Addition of Cord Blood Tissue-Derived Mesenchymal Stromal Cells to Ruxolitinib for the Treatment of Steroid-Refractory Acute Graft Versus Host Disease

NCT04744116 | Recruiting Early Phase 1 DMC FDA Drug

• 3 other identifiers: 2019-1122NCI-2020-13889P01CA148600
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (3)

• Death from any cause

  Within 28 days from the start of active study treatment

• Response

  Will compare the patient's 28-day graft versus host disease (GVHD) status to the patient's baseline GVHD status when steroid refractory acute GVHD was diagnosed.

  At day 28 from start of therapy on study

• Incidence of adverse events

  Within 28 days from the start of active study treatment

Secondary Outcomes (19)

• Graft versus host disease status

  At days 7, 14, 21 and 28 post treatment

• Proportion of response

  At days 7, 14, 21 and 28 post treatment

• Time to complete response

  Up to 6 months

• Time to very good partial response

  Up to 6 months

• Time to partial response

  Up to 6 months

Other Outcomes (2)

• Cytokine biomarker analysis (optional)

  Up to 6 months

• Fecal samples analysis (optional)

  Up to 6 months

Study Arms (3)

Arm 1 (ruxolitinib)

ACTIVE COMPARATOR

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.

Drug: Ruxolitinib

Arm 2 (ruxolitinib, lower dose ds-MSCs)

EXPERIMENTAL

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.

Other: Cellular Therapy; Drug: Ruxolitinib

Arm 3 (ruxolitinib, higher dose ds-MSCs)

EXPERIMENTAL

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.

Other: Cellular Therapy; Drug: Ruxolitinib

Interventions

Cellular Therapy OTHER

Given ds-MSCs IV

Also known as: Cell Therapy

Arm 2 (ruxolitinib, lower dose ds-MSCs); Arm 3 (ruxolitinib, higher dose ds-MSCs)

Ruxolitinib DRUG

Given PO

Also known as: INCB-18424, INCB18424, Jakafi, Oral JAK Inhibitor INCB18424

Arm 1 (ruxolitinib); Arm 2 (ruxolitinib, lower dose ds-MSCs); Arm 3 (ruxolitinib, higher dose ds-MSCs)

Eligibility Criteria

• Age: 12 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Participants between the ages of 12 years and 80 years (inclusive).
• 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.
• Karnofsky/Lansky Performance score of at least 30 at the time of study entry.
• Participants who are women of childbearing potential, must be non-pregnant, not breast-feeding, and use adequate contraception. Male patients must use adequate contraception
• Participants (or legal representative where appropriate) must be capable of providing written informed consent, and assent if indicated.

You may not qualify if:

• De novo chronic GVHD
• Isolated acute GVHD of skin
• Secondary systemic therapy for acute GVHD ruxolitinib greater than 96 hours before initiation of therapy.
• Primary treatment with agents other than alpha-1 antitrypsin (AAT) glucocorticoids and ruxolitinib.
• 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.
• 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.
• Participants with significant supplemental oxygen requirement defined as \>6 L oxygen by nasal cannula.
• Participants with known allergy to bovine or porcine products.

Sponsors & Collaborators

• M.D. Anderson Cancer Center: lead
• National Cancer Institute (NCI): collaborator

Study Sites (1)

M D Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Related Links

• M D Anderson Cancer Center

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

Cell- and Tissue-Based Therapy; ruxolitinib

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Biological Therapy; Therapeutics

Study Officials

• Partow Kebriaei, MD

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Partow Kebriaei, MD

CONTACT

713-745-0663; pkebriae@mdanderson.org

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: February 3, 2021
• First Posted: February 8, 2021
• Study Start: February 17, 2021
• Primary Completion (Estimated): March 31, 2032
• Study Completion (Estimated): March 31, 2032
• Last Updated: May 5, 2026

Record last verified: 2026-04

Locations

US (1)