Down SyndromePhase 2NCT04751136
the Effect of Cerebrolysin on Physical and Mental Functions of Down Syndrome
A Phase 2 study testing cerebrolysin in people with Down Syndrome, run by Mansoura University Children Hospital at 1 site in Egypt.
Trial summary
Down syndrome is a genetic disorder that causes delay in both physical growth and mental development. It is the most frequently reported chromosomal abnormality and the most common genetic syndrome. Down syndrome is caused by trisomy of all or part of the genetic material of human chromosome 21. Read more about this study
- Who is it for?
- Ages 6 to 7 monthsHealthy volunteers can take part
- Conditions the study is for
- Down Syndrome
- What is being tested?
- cerebrolysin
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 64 participants
- Where
- 1 siteEgypt
- Study dates
- 30 Sep 2016to 30 Aug 2019
- Phase
- Phase 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 6 to 7 months
- All sexes
- Healthy volunteers accepted
- Child
Who may be able to join
- Infants were confirmed to have Down syndrome of non disjunction genotype.
- Age was around 6 month, at time of recruitment.
- Legal guardians accepted to participate in the study and sign the informed consent.
Who cannot join
- Patients having uncontrolled hypothyroidism
- Patients with brain malformations other than the expected in infant with Down syndrome.
- Patients with hearing and / or vision impairments.
- severe congenital heart disease
- Patients having contraindications for the use of Cerebrolysin
Trial locations and contacts
1 site in 1 country.
Egypt1 site
Mansoura University Children Hospital
Al Mansurah, Dakahlia Governorate, 35516
About this study
Official title: Phase (2) Clinical Trial Studying the Effect of Cerebrolysin on Physical and Mental Functions of Down Syndrome
Down syndrome is a genetic disorder that causes delay in both physical growth and mental development. It is the most frequently reported chromosomal abnormality and the most common genetic syndrome.
Down syndrome is caused by trisomy of all or part of the genetic material of human chromosome 21. It is now estimated that 94% of individuals with Down syndrome have an extra chromosome 21 as a result of meiotic non-disjunction, or the abnormal segregation of chromosomes during maternal gamete formation and of the remaining 5%, less than 1% is due to somatic mosaicism and the rest is due to chromosome 21 translocations.
Read more
The estimated incidence of Down syndrome is between 1 / 1,000 to 1 / 1,100 live births worldwide. In Egypt, the incidence of Down syndrome has been reported to be 1 / 1000 live births.
Down syndrome is characterized by intellectual disability, short stature, distinctive facial characters and a number of co-morbidities including cardiac and digestive anomalies, thyroid problems, and childhood leukemia.
Down syndrome infants will likely experience delays in certain areas and aspects of development. However, they will achieve all of the same milestones as other normal children, just on their own timetable.
According to recent studies, the Down syndrome behavioral phenotype includes relative strengths in some aspects of visuo-spatial processing and social functioning as well as relative deficits in verbal processing. Language has been described as a "major area of deficit" in Down syndrome individuals with particular difficulties manifested in expressive language.
Due to this high incidence of Down syndrome in Egypt and the associated co-morbidities, governmental care directed to this syndrome and other handicapping conditions has increased tremendously in the past few years to the extent that Down syndrome phenotype has become a phobia and many parents and/or physicians referred normal babies for karyotype due to either suspicion of chromosomal anomalies or just for reassurance of their parents.
Although there has been enormous progress in the management of the physical aspects of Down syndrome e.g. repair of heart defects, little advancement has been made to prevent deterioration of cognitive function in these individuals. As a result, the dramatic increase in life expectancy of children with Down syndrome in the past few decades has not been paralleled with concurrent treatment for cognitive disabilities. Therefore, it has remained the most common cause of cognitive dysfunction in children.
The pathogenesis of cognitive deficits and motor disabilities in Down syndrome individuals can be attributed to diminished number and size of neuronal density, progressive neuronal degeneration, impairment of neurogenesis, and reduction in dendrite formation as well as spine density which results in disruption of synaptic function and plasticity. Therefore, many of these individuals develop increasing problems with learning and memory in later life.
Cerebrolysin® is a neurotrophic peptidergic mixture isolated from pig brain. It is produced by standardized enzymatic breakdown of lipid-free porcine brain proteins .
