Parkinson DiseasePhase 1/2NeuroEPONCT04110678
Tolerance to NeuroEPO in Parkinson Disease
A Phase 1/2 study testing NeuroEPO in people with Parkinson Disease, run by International Center for Neurological Restoration, Cuba at 2 sites in Cuba.
Trial summary
Treatment strategies in Parkinson's disease (PD) can improve a patient's quality of life but cannot stop the progression of PD. The investigators are looking for different alternatives that modify the natural course of the disease and recent research has demonstrated the neuroprotective properties of erythropoietin. Read more about this study
- Who is it for?
- Ages 40 to 70 years
- Conditions the study is for
- Parkinson Disease
- What is being tested?
- NeuroEPO
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 26 participants
- Where
- 2 sitesCuba
- Study dates
- 1 Nov 2015to 1 Jan 2017
- Phase
- Phase 1/2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 40 to 70 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Patient who fulfilled the London Brain Bank's operational criteria for PD
- Willing to participate in the study;
- ≥1 year since disease onset;
- Good response to antiparkinsonian treatment with levodopa (>30% change in motor score on the motor section of the Unified Parkinson's Disease
- no prior poly globulin (hematocrit ≤50%);
Who cannot join
- Refusal to participate;
- Known hypersensitivity to products derived from eukaryotes or hypersensitivity to human albumin;
- Pregnancy or breastfeeding;
- Hypertension;
- Immunosuppressant, androgen or anabolic steroid treatment in the month prior to recruitment;
- Sepsis or active infection;
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- Active acute or chronic inflammatory diseases;
- Haematological diseases, such as sickle cell disease, myelodysplastic syndromes, active clotting or bleeding disorders;
- Malignant tumor or cancer treatment;
- Alcoholism or drug addiction in the two years prior to inclusion assessment.
- Significant cognitive decline as measured by clinical assessment, DRS (Dementia Rating Scale) and the Diagnostic and Statistical Manual of Mental Disorders (DSM-IV).
Trial locations and contacts
2 sites in 1 country.
Cuba2 sites
Clinic of Movement Disorders, International Center for Neurological Restoration
Havana
Centro Inmunologia Molecular CIM
La Habana, 11300
About this study
Official title: Nasal Administration of the NeuroEPO in Parkinson Disease: Short-term Tolerance Physician Lead Trial
Treatment strategies in Parkinson's disease (PD) can improve a patient's quality of life but cannot stop the progression of PD. The investigators are looking for different alternatives that modify the natural course of the disease and recent research has demonstrated the neuroprotective properties of erythropoietin. In Cuba, the Center for Molecular Immunology (CIM) is a cutting edge scientific center where the recombinant form (EPOrh) and recombinant human erythropoietin with low sialic acid (NeuroEPO) are produced.
Read the detailed description
Delivery of therapeutic agents as erythropoietin (EPO) into Central Nervous System through intranasal route could benefit patients with neurological disorders. A new nasal formulation containing a non-hematopoietic recombinant EPO (NeuroEPO) has shown neuroprotective actions in preclinical models and the safety trial of NeuroEPO was evaluated for the first time in humans in Cuba.
The researchers previously conducted a proof of concept clinical trial, administering EPOrh subcutaneously where the neuropsychological performance was measured as a secondary endpoint. This protocol has the register number NCT01010802. Results showed an increased neuropsychological performance of patients after administration as compared to before administration results.
In this protocol two institutions were leading a physician clinical trial.The two institutions: International Center for Neurological Restoration (CIREN from Centro Internacional de Restauracion Neurologica from Spanish) where the recruitment and clinical evaluation of the PD patients will be done and the Center for Molecular Immunology, who was the promoter.
Randomisation will be performed by the CIM to assign the patients to groups. The groups will receive neuroEPO or placebo with identical organoleptic characteristics. The informed consent of all patients will be obtained before the start of the trial after the selection and primary evaluation of the inclusion criteria.
The dose of neuroEPO will be a vial with a dose of 1 mL/1mg administered intra-nasally for five consecutive weeks. The placebo group was administered 1 mL of an intranasal inert solution for the same period of time.
