Graft Vs Host DiseasePhase 2NCT00136903

Safety and Efficacy Study of Adult Human Mesenchymal Stem Cells to Treat Acute Graft Versus Host Disease (GVHD)

A Phase 2 study testing Prochymal® - 2 Million cells/kg, Prochymal®- 8 Million cells/kg and Methylprednisolone in people with Graft Vs Host Disease, run by Mesoblast, Inc. at 9 sites 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

To establish the safety and efficacy of two dose levels of ex-vivo cultured adult human mesenchymal stem cells (hMSCs) (Prochymal®) in participants experiencing acute GVHD, Grades II-IV, post hematopoietic stem cells (HSC) transplant. Read more about this study

Who is it for?: Ages 18 to 70 years
Conditions the study is for: Graft Vs Host Disease
What is being tested?: Prochymal® - 2 Million cells/kg, Prochymal®- 8 Million cells/kg and Methylprednisolone
Type of treatment: Pill or oral medicine, Injection or infusion
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 32 participants
Where: 9 sitesUnited States
Study dates: 27 Apr 2005to 14 Jul 2008
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 18 to 70 years
All sexes
No healthy volunteers
Adult, Older adult

Protocol 260 Inclusion Criteria:

Participant must be 18 to 70 years of age inclusive.
If female and of child-bearing age, participant must be non-pregnant, not breast feeding, and use adequate contraception. Males must use adequate contraception.
Participant must have newly diagnosed, Grade II-IV acute GVHD requiring therapy. Biopsy for confirmation of GVHD is not mandatory, but is recommended when feasible. Enrollment should not be delayed awaiting biopsy results.
Participant must have received either full or reduced intensity myeloablative regimens followed by an allogeneic hematopoietic stem cell transplant using bone marrow, peripheral blood stem cell, or cord blood, including donor lymphocyte infusion (DLI).
Participant must have minimal renal and hepatic function as defined by:
Calculated creatinine clearance (CLcr) of > 30 mL/min using the Cockroft-Gault equation.
Participant must be available for all specified assessments at the study site through study Day 28.
Participant must provide written informed consent and authorization for use and disclosure of protected health information (PHI).

Protocol 260 Exclusion Criteria:

Participant has received previous treatment for Grade II-IV acute GVHD (except as noted in Criterion 2).
Participant has been treated for GVHD with methylprednisolone, > 2mg/kg/day, for more than 72 hours prior to receiving Prochymal®.
Participant has uncontrolled alcohol or substance abuse within 6 months of randomization.
Participant has received an investigational agent (not approved by food and drug administration (FDA) for marketed use in any indication) within 30 days of randomization. Participant may not receive an investigational agent during the 28-day study period.
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 unstable arrhythmia.
Participant is unwilling to sign consent form for the long-term follow-up study, Protocol 261.
Participant has a known allergy to bovine or porcine products.
Participant had received transplant for a solid tumor disease.

Trial locations and contacts

9 sites in 1 country.

City, postcode or hospital

9 sites in 1 country

United States9 sites

Roswell Park Cancer Institute
Buffalo, New York, 14263
The Cancer Center at Hackensack University
Hackensack, New Jersey, 07601
MD Anderson Cancer Center
Houston, Texas, 77030
St. Francis Hospital
Indianapolis, Indiana, 46237
Kansas City Cancer Centers - BMT
Kansas City, Missouri, 64111
Medical College of Wisconsin, FEC
Milwaukee, Wisconsin, 53226
Mt. Sinai Hospital
New York, New York, 10029
University of Rochester
Rochester, New York, 14642
New York Medical College
Valhalla, New York, 10595

Other trials in United States

About this study

Official title: A Phase II, Randomized Study to Evaluate the Safety and Efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) For the Treatment of Acute GVHD in Patients Who Receive Allogeneic Hematopoietic Stem Cell Transplantation

Read the detailed description

Protocol 260 - Participants will be randomized with equal probability to the treatment arms (2 million cells/kilogram (kg) of Prochymal® or 8 million cells/kg of Prochymal®) using a stratified block design. The stratification factor is acute GVHD grade. For the purpose of stratification, the GVHD grades are II and III-IV. Treatment with investigational agent will be administered on study Days 1 and 4. Participants will be followed for safety and efficacy until Day 28 after initiation of treatment with the investigational agent, or until withdrawal or death, whichever occurs first.

Protocol 261- This study is designed as a long-term safety follow-up of participants who take part in the preceding clinical study of Prochymal® (Protocol 260) for the treatment of acute GVHD. Participants will be enrolled in Study 261 upon completion of the preceding Study 260.

