Acute Graft Versus Host DiseasePhase 1NCT03158896
Evaluation of Umbilical Cord-Derived Wharton's Jelly Stem Cells for the Treatment of Acute Graft Versus Host Disease
A Phase 1 study testing MSCTC-0010 Dose Escalation in people with Acute Graft Versus Host Disease, run by University of Kansas Medical Center at 1 site in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Acute Graft Versus Host Disease
- What is being tested?
- MSCTC-0010 Dose Escalation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 9 Jul 2018to 8 Dec 2025
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
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- 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).
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Kansas University Cancer Center
Kansas City, Kansas, 66160
About this study
Official title: A Phase I Study To Evaluate the Safety of Umbilical Cord - Derived, Ex-Vivo Cultured and Expanded Wharton's Jelly Mesenchymal Stem Cells for the Treatment of De Novo High Risk Acute or Steroid Refractory Acute Graft Versus Host Disease
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.
Read the detailed description
The curative potential of Allogeneic hematopoietic stem cell transplantation (allo-HCT), when applied as a therapy in the management of hematologic malignancies, specifically, derives from an immunologically driven, graft-versus-tumor effect mediated principally by donor T-cells, and is associated with a lesser risk for relapse when compared to high dose chemo-radio therapy and autologous HCT. Donor derived T-cells are also responsible for mediating the occurrence of GVHD, a common transplant-related complication, affecting a significant percentage of patients undergoing allo-HCT leading to the destruction of host tissues. The standard initial treatment for both acute and chronic GVHD is steroid-based therapy. Unfortunately, many of these patients will become resistant to steroid therapy and will subsequently be treated with second-line immunosuppressive agents. De novo high-risk aGVHD and steroid-refractory aGVHD portends a very poor prognosis; second-line agents frequently prove ineffective, and as a result, survival is < 10% at 5 years. Therefore, alternative therapies are needed to treat aGVHD following allo-HCT, particularly in the setting of de novo high-risk acute or steroid-resistant disease.
Due to the large numbers of Mesenchymal stem cells (MSC) that can be obtained from the umbilical cord, the availability of this tissue, their higher growth rates and expansion capacity, and their immune properties, including: (1) low immunogenicity and lack of stimulation of allogeneic T-lymphocyte proliferation, (2) suppression of the proliferation of activated T-lymphocytes, (3) increased production of regulatory T-cells, and (4) a shift in the immune response towards tolerance, Wharton's jelly mesenchymal stem cells (WJMSC) may be a preferred option for MSC.
The rationale for cell dosing in this protocol is based on published data from Kebriaei, et al. Dosing at 2 × 10^6 MSC/kg body weight produced a complete response in 87.5% of the treated patients. Dosing at a level 4 times higher (8 × 10^6 MSC/kg body weight) produced no improvement in complete response results. However, the higher dose produced some partial response and no patient failed to respond to therapy. Therefore, the Phase I study for MSCTC-0010 is designed to increase the dose of WJMSC from 2 × 10^6 MSC to 10 × 10^6 MSC/kg body weight, assuming no dose-limiting toxicity (DLT) is observed at the lower dose.
- Conditions
- Acute Graft Versus Host Disease
- Being tested
- MSCTC-0010 Dose Escalation
- 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
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
MSCTC-0010 Dose EscalationBiological
Also called MSCTC-0010
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
| Study group | Role | What they receive |
|---|---|---|
| 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. Receives: Biological: MSCTC-0010 Dose Escalation |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: 45 days
Secondary outcomes (4)
Proportion of participants who achieve a complete response (CR) of aGVHD by study day 42
Complete response is defined as a complete resolution of GVHD.
Measured: 42 days
Proportion of participants with improvement of GVHD in 1 or more organs involved with GVHD by day 42
GVHD will be graded by organ (skin, liver, and gastrointestinal [GI])
Measured: 42 days
Occurrence of addition of escalated immunosuppressive therapy by day 90
Starting at the day 14 evaluation, if the acute GVHD continues to be unresponsive or worsens, additional agents or changes in immunosuppresive therapy will be at discretion of the Investigator per institutional standards.
Measured: 90 days
Occurrence of Formation of ectopic tissue foci at day 90
Defined as ectopic tissue formation of greater than 1.0 centimeter (cm) evaluated by comparison CT scan from screening to day 90.
Measured: 90 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2017
- Study start9 Jul 2018
- Primary completion8 Dec 2025
- Study completion8 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Acute Graft Versus Host DiseaseStudies of the same condition, open and under way.
- More trials run by University of Kansas Medical CenterThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Kansas Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Joseph McGuirk, DO, Principal investigator, The University of Kansas - Cancer Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03158896
- Sponsor's protocol ID
- IIT-2016-aGvHD-MSCTC-0010
- Status last verified
- Feb 2026
- First submitted
- 16 May 2017
- Last updated
- 24 Feb 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Soder RP, Dudley NR, Dawn B. Microcarrier-based clinical-grade manufacturing of therapeutic Wharton's jelly mesenchymal stromal cells. Cytotherapy. 2024 Dec;26(12):1556-1565. doi: 10.1016/j.jcyt.2024.07.003. Epub 2024 Jul 6.
