NeuroEPO-Ataxia-Adults-Phase III

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:

NESCA

Scientific title:

Phase III clinical trial, placebo-controlled, randomized and double-blind to assess the efficacy and safety of NeuroEPO nasal administration in adult patients with spinocerebellar ataxias.

Acronym of Scientific Title:

NESCA

Secondary indentifying numbers:

Not aplicable

Issuing authority of the secondary identifying numbers:

Not applicable

Source(s) of monetary or material support:

Center of Molecular Immunology (CIM), 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 :

2nd Degree Specialist in Neurophysiology

Affiliation:

Academy of Sciences of Cuba (ACC)

Postal address:

Cuba street 460 between Amargura and Teniente Rey, Old Havana

City:

Havana

Country:

Cuba

Zip Code:

10100

Telephone:

+53-78670599

Email address:

velazq63@gmail.com

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:

Holguin, Hospital Lucia Inniguez, Marisleydis Verdecia Ramirez, MD. 1st Degree Neurophysiology Specialist

Villa Clara, General Teaching Hospital “Arnaldo Milian Castro, Maria Ofelia Hernandez Oliver, MD. Specialist in Neurology

Havana, Institute of Neurology and Neurosurgery (INN), Yamile Garcia Nodarse, MD. Specialist in Neurology

Recruitment status

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

Recruitment status:

Pending

Date of first enrollment:

01/03/2021

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:

Spinocerebellar Ataxia

Health condition(s) code:

Spinocerebellar Ataxias

Cerebellar Ataxia

Ataxia

Spinocerebellar Degenerations

Cerebellar Diseases

Heredodegenerative Disorders, Nervous System

Neurodegenerative Diseases

Central Nervous System Diseases

Nervous System Diseases

Spinal Cord Diseases

Dyskinesias

Neurologic Manifestations

Genetic Diseases, Inborn

Brain Diseases

Health condition keyword:

Intervention(s):

Group I (Experimental): NeuroEPO 1mg (1ml) 3 times per week (alternate days) administered intranasally for 12 months. At first a volume of 0.25 ml will be administered in a nostril, putting pressure on the nostril on the opposite side. It will wait one minute and the same volume will be administered in the other nostril. After 5 minutes the above procedure will be repeated. The final volume administered shall be 1 mL (0.5 ml in each pit). The product will be administered drop by drop slowly. Group II (Control): Placebo (1ml) 3 times per week (alternate days) administered intranasally for 12 months. At first a volume of 0.25 ml will be administered in a nostril, putting pressure on the nostril on the opposite side. It will wait one minute and the same volume will be administered in the other nostril. After 5 minutes the above procedure will be repeated. The final volume administered shall be 1 mL (0.5 ml in each pit). The product will be administered drop by drop slowly.

Intervention code:

Erythropoietin

Neuroprotective Agents

Administration, Intranasal

Placebos

Intervention keyword:

NeuroEPO

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):

Total SARA scale score (total value of the score). Measurement time: in the initial evaluation (baseline) and in the evaluations of month 6 and final (month 12).

Key secondary outcomes:

1. Cognitive affective functions (score value obtained after application of the Affective Cognitive Cerebellar Syndrome scale (SCHMAHMANN syndrome). Measurement time: At baseline and, 6 and 12 months. 2. Volume and area of the cerebral cortex (obtained through MRI imaging studies). Measurement time: At baseline and, 6 and 12 months. 3. Volume and area of the cerebellum (obtained through MRI imaging studies). Measurement time: At baseline and, 6 and 12 months. 4. Volume and area of the brain stem (obtained through MRI imaging studies). Measurement time: At baseline and, 6 and 12 months. 5. Adverse Events-AE (Occurrence (yes/no); Type of AE (AE name); Severity of the AE (Severe, Not Serious); Intensity of the AE (Mild, Moderate, Severe); Duration of the AE (Difference of dates between the start and end of the event); Causation Ratio (Very Likely, Probable, Possible, Unlikely, Unrelated, Not evaluable); Attitude to treatment (unchanged, dose modification, temporary interruption, or definitive); Treatment of the AE (treatment received); AE result (recovered, improved, persists or sequels)). Measurement time: During a time patient study until 12 months. 6. Vital signs (Blood pressure (systolic/diastolic), heart rate (FC), respiratory rate (FR)). Measurement time: Before and after each intranasal application. 7. Cardiovascular function (result of the electrocardiogram). Measurement time: At baseline and, 6 and 12 months. 8. Laboratory tests (Hematology [values of hemoglobin, differential leukogram, hematocrit, platelets]; Blood chemistry [values of urea, creatinine, glycemia, glutamic-oxalacetic transaminase (GOT), glutamic-pyruvic transaminase (GPT)]; Coagulation tests [partial thromboplastin time (PTT), prothrombin time (PT)]). Measurement time: At baseline and, 6 and 12 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:

