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Graft Versus Host DiseasePhase 2NCT00284986

Safety and Efficacy of Prochymal for the Salvage of Treatment-Refractory Acute GVHD Participants

A Phase 2 study testing Prochymal in people with Graft Versus Host Disease, run by Mesoblast, Inc. at 1 site in United States.

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

This study is designed to evaluate the safety and efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) in participants experiencing treatment-refractory acute GVHD, Grades III-IV, that is refractory to standard first-line therapies and at least one second-line therapy. Read more about this study

Who is it for?
Ages 6 months to 70 years
Conditions the study is for
Graft Versus Host Disease
What is being tested?
Prochymal
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
11 participants
Where
1 siteUnited States
Study dates
18 Nov 2005to 8 Feb 2007
Phase
Phase 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 6 months to 70 years
  • All sexes
  • No healthy volunteers
  • Child, Adult, Older adult

Who may be able to join

  • Participants must be 6 months to 70 years of age inclusive.
  • If female and of childbearing age, participants must be non-pregnant, not breast-feeding, and use adequate contraception. Male participants must use adequate contraception.
  • Participants must have Grade III-IV acute GVHD that has failed to respond to standard first and at least one second-line therapy. Biopsy for confirmation of both skin and gastrointestinal GVHD is not mandatory, but is recommended when feasible. Enrollment should not be delayed awaiting biopsy results.
  • Participants must have minimal renal function as defined by: Calculated creatinine clearance (CrCl) of > 30 milliliters/minute (mL/min) using the Cockcroft-Gault equation.
  • Participant must provide written informed consent and authorization for use and disclosure of protected health information (PHI).

Who cannot join

  • Participant has uncontrolled alcohol or substance abuse within 6 months of treatment.
  • 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 (e.g., uncontrolled infection, right heart failure, pulmonary hypertension, etc.).
  • Participant has a clinically significant, unstable arrhythmia.
  • Participant has a known allergy to bovine or porcine products.
  • Participant is unwilling to sign consent form for the long-term follow-up study, protocol 271.

Trial locations and contacts

1 site in 1 country.

United States1 site

  • Duke University

    Durham, North Carolina, 27708

Other trials in United States

About this study

Official title: A Phase II Open Label Study to Evaluate the Safety and Efficacy of Prochymal (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) Infusion for the Salvage of Treatment-Refractory Acute GVHD Patients

This study is designed to evaluate the safety and efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) in participants experiencing treatment-refractory acute GVHD, Grades III-IV, that is refractory to standard first-line therapies and at least one second-line therapy.

Read the detailed description

Allogeneic HSCT is used in the treatment of a variety of hematological, myeloproliferative and lymphoproliferative disorders, and malignancies involving solid tumors. Participants receiving HSCT can develop a life-threatening condition called GVHD. GVHD occurs when donor T cells from the donor bone marrow recognize host cells as "foreign" and initiate an inflammatory immunological response. The standard of care for treatment of acute GVHD consists of intravenous delivery of methylprednisolone starting on Day 1 and continuation of either cyclosporine or tacrolimus. This regimen of steroids and immunosuppressive drugs may relieve symptoms of GVHD, but some participants are refractory to current standard of care treatment. For treatment-refractory participants with grades III-IV GVHD mortality is approximately 80%. A therapy that could effectively suppress the immunological response from GVHD and help repair the damaged tissue could significantly decrease the mortality rate from this disease.

Being tested
Prochymal
Keywords
GVHD, Graft vs Host Disease, Graft versus Host Disease
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.

  • ProchymalDrug

    Intravenous infusion of ex-vivo cultured adult human mesenchymal stem cells

Study groups in NCT00284986
Study groupRoleWhat they receive
PROCHYMAL™ExperimentalNot described
Receives: Drug: Prochymal

What is being measured

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

  1. Response by Day 28

    Responses included complete response (CR), partial response (PR), failure to respond. CR is defined as a complete resolution of graft-versus-host disease (GVHD). PR is defined as improvement in at least one organ by at least one full stage in the absence of progression in any other organ, or resolution of GVHD in at least one organ with a need for additional treatment because of abnormalities persisting in another organ. Failure to respond is defined as progression of GVHD.

    Measured: Day 28

Secondary outcomes (9)

  1. Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1

    Measured: Day 1

  2. Best stage of each involved organ by Day 28

    Measured: Day 28

  3. Time to improvement or resolution of GVHD in one or more organs

    Measured: Up to approximately 12 months

  4. Adverse events

    Measured: Up to approximately 12 months

  5. Infusional toxicity

    Measured: Up to approximately 12 months

  6. Overall relapse of underlying disease

    Measured: Up to approximately 12 months

  7. Overall survival

    Measured: Up to approximately 12 months

  8. Formation of ectopic tissue foci

    Measured: Up to approximately 12 months

  9. Incidence of infection

    Measured: Up to approximately 12 months

Timeline

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

  1. First posted1 Feb 2006 (estimated)
  2. Study start18 Nov 2005
  3. Primary completion25 Apr 2006
  4. Study completion8 Feb 2007

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 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
11 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT00284986
Sponsor's protocol ID
Osiris 270-271
Status last verified
Apr 2023
First submitted
31 Jan 2006
Last updated
30 Oct 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Prochymal in people with Graft Versus Host Disease, run by Mesoblast, Inc. 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 5 inclusion and 5 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 30 Oct 2025. 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 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?

Response by Day 28 (time frame: Day 28). 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 NCT00284986.

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 30 Oct 2025.

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