PubMed 39093227
Study at a glance
From the registry record, last updated by the sponsor on 24 Feb 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I Study To Evaluate the Safety of Umbilical Cord - Derived, Ex-Vivo Cultured and Expanded Wharton's Jelly Mesenchymal Stem Cells for the Treatment of De Novo High Risk Acute or Steroid Refractory Acute Graft Versus Host Disease
- ClinicalTrials.gov ID
- NCT03158896
- Acronym / sponsor ID
- IIT-2016-aGvHD-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
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- University of Kansas Medical CenterUniversity, hospital or other
- Study start
- 9 Jul 2018 (actual)
- Primary completion
- 8 Dec 2025 (actual)
- Study completion
- 8 Dec 2025 (actual)
- First posted
- 18 May 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
The curative potential of Allogeneic hematopoietic stem cell transplantation (allo-HCT), when applied as a therapy in the management of hematologic malignancies, specifically, derives from an immunologically driven, graft-versus-tumor effect mediated principally by donor T-cells, and is associated with a lesser risk for relapse when compared to high dose chemo-radio therapy and autologous HCT. Donor derived T-cells are also responsible for mediating the occurrence of GVHD, a common transplant-related complication, affecting a significant percentage of patients undergoing allo-HCT leading to the destruction of host tissues. The standard initial treatment for both acute and chronic GVHD is steroid-based therapy. Unfortunately, many of these patients will become resistant to steroid therapy and will subsequently be treated with second-line immunosuppressive agents. De novo high-risk aGVHD and steroid-refractory aGVHD portends a very poor prognosis; second-line agents frequently prove ineffective, and as a result, survival is < 10% at 5 years. Therefore, alternative therapies are needed to treat aGVHD following allo-HCT, particularly in the setting of de novo high-risk acute or steroid-resistant disease.
Due to the large numbers of Mesenchymal stem cells (MSC) that can be obtained from the umbilical cord, the availability of this tissue, their higher growth rates and expansion capacity, and their immune properties, including: (1) low immunogenicity and lack of stimulation of allogeneic T-lymphocyte proliferation, (2) suppression of the proliferation of activated T-lymphocytes, (3) increased production of regulatory T-cells, and (4) a shift in the immune response towards tolerance, Wharton's jelly mesenchymal stem cells (WJMSC) may be a preferred option for MSC.
The rationale for cell dosing in this protocol is based on published data from Kebriaei, et al. Dosing at 2 × 10^6 MSC/kg body weight produced a complete response in 87.5% of the treated patients. Dosing at a level 4 times higher (8 × 10^6 MSC/kg body weight) produced no improvement in complete response results. However, the higher dose produced some partial response and no patient failed to respond to therapy. Therefore, the Phase I study for MSCTC-0010 is designed to increase the dose of WJMSC from 2 × 10^6 MSC to 10 × 10^6 MSC/kg body weight, assuming no dose-limiting toxicity (DLT) is observed at the lower dose.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Sequential
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| MSCTC-0010 Dose Escalation | Experimental | Biological: MSCTC-0010 Dose EscalationCohort 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. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (11 listed)
- 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.
- 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).
Key exclusion criteria (2 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| MSCTC-0010 Dose EscalationAlso: MSCTC-0010Cohort 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 | Biological | MSCTC-0010 Dose Escalation |
Primary outcome
proportion of participants who have a TRSAE after infusion of MSCTC 0010
Time frame: 45 days
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.
4 secondary outcomes
Proportion of participants who achieve a complete response (CR) of aGVHD by study day 42
Time frame: 42 days
Proportion of participants with improvement of GVHD in 1 or more organs involved with GVHD by day 42
Time frame: 42 days
Occurrence of addition of escalated immunosuppressive therapy by day 90
Time frame: 90 days
Occurrence of Formation of ectopic tissue foci at day 90
Time frame: 90 days
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Feb 2026.
- Derived from this study: PubMed 39093227Soder RP, Dudley NR, Dawn B. Microcarrier-based clinical-grade manufacturing of therapeutic Wharton's jelly mesenchymal stromal cells. Cytotherapy. 2024 Dec;26(12):1556-1565. doi: 10.1016/j.jcyt.2024.07.003. Epub 2024 Jul 6.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Joseph McGuirk, DO principal investigator
The University of Kansas - Cancer Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing MSCTC-0010 Dose Escalation in people with Acute Graft Versus Host Disease, run by University of Kansas Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 24 Feb 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
proportion of participants who have a TRSAE after infusion of MSCTC 0010 (time frame: 45 days). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03158896, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 24 Feb 2026.