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CompletedNCT02172885TERCELOIUpdated Oct 3, 2023

Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

A Phase 1 interventional study of Mesenchymal Stem Cells in Osteogenesis Imperfecta, sponsored by Hospital de Cruces. Completed at 2 sites in Spain. Open to participants aged 6 Months to 12 Years. Per ClinicalTrials.gov, last updated 2023-10-03.

Sponsored by Hospital de Cruces · Phase 1, Interventional, and Treatment

Phase
Phase 1
Study type
Interventional
Enrollment
2
Allocation
Not applicable
Ages
6 Months to 12 Years
Sex
All
01

Study 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).

Read the 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.

02

Conditions studied

  • Osteogenesis Imperfecta

Keywords

  • Mesenchymal stem cell based therapy
  • cell infusion
03

In context

Osteogenesis Imperfecta

84 studies on the registry are indexed under Osteogenesis Imperfecta; 20 are open to participants now.

This study's enrollment of 2 is below the median of 28 across 60 interventional studies indexed under Osteogenesis Imperfecta.

Browse Osteogenesis Imperfecta studies →

Lead sponsor

Hospital de Cruces is the lead sponsor of 10 studies on the registry; none are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
6 Months to 12 Years
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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

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 .
  • 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
Terms in these criteria, in plain language(1)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

05

Study design

Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
2 participants (actual)

Study arms

  • Experimental
    Mesenchymal stem cells

    Five Mesenchymal Stem Cell infusions

    Biological: Mesenchymal Stem Cells

Interventions

  • BiologicalMesenchymal Stem Cells

    Mesenchymal Stem Cell Infusions

06

What researchers measure

Primary outcomes

  1. Adverse Events as a Measure of Safety

    Time frame: up to 2 years post last MSCs infusion

Secondary outcomes

  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

    9 question survey using a Bleck functional scale

    Time frame: up to 2 years post last MSCs infusion

  5. change from baseline in well-being

    A 20 item questionnaire designed to evaluate the well-being will be used

    Time frame: up to 2 years post last MSCs infusion

07

Study locations

2 sites
  • Hospital Universitario Cruces
    Barakaldo, Bizkaia 48903, Spain
  • Hospital Universitario Getafe
    Getafe, Madrid, Spain
08

References and documents

Publications

  • 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. PubMed 33463067 ↗
  • 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. PubMed 35223854 ↗

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Oct 3, 2023, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT02172885
Lead sponsor
Hospital de Cruces
Collaborators
Hospital Universitario Getafe, Hospital Infantil Universitario Niño Jesús, Madrid, Spain
Responsible party
Clara I. Rodríguez (PI Stem Cell Laboratory, Hospital de Cruces) — Principal investigator
First posted
Jun 24, 2014
Start date
Apr 2014
Primary completion
Dec 2018
Completion
Dec 2018
Last update
Oct 3, 2023

Study contacts

Clara I. Rodríguez, Ph. D.
principal investigator · BioCruces Health Research Institute/Cruces University Hospital

Oversight

Data monitoring committee
Yes
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Sep 2023. You cannot join it, but the record below documents what was studied.

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Discussion

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