Ignite Creation Date:
2024-05-05 @ 11:31 AM
Last Modification Date:
2024-10-26 @ 9:09 AM
Study NCT ID:
NCT00069550
Status:
UNKNOWN
Last Update Posted:
2005-06-24
First Post:
2003-09-29
Brief Title:
Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Study Overview
Official Title:
Pathogenesis of Rett Syndrome Natural History and Treatment
Status:
UNKNOWN
Status Verified Date:
2004-12
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Rett syndrome RTT is a disorder in which the nervous system does not develop properly RTT generally affects girls but there are some boys who have been diagnosed with RTT Symptoms of RTT include small brain size poor language skills repetitive hand movements and seizures This study will evaluate the effectiveness of two drugs in treating the symptoms of RTT
Detailed Description:
RTT is a neurodevelopmental disorder characterized by apparently normal early development followed by loss of purposeful hand use distinctive hand stereotypies slowed brain growth loss of language respiratory irregularities GI disturbances gait abnormalities seizures and mental retardation These symptoms appear between ages 6 and 18 months stage 2 of the disease following apparently normal development stage 1 Subsequently there is gradual stabilization of severe mental retardation and motor compromise stage 3 The majority 70 to 80 of patients demonstrate mutations in the methyl-CpG-binding-protein-2 MeCP2 gene a transcription repressor located on chromosome Xq28 The disorder predominantly affects females but a few males with mutations in MeCP2 have been identified even though many of them do not have the classic symptoms recognized in females
Recent studies demonstrate increased brain N-methyl-D-aspartate NMDA receptors in stages 2 and 3 of the disease This age-specific increase in glutamate levels and their receptors contribute to brain damage This first study will examine the effectiveness of dextromethorphan an NMDA receptor antagonist to ameliorate symptoms Participants will be randomized to receive one of three doses of dextromethorphan All participants will be admitted to the hospital for three days at the beginning of the study During the hospitalization participants will undergo physical exam Dexascan MRI EEG behavioral assessment laboratory testing and neuropsychological evaluations Six months after baseline assessment participants will be rehospitalized for 3 days for similar assessments
Reduction in choline acetyltransferase activity in RTT patients may also contribute to disturbed cortical development and psychomotor retardation in RTT Therefore the second part of the study will evaluate the effect of donepezil hydrochloride an inhibitor of acetylcholine-esterase on acetylcholine levels This portion of the study will not begin until pharmacokinetic data for donepezil in children is available
Recent studies demonstrate increased brain N-methyl-D-aspartate NMDA receptors in stages 2 and 3 of the disease This age-specific increase in glutamate levels and their receptors contribute to brain damage This first study will examine the effectiveness of dextromethorphan an NMDA receptor antagonist to ameliorate symptoms Participants will be randomized to receive one of three doses of dextromethorphan All participants will be admitted to the hospital for three days at the beginning of the study During the hospitalization participants will undergo physical exam Dexascan MRI EEG behavioral assessment laboratory testing and neuropsychological evaluations Six months after baseline assessment participants will be rehospitalized for 3 days for similar assessments
Reduction in choline acetyltransferase activity in RTT patients may also contribute to disturbed cortical development and psychomotor retardation in RTT Therefore the second part of the study will evaluate the effect of donepezil hydrochloride an inhibitor of acetylcholine-esterase on acetylcholine levels This portion of the study will not begin until pharmacokinetic data for donepezil in children is available
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
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 5P01HD024448 | NIH | None | httpsreporternihgovquickSearch5P01HD024448 |