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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 9 September 2013
Main ID:  EUCTR2012-002553-38-ES
Date of registration: 09/04/2013
Prospective Registration: Yes
Primary sponsor: Itziar Astigarraga Aguirre
Public title: Mesenchymal stem cell based therapy for the treatment of osteogenesis imperfecta
Scientific title: Mesenchymal stem cell based therapy for the treatment of osteogenesis imperfecta - TERCELOI
Date of first enrolment: 09/08/2013
Target sample size:
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2012-002553-38
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: yes Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Other: no  
Phase: 
Countries of recruitment
Spain
Contacts
Name: U. EPIDEMIOLOGIA CLINICA- APOYO INV   
Address:  PLAZA DE CRUCES S/N 48903 BARAKALDO (BIZKAIA) Spain
Telephone: 34946000002368
Email: ana.irasarrisebastian@osakidetza.net
Affiliation:  HOSPITAL UNIVERSITARIO CRUCES
Name: U. EPIDEMIOLOGIA CLINICA- APOYO INV   
Address:  PLAZA DE CRUCES S/N 48903 BARAKALDO (BIZKAIA) Spain
Telephone: 34946000002368
Email: ana.irasarrisebastian@osakidetza.net
Affiliation:  HOSPITAL UNIVERSITARIO CRUCES
Key inclusion & exclusion criteria
Inclusion criteria:
1.-Patient age: older than 6 months and younger than 12 years old.
2.-Patients with molecular confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III).
3.-Patiens with HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
4.-All patients that fulfil the inclusion criteria regardless of whether or not they are undergoing biphosphonate treatment
5.-Patients whose parents or the legal guardians are willing to sign the consent forms to participate in this clinical trial.
Are the trial subjects under 18? yes
Number of subjects for this age range: 3
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1.-Patient age: older than 12 years old
2.-Patients lacking confirmation of mutation in either COL1A1 or COL1A2 genes associated with severe deforming OI (type III).
3.-Other pathological subtypes of OI.
4.-Patiens lacking of HLA identical (that shared at least 5/6 antigens) siblings willing to donate bone marrow-MSCs.
5.-Immunodeficiencies and any other malignancies
6.-Participation in other clinical trial
7.-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.
8.-Patients whose parents or the legal guardians do not sign the consent forms


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Therapeutic area: Body processes [G] - Cell Physiological Phenomena [G04]
Osteogenesis imperfecta (OI) is a rare genetic disorder with increased bone fragility of varying severity. In the majority of patients the disease is caused by mutations in collagen type I. Severe OI is characterized by osteopenia, frequent fractures, progressive deformity, short stature, loss of mobility, chronic pain and can lead to premature death. At present a cure does not exist.
Intervention(s)

Product Name: celulas madre mesenquimales troncales adultas alogenicas de médula ósea no expandida
Product Code: REF CRUZADA PEI Nº 12-088
Pharmaceutical Form: Living tissue equivalent
INN or Proposed INN: células mesenquimales troncales adultas alogénicas de médula ósea no expandidas
Current Sponsor code: células mesenquimales troncales adultas alogénicas de médula ósea no expa (ref cruzada PEI Nº 12-088
Other descriptive name: células mesenquimales troncales adultas alogénicas de médula ósea no expandidas(ref cruzada PEI nº 12-088)
Concentration unit: Other
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)
Timepoint(s) of evaluation of this end point: The clinical and analytical evaluations as well as immune response evaluation will take place in the initial visit (before the cell infusion which will be visit 1) to establish baselines and at the following intervals:
.-visit 2, 7 days post-first infusion
.-visit 3, 28 days post-first infusion
.-visit 4, 4 months post-first infusion
.-visit 5, 5 months post-first infusion, second infusion
.-visit 6, 7 days post-second infusion
.-visit 7, 28 days post-second infusion
.-visit 8, 9 months post-first infusion
The proliferation of allogeneic lymphocytes in co-culture experiments will be evaluated at the initial visit, visit number 4 and visit number 8.
Secondary Objective: To demonstrate the efficacy of the MSCs procedure, to verify that treatment of OI patients with several HLA-identical MSC injections, is not only a safe procedure (primary objective), but will increase the benefits observed with only one HLA-identical MSC injection
Primary end point(s): The principal aim of this project is to assess the safety of non-mutated HLA-identical MSC transplantation for OI pediatric patients irrespective of treatment with pamidronate.
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 allogenic MSC or that they shared at least 5 out of 6 HLA antigens it could be accomplished without subjecting the patients to immunosupressor treatment. Adverse secondary effects due to immunosopressor treatment could be avoided using this strategy and patients may reap the benefits of two cellular infusions instead of one. The beneficial effects observed in patients subjected to cell therapy seem to last for approximately six months after the infusion.
In addition to clinical and analytical analysis of blood and urine tests, the immune responses of treated patients will be evaluated. Humoral and cell-mediated responses will be determined.
Main Objective: To demonstrate the safety of the stem cell transplantation process using characterized HLA-identical MSC in OI infants already subjected to pamidronate treatment, but who are not receiving immunosuppresion treatment
Secondary Outcome(s)
Secondary end point(s): The secondary end point is to demonstrate the efficacy of MSC transplantation for OI patients.
The evaluation of treatment efficacy will be based on physical and clinical evaluation, analytical and radiological parameters associated with bone metabolism, reduction in the frequencies of bone fracture, increase in growth velocity, positive impact in functionality and an improvement in well being.
Timepoint(s) of evaluation of this end point: The clinical and analytical evaluations will take place in the initial visit (before the cell infusion on visit 1) and at the following visits:
.-visit 2, 7 days post-first infusion
.-visit 3, 28 days post-first infusion
.-visit 4, 4 months post-first infusion
.-visit 5, 5 months post-first infusion, second infusion
.-visit 6, 7 days post-second infusion
.-visit 7, 28 days post-second infusion
.-visit 8, 9 months post-first infusion
The radiological tests only will be performed at the initial and last visits.
Secondary ID(s)
TERCELOI
Source(s) of Monetary Support
HOSPITAL UNIVERSITARIO CRUCES
MINISTERIO DE SANIDAD- Convocatoria EC Independientes 2010
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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