It acts similar to endogenous neurotrophic factors in the form of promoting neuronal sprouting, stimulating neurogenesis, enhancing neuronal plasticity, and improving learning and memory.
Several studies demonstrated that Cerebrolysin® can be used safely in the management of children with any of the following medical conditions: minimal cerebral dysfunction, resistant forms of nocturnal enuresis, neurosensory hypoacusis, attention deficit hyperkinetic disorder, autism and Asperger syndrome.
The overall aim of the study is to assess the effect of Cerebrolysin® on neurocognitive development of infants with Down syndrome.
- Conditions
- Down Syndrome
- Being tested
- cerebrolysin
- Medical subject headings
- Down Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
cerebrolysinDrug
Infants of the treatment group were given Cerebrolysin in a dose of 0.1 ml / kg body weight once weekly intramuscular injection for 12 month (total of 48 injections).Each selected infant, in both treatment and control group, was assessed as regard: Socioeconomic status, Nutritional status and feeding practice, assessed for neurocognitive development
| Study group | Role | What they receive |
|---|---|---|
| Intervention Arm | Active comparator | infants were given Cerebrolysin®, manufactured by Neuro Pharma Gmbh, in a dose of 0.1 ml / kg body weight once weekly intramuscular injection for 12 month (total of 48 injections). Receives: Drug: cerebrolysin |
| Non-intervention Arm | No intervention | No medication was given |
What is being measured
The primary outcome is the main question the study is designed to answer.
Rate of neurocognitive development
We measure neurocognitive development using Bayley Scales of infant and toddler development®, 3rd Edition (BSID -III®) for infants of both groups, after 6 and 12 month of Cerebrolysin® injection. This scale assesses 5 subsets: receptive communication, expressive communication, fine motor, gross motor and cognitive development. Tests are implemented using the child's age (in months) to determine the starting test item for each subtest, and applying the reversal and discontinue rules. Then, the raw score is calculated for each subset as the number of test items that precede the starting item plus the number of items completed by the infant. Finally, test raw scores are recorded on standard record forms. Then, Z scores of scores are calculated according to USA norms. These calculated z scores are used as a standard to compare the rate of development for each subset.
Measured: Time frame: 12 month
Secondary outcomes (1)
Number of participants with side effects of cerebrolysin
We measure side effects that might occur during Cerebrolysin® therapy (one course with total of 48 injections).This is done, with every follow up visit, by physical examination of infants, and history taking from parents and care givers about development of irritability or convulsions during Cerebrolysin® therapy.
Measured: Time frame: 12 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Feb 2021
- Study start30 Sep 2016
- Primary completion28 Feb 2019
- Study completion30 Aug 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (10)Papers the sponsor has linked to this study.
- Other trials for Down SyndromeStudies 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Participant
- Enrolment
- 64 participants (actual)
Sponsor and investigators
- Lead sponsor
- Mansoura University Children Hospital
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Yahya Wahba, Mansoura University Children Hospital
- Investigators
- Sohier Yahia, MD, Study director, Mansoura University Children Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04751136
- Sponsor's protocol ID
- MansouraUCH1
- Status last verified
- Feb 2021
- First submitted
- 4 Feb 2021
- Last updated
- 11 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Valenti D, Braidy N, De Rasmo D, Signorile A, Rossi L, Atanasov AG, Volpicella M, Henrion-Caude A, Nabavi SM, Vacca RA. Mitochondria as pharmacological targets in Down syndrome. Free Radic Biol Med. 2018 Jan;114:69-83. doi: 10.1016/j.freeradbiomed.2017.08.014. Epub 2017 Aug 31.
PubMed 28838841Baburamani AA, Patkee PA, Arichi T, Rutherford MA. New approaches to studying early brain development in Down syndrome. Dev Med Child Neurol. 2019 Aug;61(8):867-879. doi: 10.1111/dmcn.14260. Epub 2019 May 17.
PubMed 31102269El-Gilany AH, Yahia S, Shoker M, El-Dahtory F. Cytogenetic and comorbidity profile of Down syndrome in Mansoura University Children's Hospital, Egypt. Indian J Hum Genet. 2011 Sep;17(3):157-63. doi: 10.4103/0971-6866.92092.
