Evaluation of Umbilical Cord-Derived Wharton's Jelly Stem Cells for the Treatment of Acute Graft Versus Host Disease

NCT03158896 | Completed Phase 1 DMC FDA Drug

• 1 other identifier: IIT-2016-aGvHD-MSCTC-0010
• Study Type: interventional
• Target: 24
• Locations: 1 country
• Sites: 1

Brief Summary

This study evaluates the safety and effectiveness of two different doses of umbilical cord derived, ex-vivo cultured and expanded Wharton's jelly mesenchymal stem cells (MSCTC-0010) in the treatment of acute Graft versus Host Disease (aGVHD). The first 5 participants enrolled in the study will receive a lower dose of MSCTC-0010. If none of the first 5 participants have treatment-related serious adverse events (TRSAEs) for 42 days, then the next 5 participants will receive a slightly higher dose of MSCTC-0010.

Conditions

Acute Graft Versus Host Disease

Keywords

mesenchymal stem cell; hematopoietic stem cell transplant; Allogeneic hematopoietic stem cell transplant; Autologous hematopoietic stem cell transplant; Wharton's jelly; umbilical cord blood; steroid refractory graft versus host disease

Outcome Measures

Primary Outcomes (1)

• proportion of participants who have a TRSAE after infusion of MSCTC 0010

  TRSAE is defined as a serious adverse event (SAE) that has a "probable" or "definite" relation to the MSCTC-0010 infusion. This study will use the descriptions and grading scales from Common Terminology Criteria for Adverse Events version 4.03 (CTCAE v4.03) for hematologic and non-hematologic toxicities.

  45 days

Secondary Outcomes (4)

• Proportion of participants who achieve a complete response (CR) of aGVHD by study day 42

  42 days

• Proportion of participants with improvement of GVHD in 1 or more organs involved with GVHD by day 42

  42 days

• Occurrence of addition of escalated immunosuppressive therapy by day 90

  90 days

• Occurrence of Formation of ectopic tissue foci at day 90

  90 days

Study Arms (1)

MSCTC-0010 Dose Escalation

EXPERIMENTAL

Cohort 1: First 5 participants will receive a lower dose of cord-blood derived Wharton's jelly mesenchymal stem cells (MSCTC-0010) and they will be observed for 42 days after the dose for treatment-related serious adverse events (TRSAE) and response. Cohort 2: Second 5 participants will receive an increased dose of MSCTC-0010 and will be observed for 42 days after the dose for TRSAE and response.

Biological: MSCTC-0010 Dose Escalation

Interventions

MSCTC-0010 Dose Escalation BIOLOGICAL

Cohort 1: 2.0 × 106 cells per kilogram (cells/kg) body weight, given on day 0 and on day 7 in Participants having de novo High Risk Acute or Steroid Refractory Acute Graft Versus Host Disease (HR/SR aGvHD) Cohort 2: 10 × 106 cells/kg of body weight, given on day 0 and on day 7 in Participants having de novo HR/SR aGvHD

Also known as: MSCTC-0010

MSCTC-0010 Dose Escalation

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Age: ≥ 18 years of age and ≤ 75 years of age.
• Women of child-bearing potential and men with partners of child-bearing potential must agree to practice sexual abstinence, or to use two forms of adequate contraception (hormonal AND barrier method of birth control) prior to study entry, for the duration of study participation, and for 90 days following completion of therapy. If a woman becomes pregnant or suspects she is pregnant while participating in this study, she should inform her treating physician immediately.
• A woman of child-bearing potential is any female (regardless of sexual orientation, having undergone a tubal ligation, or remaining celibate by choice) who meets the following criteria:
• Has not undergone a hysterectomy or bilateral oophorectomy; OR
• Has not been naturally postmenopausal for at least 12 consecutive months (i.e., has had menses at any time in the preceding 12 consecutive months)
• Men of child-bearing potential must not donate sperm while on this study and for 90 days after their last study treatment.
• NOTE: Acceptable forms of birth control are listed below:
• One Barrier method (cervical cap with spermicide plus male condom; diaphragm with spermicide plus male condom) PLUS
• Hormonal method (oral contraceptives, implants, or injections) or an intrauterine device (e.g., Copper-T).
• Participant must have de novo HR or steroid refractory, Grade II-IV aGVHD as defined in Appendix 1. NOTE: Biopsy at screening only for evaluation of aGVHD is not mandatory, but is recommended when feasible. Enrollment should not be delayed awaiting biopsy results.
• Participant must have received an allogenic transplant at Kansas University Cancer Center/University of Kansas Medical Center (KUCC / KUMC).

You may not qualify if:

• Participants may not have received any other investigational agent used to treat acute GVHD for 30 days prior to enrollment.
• Participant has any underlying or current medical or psychiatric condition that, in the opinion of the Investigator, would interfere with the evaluation of the participant.

Sponsors & Collaborators

• University of Kansas Medical Center: lead

Study Sites (1)

Kansas University Cancer Center

Kansas City, Kansas, 66160, United States

Location

Study Officials

• Joseph McGuirk, DO

  The University of Kansas - Cancer Center

  PRINCIPAL INVESTIGATOR

Study Design

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

Study Record Dates

• First Submitted: May 16, 2017
• First Posted: May 18, 2017
• Study Start: July 9, 2018
• Primary Completion: December 8, 2025
• Study Completion: December 8, 2025
• Last Updated: February 24, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)