NeuroEPO – Type 2 Spinocerebellar Ataxia Phase I-II | Registro Público Cubano de Ensayos Clínicos

NeuroEPO – Type 2 Spinocerebellar Ataxia Phase I-II

General information

Section to complete general information about the trial: scientific and public title, protocol identifiers, sponsors and Source(s) of Monetary or Material Support.

Acronym of Public Title:

NeuroEPO-Ataxia

Scientific title:

“Evaluation of the safety and effect of treatment with intranasal NeuroEPO in patients with type 2 Spinocerebellar Ataxia”

Acronym of Scientific Title:

NeuroEPO-Ataxia

Secondary indentifying numbers:

Not applicable

Issuing authority of the secondary identifying numbers:

Not applicable

Source(s) of monetary or material support:

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

Authorization for beginning

Section to complete information about the regulatory approval of clinical trial: regulatory agency name, approval date and reference number in the agency.

Regulatory instance to authorize the initiation of the study:

Center for State Control of Drugs, Medical Devices and Equipment (CECMED)

Reference number:

In process

Principal investigator

Section to complete information about Email address, telephone number and postal address of the Principal Investigator.

First name:

Luis

Last name:

Velazquez Perez

Medical Specialty :

Second degree specialist in Neurophysiology

Affiliation:

Center for Research and Rehabilitation of Hereditary Ataxias (CIRAH)

Postal address:

Carretera Central Km 5 1/2, Via Habana

City:

Holguin

Country:

Cuba

Zip Code:

80100

Telephone:

+53-024424090

Email address:

lvp@cristal.hlg.sld.cu

Clinical sites to participate

Section to complete the data related to the clinical sites involved in the trial: site and responsible investigator for every site.

Countries of recruitment:

Cuba

Clinical sites:

Not applicable

Research ethics committees:

Center for Research and Rehabilitation of Hereditary Ataxias (CIRAH), February 5th, 2014

Recruitment status

Section to complete information about the recruitment status and the date of first enrolment subject

Recruitment status:

Pending

Date of first enrollment:

03/11/2014

Health condition and Intervention

Section to complete information about the primary medical condition(s) or problem(s) studied and, a characteristics of the intervention(s).

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.

Outcomes and Timepoint

Section to complete information about primary and secondary outcomes including. It includes the metric or method of measurement used and, the time point for every outcome.

Primary outcome(s):

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

Key secondary outcomes:

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.

Selection criterias

Section to complete information about the inclusion and exclusion criteria for participant selection, including age and gender.

Gender:

Male/Female

Minimum age:

18 years

Maximum age:

80 years

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.

Type of population:

Adults

Type of participant:

Patients

Study design

Section to complete information about the characteristics of the study design.

Type study:

Interventional

Purpose:

Treatment

Allocation:

Randomized controlled trial

Masking:

Double Blind

Control group:

Placebo

Study design:

Parallel

Phase:

1-2

Target sample size:

34

Contact for public queries

Section to complete information about Email address, telephone number and postal address of the contact who will respond to general queries, including information about current recruitment status

First Name:

Orestes

Last Name:

Santos Morales, MD

Specialty:

Second degree specialist in Pharmacology

Affiliation:

Center of Molecular Immunology (CIM), CIMAB S.A.

Postal Address:

206 Street, No.1926 between 19 and 21, Atabey, Playa

City:

Havana

Country:

Cuba

Zip Code:

11600

Telephone:

+53 (7) 2715057 ext 131.

Email :

orestesm@cim.sld.cu

Contact for scientific queries

Section to complete information about Email address, telephone number and postal address of the contact who will respond to scientific queries.

First Name:

Orestes

Last Name:

Santos Morales, MD

Specialty:

Second degree specialist in Pharmacology

Affiliation:

Center of Molecular Immunology (CIM), CIMAB S.A.

Postal Address:

206 Street, No.1926 between 19 and 21, Atabey, Playa

City:

Havana

Country:

Cuba

Zip Code:

11600

Telephone:

+53 (7) 2715057 ext 131

Email :

orestesm@cim.sld.cu

Registration and Update

Section to complete information about the name of Primary Registry, date of registration and the unique ID number assigned by the registry (RPCEC).

Primary registry:

RPCEC

Unique ID number:

RPCEC00000187

Date of Registration in Primary Registry:

02/11/2014

Record Verification Date:

2014/11/02

Next update date:

2015/11/02

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NeuroEPO – Type 2 Spinocerebellar Ataxia Phase I-II

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