PubMed 22345986Sheets KB, Crissman BG, Feist CD, Sell SL, Johnson LR, Donahue KC, Masser-Frye D, Brookshire GS, Carre AM, Lagrave D, Brasington CK. Practice guidelines for communicating a prenatal or postnatal diagnosis of Down syndrome: recommendations of the national society of genetic counselors. J Genet Couns. 2011 Oct;20(5):432-41. doi: 10.1007/s10897-011-9375-8. Epub 2011 May 27.
PubMed 21618060Grieco J, Pulsifer M, Seligsohn K, Skotko B, Schwartz A. Down syndrome: Cognitive and behavioral functioning across the lifespan. Am J Med Genet C Semin Med Genet. 2015 Jun;169(2):135-49. doi: 10.1002/ajmg.c.31439. Epub 2015 May 18.
PubMed 25989505Tsao R, Kindelberger C. Variability of cognitive development in children with Down syndrome: relevance of good reasons for using the cluster procedure. Res Dev Disabil. 2009 May-Jun;30(3):426-32. doi: 10.1016/j.ridd.2008.10.009. Epub 2008 Nov 25.
PubMed 19036558Weston NM, Sun D. The Potential of Stem Cells in Treatment of Traumatic Brain Injury. Curr Neurol Neurosci Rep. 2018 Jan 25;18(1):1. doi: 10.1007/s11910-018-0812-z.
PubMed 29372464Muresanu DF, Heiss WD, Hoemberg V, Bajenaru O, Popescu CD, Vester JC, Rahlfs VW, Doppler E, Meier D, Moessler H, Guekht A. Cerebrolysin and Recovery After Stroke (CARS): A Randomized, Placebo-Controlled, Double-Blind, Multicenter Trial. Stroke. 2016 Jan;47(1):151-9. doi: 10.1161/STROKEAHA.115.009416. Epub 2015 Nov 12.
PubMed 26564102Kim JY, Kim HJ, Choi HS, Park SY, Kim DY. Effects of Cerebrolysin(R) in Patients With Minimally Conscious State After Stroke: An Observational Retrospective Clinical Study. Front Neurol. 2019 Aug 2;10:803. doi: 10.3389/fneur.2019.00803. eCollection 2019.
PubMed 31428035Krasnoperova MG, Bashina VM, Skvortsov IA, Simashkova NV. [The effect of cerebrolysin on cognitive functions in childhood autism and in Asperger syndrome]. Zh Nevrol Psikhiatr Im S S Korsakova. 2003;103(6):15-8. Russian.
PubMed 12872620
Study at a glance
From the registry record, last updated by the sponsor on 11 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase (2) Clinical Trial Studying the Effect of Cerebrolysin on Physical and Mental Functions of Down Syndrome
- ClinicalTrials.gov ID
- NCT04751136
- Acronym / sponsor ID
- MansouraUCH1
- Conditions
- Down Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 64 participants (actual)
- Lead sponsor
- Mansoura University Children HospitalUniversity, hospital or other
- Study start
- 30 Sep 2016 (actual)
- Primary completion
- 28 Feb 2019 (actual)
- Study completion
- 30 Aug 2019 (actual)
- First posted
- 11 Feb 2021 (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.
Down syndrome is a genetic disorder that causes delay in both physical growth and mental development. It is the most frequently reported chromosomal abnormality and the most common genetic syndrome.
Down syndrome is caused by trisomy of all or part of the genetic material of human chromosome 21. It is now estimated that 94% of individuals with Down syndrome have an extra chromosome 21 as a result of meiotic non-disjunction, or the abnormal segregation of chromosomes during maternal gamete formation and of the remaining 5%, less than 1% is due to somatic mosaicism and the rest is due to chromosome 21 translocations.
The estimated incidence of Down syndrome is between 1 / 1,000 to 1 / 1,100 live births worldwide. In Egypt, the incidence of Down syndrome has been reported to be 1 / 1000 live births.
Down syndrome is characterized by intellectual disability, short stature, distinctive facial characters and a number of co-morbidities including cardiac and digestive anomalies, thyroid problems, and childhood leukemia.
