Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

COMPLETED

Clinical Phase

PHASE1

Total Enrollment

2 participants

Study Classification

INTERVENTIONAL

Study Start Date

2014-04-30

Study Completion Date

2018-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

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).

Related Clinical Trials

Explore similar clinical trials based on study characteristics and research focus.

Safety and Efficacy Study of Umbilical Cord Blood-Drived Mesenchymal Stem Cells to Promote Engraftment of Unrelated Hematopoietic Stem Cell Transplantation

NCT00823316

Acute Leukemia

COMPLETED PHASE1/PHASE2

Mesenchymal Stem Cell Therapy for Lung Rejection

NCT02181712

Lung Transplant Reject Bronchiolitis Obliterans

COMPLETED PHASE1

Stem Cell Transplantation to Treat Systemic Mastocytosis

NCT00006413

Mastocytosis

COMPLETED PHASE2

MSCs With or Without Peripheral Blood Stem Cell for Treatment of Poor Graft Function and Delayed Platelet Engraftment

NCT02240992

Stem Cell Transplantation, Hematopoietic Poor Graft Function Delayed Platelet Engraftment +1 more

UNKNOWN PHASE2/PHASE3

Safety and Tolerance of Immunomodulating Therapy With Donor-specific MSC in Pediatric Living-Donor Liver Transplantation

NCT02957552

Pediatric Liver Transplantation

UNKNOWN PHASE1

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

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.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Osteogenesis Imperfecta

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Mesenchymal stem cells

Five Mesenchymal Stem Cell infusions

Group Type EXPERIMENTAL

Mesenchymal Stem Cells

Intervention Type BIOLOGICAL

Mesenchymal Stem Cell Infusions

Interventions

Learn about the drugs, procedures, or behavioral strategies being tested and how they are applied within this trial.

Mesenchymal Stem Cells

Mesenchymal Stem Cell Infusions

Intervention Type BIOLOGICAL

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

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.
* 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

Minimum Eligible Age

6 Months

Maximum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No