|
Main
|
|
Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
|
Register:
|
ClinicalTrials.gov |
|
Last refreshed on:
|
30 March 2015 |
|
Main ID: |
NCT01619644 |
|
Date of registration:
|
12/06/2012 |
|
Prospective Registration:
|
No |
|
Primary sponsor: |
|
|
Public title:
|
Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial
RUBIVAL |
|
Scientific title:
|
Rubinstein-Taybi Syndrome: Functional Imaging and Therapeutic Trial |
|
Date of first enrolment:
|
April 2012 |
|
Target sample size:
|
41 |
|
Recruitment status: |
Completed |
|
URL:
|
http://clinicaltrials.gov/show/NCT01619644 |
|
Study type:
|
Interventional |
|
Study design:
|
Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment
|
|
Phase:
|
Phase 2
|
|
|
Countries of recruitment
|
|
France
| | | | | | | |
|
Contacts
|
|
Name:
|
Didier LACOMBE, PU-PH |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University Hospital Bordeaux, France |
|
|
Name:
|
Paul PEREZ, PH |
|
Address:
|
|
|
Telephone:
|
|
|
Email:
|
|
|
Affiliation:
|
University Hospital Bordeaux, France |
| |
|
Key inclusion & exclusion criteria
|
Inclusion Criteria:
- Children over 6 and under 21
- RTS confirmed by a genetic study with a CBP gene or EP300 gene mutation
- Sufficient cognitive capacities for neuropsychological evaluation
- Free and informed consent of the parents or guardians
- Children affiliated to or benefiting of the French social welfare system
Exclusion Criteria:
- Contraindication to sodium valproate
- Women of reproductive age without effective contraception means
- Case history of sodium valproate treatment
- Monotherapy treatment for epilepsy with Lamictal with a dosage superior to 5 mg/kg/j
- Family history of severe hepatitis including drug
- Acute or chronic hepatitis
- Pregnancy
Age minimum:
6 Years
Age maximum:
21 Years
Gender:
Both
|
|
Health Condition(s) or Problem(s) studied
|
|
Rubinstein-Taybi Syndrome
|
|
Intervention(s)
|
|
Drug: sodium valproate
|
|
Drug: Placebo
|
|
Primary Outcome(s)
|
|
Memory tests (assessing memory learning)
[Time Frame: 1 year]
|
|
Secondary Outcome(s)
|
|
Special brain imaging profile and motor skills (posturology and motor coordination in a visio-manual pointing task)
[Time Frame: 1 year]
|
|
Acetylation level of selected gene
[Time Frame: 1 year]
|
|
Cognitive and developmental profile
[Time Frame: 1 year]
|
|
Global acetylation level
[Time Frame: 1 year]
|
|
Histone acetylation profile
[Time Frame: 1 year]
|
|
Measurement of selected gene expression
[Time Frame: 1 year]
|
|
Secondary ID(s)
|
|
CHUBX 2011/20
|
|
Source(s) of Monetary Support
|
|
Please refer to primary and secondary sponsors
|
|
Results
|
|
Results available:
|
|
|
Date Posted:
|
|
|
Date Completed:
|
|
|
URL:
|
|
|
|