Down syndrome infants will likely experience delays in certain areas and aspects of development. However, they will achieve all of the same milestones as other normal children, just on their own timetable.
According to recent studies, the Down syndrome behavioral phenotype includes relative strengths in some aspects of visuo-spatial processing and social functioning as well as relative deficits in verbal processing. Language has been described as a "major area of deficit" in Down syndrome individuals with particular difficulties manifested in expressive language.
Due to this high incidence of Down syndrome in Egypt and the associated co-morbidities, governmental care directed to this syndrome and other handicapping conditions has increased tremendously in the past few years to the extent that Down syndrome phenotype has become a phobia and many parents and/or physicians referred normal babies for karyotype due to either suspicion of chromosomal anomalies or just for reassurance of their parents.
Although there has been enormous progress in the management of the physical aspects of Down syndrome e.g. repair of heart defects, little advancement has been made to prevent deterioration of cognitive function in these individuals. As a result, the dramatic increase in life expectancy of children with Down syndrome in the past few decades has not been paralleled with concurrent treatment for cognitive disabilities. Therefore, it has remained the most common cause of cognitive dysfunction in children.
The pathogenesis of cognitive deficits and motor disabilities in Down syndrome individuals can be attributed to diminished number and size of neuronal density, progressive neuronal degeneration, impairment of neurogenesis, and reduction in dendrite formation as well as spine density which results in disruption of synaptic function and plasticity. Therefore, many of these individuals develop increasing problems with learning and memory in later life.
Cerebrolysin® is a neurotrophic peptidergic mixture isolated from pig brain. It is produced by standardized enzymatic breakdown of lipid-free porcine brain proteins .
It acts similar to endogenous neurotrophic factors in the form of promoting neuronal sprouting, stimulating neurogenesis, enhancing neuronal plasticity, and improving learning and memory.
Several studies demonstrated that Cerebrolysin® can be used safely in the management of children with any of the following medical conditions: minimal cerebral dysfunction, resistant forms of nocturnal enuresis, neurosensory hypoacusis, attention deficit hyperkinetic disorder, autism and Asperger syndrome.
The overall aim of the study is to assess the effect of Cerebrolysin® on neurocognitive development of infants with Down syndrome.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (participant)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Intervention Arm | Active comparator | Drug: cerebrolysininfants were given Cerebrolysin®, manufactured by Neuro Pharma Gmbh, in a dose of 0.1 ml / kg body weight once weekly intramuscular injection for 12 month (total of 48 injections). |
| Non-intervention Arm | No intervention | –No medication was given |
Who may qualify
- Ages
- 6 to 7 months
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child
Key inclusion criteria (3 listed)
- Infants were confirmed to have Down syndrome of non disjunction genotype.
- Age was around 6 month, at time of recruitment.
- Legal guardians accepted to participate in the study and sign the informed consent.
Key exclusion criteria (5 listed)
- Patients having uncontrolled hypothyroidism
- Patients with brain malformations other than the expected in infant with Down syndrome.
- Patients with hearing and / or vision impairments.
- severe congenital heart disease
- Patients having contraindications for the use of Cerebrolysin
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 |
|---|---|---|
| cerebrolysinInfants of the treatment group were given Cerebrolysin in a dose of 0.1 ml / kg body weight once weekly intramuscular injection for 12 month (total of 48 injections).Each selected infant, in both treatment and control group, was assessed as regard: Socioeconomic status, Nutritional status and feeding practice, assessed for neurocognitive development | Drug | Intervention Arm |
Primary outcome
Rate of neurocognitive development
Time frame: Time frame: 12 month
We measure neurocognitive development using Bayley Scales of infant and toddler development®, 3rd Edition (BSID -III®) for infants of both groups, after 6 and 12 month of Cerebrolysin® injection. This scale assesses 5 subsets: receptive communication, expressive communication, fine motor, gross motor and cognitive development. Tests are implemented using the child's age (in months) to determine the starting test item for each subtest, and applying the reversal and discontinue rules. Then, the raw score is calculated for each subset as the number of test items that precede the starting item plus the number of items completed by the infant. Finally, test raw scores are recorded on standard record forms. Then, Z scores of scores are calculated according to USA norms. These calculated z scores are used as a standard to compare the rate of development for each subset.
