Clinical study to assess treatment effects of Cerebrolysin in amyotrophic lateral sclerosis
| ISRCTN | ISRCTN38393880 |
|---|---|
| DOI | https://doi.org/10.1186/ISRCTN38393880 |
- Submission date
- 01/12/2023
- Registration date
- 08/01/2024
- Last edited
- 09/01/2024
- Recruitment status
- No longer recruiting
- Overall study status
- Completed
- Condition category
- Nervous System Diseases
Plain English Summary
Background and study aims
Amyotrophic lateral sclerosis (ALS) is a progressive condition that causes you to lose control of your muscles in stages. Cerebrolysin is a drug that supports the nerves and may therefore have a beneficial effect in the treatment of ALS. This study assessed whether patients have a greater therapeutic benefit in treating the symptoms of ALS when Cerebrolysin is added to the standard treatment (riluzole).
Who can participate?
Patients aged 18 years and over with a confirmed diagnosis of ALS and a considerable increase in muscle tone
What does the study involve?
Patients received intravenous injections of 10 ml Cerebrolysin once a day, 5 days a week for the first month, then 3 days a week for the next 2 months, administered at home by a specialist nurse. Patients in the placebo group received the same treatment with 10 ml of normal saline. All patients received 50 mg of riluzole by mouth twice daily. Clinical assessments of motor and functional deterioration, spasticity and depressive symptoms were made before the start of treatment, at month 1, month 2 and at the end of the treatment period at month 3.
What are the possible benefits and risks of participating?
The potential benefit was an improvement in ALS symptoms, and no safety issues were expected.
Where is the study run from?
Instituto Cardiológico Banfield (ICB) (Argentina)
When is the study starting and how long is it expected to run for?
July 2020 to June 2022
Who is funding the study?
Instituto Cardiológico Banfield (ICB) (Argentina) and the study drug was provided by EVER Neuro Pharma GmbH
Who is the main contact?
Dr Afredo José Firstenfeld, alfredo.firstenfeld@gmail.com
Contact information
Public, Scientific, Principal Investigator
Maipú 660, B1828IJN Gran Buenos Aires
Buenos Aires
B1828IJN
Argentina
| Phone | +54 (0)11 4202-5925 |
|---|---|
| alfredo.firstenfeld@gmail.com |
Study information
| Study design | Investigator-initiated interventional prospective single-center placebo-controlled randomized double-blind Phase II study |
|---|---|
| Primary study design | Interventional |
| Secondary study design | Randomised controlled trial |
| Study setting(s) | Hospital |
| Study type | Quality of life, Treatment, Safety, Efficacy |
| Participant information sheet | Not available in web format, please use the contact details to request a participant information sheet |
| Scientific title | Add-on treatment with Cerebrolysin improves clinical symptoms in amyotrophic lateral sclerosis patients: results of a prospective, single-center, placebo-controlled, randomized, double-blind, Phase II study |
| Study hypothesis | Patients with amyotrophic lateral sclerosis (ALS) benefit more from treatment with a combination of Cerebrolysin and riluzole than they do from treatment with riluzole alone. |
| Ethics approval(s) |
Approved 12/01/2021, Comité De Ética De Clínica Privada Banfield Bancei (Félix de Azara 1780 – Banfield, Pcia de Bs. As, Buenos Aires, Banfield, Austria; +54 (0)11 3754-0050; info@clinicabanfield.com), ref: Carta de dictamen - Cerebrolysin - 12ene21 |
| Condition | Amyotrophic lateral sclerosis |
| Intervention | Patients were randomized 1:1 to Cerebrolysin or placebo (0.9% NaCl). An Excel-generated randomization list was used with blocks of 10 patients to account for balancing the sample size. Patients received intravenous injections of 10 ml Cerebrolysin once a day, 5 days a week for the first month, then 3 days a week for the next 2 months, administered at home by a specialist nurse. Patients in the placebo group received the same treatment with 10 ml of normal saline. All patients received 50 mg of riluzole by mouth twice daily. Clinical assessments of motor and functional deterioration, spasticity and depressive symptoms were made before the start of treatment, at month 1, month 2 and at the end of the treatment period at month 3. |
| Intervention type | Drug |
| Pharmaceutical study type(s) | Clinical efficacy and safety study |
| Phase | Phase II |
| Drug / device / biological / vaccine name(s) | Cerebrolysin, riluzole |
| Primary outcome measure | Functional impairment is measured using the Amyotrophic Lateral Sclerosis Functional Rating Scale – revised (ALSFRS-R) at baseline and month 1 |
| Secondary outcome measures | 1. Functional impairment is measured using the Amyotrophic Lateral Sclerosis Functional Rating Scale – revised (ALSFRS-R) at baseline to months 2 and 3 2. Depressive symptoms are measured using the Beck’s Depression Inventory-II (BDI-II) at baseline to months 1, 2, and 3 3. Spasticity is measured by the Modified Ashworth Scale (MAS) at baseline to months 1, 2, and 3 4. Gross motor skills are measured by the time taken to walk four meters, the walked distance in 120 seconds, and the number of knee bends to the opposite arm at baseline to months 1, 2, and 3 5. Hand strength is measured by an handheld dynamometer at baseline to months 1, 2, and 3 |
| Overall study start date | 08/07/2020 |
| Overall study end date | 02/06/2022 |
Eligibility
| Participant type(s) | Patient |
|---|---|
| Age group | Adult |
| Lower age limit | 18 Years |
| Sex | Both |
| Target number of participants | 20 |
| Total final enrolment | 20 |
| Participant inclusion criteria | 1. Either sex and at least 18 years of age 2. Clinically definite diagnosis of ALS according to the El Escorial and revised Airlie House diagnostic criteria 3. Limb onset and/or bulbar onset with pyramidal signs 4. Modified Ashworth Spasticity Scale score of 3 5. Informed consent |
| Participant exclusion criteria | 1. Co-morbidities such as hepatic disease, renal failure or severe renal impairment, coronary disease, epilepsy, Parkinson’s disease, or dementia 2. Any condition that might interfere with compliance with study procedures or influence outcome assessment 3. Pregnant or breastfeeding 4. Participation in another interventional study within the previous 2 months 5. Contraindication to Cerebrolysin 6. Concomitant use of ginkgo biloba, erythropoietin, citicoline, and amantadine |
| Recruitment start date | 29/07/2021 |
| Recruitment end date | 10/03/2022 |
Locations
Countries of recruitment
- Argentina
Study participating centre
Buenos Aires
B1828IJN
Argentina
Sponsor information
Hospital/treatment centre
Maipú 660
Buenos Aires
B1828IJN
Argentina
| Phone | +54 (0)11 4202-5925 |
|---|---|
| mccomeba@hotmail.es | |
| Website | http://www.institutocardiologicobanfield.com/ |
Funders
Funder type
Hospital/treatment centre
No information available
Results and Publications
| Intention to publish date | 31/12/2023 |
|---|---|
| Individual participant data (IPD) Intention to share | No |
| IPD sharing plan summary | Data sharing statement to be made available at a later date |
| Publication and dissemination plan | The publication is in the submission process. |
| IPD sharing plan | The data-sharing plans for the current study are unknown and will be made available at a later date. |
Editorial Notes
20/12/2023: Study's existence confirmed by the Comité De Ética De Clínica Privada Banfield Bancei.
