An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome

NCT02562820 | Terminated Phase 1 DMC

• 1 other identifier: 15-627
• Study Type: interventional
• Target: 4
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Rett Syndrome

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Exploratory - Rett Syndrome Behavior Questionnaire (RSBQ)

  On the day following study drug infusion at each visit (30 - 36 hours after admission for each visit).

Study Arms (2)

Ketamine

EXPERIMENTAL

Subjects will receive intravenous infusion of a 0.1, 0.5, 1.0, 2.0 or 4.5 mg/kg dose over the course of 40 minutes

Procedure: Intravenous infusion

Placebo

PLACEBO COMPARATOR

Subjects will receive an intravenous infusion of normal saline over the course of 40 minutes

Procedure: Intravenous infusion

Interventions

Intravenous infusion PROCEDURE

Intravenous infusion of Ketamine

Ketamine

Eligibility Criteria

• Age: 5 Years - 25 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Rett syndrome diagnosis based on the 2010 RTT diagnostic criteria, as determined by review of clinical records, and
• Pathogenic mutation in the MECP2 gene, and
• Breathing score of 3 or greater on RSBQ, and
• Age 5-25 years.

You may not qualify if:

• Ongoing clinical regression as determined by review of clinical records and consultation with parents, or
• Seizure within one week of study session, or
• Unstable systemic illness other than Rett syndrome, or
• Clinically important variations in medication use.

Sponsors & Collaborators

• The Cleveland Clinic: lead

Study Sites (1)

Cleveland Clinic

Cleveland, Ohio, 44195, United States

Location

MeSH Terms

Conditions

Rett Syndrome

Interventions

Infusions, Intravenous

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Heredodegenerative Disorders, Nervous System

Intervention Hierarchy (Ancestors)

Administration, Intravenous; Drug Administration Routes; Drug Therapy; Therapeutics; Infusions, Parenteral

Study Officials

• Daniel Sessler, MD

  The Cleveland Clinic

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 28, 2015
• First Posted: September 29, 2015
• Study Start: November 1, 2015
• Primary Completion: July 1, 2017
• Study Completion: July 1, 2017
• Last Updated: August 30, 2017

Record last verified: 2017-08

Locations

US (1)