NCT02824653

Brief Summary

Graft versus host disease is a serious and often life-threatening complication in allogeneic haematopoietic stem cell transplantation. Although corticosteroids are the first-choice of treatment in these patients, but about 30-50% patients do not respond to it and develop steroid-refractory GVHD. Mesenchymal stem cells (MSC) have emerged as a promising treatment option in these patients. This phase-I/II clinical trial aims at establishing the safety and clinical efficacy of allogenic ex-vivo cultured MSCs to treat steroid-refractory GVHD in a Pakistani HSCT cohort.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Jan 2015

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2015

Completed
1.5 years until next milestone

First Submitted

Initial submission to the registry

June 28, 2016

Completed
3 days until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2016

Completed
6 days until next milestone

First Posted

Study publicly available on registry

July 7, 2016

Completed
5 months until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2016

Completed
Last Updated

January 25, 2017

Status Verified

January 1, 2017

Enrollment Period

1.5 years

First QC Date

June 28, 2016

Last Update Submit

January 24, 2017

Conditions

Graft Versus Host Disease

Keywords

acute GVHDchronic GVHDsteroid-refractoryMesenchymal Stem Cells

Outcome Measures

Primary Outcomes (3)

  • Incidence of treatment-emergent adverse events and complications [Safety and Tolerability]

    Safety will be evaluated by reporting any incidence of adverse event, complication and toxicity related to administration of MSCs within 4 weeks of last infusion .

    4 weeks

  • Acute GVHD-restaging [Efficacy]

    Efficacy of ex-vivo expanded allogenic mesenchymal stem cell infusion will be determined by organ-specific re-staging and global grading of Acute GVHD as per consensus group criteria. Response will be measured as complete response (CR), partial response (PR), stable disease(SD) and progressive disease(PD). CR:acute GVHD symptoms and signs disappear; PR:aGVHD symptoms and signs improve; SD:aGVHD symptoms and signs remain (without improvement or deterioration);PD: aGVHD symptoms and signs deteriorate.

    4 weeks

  • Chronic GVHD-restaging [Efficacy]

    Organ-specific scoring and global scoring of chronic GVHD will be done as per NIH consensus criteria of 2014. Treatment response will be measured as complete response (CR), partial response (PR), stable disease(SD) and progressive disease(PD). CR:chronic GVHD symptoms and signs disappear; PR:cGVHD symptoms and signs improve; SD:cGVHD symptoms and signs remain (without improvement or deterioration);PD: cGVHD symptoms and signs deteriorate.

    3 months

Secondary Outcomes (2)

  • Survival analysis at six months after MSC infusion

    6 months

  • Incidence free survival at six months after MSC infusion

    6 months

Study Arms (1)

Mesenchymal Stem Cells

EXPERIMENTAL

The experimental arm is comprised of GVHD patients receiving allogenic bone marrow mesenchymal stem cells

Biological: Mesenchymal Stem Cells

Interventions

Mesenchymal Stem CellsBIOLOGICAL

Allogenic Human Bone Marrow Mesenchymal Stem Cells (ex-vivo expanded in pooled human platelet lysate)

Also known as: Allogenic Bone Marrow MSCs
Mesenchymal Stem Cells

Eligibility Criteria

Age1 Year - 65 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Must fill Informed consent.
  • Patient who has undergone allogeneic HSCT and has steroid-refractory grade II-IV acute GVHD.
  • Patients who did not respond to treatment options including methylprednisolone, and or cyclosporine A or ATG.
  • Patients who despite above mentioned treatment have unresponsive GVHD after 5 days or progressive acute GVHD for more than 72 hours.
  • Patients who have received any other treatment and had discontinued for non-responsiveness.

You may not qualify if:

  • Poor performance status, not expected to survive 5 days.
  • Patients with hypersensitivity to penicillin and/or gentamycin.
  • Poor compliance.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Armed Forces Bone Marrow Transplant Centre

Rawalpindi, Punjab Province, 46000, Pakistan

Location

MeSH Terms

Conditions

Graft vs Host DiseaseBronchiolitis Obliterans Syndrome

Condition Hierarchy (Ancestors)

Immune System DiseasesOrganizing PneumoniaBronchiolitis ObliteransBronchiolitisBronchitisBronchial DiseasesRespiratory Tract DiseasesLung Diseases, ObstructiveLung Diseases

Study Officials

  • Parvez Ahmed, FCPS, MCPS

    Armed Forces Bone Marrow Transplant Centre, Rawalpindi, Pakistan

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER GOV
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 28, 2016

First Posted

July 7, 2016

Study Start

January 1, 2015

Primary Completion

July 1, 2016

Study Completion

December 1, 2016

Last Updated

January 25, 2017

Record last verified: 2017-01

Locations

PA(1)