Brief Summary
This 2 cohort, sequential, ascending dose study will assess the safety, tolerability and efficacy of oral ketamine dosed in a single 5-day BID regimen in addition to placebo, in a 4-week cross-over design in patients with Rett Syndrome. Approximately 12 patients per cohort are anticipated to participate for approximately 8-10 weeks at approximately 7 US study centers.
Brief Title
A Study to Evaluate Ketamine for the Treatment of Rett Syndrome
Detailed Description
This study is designed to assess oral ketamine for the treatment of Rett Syndrome and consists of up to 4 ascending dose cohorts, each assessing 1 dose level of ketamine vs placebo. Patients will receive in either order, a 5-day BID regimen of both placebo and the cohort-specified dose level of oral ketamine. Patients may only participate in 1 cohort. Safety and tolerability will be assessed via patient disposition, vital signs, physical examination, adverse events and concomitant medication use. Efficacy will be assessed via physician and caregiver questionnaires and assessments, and continuous, wearable, at-home biosensor data collection. An independent safety committee will review safety data from each cohort to determine if the subsequent ascending dose cohort is warranted. A total of 12 patients per cohort is anticipated at approximately 7 sites. The screening period will last between 2 and 4 weeks, the cross-over treatment period will last 4 weeks, and the safety follow-up period will last 2 weeks. Total patient participation is approximately 8-10 weeks.
Categories
Completion Date
Completion Date Type
Actual
Conditions
Rett Syndrome
Eligibility Criteria
Inclusion Criteria:
* Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
* between the ages of 6 and 12, inclusive, who have not achieved menarche
* ability to take oral medications
* are generally healthy.
Exclusion Criteria:
* Patients not on stable medication regimens/other types of behavioral, educational, or dietary interventions for at least 4 weeks,
* are taking medications that may interact with ketamine,
* have a condition where increased blood pressure, spinal fluid pressure, or ocular pressure may put the patient at increased risk.
* Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
* between the ages of 6 and 12, inclusive, who have not achieved menarche
* ability to take oral medications
* are generally healthy.
Exclusion Criteria:
* Patients not on stable medication regimens/other types of behavioral, educational, or dietary interventions for at least 4 weeks,
* are taking medications that may interact with ketamine,
* have a condition where increased blood pressure, spinal fluid pressure, or ocular pressure may put the patient at increased risk.
Inclusion Criteria
Inclusion Criteria:
* Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
* between the ages of 6 and 12, inclusive, who have not achieved menarche
* ability to take oral medications
* are generally healthy.
* Female Rett Syndrome patients diagnosed with Rett Syndrome with a confirmed MECP2 mutation
* between the ages of 6 and 12, inclusive, who have not achieved menarche
* ability to take oral medications
* are generally healthy.
Gender
Female
Gender Based
false
Keywords
MECP2
RTT
Retts
ketamine
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
Maximum Age
12 Years
Minimum Age
6 Years
NCT Id
NCT03633058
Org Class
Other
Org Full Name
Rett Syndrome Research Trust
Org Study Id
Ket-101-RSRT
Overall Status
Completed
Phases
Phase 2
Primary Completion Date
Primary Completion Date Type
Actual
Official Title
A Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett Syndrome
Primary Outcomes
Outcome Description
The Number of Participants with Treatment-emergent adverse events on ketamine compared to placebo will be summarized
Outcome Measure
Dose-Limiting Adverse Events
Outcome Time Frame
6 weeks
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Maximum Age Number (converted to Years and rounded down)
12
Minimum Age Number (converted to Years and rounded down)
6
Investigators
Investigator Type
Principal Investigator
Investigator Name
Aleksandra Djukic
Investigator Email
adjukic@montefiore.org
Investigator Phone