Type 2 Spinocerebellar Ataxia
Conditions
Interventions
Group I (Study): 17 patients will be included to receive a dose of 1 mg/ml of NeuroEPO by intranasal route, three times per week (Monday, Wednesday and Friday), during 6 months.
Group II (Control): 17
Sponsors
Center of Molecular Immunology (CIM), CIMAB
Drug Research and Development Center (CIDEM)
Eligibility
Sex/Gender
All
Age
18 Years to 80 Years
Inclusion criteria
Inclusion criteria: 1.Patients with clinical and molecular diagnosis of stage I-II Type 2 Spinocerebellar Ataxia. 2.Patients of both gender, with age between 18 and 80 years (both included). 3.Written informed consent given by the patient. 4.Vital signs within normal limits. Systolic blood pressure until 140 mm Hg Diastolic blood pressure until 90 mm Hg Heart rate 60-100 x minute Respiratory rate 16-20 x minute
Exclusion criteria
Exclusion criteria: 1.Antecedents of alcoholism and drug dependency. 2.Antecedents of any other degenerative or systemic neurological disease with repercussion on the nervous system. 3.Antecedents of psychiatric diseases. 4.Hematological diseases such as thrombocytosis, anticoagulation, antecedents of thrombotic events. 5.Severe acute diseases at entry. 6.Decompensate chronic diseases associated to SCA2. 7.Chronic inflammatory diseases, Epilepsy, diabetes mellitus, cardiac insufficiency. 8.Patient with folic iron and vitamin B12 supplementation. 9.Pregnancy or nursing. 10.Patients with dementia symptoms. 11.Had been operated in the previous six months, 12.Patients with hypersensibility to human recombinant erythropoietin or some ingredient of the formulation. 13.Rhinitis 14.Patients with deviated nasal septum. 15.Parallel participation in another clinical trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Spinocerebellar Ataxia Functional Index (SCAFI). Measuring time: Before and after NeuroEPO treatment (6 months). | — |
Secondary
| Measure | Time frame |
|---|---|
| Safety variables: - Presence of adverse events (AE) (distribution frequency for the appearance of adverse events (Yes, No), type of event (name of the AE), duration (time from appearance to end of the event), intensity of AE (mild, moderate, severe), seriousness of AE (serious, no serious), relation of causality (very probable, probable, possible, improbable, no related, no evaluated), result of AE (recuperate, improvement, persist, sequels), attitude concerning the studied treatment (without changes, dose modification, temporal or definitive treatment discontinuation), treatment to control AE. Measuring time: At each NeuroEPO administration and during the whole study. - Vital signs (body temperature (Celsius degrees), heart rate (beats per minute), blood pressure (mm Hg) and respiratory rate (breaths per minute)). Measuring time: Before and after each NeuroEPO administration. - Laboratory tests (hemoglobin, hematocrit, white blood cells counts, reticulocytes count, platelet count, coagulation parameters, glucemy, transaminases, urea, creatinine). Measuring time: Before treatment and monthly during 6 months. - Tolerance at the administration site (nasal mucous) Signs of local toxicity as redden, edema (Yes, No), intensity (mild, moderate, severe), reversible (Yes, No). Measuring time: Before treatment, 3 months, and 6 months. - Cardiovascular monitoring (electrocardiogram) Measuring time: Before treatment, 3 months, and 6 months. Therapeutic response: - Electronystagmography variable (change in the error rate of the anti-saccadic eye movements). Measuring time: At the beginning and 6 months. - Markers of oxidative stress or damage (Relation CAT/SOD, GSH, GST, TBARS). Measuring time: At the beginning and 6 months. - EPO concentration in cerebrospinal fluid. (By ELISA). Measuring time: Before first NeuroEPO dose and after last dose at month 6, both cases one hour after intranasal administration. - Motor coordination (SARA Scale). Measuring time: At the begin | — |
Countries
Cuba
Contacts
Public ContactOrestes Santos Morales, MD
Center of Molecular Immunology (CIM), CIMAB S.A.
Outcome results
None listed