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

Graft Versus Host Disease Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00284986
• Other study ID #: Osiris 270-271
• Status: Completed
• Phase: Phase 2
• Start date: November 18, 2005
• Est. completion date: February 8, 2007

Study information

• Verified date: January 2022
• Source: Mesoblast, Ltd.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Description:

Allogeneic hematopoetic stem cell transplantation (HCT) is used in the treatment of a variety of hematological, myeloproliferative and lymphoproliferative disorders, and malignancies involving solid tumors. Patients receiving HCT can develop a life-threatening condition called graft versus host disease (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 patients are refractory to current standard of care treatment. For treatment-refractory patients 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 11
• Est. completion date: February 8, 2007
• Est. primary completion date: April 25, 2006
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 70 Years

Eligibility

Inclusion Criteria:

• 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.

• Participants must provide written informed consent and authorization for use and disclosure of protected health information (PHI).

Exclusion Criteria:

• 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.

Related Conditions & MeSH terms

• Graft Versus Host Disease
• Graft vs Host Disease

Intervention

Drug:
• Prochymal®
Intravenous infusion of ex-vivo cultured adult human mesenchymal stem cells

Locations

Country	Name	City	State
United States	Duke University	Durham	North Carolina

Sponsors (1)

Lead Sponsor	Collaborator
Mesoblast, Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Response by Day 28		Day 28
Secondary	Improvement of GVHD by Day 28 in one or more organs involved with GVHD symptoms at day 1		Day 1
Secondary	Best stage of each involved organ by Day 28		Day 28
Secondary	Time to improvement or resolution of GVHD in one or more organs		Up to approximately 12 months
Secondary	Adverse events		Up to approximately 12 months
Secondary	Infusional toxicity		Up to approximately 12 months
Secondary	Overall relapse of underlying disease		Up to approximately 12 months
Secondary	Overall survival		Up to approximately 12 months
Secondary	Formation of ectopic tissue foci		Up to approximately 12 months
Secondary	Incidence of infection		Up to approximately 12 months