Rett Syndrome Clinical Trial
Official title:
Pilot Study of the Effects of the Desipramine on the Neurovegetative Parameters of the Child With Rett Syndrome
Verified date | July 2018 |
Source | Assistance Publique Hopitaux De Marseille |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Rett syndrome is a neurodevelopmental disorder characterized by cognitive impairment,
communication dysfunction, stereotypic movement disorder, and growth failure. Rett syndrome
is caused by mutations in the Methyl CpG-Binding Protein-2 (MECP2) gene and has no treatment.
A mouse experimental model of Rett syndrome created by genetic invalidation of the MECP2 gene
is available. It had been then observed that adult MECP2-deficient mice show respiratory
alterations and found that endogenous noradrenaline helps to maintain a normal respiratory
rhythm. Desipramine, a selective inhibitor of norepinephrine reuptake, seems to be efficient
to reduce the respiratory alteration occuring in MECP2-deficient mice (Insem patent 2005,
Villard and Roux 2006).
The aim of the study is to evaluate these obtained results in MECP2-deficient mice on
patients with Rett syndrome.
Status | Completed |
Enrollment | 36 |
Est. completion date | August 21, 2017 |
Est. primary completion date | August 11, 2014 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | 4 Years to 18 Years |
Eligibility |
Inclusion Criteria: - Rett syndrome; - Girls weighing less than 60 kg; - Respiratory alteration; - Diagnosis of Rett syndrome confirmed by MECP2 genotyping (Xq28). Exclusion Criteria: - Boys; - Pregnancy and breath feeding; - Case history of status epilepticus; - Patient treated by IMAO or sultopride; - Hepatic or renal failure. |
Country | Name | City | State |
---|---|---|---|
France | Assistance Publique - Hopitaux de Marseille | Marseille |
Lead Sponsor | Collaborator |
---|---|
Assistance Publique Hopitaux De Marseille |
France,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | To study the efficacy of the desipramine on the respiratory disturbations | 2 years | ||
Secondary | To study the safety of the desipramine in the studied population | 2 years |
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