Umbilical Cord Derived Mesenchymal Stromal Cells For The Treatment of Severe Steroid-resistant Graft Versus Host Disease (PTC-UC-MSC)
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ClinicalTrials.gov Identifier: NCT02032446 |
Recruitment Status : Unknown
Verified January 2019 by Rambaldi Alessandro, A.O. Ospedale Papa Giovanni XXIII.
Recruitment status was: Recruiting
First Posted : January 10, 2014
Last Update Posted : January 18, 2019
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Condition or disease | Intervention/treatment | Phase |
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Hematologic Malignancies | Biological: UMBILICAL CORD DERIVED MESENCHYMAL STROMAL CELLS (UC-MSC) | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Estimated Enrollment : | 47 participants |
Allocation: | N/A |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | UMBILICAL CORD DERIVED MESENCHYMAL STROMAL CELLS (UC-MSC) FOR THE TREATMENT OF SEVERE (GRADE III-IV) STEROID-RESISTANT GRAFT VERSUS HOST DISEASE (GvHD): A PHASE I/II TRIAL |
Study Start Date : | September 2013 |
Actual Primary Completion Date : | September 2016 |
Estimated Study Completion Date : | September 2019 |

Arm | Intervention/treatment |
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Experimental: Umbilical Cord Mesenchymal stromal cells (UC-MSC)
Pentostatin will be given by intravenous infusion at a dose of 1 mg/m2 for 3 consecutive days. Thereafter, three Umbilical Cord Mesenchymal stromal cells (UC-MSC) infusions will be given at weekly interval starting from day 5. We will follow a dose escalating programme with progressively increasing doses of cells until the maximally tolerated dose (MTD) is achieved. The dose escalating design will be characterised by the administration of 1x106 /kg UC-MSC per dose per three doses for the first three patients (total up to 3x106/kg). The second three patients will receive 2x106/kg UC-MSC per dose per three doses (total up to 6x106/kg). The third three patients will receive 3x106/kg UC-MSC per dose per three doses (total up to 9x106 cells/kg). Since three dosages of cells are programmed for each group of 3 patients, a minimum of 9 patients should be studied, unless unacceptable acute infusion related toxicity is observed. |
Biological: UMBILICAL CORD DERIVED MESENCHYMAL STROMAL CELLS (UC-MSC)
pentostatin, dose 1 mg/m2 § MSC doses:
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- vital parameters [ Time Frame: one year ]Following infusion of UC-MSC, the patient will be monitored for acute infusion-related toxicity. Any toxicity will be treated at the discretion of the attending physician. Infusional toxicity is defined as any alteration of the vital parameters of the patient if they have appeared acutely and may be directly correlated to the UC-MSC infusion
- assessed of acute graft versus host disease (GvHD) [ Time Frame: one year ]graft versus host disease will be assessed at day +7, +9, +12, +14, +16, +19, +21, +28, + 35, +42 e +49 and after 6 months and 1 year from the last UC-MSC infusion. Efficacy on acute graft versus host disease is defined as complete or partial resolution of acute GvHD evaluated according to conventional staging and grading score systems.The efficacy will be evaluated at day +30 after the third UC-MSC infusion or, if less, at day +30 after the last UC-MSC infusion.

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Ages Eligible for Study: | up to 70 Years (Child, Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Patients are required to meet the following inclusion criteria:
- Patients with steroid refractory grade III-IV classic acute graft versus host disease (GvHD)occurring within 100 days after transplant or induced by donor lymphocyte infusions (DLI) or T-cell add back. Steroid refractory graft versus host disease (GvHD)is defined according to Pidala and Anasetti10 as follows: a) progression of at least 1 overall grade within 3 days of optimal steroid treatment; b) failure to demonstrate any overall grade improvement over 5 to 7 days; c) incomplete response by 14 days of 2 mg/kg/day of steroid therapy.
- Patients with persistent, recurrent, or late acute graft versus host disease (GvHD) (features of acute graft versus host disease occurring beyond 100 days, often during withdrawal of immune suppression).
- Patients with an overlap syndrome in which diagnostic or distinctive features of chronic graft versus host disease (GvHD) and acute graft versus host disease (GvHD) appear together79.
Exclusion Criteria:
1. Inability to obtain written informed consent

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02032446
Contact: ALESSANDRO RAMBALDI, MD | 035.2673681 ext 0039 | arambaldi@asst-pg23.it |
Italy | |
A O Papa Giovanni XXIII | Recruiting |
Bergamo, Italy, 24127 | |
Contact: Maria Luisa Ferrari, Study coordinator 035.2673681 ext 0039 mlferrari@asst-pg23.it | |
Sub-Investigator: Alessandra Algarotti, MD | |
Sub-Investigator: Caterina Micò, MD | |
Sub-Investigator: Anna Grassi, MD | |
Ao S Croce E Carle | Recruiting |
Cuneo, Italy, 12100 | |
Contact: NICOLA MORANDINI, MD +3901716424478 MORANDININ@GMAIL.COM | |
AO Careggi | Not yet recruiting |
Firenze, Italy, 50134 | |
Contact: Riccardo Saccardi, MD 0557947672 riccardo.saccardi@aou.unifi.it | |
IRCCS G Gaslini | Not yet recruiting |
Genova, Italy, 16147 | |
Contact: Edoardo Lanino, MD 01056362405 edoardolanino@gaslini.org | |
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico | Active, not recruiting |
Milano, Italy, 20122 | |
Clinica Pediatrica San Gerardo | Active, not recruiting |
Monza, Italy, 20900 | |
Azienda Ospedaliero-Universitaria Di Udine | Not yet recruiting |
Udine, Italy, 33100 | |
Contact: RENATO FANIN, MD +39.0432559662 RENATO.FANIN@UNIUD.IT | |
Ospedale San Bortolo | Recruiting |
Vicenza, Italy, 36100 | |
Contact: ROBERTO RAIMONDI, MD +39.0444753518 RAIMONDI@HEMATO.VEN.IT |
Principal Investigator: | Alessandro Rambaldi, MD | A.O. Ospedale Papa Giovanni XXIII |
Publications of Results:
Responsible Party: | Rambaldi Alessandro, Head Hematology and Bone Marrow Transplant Unit, A.O. Ospedale Papa Giovanni XXIII |
ClinicalTrials.gov Identifier: | NCT02032446 |
Other Study ID Numbers: |
EudraCT 2012-000582-21 |
First Posted: | January 10, 2014 Key Record Dates |
Last Update Posted: | January 18, 2019 |
Last Verified: | January 2019 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | Yes |
Plan Description: | interim analysis |
Hematologic Neoplasms Graft vs Host Disease Neoplasms |
Immune System Diseases Neoplasms by Site Hematologic Diseases |