participants must have had a biopsy that suggests GVHD; a repeat biopsy to enroll on the study is not necessary.
3. Karnofsky/Lansky Performance score of at least 30 at the time of study entry.
4. Participants who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male patients must use adequate contraception
5. Participants (or legal representative where appropriate) must be capable of providing written informed consent, and assent if indicated.

Exclusion Criteria:

1. De novo chronic GVHD
2. Isolated acute GVHD of skin
3. Secondary systemic therapy for acute GVHD ruxolitinib greater than 96 hours before initiation of therapy.
4. Primary treatment with agents other than alpha-1 antitrypsin (AAT) glucocorticoids and ruxolitinib.
5. Participants with uncontrolled infections will be excluded. Infections are considered controlled if appropriate therapy has been instituted and, at the time of enrollment, no signs of progression are present. Progression of infection is defined as hemodynamic instability attributable to sepsis, new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
6. Adult and pediatric patients with cognitive impairments and/or any serious unstable pre-existing medical condition or psychiatric disorder that can interfere with safety or with obtaining informed consent or compliance with study procedures.
7. Participants with significant supplemental oxygen requirement defined as >6 L oxygen by nasal cannula.
8. Participants with known allergy to bovine or porcine products.

Study Design

Enrollment

24 participants

Anticipated

Allocation

Randomized

Intervention Model

Parallel Assignment

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: Arm 1 (ruxolitinib)

Patients receive ruxolitinib PO BID for at least 3 days and may consider tapering after 6 months of therapy if response occurs and therapeutic corticosteroid doses have been discontinued.

experimental: Arm 2 (ruxolitinib, lower dose ds-MSCs)

Patients receive ruxolitinib PO BID as in Arm 1. Patients also receive lower dose of cb-MSCs IV for up to 60 minutes twice weekly (at least 3 days apart) over 4 consecutive weeks for 8 total doses.

experimental: Arm 3 (ruxolitinib, higher dose ds-MSCs)

Patients receive ruxolitinib PO BID as in Arm 1. Patients also receive higher dose of cb-MSCs IV for up to 60 minutes twice weekly (at least 3 days apart) over 4 consecutive weeks for 8 total doses.

Interventions

Cellular Therapy

Given ds-MSCs IV

Ruxolitinib

Given PO

Primary outcome measure

  • Death from any cause [ Time Frame: Within 28 days from the start of active study treatment ]
  • Response [ Time Frame: At day 28 from start of therapy on study ]
  • Incidence of adverse events [ Time Frame: Within 28 days from the start of active study treatment ]

Central Contacts and Locations

Central contacts

Locations

M D Anderson Cancer Center

Recruiting

Houston, Texas, United States, 77030

Contacts

Principal Investigator:

Partow Kebriaei, MD

More Information

Sponsor

M.D. Anderson Cancer Center

Last update posted

Aug 18, 2026

Last verified

Aug, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by M.D. Anderson Cancer Center on 2026-08-18.