The IBM SPSS Statistics V 21 package will be used for the statistical analysis of the data. The investigators will use tables of frequency analysis and descriptive statistics to analyse the demographic characteristics of the sample.
The analysis of the results obtained will be done studying the differences between quantitative variables for paired and unpaired samples, the Wilcoxon and U of Mann-Whitney tests will be used respectively. For the qualitative variables Chi square (X2) test will be used. All values of p < 0.05 will be considered significant.
- Conditions
- Parkinson Disease
- Being tested
- NeuroEPO
- Keywords
- Parkinson Disease, Tolerance, NeuroEPO, side effects, UPDRS, Cognition
- Medical subject headings
- Parkinson Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
NeuroEPODrug
Also called Placebo
NeuroEPO [CIMAB S.A, Havana, Cuba]
| Study group | Role | What they receive |
|---|---|---|
| NeuroEPO | Active comparator | The dose of NeuroEPO was a vial with a dose of 1 mL/1mg administered intra-nasally for five consecutive weeks. Receives: Drug: NeuroEPO |
| Placebo | Placebo comparator | The dose of placebo was a vial with a dose of 1 mL/1mg of an intranasal inert solution administered intra-nasally for five consecutive weeks. Receives: Drug: NeuroEPO |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of participants with local nasal events
the administration of the drug is intra-nasal for that reason the first adverse event to study is the irritation of the nose. The presence of toxicity signs in the nasal mucous, such as: redness, swelling and nasal congestion was evaluated by means of thorough medical examination of the nasal cavity by the same Otorhinolaryngology Specialist.
Measured: day 1-5
Number or participants with increment in the haematological and biochemistry parameters
the blood tests were obtained to evaluate hematological counts (reticulocytes, hemoglobin, hematocrit, leukocytes), coagulation parameters (platelet count, partial thromboplastin and prothrombin times) and blood chemistry (glycemia, creatinine, urea, liver enzymes)
Measured: day 0 day 5 and day 14
Change from Baseline Systolic Blood Pressure after each intervention
The vital signs and the physical checkup was permanent before and after each application. The change of the baseline systolic blood pressure could be a criteria for suspension of the treatment
Measured: day 1-5
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Oct 2019
- Study start1 Nov 2015
- Primary completion11 Feb 2016
- Study completion1 Jan 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (3)Papers the sponsor has linked to this study.
- Other trials for Parkinson DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- Randomized, blinded, Physician lead trial
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 26 participants (actual)
Sponsor and investigators
- Lead sponsor
- International Center for Neurological Restoration, Cuba
- Sponsor type
- University, hospital or other
- Collaborators
- Center of Molecular Immunology, Cuba, University of Electronic Science and Technology of China
- Responsible party
- Principal investigator, Ivonne Pedroso Ibanez, International Center for Neurological Restoration, Cuba
- Investigators
- Daniel E Amaro Gonzalez, PhD Eng, Principal investigator, Centro Inmunologia Molecular CIM Havana CubaTeresita Rodríguez Obaya, PhD, Study chair, Centro Immunologia Molecular CIM Havana CubaIliana Sosa Teste, PhD, Principal investigator, Centro Nacional Producción Animales de Laboratorio (CENPALAB) Cuba
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04110678
- Sponsor's protocol ID
- NeuroEPO-001-PD
- Acronym
- NeuroEPO
- Status last verified
- Jul 2020
- First submitted
- 18 Sep 2019
- Last updated
- 15 Jul 2020
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
Pedroso I, Garcia M, Casabona E, Morales L, Bringas ML, Perez L, Rodriguez T, Sosa I, Ricardo Y, Padron A, Amaro D. Protective Activity of Erythropoyetine in the Cognition of Patients with Parkinson's Disease. Behav Sci (Basel). 2018 May 21;8(5):51. doi: 10.3390/bs8050051. eCollection 2018 May.
PubMed 29862060Bringas Vega ML, Pedroso Ibanez I, Razzaq FA, Zhang M, Morales Chacon L, Ren P, Galan Garcia L, Gan P, Virues Alba T, Lopez Naranjo C, Jahanshahi M, Bosch-Bayard J, Valdes-Sosa PA. The Effect of Neuroepo on Cognition in Parkinson's Disease Patients Is Mediated by Electroencephalogram Source Activity. Front Neurosci. 2022 Jun 30;16:841428. doi: 10.3389/fnins.2022.841428. eCollection 2022.
