Osteogenesis ImperfectaPhase 1TERCELOINCT02172885
Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
A Phase 1 study testing Mesenchymal Stem Cells in people with Osteogenesis Imperfecta, run by Hospital de Cruces at 2 sites in Spain.
Trial 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 more about this study
- Who is it for?
- Ages 6 months to 12 years
- Conditions the study is for
- Osteogenesis Imperfecta
- What is being tested?
- Mesenchymal Stem Cells
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 2 participants
- Where
- 2 sitesSpain
- Study dates
- Apr 2014to Dec 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 months to 12 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
Spain2 sites
Hospital Universitario Cruces
Barakaldo, Bizkaia, 48903
Hospital Universitario Getafe
Getafe, Madrid
About this study
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.
- Conditions
- Osteogenesis Imperfecta
- Being tested
- Mesenchymal Stem Cells
- Keywords
- Mesenchymal stem cell based therapy, cell infusion
- Medical subject headings
- Osteogenesis Imperfecta
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Mesenchymal Stem CellsBiological
Mesenchymal Stem Cell Infusions
| Study group | Role | What they receive |
|---|---|---|
| Mesenchymal stem cells | Experimental | Five Mesenchymal Stem Cell infusions Receives: Biological: Mesenchymal Stem Cells |
What is being measured
The primary outcome is the main question the study is designed to answer.
Adverse Events as a Measure of Safety
Measured: up to 2 years post last MSCs infusion
Secondary outcomes (5)
bone mineral density
Measured: up to 2 years post last MSCs infusion
fracture rate
Measured: up to 2 years post last MSCs infusion
growth velocity
Measured: up to 2 years post last MSCs infusion
change from baseline in degree of functionality
9 question survey using a Bleck functional scale
Measured: up to 2 years post last MSCs infusion
change from baseline in well-being
A 20 item questionnaire designed to evaluate the well-being will be used
Measured: up to 2 years post last MSCs infusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jun 2014 (estimated)
- Study startApr 2014
- Primary completionDec 2018
- Study completionDec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Osteogenesis ImperfectaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 2 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hospital de Cruces
- Sponsor type
- University, hospital or other
- Collaborators
- Hospital Universitario Getafe, Hospital Infantil Universitario Niño Jesús, Madrid, Spain
- Responsible party
- Principal investigator, Clara I. Rodríguez, Hospital de Cruces
- Investigators
- Clara I. Rodríguez, Ph. D., Principal investigator, BioCruces Health Research Institute/Cruces University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02172885
- Sponsor's protocol ID
- EudraCT Number:2012-002553-38
- Other IDs
- Health Department of Spain
- Acronym
- TERCELOI
- Status last verified
- Sep 2023
- First submitted
- 12 Jun 2014
- Last updated
- 3 Oct 2023
- Results posted
- No
- Data monitoring committee
- Yes
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 33463067Infante 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 at a glance
From the registry record, last updated by the sponsor on 3 Oct 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta
- ClinicalTrials.gov ID
- NCT02172885
- Acronym / sponsor ID
- TERCELOI / EudraCT Number:2012-002553-38
- Conditions
- Osteogenesis Imperfecta
- Keywords
- Mesenchymal stem cell based therapy, cell infusion
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 2 participants (actual)
- Lead sponsor
- Hospital de CrucesUniversity, hospital or other
- Collaborators
- Hospital Universitario Getafe, Hospital Infantil Universitario Niño Jesús, Madrid, Spain
- Study start
- Apr 2014
- Primary completion
- Dec 2018 (actual)
- Study completion
- Dec 2018 (actual)
- First posted
- 24 Jun 2014 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Mesenchymal stem cells | Experimental | Biological: Mesenchymal Stem CellsFive Mesenchymal Stem Cell infusions |
Who may qualify
- Ages
- 6 months to 12 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Mesenchymal Stem CellsMesenchymal Stem Cell Infusions | Biological | Mesenchymal stem cells |
Primary outcome
Adverse Events as a Measure of Safety
Time frame: up to 2 years post last MSCs infusion
5 secondary outcomes
bone mineral density
Time frame: up to 2 years post last MSCs infusion
fracture rate
Time frame: up to 2 years post last MSCs infusion
growth velocity
Time frame: up to 2 years post last MSCs infusion
change from baseline in degree of functionality
Time frame: up to 2 years post last MSCs infusion
change from baseline in well-being
Time frame: up to 2 years post last MSCs infusion
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Oct 2023.
- Background: PubMed 33463067Infante 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.
- Results publication: PubMed 35223854Infante 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.
- Reiterative infusions of MSCs improve pediatric osteogenesis imperfecta eliciting a pro-osteogenic paracrine response: TERCELOI clinical trialLink provided by the sponsor.
Related trials for Osteogenesis Imperfecta
RecruitingPhase 2
Osteogenesis Imperfecta Trial of AGA2115 for ADUlts With COL1A1 and/or COL1A2 GeNetic Variations (IDUN)
Osteogenesis Imperfecta (OI)
Active, not recruitingObservational study
Cardiopulmonary Outcomes in Osteogenesis Imperfecta: BBD7708
Osteogenesis Imperfecta
Enrolling by invitationObservational study
GRACE II (General Retrospective Analysis of Commercial Experience With AGN1 LOEP)
Osteoporosis
Active, not recruitingPhase 3
Setrusumab vs Bisphosphonates in Pediatric Subjects With Osteogenesis Imperfecta
Osteogenesis Imperfecta
Active, not recruitingPhase 2/3
Setrusumab vs Placebo for Osteogenesis Imperfecta
Osteogenesis Imperfecta
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clara I. Rodríguez, Ph. D. principal investigator
BioCruces Health Research Institute/Cruces University Hospital
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing Mesenchymal Stem Cells in people with Osteogenesis Imperfecta, run by Hospital de Cruces at 2 sites in Spain.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Oct 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Spain. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Adverse Events as a Measure of Safety (time frame: up to 2 years post last MSCs infusion). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02172885, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Oct 2023.