Conditions: Graft Vs Host Disease
Being tested: Prochymal® - 2 Million cells/kg, Prochymal®- 8 Million cells/kg, Methylprednisolone, Prednisone, Cyclosporine
Keywords: Graft vs Host Disease, (GVHD), Graft Versus Host Disease, Bone marrow transplant, Stem cells, Mesenchymal stem cells (MSCs), Adult stem cells, Leukemia, Lymphoma
Medical subject headings: Graft vs Host Disease, Leukemia, Lymphoma

What participants receive

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

Prochymal® - 2 Million cells/kgDrug
Also called Remestemcel-L, ex-vivo cultured adult human mesenchymal stem cells, hMSCs
2 million hMSCs/kg actual body weight, IV on study Days 1 and 4
Prochymal®- 8 Million cells/kgDrug
Also called Remestemcel-L, ex-vivo cultured adult human mesenchymal stem cells, hMSCs
8 million hMSCs/kg actual body weight IV on study Days 1 and 4
MethylprednisoloneDrug
Methylprednisolone 2 mg/kg administered intravenously.
PrednisoneDrug
Prednisone 2.5 mg/kg administered orally.
CyclosporineDrug
Administered as prescribed by the caregiver.
TacrolimusDrug
Administered as prescribed by the caregiver.
Mycophenolate MofetilDrug
Administered as prescribed by the caregiver.

Study groups in NCT00136903
Study group	Role	What they receive
Prochymal® - 2 Million cells/kg	Active comparator	Participants will receive Prochymal® consisting of 2 million hMSCs/kg actual body weight, intravenously (IV) on Days 1 and 4 along with daily standard of care which includes methylprednisolone 2 milligrams (mg)/kg IV or prednisone 2.5 mg/kg orally. Participants will also continue cyclosporine, tacrolimus, and/or mycophenolate mofetil (MMF) at full therapeutic doses. Receives: Drug: Prochymal® - 2 Million cells/kg; Drug: Methylprednisolone; Drug: Prednisone; Drug: Cyclosporine; Drug: Tacrolimus; Drug: Mycophenolate Mofetil
Prochymal® - 8 Million cells/kg	Active comparator	Participants will receive Prochymal® consisting of 8 million hMSCs/kg actual body weight IV on Days 1 and 4 along with daily standard of care which includes methylprednisolone 2 mg/kg IV or prednisone 2.5 mg/kg orally. Participants will also continue cyclosporine, tacrolimus, and/or mycophenolate mofetil (MMF) at full therapeutic doses. Receives: Drug: Prochymal®- 8 Million cells/kg; Drug: Methylprednisolone; Drug: Prednisone; Drug: Cyclosporine; Drug: Tacrolimus; Drug: Mycophenolate Mofetil

What is being measured

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

Protocol 260 - Response by Day 28, also called Overall Response (OR). OR includes complete response (CR) and partial response (PR)
Measured: 28 Days
Protocol 261-The incidence rate of different adverse events among participants treated with either dose of Prochymal® in the preceding study (Protocol No. 260).
Measured: 2 Years

Secondary outcomes (4)

Protocol 260 - Partial Response or Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at Day 1,
Measured: 28 Days
Protocol 260 - Time to best response of GVHD
Measured: 28 Days
Protocol 260 - Time to improvement of GVHD in one or more organs
Measured: 28 Days
Protocol 261 - Survival through study 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.

27 Apr 200514 Jul 2008

First posted29 Aug 2005 (estimated)
Study start27 Apr 2005
Primary completion28 Jul 2006
Study completion14 Jul 2008

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Publications (6)Papers the sponsor has linked to this study.
Other trials for Graft Vs Host DiseaseStudies of the same condition, open and under way.
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 2
Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Enrolment: 32 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT00136903
Sponsor's protocol ID: 260-261
Status last verified: Jan 2022
First submitted: 25 Aug 2005
Last updated: 31 Jan 2022
Results posted: No
Data monitoring committee: No
FDA-regulated drug: Yes
FDA-regulated device: No

