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Mesenchymal Stem Cells as a Treatment for Oral Complications of Graft-versus-host Disease

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ClinicalTrials.gov Identifier: NCT02055625
Recruitment Status : Suspended
First Posted : February 5, 2014
Last Update Posted : March 13, 2018
Sponsor:
Collaborator:
Stockholm County Council, Sweden
Information provided by (Responsible Party):
Rachael Sugars, Karolinska Institutet

Brief Summary:
Graft-versus-host disease (GVHD) is a major complication of allogeneic hematopoietic stem cell transplantation (HSCT), and is classified as acute (aGVHD) or chronic (cGVHD). aGVHD onsets within the first 100 days after transplant or with clinical features including erythema, liver dysfunction and oral mucositis, whilst cGVHD or persistent GVHD occurs in approximately 30-60% of transplant patients who survive their first year . Long-term five-year prognosis for cGVHD patients is poor with a 70% mortality rate. cGVHD manifests as an autoimmune-like disease affecting multiple sites, including skin, mouth, eyes, gastrointestinal tract, liver, and joints. The oral cavity is the second most common site to be affected with symptoms in 45-83% of cases. In the mouth a diverse spectrum of clinical features can be found for example mucosal lesions can affect almost any site, salivary gland dysfunction and restricted mouth opening. Short-term patients can experience mucosal sensitivity, malnutrition, problems speaking, increased caries risk, xerostomia, oral pain and a diminished quality-of-life. Long-term complications include secondary malignancies and perhaps early death. Clinical management seeks to alleviate the symptoms and improve quality-of-life but 50% of patients fail front-line systemic steroid therapy. Oral cGVHD can be treated with topical high potency corticosteroids and oral rinses, however these treatments are not always effective and carry a risk of systemic absorption. Mesenchymal stem/stromal cells (MSCs) resident in adult and fetal tissues, such as the bone marrow have the capacity to form bone, cartilage, stroma, muscle and fat, are known to exhibit immunosuppressive and immunoregulatory properties both in vivo and in vitro. MSC infusions have been used to treat disorders such as osteogenic imperfecta, cardiovascular disease and to heal large bony defects. Indeed, the immunosuppressive capacity of MSCs have led to infusions being used as a second-line treatment for GVHD patients, and our group has shown within a Phase II clinical trial, 55% aGVHD patients who failed front-line steroid treatment responded to MSC infusion these studies are going with cGVHD patients. The goal of this project is to perform a pilot study to determine whether MSC injections directly into mucosal lesions in patients with oral cGVHD are able to alleviate the symptoms and facilitate the reparative process.

Condition or disease Intervention/treatment Phase
Graft -Versus-host-disease Biological: Mesenchymal stromal cells Phase 1 Phase 2

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 12 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Treatment of Oral Mucosa in Patients With Graft-versus-host Disease Following Injection of Mesenchymal Stem Cells - Human Pilot Study
Study Start Date : January 2014
Estimated Primary Completion Date : December 2018

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Mesenchymal stromal cell treatment
Biological: Mesenchymal stromal cells
Biological: Mesenchymal stromal cells
Allogenous mesenchymal stromal cells will be injected directly underneath the mucosal lesions at one side under local anesthetic at 2.5-4 million cells/ml. A total of approximately 1.7 ml will be injected divided in 2-3 injections around the lesion. Patients will be followed every second day up to 7 days afterwards and then routinely at 14 days, 1 month, 2 months and 6 months to assess the healing process.




Primary Outcome Measures :
  1. Change in disease activity according to National Institutes of Health criteria for oral graft-versus-host disease [ Time Frame: Baseline to 6 months ]

Secondary Outcome Measures :
  1. Change in self-assessed disease activity and quality of life [ Time Frame: Baseline to 6 months ]
  2. Safety [ Time Frame: Baseline to 21 months ]
    Frequency of complications, infections and relapse



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Ages Eligible for Study:   18 Years to 80 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Chronic graft-versus-host disease and oral manifestations grade 3 exhibiting severe symptoms, according to the NIH Consensus Working Group for Diagnosis and Staging of cGVHD and have failed frontline therapy

Exclusion Criteria:

  • Active malignancy
  • Fulfilling criteria for previously initiated study for treatment of chronic graft-versus-host disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02055625


Locations
Sweden
Oral and Maxillofacial Surgery, Karolinska Univeristy Hospital
Stockholm, Huddinge, Sweden, 14186
Sponsors and Collaborators
Karolinska Institutet
Stockholm County Council, Sweden
Investigators
Principal Investigator: Katarina Le Blanc, Professor Karolinska Institutet
Principal Investigator: Karin Garming-Legert, DDS, PhD Karolinska Institutet
Principal Investigator: Rachael Sugars, BSc, PhD Karolinska Institutet

Responsible Party: Rachael Sugars, Associate Professor, Karolinska Institutet
ClinicalTrials.gov Identifier: NCT02055625     History of Changes
Other Study ID Numbers: 2013/1241-31/1
First Posted: February 5, 2014    Key Record Dates
Last Update Posted: March 13, 2018
Last Verified: March 2018

Keywords provided by Rachael Sugars, Karolinska Institutet:
Graft-versus-host-disease
Immune system diseases
Oral

Additional relevant MeSH terms:
Graft vs Host Disease
Immune System Diseases