PubMed 35844232Garcia-Llano M, Pedroso-Ibanez I, Morales-Chacon L, Rodriguez-Obaya T, Perez-Ruiz L, Sosa-Teste I, Amaro-Gonzalez D, Bringas-Vega ML. Short-term Tolerance of Nasally-administered NeuroEPO in Patients with Parkinson Disease. MEDICC Rev. 2021 Jan;23(1):49-54. doi: 10.37757/MR2021.V23.N1.10. Epub 2021 Jan 30.
PubMed 33780423
Study at a glance
From the registry record, last updated by the sponsor on 15 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Nasal Administration of the NeuroEPO in Parkinson Disease: Short-term Tolerance Physician Lead Trial
- ClinicalTrials.gov ID
- NCT04110678
- Acronym / sponsor ID
- NeuroEPO / NeuroEPO-001-PD
- Conditions
- Parkinson Disease
- Keywords
- Parkinson Disease, Tolerance, NeuroEPO, side effects, UPDRS, Cognition
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 26 participants (actual)
- Lead sponsor
- International Center for Neurological Restoration, CubaUniversity, hospital or other
- Collaborators
- Center of Molecular Immunology, Cuba, University of Electronic Science and Technology of China
- Study start
- 1 Nov 2015 (actual)
- Primary completion
- 11 Feb 2016 (actual)
- Study completion
- 1 Jan 2017 (actual)
- First posted
- 1 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Treatment strategies in Parkinson's disease (PD) can improve a patient's quality of life but cannot stop the progression of PD. The investigators are looking for different alternatives that modify the natural course of the disease and recent research has demonstrated the neuroprotective properties of erythropoietin. In Cuba, the Center for Molecular Immunology (CIM) is a cutting edge scientific center where the recombinant form (EPOrh) and recombinant human erythropoietin with low sialic acid (NeuroEPO) are produced.
Detailed description
Delivery of therapeutic agents as erythropoietin (EPO) into Central Nervous System through intranasal route could benefit patients with neurological disorders. A new nasal formulation containing a non-hematopoietic recombinant EPO (NeuroEPO) has shown neuroprotective actions in preclinical models and the safety trial of NeuroEPO was evaluated for the first time in humans in Cuba.
The researchers previously conducted a proof of concept clinical trial, administering EPOrh subcutaneously where the neuropsychological performance was measured as a secondary endpoint. This protocol has the register number NCT01010802. Results showed an increased neuropsychological performance of patients after administration as compared to before administration results.
In this protocol two institutions were leading a physician clinical trial.The two institutions: International Center for Neurological Restoration (CIREN from Centro Internacional de Restauracion Neurologica from Spanish) where the recruitment and clinical evaluation of the PD patients will be done and the Center for Molecular Immunology, who was the promoter.
Randomisation will be performed by the CIM to assign the patients to groups. The groups will receive neuroEPO or placebo with identical organoleptic characteristics. The informed consent of all patients will be obtained before the start of the trial after the selection and primary evaluation of the inclusion criteria.
The dose of neuroEPO will be a vial with a dose of 1 mL/1mg administered intra-nasally for five consecutive weeks. The placebo group was administered 1 mL of an intranasal inert solution for the same period of time.
The IBM SPSS Statistics V 21 package will be used for the statistical analysis of the data. The investigators will use tables of frequency analysis and descriptive statistics to analyse the demographic characteristics of the sample.
