Rett Syndrome Clinical Trial
Official title:
Ketamine for the Treatment of Rett Syndrome: An Exploratory Trial
Verified date | August 2017 |
Source | The Cleveland Clinic |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
Rett Syndrome (RTT) is a complex disorder resulting from mutations in a gene encoding the
MeCP2 protein. Currently, there are no methods to fix the abnormal gene, however, animal
studies suggest that the symptoms of RTT can be treated.
Ketamine is a sedative or anesthetic, depending on the dose. The drug is approved by the US
Food and Drug Administration (FDA) and is commonly used in children and adults. Animal
studies and case reports in humans suggest that ketamine may reduce the symptoms of Rett
syndrome. The purpose of this study is to determine the safety and efficacy of ketamine for
treating breathing and behavioral symptoms of RTT.
Status | Terminated |
Enrollment | 4 |
Est. completion date | July 2017 |
Est. primary completion date | July 2017 |
Accepts healthy volunteers | No |
Gender | Female |
Age group | 5 Years to 25 Years |
Eligibility |
Inclusion Criteria: 1. Rett syndrome diagnosis based on the 2010 RTT diagnostic criteria, as determined by review of clinical records, and 2. Pathogenic mutation in the MECP2 gene, and 3. Breathing score of 3 or greater on RSBQ, and 4. Age 5-25 years. Exclusion Criteria: 1. Ongoing clinical regression as determined by review of clinical records and consultation with parents, or 2. Seizure within one week of study session, or 3. Unstable systemic illness other than Rett syndrome, or 4. Clinically important variations in medication use. |
Country | Name | City | State |
---|---|---|---|
United States | Cleveland Clinic | Cleveland | Ohio |
Lead Sponsor | Collaborator |
---|---|
The Cleveland Clinic |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Behavioral phenomena requiring intervention | Occurrence of agitation, irritability, or worsening of pre-existing symptoms. | From time of admission until time of discharge during any particular study visit, typically within 36 hours of admission. | |
Secondary | Exploratory - Rett Syndrome Behavior Questionnaire (RSBQ) | A formal score obtained by administering the RSBQ to the subject's parent/guardian before and after every study visit. | On the day following study drug infusion at each visit (30 - 36 hours after admission for each visit). |
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