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

September 29, 2023 updated by: Clara I. Rodríguez, Hospital de Cruces
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).

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Enrollment (Actual)

2

Phase

  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Spain
    • Bizkaia
      • Barakaldo, Bizkaia, Spain, 48903
        • Hospital Universitario Cruces
    • Madrid
      • Getafe, Madrid, Spain
        • Hospital Universitario Getafe

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

6 months to 12 years (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Mesenchymal stem cells
Five Mesenchymal Stem Cell infusions
Biological: Mesenchymal Stem Cells
Mesenchymal Stem Cell Infusions

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
Adverse Events as a Measure of Safety
Time Frame: up to 2 years post last MSCs infusion
up to 2 years post last MSCs infusion

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
bone mineral density
Time Frame: up to 2 years post last MSCs infusion
up to 2 years post last MSCs infusion
fracture rate
Time Frame: up to 2 years post last MSCs infusion
up to 2 years post last MSCs infusion
growth velocity
Time Frame: up to 2 years post last MSCs infusion
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
up to 2 years post last MSCs infusion
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
up to 2 years post last MSCs infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Hospital de Cruces

Collaborators

Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Hospital Universitario Getafe

Investigators

  • Principal Investigator: Clara I. Rodríguez, Ph. D., BioCruces Health Research Institute/Cruces University Hospital

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

April 1, 2014

Primary Completion (Actual)

December 1, 2018

Study Completion (Actual)

December 1, 2018

Study Registration Dates

First Submitted

June 12, 2014

First Submitted That Met QC Criteria

June 23, 2014

First Posted (Estimated)

June 24, 2014

Study Record Updates

Last Update Posted (Actual)

October 3, 2023

Last Update Submitted That Met QC Criteria

September 29, 2023

Last Verified

September 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • EudraCT Number:2012-002553-38
  • Health Department of Spain (Other Grant/Funding Number: EC10-219)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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