Decidual Stromal Cells as Treatment for Acute Graft Versus Host Disease, a Phase 1-2 Study

Decidual Stromal Cells as Treatment for Acute Graft Versus Host Disease

The purpose of this study is to evaluate safety and efficacy using decidual stromal cell therapy for graft versus host disease after allogeneic hematopoietic stem cell transplantation. The hypothesis to be tested is that the cells are safe to infuse and that they have a positive clinical effect.

Study Overview

• Status: Unknown
• Conditions: Graft vs Host Disease
• Intervention / Treatment: Biological: Decidual Stromal Cells thawed in plasma; Biological: Decidual Stromal Cells thawed in human albumin

Detailed Description

Patients with GVHD grade 2-4, will receive decidual stromal cells at approximately 1-2x10^6 cells/kg at one or more occasions dependent on clinical response.

• Study Type: Interventional
• Enrollment (Anticipated): 50
• Phase: Phase 2; Phase 1

Contacts and Locations

• Study Contact: Name: Olle Ringdén, MD, PhD; Phone Number: +46858582672; Email: olle.ringden@ki.se
• Study Contact Backup: Name: Helen Kaipe, PhD; Phone Number: +46700901052; Email: helen.kaipe@ki.se

Study Locations

• Sweden
  • Stockholm, Sweden, 14186
    • Recruiting
    • Karolinska Institutet
    • Principal Investigator: Olle Ringdén, MD, PhD
    • Contact: Olle Ringdén, MD, PhD; Phone Number: +858582672; Email: olle.ringden@ki.se

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult; Older Adult
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

• Acute graft versus host disease grade 2-4.
• Are on calcineurin inhibitor and high dose corticosteroids.

Exclusion Criteria:

None.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Decidual Stromal Cells as last line treatment; Patients with therapy-refractory GVHD and on calcineurin inhibitor and high dose corticosteroids without any signs of improvement will be given DSCs to evaluate a possible effect. DSCs will be thawed from the freezer in plasma.	Biological: Decidual Stromal Cells thawed in plasma
Active Comparator: Decidual Stromal Cells; Patients with therapy-refractory GVHD and on calcineurin inhibitor and high dose corticosteroids will be given DSCs as early as possible at one or more occasions at weekly intervals dependent on clinical response. DSCs will be thawed from the freezer in plasma.	Biological: Decidual Stromal Cells thawed in human albumin

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Actuarial survival at six months after first DSC infusion	6 months after inclusion

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Response at 28 days after onset of graft versus host disease	Response will be measured as: Partial response (PR) if the patient has improved one grade in the overall GvH.; Complete response (CR) if the patient is free of GvH.; Non responder (NR) if the patient does not improve after treatment.	28 days after inclusion
Response at 84 days after onset of graft versus host disease	Response will be measured as: Partial response (PR) if the patient has improved one grade in the overall GvH.; Complete response (CR) if the patient is free of GvH.; Non responder (NR) if the patient does not improve after treatment.	Up to 84 days after inclusion
Response at 168 days after onset of graft versus host disease	Response will be measured as: Partial response (PR) if the patient has improved one grade in the overall GvH.; Complete response (CR) if the patient is free of GvH.; Non responder (NR) if the patient does not improve after treatment.	Up to 168 days after inclusion
Side effects	Adverse effects related to the treatment.	Up to 6 months after inclusion
Incidence of severe infections	Severe bacterial, viral and fungal infections.	Up to one year after inclusion
Disease free survival	Survival free from relapse.	Up to one year after inclusion

Collaborators and Investigators

• Sponsor: Karolinska Institutet
• Collaborators: Karolinska University Hospital
• Investigators: Principal Investigator: Olle Ringdén, MD, PhD, Karolinska Institutet

Publications and helpful links

General Publications

• Erkers T, Nava S, Yosef J, Ringden O, Kaipe H. Decidual stromal cells promote regulatory T cells and suppress alloreactivity in a cell contact-dependent manner. Stem Cells Dev. 2013 Oct 1;22(19):2596-605. doi: 10.1089/scd.2013.0079. Epub 2013 Jul 2.
• Ringden O, Erkers T, Nava S, Uzunel M, Iwarsson E, Conrad R, Westgren M, Mattsson J, Kaipe H. Fetal membrane cells for treatment of steroid-refractory acute graft-versus-host disease. Stem Cells. 2013 Mar;31(3):592-601. doi: 10.1002/stem.1314.
• Sadeghi B, Remberger M, Gustafsson B, Winiarski J, Moretti G, Khoein B, Klingspor L, Westgren M, Mattsson J, Ringden O. Long-Term Follow-Up of a Pilot Study Using Placenta-Derived Decidua Stromal Cells for Severe Acute Graft-versus-Host Disease. Biol Blood Marrow Transplant. 2019 Oct;25(10):1965-1969. doi: 10.1016/j.bbmt.2019.05.034. Epub 2019 Jun 4.

Study record dates

Study Major Dates

• Study Start: January 1, 2011
• Primary Completion (Anticipated): December 1, 2016
• Study Completion (Anticipated): December 1, 2016

Study Registration Dates

• First Submitted: June 17, 2014
• First Submitted That Met QC Criteria: June 23, 2014
• First Posted (Estimate): June 24, 2014

Study Record Updates

• Last Update Posted (Estimate): November 11, 2014
• Last Update Submitted That Met QC Criteria: November 8, 2014
• Last Verified: November 1, 2014

More Information

Terms related to this study

• Keywords: Stem Cell Transplantation; Decidual Stromal Cells
• Additional Relevant MeSH Terms: Immune System Diseases; Graft vs Host Disease
• Other Study ID Numbers: DSCGVHD002