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

NCT00284986 | Completed Phase 2 FDA Drug

• 1 other identifier: Osiris 270-271
• Study Type: interventional
• Target: 11
• Locations: 1 country
• Sites: 1

Brief 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.

Conditions

Graft Versus Host Disease

Keywords

GVHD; Graft vs Host Disease; Graft versus Host Disease

Outcome Measures

Primary Outcomes (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.

  Day 28

Secondary Outcomes (9)

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

  Day 1

• Best stage of each involved organ by Day 28

  Day 28

• Time to improvement or resolution of GVHD in one or more organs

  Up to approximately 12 months

• Adverse events

  Up to approximately 12 months

• Infusional toxicity

  Up to approximately 12 months

Study Arms (1)

PROCHYMAL™

EXPERIMENTAL

Drug: Prochymal

Interventions

Prochymal DRUG

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

PROCHYMAL™

Eligibility Criteria

• Age: 6 Months - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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).

You may not qualify if:

• 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.

Sponsors & Collaborators

• Mesoblast, Inc.: lead

Study Sites (1)

Duke University

Durham, North Carolina, 27708, United States

Location

MeSH Terms

Conditions

Graft vs Host Disease

Interventions

remestemcel-l

Condition Hierarchy (Ancestors)

Immune System Diseases

Study Officials

• Christopher James, PA

  Mesoblast, Inc.

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 31, 2006
• First Posted: February 1, 2006
• Study Start: November 18, 2005
• Primary Completion: April 25, 2006
• Study Completion: February 8, 2007
• Last Updated: October 30, 2025

Record last verified: 2023-04

Locations

US (1)