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Trial record 32 of 799 for:    Interventional Studies | mesenchymal

Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta (TERCELOI)

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ClinicalTrials.gov Identifier: NCT02172885
Recruitment Status : Active, not recruiting
First Posted : June 24, 2014
Last Update Posted : February 23, 2018
Sponsor:
Collaborators:
Hospital Universitario Getafe
Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Information provided by (Responsible Party):
Clara I. Rodríguez, Hospital de Cruces

Brief Summary:
The purpose of this study is to determine the safety and effectiveness of five infusions of characterized HLA-identical MSC in non immunosuppressed children with Osteogenesis Imperfecta (OI).

Condition or disease Intervention/treatment Phase
Osteogenesis Imperfecta Biological: Mesenchymal Stem Cells Phase 1

Detailed Description:
The principal aim of this trial is to assess the safety of non-mutated HLA-identical Mesenchymal stem cell (MSC) transplantation for OI pediatric patients irrespective of treatment with biphosphonates. Since MSC are inherently non-immunogenic and do not elicit proliferation of allogeneic lymphocytes (in co-culture experiments), a cell therapy based on HLA-identical or histocompatible (at least 5 shared out of 6 HLA antigens) allogenic MSC may be accomplished without subjecting the patients to immunosuppressor treatment. Adverse secondary effects due to immunosuppressor treatment will be avoided using this strategy thus patients may benefit from two cellular infusions. The patients will be followed for 2 years post their fifth and last MSC infusion.

Study Type : Interventional
Estimated Enrollment : 5 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Study Start Date : April 2014
Estimated Primary Completion Date : December 2018
Estimated Study Completion Date : December 2018


Arm Intervention/treatment
Experimental: Mesenchymal stem cells
Five Mesenchymal Stem Cell infusions
Biological: Mesenchymal Stem Cells
Mesenchymal Stem Cell Infusions




Primary Outcome Measures :
  1. Adverse Events as a Measure of Safety [ Time Frame: up to 2 years post last MSCs infusion ]

Secondary Outcome Measures :
  1. bone mineral density [ Time Frame: up to 2 years post last MSCs infusion ]
  2. fracture rate [ Time Frame: up to 2 years post last MSCs infusion ]
  3. growth velocity [ Time Frame: up to 2 years post last MSCs infusion ]
  4. change from baseline in degree of functionality [ Time Frame: up to 2 years post last MSCs infusion ]
    9 question survey using a Bleck functional scale

  5. change from baseline in well-being [ Time Frame: up to 2 years post last MSCs infusion ]
    A 20 item questionnaire designed to evaluate the well-being will be used



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Ages Eligible for Study:   6 Months to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Patient age: older than 6 months and younger than 12 years old.
  • Patients with molecular confirmation of mutation in either COL1A1 or COL1A2 genes associated with OI (type III).
  • Patients with HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
  • All patients that fulfil the inclusion criteria regardless of whether or not they are undergoing biphosphonate treatment.
  • Patients whose parents or the legal guardians are willing to sign the consent forms to participate in this clinical trial.

Exclusion Criteria:

  • Patient age: older than 12 years old
  • Patients lacking confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III).
  • Other pathological subtypes of OI.
  • Patients lacking of HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
  • Immunodeficiencies and any other malignancies.
  • Participation in other clinical trial.
  • Any medical or psychiatric condition that in the researcher´s opinion could affect the patient´s ability to complete the trial or hamper the participation in the trial.
  • Patients whose parents or the legal guardians do not sign the consent forms

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): NCT02172885


Locations
Spain
Hospital Universitario Cruces
Barakaldo, Bizkaia, Spain, 48903
Hospital Universitario Getafe
Getafe, Madrid, Spain
Sponsors and Collaborators
Hospital de Cruces
Hospital Universitario Getafe
Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Investigators
Principal Investigator: Clara I. Rodríguez, Ph. D. BioCruces Health Research Institute/Cruces University Hospital

Responsible Party: Clara I. Rodríguez, PI Stem Cell Laboratory, Hospital de Cruces
ClinicalTrials.gov Identifier: NCT02172885     History of Changes
Other Study ID Numbers: EudraCT Number:2012-002553-38
Health Department of Spain ( Other Grant/Funding Number: EC10-219 )
First Posted: June 24, 2014    Key Record Dates
Last Update Posted: February 23, 2018
Last Verified: February 2018

Keywords provided by Clara I. Rodríguez, Hospital de Cruces:
Mesenchymal stem cell based therapy
cell infusion

Additional relevant MeSH terms:
Osteogenesis Imperfecta
Osteochondrodysplasias
Bone Diseases, Developmental
Bone Diseases
Musculoskeletal Diseases
Genetic Diseases, Inborn
Collagen Diseases
Connective Tissue Diseases