A Study to Evaluate Ketamine for the Treatment of Rett Syndrome



Status:Recruiting
Conditions:Other Indications, Neurology
Therapuetic Areas:Neurology, Other
Healthy:No
Age Range:6 - 12
Updated:3/28/2019
Start Date:March 12, 2019
End Date:December 2019
Contact:Jana von Hehn, PhD
Email:jana@rsrt.org
Phone:203-444-1587

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A Randomized, Double-blind, Placebo-controlled, Cross-over Study to Assess the Safety, Tolerability and Efficacy of Oral Ketamine for Patients With Rett Syndrome

This 4 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
and up to approximately 48 patients in total are anticipated to participate for approximately
8-10 weeks at approximately 7 US study centers.

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 and up to 48 patients in total 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.

Inclusion Criteria:

- Female Rett Syndrome patients diagnosed with typical 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.
We found this trial at
7
sites
1211 Medical Center Dr
Nashville, Tennessee 37232
(615) 322-5000
Principal Investigator: Jeffrey Neul, MD, PhD
Phone: 615-875-2539
Vanderbilt Univ Med Ctr Vanderbilt University Medical Center (VUMC) is a comprehensive healthcare facility dedicated...
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Nashville, TN
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South 34th Street
Philadelphia, Pennsylvania 19104
 215-590-1000
Principal Investigator: Eric Marsh, MD, PhD
Phone: 267-426-5171
Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Philadelphia, PA
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13123 E 16th Ave
Aurora, Colorado 80045
(720) 777-1234
Principal Investigator: Timothy Benke, MD, PhD
Phone: 720-777-8499
Children's Hospital Colorado At Children's Hospital Colorado, we see more, treat more and heal more...
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Aurora, CO
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Birmingham, Alabama 35233
Principal Investigator: Alan Percy, MD
Phone: 205-996-4935
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Birmingham, AL
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300 Longwood Ave
Boston, Massachusetts 02115
(617) 355-6000
Principal Investigator: David Lieberman, MD, PhD
Phone: 617-355-1495
Boston Children's Hospital Boston Children's Hospital is a 395-bed comprehensive center for pediatric health care....
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Boston, MA
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1653 W. Congress Parkway
Chicago, Illinois 60612
(312) 942-5000
Principal Investigator: Elizabeth Berry-Kravis, MD, PhD
Phone: 312-942-2815
Rush University Medical Center Rush University Medical Center encompasses a 664-bed hospital serving adults and...
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Chicago, IL
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5600 Oakbrook Parkway
Norcross, Georgia 30093
Principal Investigator: Daniel Tarquinio, DO
Phone: 617-984-9091
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Norcross, GA
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