1 secondary outcome
Number of participants with side effects of cerebrolysin
Time frame: Time frame: 12 month
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 11 Feb 2021.
- Background: PubMed 28838841Valenti D, Braidy N, De Rasmo D, Signorile A, Rossi L, Atanasov AG, Volpicella M, Henrion-Caude A, Nabavi SM, Vacca RA. Mitochondria as pharmacological targets in Down syndrome. Free Radic Biol Med. 2018 Jan;114:69-83. doi: 10.1016/j.freeradbiomed.2017.08.014. Epub 2017 Aug 31.
- Background: PubMed 31102269Baburamani AA, Patkee PA, Arichi T, Rutherford MA. New approaches to studying early brain development in Down syndrome. Dev Med Child Neurol. 2019 Aug;61(8):867-879. doi: 10.1111/dmcn.14260. Epub 2019 May 17.
- Background: PubMed 22345986El-Gilany AH, Yahia S, Shoker M, El-Dahtory F. Cytogenetic and comorbidity profile of Down syndrome in Mansoura University Children's Hospital, Egypt. Indian J Hum Genet. 2011 Sep;17(3):157-63. doi: 10.4103/0971-6866.92092.
- Background: PubMed 21618060Sheets KB, Crissman BG, Feist CD, Sell SL, Johnson LR, Donahue KC, Masser-Frye D, Brookshire GS, Carre AM, Lagrave D, Brasington CK. Practice guidelines for communicating a prenatal or postnatal diagnosis of Down syndrome: recommendations of the national society of genetic counselors. J Genet Couns. 2011 Oct;20(5):432-41. doi: 10.1007/s10897-011-9375-8. Epub 2011 May 27.
- Background: PubMed 25989505Grieco J, Pulsifer M, Seligsohn K, Skotko B, Schwartz A. Down syndrome: Cognitive and behavioral functioning across the lifespan. Am J Med Genet C Semin Med Genet. 2015 Jun;169(2):135-49. doi: 10.1002/ajmg.c.31439. Epub 2015 May 18.
- Background: PubMed 19036558Tsao R, Kindelberger C. Variability of cognitive development in children with Down syndrome: relevance of good reasons for using the cluster procedure. Res Dev Disabil. 2009 May-Jun;30(3):426-32. doi: 10.1016/j.ridd.2008.10.009. Epub 2008 Nov 25.
- Background: PubMed 29372464Weston NM, Sun D. The Potential of Stem Cells in Treatment of Traumatic Brain Injury. Curr Neurol Neurosci Rep. 2018 Jan 25;18(1):1. doi: 10.1007/s11910-018-0812-z.
- Background: PubMed 26564102Muresanu DF, Heiss WD, Hoemberg V, Bajenaru O, Popescu CD, Vester JC, Rahlfs VW, Doppler E, Meier D, Moessler H, Guekht A. Cerebrolysin and Recovery After Stroke (CARS): A Randomized, Placebo-Controlled, Double-Blind, Multicenter Trial. Stroke. 2016 Jan;47(1):151-9. doi: 10.1161/STROKEAHA.115.009416. Epub 2015 Nov 12.
- Background: PubMed 31428035Kim JY, Kim HJ, Choi HS, Park SY, Kim DY. Effects of Cerebrolysin(R) in Patients With Minimally Conscious State After Stroke: An Observational Retrospective Clinical Study. Front Neurol. 2019 Aug 2;10:803. doi: 10.3389/fneur.2019.00803. eCollection 2019.
- Background: PubMed 12872620Krasnoperova MG, Bashina VM, Skvortsov IA, Simashkova NV. [The effect of cerebrolysin on cognitive functions in childhood autism and in Asperger syndrome]. Zh Nevrol Psikhiatr Im S S Korsakova. 2003;103(6):15-8. Russian.
- Individual participant dataSharing planned: No
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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.
Sohier Yahia, MD study director
Mansoura University Children Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing cerebrolysin in people with Down Syndrome, run by Mansoura University Children Hospital at 1 site in Egypt.
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 3 inclusion and 5 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 11 Feb 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Egypt. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
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?
Rate of neurocognitive development (time frame: Time frame: 12 month). 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 NCT04751136, which also lists 10 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 11 Feb 2021.