NeuroEPO – Type 2 Spinocerebellar Ataxia Phase I-II

• Register: RPCEC
• Last refreshed on: 1 April 2024
• Main ID: RPCEC00000187
• Date of registration: 02/11/2014
• Prospective Registration: Yes
• Primary sponsor: Center of Molecular Immunology (CIM), CIMAB
• Public title: NeuroEPO – Type 2 Spinocerebellar Ataxia Phase I-II
• Scientific title: “Evaluation of the safety and effect of treatment with intranasal NeuroEPO in patients with type 2 Spinocerebellar Ataxia” - NeuroEPO-Ataxia
• Date of first enrolment: 03/11/2014
• Target sample size: 34
• Recruitment status: Pending
• URL: https://rpcec.sld.cu/en/trials/RPCEC00000187-En
• Study type: Interventional
• Study design: Allocation: Randomized controlled trial. Masking: Double Blind. Control group: Placebo. Assignment: Parallel. Purpose: Treatment
• Phase: 1-2

Countries of recruitment

Cuba

Contacts

• Name: Orestes Santos Morales, MD
• Affiliation: Center of Molecular Immunology (CIM), CIMAB S.A.

• Name: Orestes Santos Morales, MD
• Affiliation: Center of Molecular Immunology (CIM), CIMAB S.A.

Key inclusion & exclusion 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: 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.


Age minimum: 18 years
Age maximum: 80 years
Gender: Male/Female

Health Condition(s) or Problem(s) studied

Type 2 Spinocerebellar Ataxia

Intervention(s)

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 patients will be included to receive a dose of 1 mg/ml of Placebo by intranasal route, three times per week (Monday, Wednesday and Friday), during 6 months.

Primary Outcome(s)

Spinocerebellar Ataxia Functional Index (SCAFI). Measuring time: Before and after NeuroEPO treatment (6 months).

Secondary Outcome(s)

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 beginning and 6 months.
- Cognitive variables Phonological test FAS (average/min of right words that begin with FAS), Stroop test (time of completing and number of mistakes for the conflict task), Evoked verbal memory test (number of remembered words, number of necessary trials to remember all the words). Measuring time: At the beginning and 6 months.

Secondary ID(s)

Not applicable

Source(s) of Monetary Support

Center of Molecular Immunology (CIM) Cuban Ministry of Public Health (MINSAP)

Secondary Sponsor(s)

Drug Research and Development Center (CIDEM)