Condition category
Injury, Occupational Diseases, Poisoning
Date applied
12/07/2017
Date assigned
18/08/2017
Last edited
07/08/2017
Prospective/Retrospective
Retrospectively registered
Overall trial status
Ongoing
Recruitment status
No longer recruiting

Plain English Summary

Background and study aims
Acute graft-versus-host disease (aGVHD) is a possible complication of a bone marrow stem cell transplant from another person (allogeneic hematopoietic stem cell transplantation). It is caused by the donated immune cells reacting against the host tissues. It is potentially lethal and around half of stem cell recipients develop it. Acute GVHD typically occurs in the first 100 days after transplant and affects three organs: skin, liver and gastrointestinal (digestive) tract. The first treatment is to use glucocorticoid drugs but half of the patients do not respond to treatment (steroid refractory). The prognosis of patients who develop acute GVHD and do not respond to treatment is dismal. Numerous strategies to treat steroid-refractory aGVHD with a second treatment have been tested, but none have shown an effect. Therefore, there is no established second-line treatment for steroid-refractory acute GvHD. Mesenchymal stromal cells (MSCs) can decrease the activity of most cells of the immune system. The aim of this study is to assess the effectiveness of MSC treatment for steroid-refractory aGVHD.

Who can participate?
Patients aged 18 and older with steroid-refractory aGVHD after hematopoietic stem cell transplantation

What does the study involve?
All participants are treated with mesenchymal stromal cells. Blood samples are taken during treatment and at follow ups. Patients are followed up to assess their response to treatment, relapse and survival rates.

What are the possible benefits and risks of participating?
There are no benefits or risks for participants, but the results could be used to improve the treatment of patients with aGVHD in the future.

Where is the study run from?
Vilnius University Hospital Santaros Klinikos (Lithuania)

When is the study starting and how long is it expected to run for?
October 2013 to November 2022

Who is funding the study?
Hematology and Oncology Research Association of Lithuania

Who is the main contact?
Mr Adomas Bukauskas

Trial website

Contact information

Type

Scientific

Primary contact

Mr Adomas Bukauskas

ORCID ID

http://orcid.org/0000-0001-5857-4638

Contact details

Seliu 29-41
Vilnius
LT-08125
Lithuania

Additional identifiers

EudraCT number

ClinicalTrials.gov number

Protocol/serial number

TPSL-LTU-2015

Study information

Scientific title

Mesenchymal stromal cells in patients with steroid refractory acute graft versus host disease after allogeneic hematopoietic stem cell transplantation or donor lymphocyte infusion

Acronym

Study hypothesis

Mesenchymal stromal cells have been shown to have immunosuppressive effects. Through various mechanisms mesenchymal stromal cells exert an effect on most cells of the immune system and inhibit proliferation, activation, and cytokine release. Donor T cell activation and inflammatory cytokines play a major role in acute graft versus host disease (aGVHD) pathogenesis. The trialists hypothesize that by immunomodulatory properties mesenchymal stromal cells will be effective in controlling steroid refractory aGVHD and will prolong overall survival.

Ethics approval

Vilnius regional Bioethics Committee, 03/11/2015, ref: 1582000-15-814-326

Study design

Observational prospective non-interventional single-center study

Primary study design

Observational

Secondary study design

Longitudinal study

Trial setting

Hospitals

Trial type

Treatment

Patient information sheet

Not available in web format, please use the contact details to request a patient information sheet

Condition

Steroid refractory acute graft versus host disease after hematopoietic stem cell transplantation or donor lymphocyte infusion

Intervention

Patients with steroid refractory acute graft versus host disease were treated with mesenchymal stromal cells as compassionate use medicinal product (standard of care). Doses have not been predetermined in the protocol and follow standard practice. Additionally, blood samples were taken during treatment phase and at follow ups.

Intervention type

Biological/Vaccine

Phase

Drug names

Primary outcome measures

Overall response rate (ORR) to mesenchymal stromal cells, defined as complete response (CR) or partial response (PR) of acute graft versus host disease, measured at D7, D14, D21, D28, D35, D42, D49, D56, 3 months

Secondary outcome measures

Measured at D7, D14, D21, D28, D35, D42, D49, D56, 3 months, 6 months, 9 months, 12 months, 18 months, 24 months and each year thereafter:
1. Overall survival (OS), defined as time from the start of treatment until death from any cause
2. Event free survival, defined as time from the start of treatment until the following events (whichever occurs first): death, hematologic malignancy relapse, no PR/CR by 3 months since the study entry, aGVHD relapse after PR/CR requiring next line treatment, progression to extensive chronic GVHD

Overall trial start date

09/10/2013

Overall trial end date

30/11/2022

Reason abandoned

Eligibility

Participant inclusion criteria

1. Steroid refractory acute graft versus host disease after hematopoietic stem cell transplantation
2. 18 years old and older
3. Signed informed consent form

Participant type

Patient

Age group

Adult

Gender

Both

Target number of participants

45

Participant exclusion criteria

Does not meet inclusion criteria

Recruitment start date

09/10/2013

Recruitment end date

10/04/2017

Locations

Countries of recruitment

Lithuania

Trial participating centre

Vilnius University Hospital Santaros Klinikos
Santariskiu 2
Vilnius
LT-08406
Lithuania

Sponsor information

Organisation

Hematology and Oncology Research Association of Lithuania (Hematologijos ir onkologijos tyrėjų asociacija LTU)

Sponsor details

Savicikio 4
Vilnius
01108
Lithuania

Sponsor type

Research organisation

Website

Funders

Funder type

Research organisation

Funder name

Hematology and Oncology Research Association of Lithuania (Hematologijos ir onkologijos tyrėjų asociacija LTU)

Alternative name(s)

Funding Body Type

Funding Body Subtype

Location

Results and Publications

Publication and dissemination plan

Planned publication in a peer-reviewed journal in 2018.

IPD sharing statement
The data sharing plans for the current study are unknown and will be made available at a later date.

Intention to publish date

31/12/2018

Participant level data

To be made available at a later date

Results - basic reporting

Publication summary

Publication citations

Additional files

Editorial Notes