The analysis of the results obtained will be done studying the differences between quantitative variables for paired and unpaired samples, the Wilcoxon and U of Mann-Whitney tests will be used respectively. For the qualitative variables Chi square (X2) test will be used. All values of p < 0.05 will be considered significant.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
- Model description
- Randomized, blinded, Physician lead trial
| Arm | Type | Receives |
|---|---|---|
| NeuroEPO | Active comparator | Drug: NeuroEPOThe dose of NeuroEPO was a vial with a dose of 1 mL/1mg administered intra-nasally for five consecutive weeks. |
| Placebo | Placebo comparator | Drug: NeuroEPOThe dose of placebo was a vial with a dose of 1 mL/1mg of an intranasal inert solution administered intra-nasally for five consecutive weeks. |
Who may qualify
- Ages
- 40 to 70 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- Patient who fulfilled the London Brain Bank's operational criteria for PD
- Willing to participate in the study;
- ≥1 year since disease onset;
- Good response to antiparkinsonian treatment with levodopa (>30% change in motor score on the motor section of the Unified Parkinson's Disease
- no prior poly globulin (hematocrit ≤50%);
Key exclusion criteria (11 listed)
- Refusal to participate;
- Known hypersensitivity to products derived from eukaryotes or hypersensitivity to human albumin;
- Pregnancy or breastfeeding;
- Hypertension;
- Immunosuppressant, androgen or anabolic steroid treatment in the month prior to recruitment;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| NeuroEPOAlso: PlaceboNeuroEPO [CIMAB S.A, Havana, Cuba] | Drug | NeuroEPO, Placebo |
Primary outcome
Number of participants with local nasal events
Time frame: day 1-5
the administration of the drug is intra-nasal for that reason the first adverse event to study is the irritation of the nose. The presence of toxicity signs in the nasal mucous, such as: redness, swelling and nasal congestion was evaluated by means of thorough medical examination of the nasal cavity by the same Otorhinolaryngology Specialist.
Number or participants with increment in the haematological and biochemistry parameters
Time frame: day 0 day 5 and day 14
the blood tests were obtained to evaluate hematological counts (reticulocytes, hemoglobin, hematocrit, leukocytes), coagulation parameters (platelet count, partial thromboplastin and prothrombin times) and blood chemistry (glycemia, creatinine, urea, liver enzymes)
Change from Baseline Systolic Blood Pressure after each intervention
Time frame: day 1-5
The vital signs and the physical checkup was permanent before and after each application. The change of the baseline systolic blood pressure could be a criteria for suspension of the treatment
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Jul 2020.
- Results publication: PubMed 29862060Pedroso I, Garcia M, Casabona E, Morales L, Bringas ML, Perez L, Rodriguez T, Sosa I, Ricardo Y, Padron A, Amaro D. Protective Activity of Erythropoyetine in the Cognition of Patients with Parkinson's Disease. Behav Sci (Basel). 2018 May 21;8(5):51. doi: 10.3390/bs8050051. eCollection 2018 May.
- Derived from this study: PubMed 35844232Bringas Vega ML, Pedroso Ibanez I, Razzaq FA, Zhang M, Morales Chacon L, Ren P, Galan Garcia L, Gan P, Virues Alba T, Lopez Naranjo C, Jahanshahi M, Bosch-Bayard J, Valdes-Sosa PA. The Effect of Neuroepo on Cognition in Parkinson's Disease Patients Is Mediated by Electroencephalogram Source Activity. Front Neurosci. 2022 Jun 30;16:841428. doi: 10.3389/fnins.2022.841428. eCollection 2022.
- Derived from this study: PubMed 33780423Garcia-Llano M, Pedroso-Ibanez I, Morales-Chacon L, Rodriguez-Obaya T, Perez-Ruiz L, Sosa-Teste I, Amaro-Gonzalez D, Bringas-Vega ML. Short-term Tolerance of Nasally-administered NeuroEPO in Patients with Parkinson Disease. MEDICC Rev. 2021 Jan;23(1):49-54. doi: 10.37757/MR2021.V23.N1.10. Epub 2021 Jan 30.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel E Amaro Gonzalez, PhD Eng principal investigator
Centro Inmunologia Molecular CIM Havana Cuba
Teresita Rodríguez Obaya, PhD study chair
Centro Immunologia Molecular CIM Havana Cuba
Iliana Sosa Teste, PhD principal investigator
Centro Nacional Producción Animales de Laboratorio (CENPALAB) Cuba
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing NeuroEPO in people with Parkinson Disease, run by International Center for Neurological Restoration, Cuba at 2 sites in Cuba.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 15 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Cuba. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of participants with local nasal events (time frame: day 1-5). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04110678, which also lists 3 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Jul 2020.