Ignite Creation Date:
2024-05-06 @ 2:32 AM
Last Modification Date:
2024-10-26 @ 11:19 AM
Study NCT ID:
NCT02061137
Status:
COMPLETED
Last Update Posted:
2018-06-15
First Post:
2013-08-27
Brief Title:
Study to Assess Safety and Efficacy of Fingolimod in Children With Rett Syndrome
Sponsor:
University Hospital Basel Switzerland
Organization:
University Hospital Basel Switzerland
Study Overview
Official Title:
A Phase 1 Clinical Study to Assess Safety and Efficacy of Oral Fingolimod FTY720 in Children With Rett Syndrome
Status:
COMPLETED
Status Verified Date:
2018-06
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
FINGORETT
Brief Summary:
The Trial Objective is to assess safety and efficacy of oral fingolimod FTY720 in children older than 6 years with Rett Syndrome So far there is no established treatment for children with Rett Syndrome Therefore a positive result in terms of safety and first indications of efficacy would path the way to a phase II clinical study with more patients to further test the hypothesis that fingolimod treatment may slow down the regression of motor and language skills
Detailed Description:
Rett syndrome is a neurodevelopmental disorder characterized by normal early psychomotor development followed by the loss of psychomotor and acquired purposeful hand skills and the onset of stereotyped movement of the hands and gait disturbance The gene was discovered in 1999 and the disease was found to be caused by a mutation of the methyl-CpGbinding protein 2 MeCP2 However in many ways this clinically peculiar condition remains a mystery with no clear correlations between the gene mutation and abnormal biological markers neuropathology andor unique clinical symptoms and signs
Rett syndrome is an X-linked Xq28 dominant postnatal severe neurodevelopmental disorder which is the second most common cause for genetic mental retardation in girls and the first pervasive disorder with a known genetic basis Its incidence is between 110000-15000 live births The classical variant is characterized by apparently normal development for the first 6-18 months accompanied usually with early deceleration of head growth followed by period of regression of motor and language skills hand stereotypes seizures autonomic dysfunction and other neurological and related symptoms
Repeated observations and experiments of the mouse models in several laboratories led to the appreciation of the role of BDNF in the disease pathophysiology BDNF is a neurotrophic factor playing a major role in neurogenesis neuronal survival differentiation and maturation during early development as well as in synaptic function and plasticity throughout life Abnormalities in BDNF homeostasis are believed to contribute to the neurological phenotype and pathophysiology in part of the symptoms in methyl-CpG binding protein 2Mecp2 null mice that show progressive deficits in its expression during the symptomatic stage
FTY720 Gilenya is an orally active modulator of four of the five sphingosine-1 phosphateS1P receptors FTY720 acts as super agonist on the S1P receptor on thymocytes and lymphocytes inducing uncouplinginternalization of that receptor
A local study group Yves-Alain Barde found that FTY720 increases the levels of brain derived neurotrophic factor and improves symptoms of mice lacking MeCP2 In addition the volume of the striatum seemed to be higher 4 week old mice were treated in 4 days intervals with 01mgkg body weight intraperitoneally
Based on these results we intend to perform a phase I clinicalstudy to assess safety and efficacy of oral fingolimod FTY720 in children with Rett Syndrome Children will be included if being older than 6 years of age fulfilling diagnostic criteria of Rett Syndrome in clinical Stages II -IV and having parents that do agree
Rett syndrome is an X-linked Xq28 dominant postnatal severe neurodevelopmental disorder which is the second most common cause for genetic mental retardation in girls and the first pervasive disorder with a known genetic basis Its incidence is between 110000-15000 live births The classical variant is characterized by apparently normal development for the first 6-18 months accompanied usually with early deceleration of head growth followed by period of regression of motor and language skills hand stereotypes seizures autonomic dysfunction and other neurological and related symptoms
Repeated observations and experiments of the mouse models in several laboratories led to the appreciation of the role of BDNF in the disease pathophysiology BDNF is a neurotrophic factor playing a major role in neurogenesis neuronal survival differentiation and maturation during early development as well as in synaptic function and plasticity throughout life Abnormalities in BDNF homeostasis are believed to contribute to the neurological phenotype and pathophysiology in part of the symptoms in methyl-CpG binding protein 2Mecp2 null mice that show progressive deficits in its expression during the symptomatic stage
FTY720 Gilenya is an orally active modulator of four of the five sphingosine-1 phosphateS1P receptors FTY720 acts as super agonist on the S1P receptor on thymocytes and lymphocytes inducing uncouplinginternalization of that receptor
A local study group Yves-Alain Barde found that FTY720 increases the levels of brain derived neurotrophic factor and improves symptoms of mice lacking MeCP2 In addition the volume of the striatum seemed to be higher 4 week old mice were treated in 4 days intervals with 01mgkg body weight intraperitoneally
Based on these results we intend to perform a phase I clinicalstudy to assess safety and efficacy of oral fingolimod FTY720 in children with Rett Syndrome Children will be included if being older than 6 years of age fulfilling diagnostic criteria of Rett Syndrome in clinical Stages II -IV and having parents that do agree
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None