NCT02172885

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).

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
2

participants targeted

Target at below P25 for phase_1

Timeline
Completed

Started Apr 2014

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

April 1, 2014

Completed
2 months until next milestone

First Submitted

Initial submission to the registry

June 12, 2014

Completed
12 days until next milestone

First Posted

Study publicly available on registry

June 24, 2014

Completed
4.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2018

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2018

Completed
Last Updated

October 3, 2023

Status Verified

September 1, 2023

Enrollment Period

4.7 years

First QC Date

June 12, 2014

Last Update Submit

September 29, 2023

Conditions

Osteogenesis Imperfecta

Keywords

Mesenchymal stem cell based therapycell infusion

Outcome Measures

Primary Outcomes (1)

  • Adverse Events as a Measure of Safety

    up to 2 years post last MSCs infusion

Secondary Outcomes (5)

  • bone mineral density

    up to 2 years post last MSCs infusion

  • fracture rate

    up to 2 years post last MSCs infusion

  • growth velocity

    up to 2 years post last MSCs infusion

  • change from baseline in degree of functionality

    up to 2 years post last MSCs infusion

  • change from baseline in well-being

    up to 2 years post last MSCs infusion

Study Arms (1)

Mesenchymal stem cells

EXPERIMENTAL

Five Mesenchymal Stem Cell infusions

Biological: Mesenchymal Stem Cells

Interventions

Mesenchymal Stem CellsBIOLOGICAL

Mesenchymal Stem Cell Infusions

Mesenchymal stem cells

Eligibility Criteria

Age6 Months - 12 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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.
  • Patients whose parents or the legal guardians are willing to sign the consent forms to participate in this clinical trial.

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Hospital Universitario Cruces

Barakaldo, Bizkaia, 48903, Spain

Location

Hospital Universitario Getafe

Getafe, Madrid, Spain

Location

Related Publications (2)

  • 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.

    PMID: 33463067BACKGROUND

  • 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.

    PMID: 35223854RESULT

Related Links

MeSH Terms

Conditions

Osteogenesis Imperfecta

Condition Hierarchy (Ancestors)

OsteochondrodysplasiasBone Diseases, DevelopmentalBone DiseasesMusculoskeletal DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesCollagen DiseasesConnective Tissue DiseasesSkin and Connective Tissue Diseases

Study Officials

  • Clara I. Rodríguez, Ph. D.

    BioCruces Health Research Institute/Cruces University Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
PI Stem Cell Laboratory

Study Record Dates

First Submitted

June 12, 2014

First Posted

June 24, 2014

Study Start

April 1, 2014

Primary Completion

December 1, 2018

Study Completion

December 1, 2018

Last Updated

October 3, 2023

Record last verified: 2023-09

Locations

ES(2)