Publications

Bartholomew A, Sturgeon C, Siatskas M, Ferrer K, McIntosh K, Patil S, Hardy W, Devine S, Ucker D, Deans R, Moseley A, Hoffman R. Mesenchymal stem cells suppress lymphocyte proliferation in vitro and prolong skin graft survival in vivo. Exp Hematol. 2002 Jan;30(1):42-8. doi: 10.1016/s0301-472x(01)00769-x.
PubMed 11823036
Deans RJ, Moseley AB. Mesenchymal stem cells: biology and potential clinical uses. Exp Hematol. 2000 Aug;28(8):875-84. doi: 10.1016/s0301-472x(00)00482-3.
PubMed 10989188
Lazarus HM, Koc ON, Devine SM, Curtin P, Maziarz RT, Holland HK, Shpall EJ, McCarthy P, Atkinson K, Cooper BW, Gerson SL, Laughlin MJ, Loberiza FR Jr, Moseley AB, Bacigalupo A. Cotransplantation of HLA-identical sibling culture-expanded mesenchymal stem cells and hematopoietic stem cells in hematologic malignancy patients. Biol Blood Marrow Transplant. 2005 May;11(5):389-98. doi: 10.1016/j.bbmt.2005.02.001.
PubMed 15846293
Le Blanc K, Rasmusson I, Sundberg B, Gotherstrom C, Hassan M, Uzunel M, Ringden O. Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. Lancet. 2004 May 1;363(9419):1439-41. doi: 10.1016/S0140-6736(04)16104-7.
PubMed 15121408
Le Blanc K, Pittenger M. Mesenchymal stem cells: progress toward promise. Cytotherapy. 2005;7(1):36-45. doi: 10.1080/14653240510018118.
PubMed 16040382
Kebriaei P, Isola L, Bahceci E, Holland K, Rowley S, McGuirk J, Devetten M, Jansen J, Herzig R, Schuster M, Monroy R, Uberti J. Adult human mesenchymal stem cells added to corticosteroid therapy for the treatment of acute graft-versus-host disease. Biol Blood Marrow Transplant. 2009 Jul;15(7):804-11. doi: 10.1016/j.bbmt.2008.03.012.
PubMed 19539211

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 2
De-escalated Cyclophosphamide (PTCy) and Ruxolitinib for Graft-versus-Host Disease (GVHD) Prophylaxis
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RecruitingEarly Phase 1
HMCT/CT2401: Abatacept GVHD Prophylaxis Following Omidubicel HCT
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RecruitingObservational study
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RecruitingPhase 1/2
The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation
Peripheral Blood Stem Cell Transplantation, Hematopoietic Stem Cell Transplantation
RecruitingPhase 1/2
Phase I/II Study of Pacritinib, A JAK2/IRAK1/CSF1R Inhibitor, in Refractory Chronic Graft-Versus-Host Disease (cGVHD) After Allogeneic Hematopoietic Stem Cell Transplantation (HSCT)
Graft vs Host Disease

All Graft Vs Host Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 31 Jan 2022. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: A Phase II, Randomized Study to Evaluate the Safety and Efficacy of Prochymal® (Ex-vivo Cultured Adult Human Mesenchymal Stem Cells) For the Treatment of Acute GVHD in Patients Who Receive Allogeneic Hematopoietic Stem Cell Transplantation
ClinicalTrials.gov ID: NCT00136903
Acronym / sponsor ID: 260-261
Conditions: Graft Vs Host Disease
Keywords: Graft vs Host Disease, (GVHD), Graft Versus Host Disease, Bone marrow transplant, Stem cells, Mesenchymal stem cells (MSCs), Adult stem cells, Leukemia
Phase: Phase 2
Study type: Interventional
Recruitment status: Completed
Enrolment: 32 participants (actual)
Lead sponsor: Mesoblast, Inc.Industry (pharma, biotech, devices)
Study start: 27 Apr 2005 (actual)
Primary completion: 28 Jul 2006 (actual)
Study completion: 14 Jul 2008 (actual)
First posted: 29 Aug 2005 (estimated)
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.

Detailed description

Study design

Primary purpose: Treatment
Allocation: Randomised
Intervention model: Parallel groups
Masking: None (open label)
Number of arms: 2

Arm	Type	Receives
Prochymal® - 2 Million cells/kg	Active comparator	Drug: Prochymal® - 2 Million cells/kg, Drug: Methylprednisolone, Drug: Prednisone, Drug: Cyclosporine, Drug: Tacrolimus, Drug: Mycophenolate MofetilParticipants will receive Prochymal® consisting of 2 million hMSCs/kg actual body weight, intravenously (IV) on Days 1 and 4 along with daily standard of care which includes methylprednisolone 2 milligrams (mg)/kg IV or prednisone 2.5 mg/kg orally. Participants will also continue cyclosporine, tacrolimus, and/or mycophenolate mofetil (MMF) at full therapeutic doses.
Prochymal® - 8 Million cells/kg	Active comparator	Drug: Prochymal®- 8 Million cells/kg, Drug: Methylprednisolone, Drug: Prednisone, Drug: Cyclosporine, Drug: Tacrolimus, Drug: Mycophenolate MofetilParticipants will receive Prochymal® consisting of 8 million hMSCs/kg actual body weight IV on Days 1 and 4 along with daily standard of care which includes methylprednisolone 2 mg/kg IV or prednisone 2.5 mg/kg orally. Participants will also continue cyclosporine, tacrolimus, and/or mycophenolate mofetil (MMF) at full therapeutic doses.

