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Graft-versus-host-diseasePhase 1/2MSCforGVHDNCT00827398

Treatment of Steroid Resistant GVHD by Infusion MSC

A Phase 1/2 study testing MSC (hPPL) in people with Graft-versus-host-disease, run by N.M. Wulffraat at 2 sites in Netherlands.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

For numerous malignant diseases allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy. One of the major complications is the occurrence of acute graft-versus-host-disease (aGVHD). Read more about this study

Who is it for?
Ages 1 month to 68 years
Conditions the study is for
Graft-versus-host-disease
What is being tested?
MSC (hPPL)
How many people
50 participants
Where
2 sitesNetherlands
Study dates
Jan 2009to Jul 2013
Phase
Phase 1/2
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 1 month to 68 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Newly diagnosed acute grade II-IV GVHD or chronic GVHD with an acute pattern matching grade II-IV after allogeneic stem cell transplantation
  • Patients must have received 2 mg/kg/day of prednisolon for at least 3 consecutive days and experience progression of GVHD or no response to at least 7 days of steroid treatment.
  • In addition to steroids the patient has received either cyclosporin
  • Written informed consent
  • MSC donor must be HIV, HTLV, hepatitis BS antigen, HCV and HBC, Treponema Pallidum antibody negative. MSC donors can be mismatched related donor, third party matched or mismatched donor.

Who cannot join

  • Patients with poor performance, not expected to survive 3 weeks.
  • Donor Chimerism below 90%
  • Active uncontrolled CMV, EBV or fungal infection

Trial locations and contacts

2 sites in 1 country.

Netherlands2 sites

  • UMC Utrecht, department of pediatrics

    Utrecht, 3508AB

  • UMCU department of Haematology

    Utrecht, 3584AB

Other trials in Netherlands

About this study

Official title: Treatment of Steroid Resistant Grade II to IV GVHD by Infusion MSCof Mesenchymal Stem Cells Expanded With Human Plasma and Platelet Lysate a Phase I/II Study

For numerous malignant diseases allogeneic hematopoietic stem cell transplantation (HSCT) is the only curative therapy. One of the major complications is the occurrence of acute graft-versus-host-disease (aGVHD). Thirty to eighty percent of patients after HSCT develop aGVHD despite the prophylactic application of different immunosuppressive drugs.

The response rates to the conventional first line treatment are only 15-35%4. In case of a steroid refractory aGVHD different therapeutic strategies have been evaluated, but with no satisfactory results so far. The mortality of patients suffering from steroid refractory aGVHD remains at 75-80%. Therefore, it remains important to search for new therapeutical strategies for the treatment of aGVHD.

Read the detailed description

For numerous malignant and non-malignant hematological diseases allogeneic hemato¬poietic stem cell transplantation (HSCT) is the only curative therapy. One of the major complications is the occurrence of acute graft-versus-host-disease (aGVHD). Thirty to eighty percent of patients after HSCT develop aGVHD despite the prophylactic application of different immunosuppressive drugs depending on risk factors such as HLA-match, donor relation, age etc.1-3.

First line therapy of aGVHD > grade I consists of steroids at a dose of 2 mg/kg. The response rates to this treatment are only 15-35%4. In case of a steroid refractory aGVHD different therapeutic strategies have been evaluated, but with no satisfactory results so far. The mortality of patients suffering from steroid refractory aGVHD remains at 75-80%, although numerous studies with different treatment strategies have been conducted2-5. Therefore, it remains important to search for new therapeutical strategies for the treatment of aGVHD.

The first patient to receive mismatched Mesenchymal Stem Cells was a twenty-year-old woman with acute myeloid leukemia treated with peripheral blood stem cells combined with MSC from her haploidentical father. Lazarus et al. reported on 46 patients who received HSCs and culture-expanded MSCs from HLA-identical siblings. Moderate to severe acute GvHD was observed in 28% of the patients, and chronic GvHD was seen in 61%. The two-year progression-free survival was observed in 53% of the patients. MSC infusion caused no acute or long-term MSC-associated adverse events.

Traditionally, for MSC isolation and expansion, fetal calf serum (FCS) supplemented media are used. The use of FCS has however several drawbacks and potential problems. We have therefore established a MSC culture protocol in animal serum free conditions using human platelet lysate and human plasma instead.

The present phase I/II study is designed to gather further insight into the clinical benefit in 50 patients (adults and children) with GvHD exerted by MSC expanded with human platelet lysate and plasma

Being tested
MSC (hPPL)
Keywords
MSC, GVHD, Adults, Children, steroid refractory acute GVHD occurring after allogeneic stem cell transplantation
Medical subject headings
Graft vs Host Disease

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • MSC (hPPL)Biological

    Also called Mesenchymal Stem Cells

    Treatment with MSCs (hPPL) is indicated as soon as steroid refractory acute GVHD is diagnosed

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. number of adverse events after infusion of MSC (hPPL)

    Measured: 6 months

  2. Number of severe infections after MSC infusion

    Measured: 6 months

Secondary outcomes (3)

  1. Response of acute GVHD

    Measured: 6 months

  2. Determination of incidence of chronic GVHD

    Measured: 6 months

  3. Survival

    Measured: 6 months

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jan 2009 (estimated)
  2. Study startJan 2009
  3. Primary completionJul 2013
  4. Study completionJul 2013

Resources

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 1/2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
50 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00827398
Sponsor's protocol ID
NL13729.000.07
Acronym
MSCforGVHD
Status last verified
Nov 2014
First submitted
20 Jan 2009
Last updated
6 Nov 2014
Results posted
No
Data monitoring committee
No

Questions about this trial

What is this clinical trial studying?

A Phase 1/2 study testing MSC (hPPL) in people with Graft-versus-host-disease, run by N.M. Wulffraat at 2 sites in Netherlands.

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 5 inclusion and 3 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 6 Nov 2014. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in Netherlands. 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 combined 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.

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

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?

number of adverse events after infusion of MSC (hPPL) (time frame: 6 months). The study lists 2 primary outcomes in all. 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 NCT00827398.

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 6 Nov 2014.

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