Mesenchymal Stem Cell Based Therapy for the Treatment of... | NCT02172885

Studies
Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

NCT02172885

Sponsor

Hospital de Cruces

Status

Completed

Last Update Posted

Oct 3, 2023Actual

Enrollment

2Actual

Phase

Phase 1

Conditions

Osteogenesis Imperfecta

Interventions

Mesenchymal Stem Cells

Countries

Spain

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

Results Section

No data available

No data is available for this block.

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Document Has No Statistical Analysis Plan (SAP)

Derived Section

Miscellaneous Info Module

Version Holder

ID	Type	Description	Link
Health Department of Spain	Other Grant/Funding Number	EC10-219

Status Module

Record Verification Date

Sep 2023

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Apr 2014

Primary Completion Date

Dec 2018Actual

Completion Date

Dec 2018Actual

First Submitted Date

Jun 12, 2014

First Submission Date that Met QC Criteria

Jun 23, 2014

First Posted Date

Jun 24, 2014Estimated

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Sep 29, 2023

Last Update Posted Date

Oct 3, 2023Actual

Name	Class
Hospital Universitario Getafe	OTHER
Hospital Infantil Universitario Niño Jesús, Madrid, Spain	OTHER

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Not provided

Is FDA Regulated Device

Not provided

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

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.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 1

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

2Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Mesenchymal stem cells	Experimental	Five Mesenchymal Stem Cell infusions	Biological: Mesenchymal Stem Cells

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Mesenchymal Stem Cells	Biological	Mesenchymal Stem Cell Infusions	Mesenchymal stem cells

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Adverse Events as a Measure of Safety		up to 2 years post last MSCs infusion

Secondary Outcomes

Measure	Description	Time Frame
bone mineral density		up to 2 years post last MSCs infusion
fracture rate		up to 2 years post last MSCs infusion
growth velocity

Other Outcomes

Not provided

Eligibility Module

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

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

6 Months

Maximum Age

12 Years

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Clara I. Rodríguez, Ph. D.	BioCruces Health Research Institute/Cruces University Hospital	Principal Investigator

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Hospital Universitario Cruces		Barakaldo	Bizkaia	48903	Spain
Hospital Universitario Getafe

References Module

Citations

PubMed Identifier	Type	Citation	Retractions
33463067	Background	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.
35223854

ID	Term
D010009	Osteochondrodysplasias
D001848	Bone Diseases, Developmental
D001847	Bone Diseases
D009140	Musculoskeletal Diseases