Who may qualify

Ages: 18 to 70 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Adult, Older adult

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
Prochymal® - 2 Million cells/kgAlso: Remestemcel-L, ex-vivo cultured adult human mesenchymal stem cells, hMSCs2 million hMSCs/kg actual body weight, IV on study Days 1 and 4	Drug	Prochymal® - 2 Million cells/kg
Prochymal®- 8 Million cells/kgAlso: Remestemcel-L, ex-vivo cultured adult human mesenchymal stem cells, hMSCs8 million hMSCs/kg actual body weight IV on study Days 1 and 4	Drug	Prochymal® - 8 Million cells/kg
MethylprednisoloneMethylprednisolone 2 mg/kg administered intravenously.	Drug	Prochymal® - 2 Million cells/kg, Prochymal® - 8 Million cells/kg
PrednisonePrednisone 2.5 mg/kg administered orally.	Drug	Prochymal® - 2 Million cells/kg, Prochymal® - 8 Million cells/kg
CyclosporineAdministered as prescribed by the caregiver.	Drug	Prochymal® - 2 Million cells/kg, Prochymal® - 8 Million cells/kg
TacrolimusAdministered as prescribed by the caregiver.	Drug	Prochymal® - 2 Million cells/kg, Prochymal® - 8 Million cells/kg
Mycophenolate MofetilAdministered as prescribed by the caregiver.	Drug	Prochymal® - 2 Million cells/kg, Prochymal® - 8 Million cells/kg

Primary outcome

Protocol 260 - Response by Day 28, also called Overall Response (OR). OR includes complete response (CR) and partial response (PR)
Time frame: 28 Days
Protocol 261-The incidence rate of different adverse events among participants treated with either dose of Prochymal® in the preceding study (Protocol No. 260).
Time frame: 2 Years

4 secondary outcomes

Protocol 260 - Partial Response or Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at Day 1,
Time frame: 28 Days
Protocol 260 - Time to best response of GVHD
Time frame: 28 Days
Protocol 260 - Time to improvement of GVHD in one or more organs
Time frame: 28 Days
Protocol 261 - Survival through study 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 31 Jan 2022.
Background: PubMed 11823036Bartholomew A, Sturgeon C, Siatskas M, Ferrer K, McIntosh K, Patil S, Hardy W, Devine S, Ucker D, Deans R, Moseley A, Hoffman R. Mesenchymal stem cells suppress lymphocyte proliferation in vitro and prolong skin graft survival in vivo. Exp Hematol. 2002 Jan;30(1):42-8. doi: 10.1016/s0301-472x(01)00769-x.
Background: PubMed 10989188Deans RJ, Moseley AB. Mesenchymal stem cells: biology and potential clinical uses. Exp Hematol. 2000 Aug;28(8):875-84. doi: 10.1016/s0301-472x(00)00482-3.
Background: PubMed 15846293Lazarus HM, Koc ON, Devine SM, Curtin P, Maziarz RT, Holland HK, Shpall EJ, McCarthy P, Atkinson K, Cooper BW, Gerson SL, Laughlin MJ, Loberiza FR Jr, Moseley AB, Bacigalupo A. Cotransplantation of HLA-identical sibling culture-expanded mesenchymal stem cells and hematopoietic stem cells in hematologic malignancy patients. Biol Blood Marrow Transplant. 2005 May;11(5):389-98. doi: 10.1016/j.bbmt.2005.02.001.
Background: PubMed 15121408Le Blanc K, Rasmusson I, Sundberg B, Gotherstrom C, Hassan M, Uzunel M, Ringden O. Treatment of severe acute graft-versus-host disease with third party haploidentical mesenchymal stem cells. Lancet. 2004 May 1;363(9419):1439-41. doi: 10.1016/S0140-6736(04)16104-7.
Background: PubMed 16040382Le Blanc K, Pittenger M. Mesenchymal stem cells: progress toward promise. Cytotherapy. 2005;7(1):36-45. doi: 10.1080/14653240510018118.
Results publication: PubMed 19539211Kebriaei P, Isola L, Bahceci E, Holland K, Rowley S, McGuirk J, Devetten M, Jansen J, Herzig R, Schuster M, Monroy R, Uberti J. Adult human mesenchymal stem cells added to corticosteroid therapy for the treatment of acute graft-versus-host disease. Biol Blood Marrow Transplant. 2009 Jul;15(7):804-11. doi: 10.1016/j.bbmt.2008.03.012.
Click here for more information on Prochymal® for treatment of GVHDLink provided by the sponsor.

Related trials for Graft Vs Host Disease

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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.

Christopher James, PA study director

Mesoblast, Inc.

9 sites in 1 country: see every site and its status.

Questions about this trial

What is this clinical trial studying?

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

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 31 Jan 2022. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites 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?

Protocol 260 - Response by Day 28, also called Overall Response (OR). OR includes complete response (CR) and partial response (PR) (time frame: 28 Days). 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 NCT00136903, which also lists 6 linked publications.

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 31 Jan 2022.