Friedreich's ataxia. Early-onset cerebellar ataxia
Conditions
Interventions
Intervention group: Bone marrow samples (allogeneic) will be collected from five children with Friedrich's Ataxia, and the cells will be processed and cultured in the transplant environment. Subsequen
Sponsors
Mashhad University of Medical Sciences
Eligibility
Sex/Gender
All
Age
5 Years to 20 Years
Inclusion criteria
Inclusion criteria: Patients with Friedrich's Ataxia Having a positive genetic test with GAA repeat sequences
Exclusion criteria
Exclusion criteria: Individuals with seizures or epilepsy IQ below 80 Sensitivity to stem cell injection
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Improvement in neurological function. Timepoint: 1, 2, 3, 4, 5, and 6 months after the first injection. Method of measurement: Regular neurological examinations are conducted (improvement in walking condition, increased pupillary response to light, partial improvement of eye nystagmus, improvement in speech, and improvement in hearing). | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety of stem cell transplantation. Timepoint: 1, 2, 3, 4, 5, and 6 months after injection. Method of measurement: Assessment of response to stem cell transplantation through immune tolerance and absence of hypersensitivity reactions.;Tolerance of stem cell transplantation. Timepoint: 1, 2, 3, 4, 5, and 6 months after injection. Method of measurement: Assessment of response to stem cell transplantation through immune tolerance and absence of hypersensitivity reactions.;Improvement in cardiac function. Timepoint: 1, 2, 3, 4, 5, and 6 months after injection. Method of measurement: improvement in cardiac function using echocardiography.;Enhancement of quality of life. Timepoint: 1, 2, 3, 4, 5, and 6 months after injection. Method of measurement: Urinary and fecal continence, and self-care ability. | — |
Countries
Iran (Islamic Republic of)
Contacts
Public ContactHamid Khalesi
Mashhad University of Medical Sciences
Outcome results
None listed