70 years

Inclusion criteria:

1. otal SARA scale score between 3 and 14 points. 2. Ages between 18 and 70 years (inclusive). 3. Expresses in writing your consent to participate in the study.

Exclusion criteria:

1. Suffering from decompensated chronic diseases (Cardiopathies, Diabetes Mellitus, Arterial Hypertension, Chronic Renal Failure, Bronchial Asthma, etc.). 2. Parkinson's manifestations and/or frontotemporal dementia. 3. History of any other degenerative or systemic neurological disease with an impact on the nervous system. 4. History of psychiatric illnesses. 5. Known bleeding disorders or active bleeding. 6. Patient who is being treated with anti-aguerants or anticoagulants. 7. Severe acute diseases at the time of inclusion. 8. Pregnant or lactating woman. 9. Patient with reproductive and sexually active capacity who does not agree to use at least one effective method of contraception prior to inclusion in the study and/or during participation in the clinical trial (in the case of the female sex: intrauterine devices, barrier methods, hormones or tubal ligation and in the case of the male sex: vasectomy, condom use). 10. Hypersensitivity to the active substance (recombinant human erythropoietin) or to any of the active substances that make up the product. 11. You are participating in another clinical trial. 12. History of alcoholism and/or pharmacodependence.

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:

3

Target sample size:

80

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

Specialty:

2nd degree specialist in Pharmacology

Affiliation:

Center of Molecular Immunology

Postal Address:

216 Street, corner to 15th Ave. Playa

City:

Havana

Country:

Cuba

Zip Code:

11600

Telephone:

+53-72717933

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

Specialty:

2nd degree specialist in Pharmacology

Affiliation:

Center of Molecular Immunology

Postal Address:

216 Street, corner to 15th Ave. Playa

City:

Havana

Country:

Cuba

Zip Code:

11600

Telephone:

+53-72717933

Email :

orestesm@cim.sld.cu

Research Ethics Committees

Section to complete the data related to the review ethics committees.

Name of Research Ethics Committees:

Lucia Iniguez Hospital

Institute of Neurology and Neurosurgery

Arnaldo Milian Castro Clinical Surgical Hospital

Status of evaluation:

In review

Status of evaluation date of Ethic Committee:

01/09/2020

Postal address of Ethic Committee :

Carretera Valle Mayabe y Circunvalacion. CP. 80100, Holguin, Cuba

Calle 29, esq D, Vedado, La Habana, Cuba

Avenida “Arnaldo Milian Castro” e/ Circunvalacion y Doble Via. CP. 50100, Villa Clara, Cuba

Telephone:

+53-024481013

+53-78345587

+53-042270000

Email:

velazq63@gmail.com

-

padilla@hamc.vcl.sld.cu

About study completion

Section to complete the data related to the study completion.

Study completion date:

01/03/2023

Date of available results:

01/03/2024

Date of first publication:

01/09/2024

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:

RPCEC00000343

Date of Registration in Primary Registry:

19/11/2020

Record Verification Date:

2020/11/20

Next update date:

2021/11/20

Link to the spanish version:

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NeuroEPO-Ataxia-Adults-Phase III

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