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Completed

NCT Number: NCT02172885

Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

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).

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Key information

Conditions

Osteogenesis Imperfecta Bone Diseases Bone Diseases, Developmental Collagen Diseases Congenital, Hereditary, and Neonatal Diseases and Abnormalities Connective Tissue Diseases Genetic Diseases, Inborn Musculoskeletal Diseases Osteochondrodysplasias Skin and Connective Tissue Diseases

Age range

6 month–12 year

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 1

Primary location

Hospital Universitario Cruces, Barakaldo, Bizkaia, Spain

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About this study

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.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Mesenchymal stem cells

Biological

Mesenchymal Stem Cell Infusions

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

    Time frame: up to 2 years post last MSCs infusion

    9 question survey using a Bleck functional scale

  5. 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

Sponsors and collaborators

Lead sponsor

Hospital de Cruces

Other

Collaborators

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

Registry information

Acronym: TERCELOI

Important dates

Study start
2014
Primary completion
2018
Study completion
2018
First posted
Jun 24, 2014
Registry last updated
Oct 3, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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