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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02172885
Recruitment Status : Completed
First Posted : June 24, 2014
Last Update Posted : March 26, 2019
Sponsor:
Collaborators:
Hospital Universitario Getafe
Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Information provided by (Responsible Party):
Clara I. Rodríguez, Hospital de Cruces

Tracking Information
First Submitted Date  ICMJE June 12, 2014
First Posted Date  ICMJE June 24, 2014
Last Update Posted Date March 26, 2019
Study Start Date  ICMJE April 2014
Actual Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 17, 2016)
Adverse Events as a Measure of Safety [ Time Frame: up to 2 years post last MSCs infusion ]
Original Primary Outcome Measures  ICMJE
 (submitted: June 23, 2014)
Adverse Events as a Measure of Safety [ Time Frame: up to 3 and a half years post last MSCs infusion ]
Change History Complete list of historical versions of study NCT02172885 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 17, 2016)
  • bone mineral density [ Time Frame: up to 2 years post last MSCs infusion ]
  • fracture rate [ Time Frame: up to 2 years post last MSCs infusion ]
  • growth velocity [ Time Frame: up to 2 years post last MSCs infusion ]
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: June 23, 2014)
  • bone mineral density [ Time Frame: up to 3 and a half years post last MSCs infusion ]
  • fracture rate [ Time Frame: up to 3 and a half years post last MSCs infusion ]
  • growth velocity [ Time Frame: up to 3 and a half years post last MSCs infusion ]
  • change from baseline in degree of functionality [ Time Frame: up to 3 and a half years post last MSCs infusion ]
    9 question survey using a Bleck functional scale
  • change from baseline in well-being [ Time Frame: up to 3 and a half years post last MSCs infusion ]
    A 20 item questionnaire designed to evaluate the well-being will be used
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Official Title  ICMJE Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
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).
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  ICMJE Interventional
Study Phase  ICMJE Phase 1
Study Design  ICMJE Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Osteogenesis Imperfecta
Intervention  ICMJE Biological: Mesenchymal Stem Cells
Mesenchymal Stem Cell Infusions
Study Arms  ICMJE Experimental: Mesenchymal stem cells
Five Mesenchymal Stem Cell infusions
Intervention: Biological: Mesenchymal Stem Cells
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: March 22, 2019)
2
Original Estimated Enrollment  ICMJE
 (submitted: June 23, 2014)
5
Actual Study Completion Date  ICMJE December 2018
Actual Primary Completion Date December 2018   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 6 Months to 12 Years   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Spain
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02172885
Other Study ID Numbers  ICMJE EudraCT Number:2012-002553-38
Health Department of Spain ( Other Grant/Funding Number: EC10-219 )
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Clara I. Rodríguez, Hospital de Cruces
Study Sponsor  ICMJE Hospital de Cruces
Collaborators  ICMJE
  • Hospital Universitario Getafe
  • Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Investigators  ICMJE
Principal Investigator: Clara I. Rodríguez, Ph. D. BioCruces Health Research Institute/Cruces University Hospital
PRS Account Hospital de Cruces
Verification Date March 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP