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

Primary Purpose

Osteogenesis Imperfecta

Status
Completed
Phase
Phase 1
Locations
Spain
Study Type
Interventional
Intervention
Mesenchymal Stem Cells
Sponsored by
Hospital de Cruces
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Osteogenesis Imperfecta focused on measuring Mesenchymal stem cell based therapy, cell infusion

Eligibility Criteria

6 Months - 12 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Hospital Universitario Cruces
  • Hospital Universitario Getafe

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Mesenchymal stem cells

Arm Description

Five Mesenchymal Stem Cell infusions

Outcomes

Primary Outcome Measures

Adverse Events as a Measure of Safety

Secondary Outcome Measures

bone mineral density
fracture rate
growth velocity
change from baseline in degree of functionality
9 question survey using a Bleck functional scale
change from baseline in well-being
A 20 item questionnaire designed to evaluate the well-being will be used

Full Information

First Posted
June 12, 2014
Last Updated
September 29, 2023
Sponsor
Hospital de Cruces
Collaborators
Hospital Universitario Getafe, Hospital Infantil Universitario Niño Jesús, Madrid, Spain
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1. Study Identification

Unique Protocol Identification Number
NCT02172885
Brief Title
Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Acronym
TERCELOI
Official Title
Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
April 2014 (undefined)
Primary Completion Date
December 2018 (Actual)
Study Completion Date
December 2018 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hospital de Cruces
Collaborators
Hospital Universitario Getafe, Hospital Infantil Universitario Niño Jesús, Madrid, Spain

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Osteogenesis Imperfecta
Keywords
Mesenchymal stem cell based therapy, cell infusion

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Mesenchymal stem cells
Arm Type
Experimental
Arm Description
Five Mesenchymal Stem Cell infusions
Intervention Type
Biological
Intervention Name(s)
Mesenchymal Stem Cells
Intervention Description
Mesenchymal Stem Cell Infusions
Primary Outcome Measure Information:
Title
Adverse Events as a Measure of Safety
Time Frame
up to 2 years post last MSCs infusion
Secondary Outcome Measure Information:
Title
bone mineral density
Time Frame
up to 2 years post last MSCs infusion
Title
fracture rate
Time Frame
up to 2 years post last MSCs infusion
Title
growth velocity
Time Frame
up to 2 years post last MSCs infusion
Title
change from baseline in degree of functionality
Description
9 question survey using a Bleck functional scale
Time Frame
up to 2 years post last MSCs infusion
Title
change from baseline in well-being
Description
A 20 item questionnaire designed to evaluate the well-being will be used
Time Frame
up to 2 years post last MSCs infusion

10. Eligibility

Sex
All
Minimum Age & Unit of Time
6 Months
Maximum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clara I. Rodríguez, Ph. D.
Organizational Affiliation
BioCruces Health Research Institute/Cruces University Hospital
Official's Role
Principal Investigator
Facility Information:
Facility Name
Hospital Universitario Cruces
City
Barakaldo
State/Province
Bizkaia
ZIP/Postal Code
48903
Country
Spain
Facility Name
Hospital Universitario Getafe
City
Getafe
State/Province
Madrid
Country
Spain

12. IPD Sharing Statement

Citations:
PubMed Identifier
33463067
Citation
Infante A, Gener B, Vazquez M, Olivares N, Arrieta A, Grau G, Llano I, Madero L, Bueno AM, Sagastizabal B, Gerovska D, Arauzo-Bravo MJ, Astigarraga I, Rodriguez CI. Reiterative infusions of MSCs improve pediatric osteogenesis imperfecta eliciting a pro-osteogenic paracrine response: TERCELOI clinical trial. Clin Transl Med. 2021 Jan;11(1):e265. doi: 10.1002/ctm2.265.
Results Reference
background
PubMed Identifier
35223854
Citation
Infante A, Cabodevilla L, Gener B, Rodriguez CI. Circulating TGF-beta Pathway in Osteogenesis Imperfecta Pediatric Patients Subjected to MSCs-Based Cell Therapy. Front Cell Dev Biol. 2022 Feb 9;10:830928. doi: 10.3389/fcell.2022.830928. eCollection 2022.
Results Reference
result
Links:
URL
http://www.ncbi.nlm.nih.gov/pmc/articles/PMC7805402/
Description
Reiterative infusions of MSCs improve pediatric osteogenesis imperfecta eliciting a pro-osteogenic paracrine response: TERCELOI clinical trial

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Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

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