A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT03633058 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: Ket-101-RSRT
• Study Type: interventional
• Target: 23
• Locations: 1 country
• Sites: 7

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.

Conditions

Rett Syndrome

Keywords

MECP2; RTT; Retts; ketamine

Outcome Measures

Primary Outcomes (1)

• Dose-Limiting Adverse Events

  The Number of Participants with Treatment-emergent adverse events on ketamine compared to placebo will be summarized

  6 weeks

Other Outcomes (9)

• Continuous Biosensor Data

  6-8 weeks

• Clinical Global Impression of Improvement

  4 weeks

• Motor Behavioral Assessment

  4 weeks

Study Arms (2)

0.75 mg/kg

EXPERIMENTAL

ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo

Drug: Ketamine

1.5 mg/kg

EXPERIMENTAL

ketamine will be dosed orally twice daily for 5 days at 0.75 mg/kg, in addition to 5 days of placebo

Drug: Ketamine

Interventions

Ketamine DRUG

oral ketamine dosed twice daily for 5 days

Also known as: Ketalar

0.75 mg/kg; 1.5 mg/kg

Eligibility Criteria

• Age: 6 Years - 12 Years
• Sex: female
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Rett Syndrome Research Trust: lead
• Vanderbilt University Medical Center: collaborator

Study Sites (7)

University of Alabama Birmingham School of Medicine

Birmingham, Alabama, 35294, United States

Location

Children's Hospital Colorado

Aurora, Colorado, 80045, United States

Location

Rush University Medical Center

Chicago, Illinois, 60612, United States

Location

Boston Children's Hospital

Boston, Massachusetts, 02115, United States

Location

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, 19104, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37203, United States

Location

Texas Children's Hospital

Houston, Texas, 77030, United States

Location

Related Publications (1)

• Campbell K, Neul JL, Lieberman DN, Berry-Kravis E, Benke TA, Fu C, Percy A, Suter B, Morris D, Carpenter RL, Marsh ED, von Hehn J. A randomized, placebo-controlled, cross-over trial of ketamine in Rett syndrome. J Neurodev Disord. 2025 Jan 24;17(1):4. doi: 10.1186/s11689-025-09591-y.

  PMID: 39856538 DERIVED

MeSH Terms

Conditions

Rett Syndrome

Interventions

Ketamine

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)

Cyclohexanes; Cycloparaffins; Hydrocarbons, Alicyclic; Hydrocarbons, Cyclic; Hydrocarbons; Organic Chemicals

Results Point of Contact

• Title: Jeffrey L. Neul, MD, PhD, Principal Investigator
• Organization: Vanderbilt University Medical Center

jeffrey.l.neul@vumc.org

615-322-8242

Study Officials

• Jeffrey Neul, MD, PhD

  Vanderbilt University Medical Center

  PRINCIPAL INVESTIGATOR

• Jana von Hehn, PhD

  Rett Syndrome Research Trust

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Masking Details: double-blind
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor of Pediatrics, Pharmacology, and Special Education

Model Details: Patients will receive placebo and ketamine for 5 days BID each, in a double-blind treatment order, and will be assessed for 2 weeks after each treatment.

Study Record Dates

• First Submitted: August 8, 2018
• First Posted: August 16, 2018
• Study Start: March 12, 2019
• Primary Completion: November 8, 2021
• Study Completion: November 22, 2021
• Last Updated: May 28, 2024
• Results First Posted: March 27, 2024

Record last verified: 2024-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (7)