Ignite Creation Date:
2024-05-05 @ 11:22 AM
Last Modification Date:
2024-10-26 @ 9:04 AM
Study NCT ID:
NCT00004807
Status:
COMPLETED
Last Update Posted:
2005-06-24
First Post:
2000-02-24
Brief Title:
Study of the Pathogenesis of Rett Syndrome
Sponsor:
Eunice Kennedy Shriver National Institute of Child Health and Human Development NICHD
Organization:
Office of Rare Diseases ORD
Study Overview
Official Title:
None
Status:
COMPLETED
Status Verified Date:
1998-12
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:
None
Brief Summary:
OBJECTIVES I Extend current knowledge of the phenotype and natural history of Rett syndrome RS
II Continue the search for a cytogenetic andor DNA marker III Study the effects of cholinergic drugs based on preliminary evidence for reduced levels of brain acetylcholine while continuing supportive care to modify seizures respiratory abnormalities and motor disturbances and improve nutrition behavior and learning
IV Identify targets for future therapeutic interventions eg growth factors to influence neurologic recovery
II Continue the search for a cytogenetic andor DNA marker III Study the effects of cholinergic drugs based on preliminary evidence for reduced levels of brain acetylcholine while continuing supportive care to modify seizures respiratory abnormalities and motor disturbances and improve nutrition behavior and learning
IV Identify targets for future therapeutic interventions eg growth factors to influence neurologic recovery
Detailed Description:
PROTOCOL OUTLINE Patients receive a comprehensive clinical evaluation including an ophthalmologic exam speech communication and developmental assessment nutritional evaluation neurologic exam respiratory monitoring and cytogenetic studies
Selected patients with malnutrition are given night feedings of Pediasure with Fiber by nasogastric tube Specific recommendations for feeding techniques and diet are made
Selected patients with seizures or severe hyperventilation and progressive rigidity are nonrandomly assigned to dextromethorphan or topiramate therapy
Oral dextromethorphan is maintained 6 months to 1 year duration of therapy depends on response Oral topiramate is given for 6 months to 1 year and Aricept for 6 months to 1 year
Concurrent anticonvulsants may require dose adjustments while on above protocols Supportive care for constipation scoliosis and weight loss is allowed
Selected patients with malnutrition are given night feedings of Pediasure with Fiber by nasogastric tube Specific recommendations for feeding techniques and diet are made
Selected patients with seizures or severe hyperventilation and progressive rigidity are nonrandomly assigned to dextromethorphan or topiramate therapy
Oral dextromethorphan is maintained 6 months to 1 year duration of therapy depends on response Oral topiramate is given for 6 months to 1 year and Aricept for 6 months to 1 year
Concurrent anticonvulsants may require dose adjustments while on above protocols Supportive care for constipation scoliosis and weight loss is allowed
Study Oversight
Has Oversight DMC:
Is a FDA Regulated Drug?:
Is a FDA Regulated Device?:
Is an Unapproved Device?:
Is a PPSD?:
Is a US Export?:
Is an FDA AA801 Violation?:
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| JHUSM-KKI-87021203 | None | None | None |