A Study to Evaluate Ketamine for the Treatment of Rett Syndrome

NCT ID: NCT03633058

Last Updated: 2024-05-28

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 23 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-03-12
• Study Completion Date: 2021-11-22

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.

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.

Conditions

Rett Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: CROSSOVER
• Primary Study Purpose: TREATMENT
• Blinding Strategy: TRIPLE

Study Groups

0.75 mg/kg

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

Group Type: EXPERIMENTAL

Ketamine

Intervention Type: DRUG

oral ketamine dosed twice daily for 5 days

1.5 mg/kg

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

Group Type: EXPERIMENTAL

Ketamine

Intervention Type: DRUG

oral ketamine dosed twice daily for 5 days

Interventions

Ketamine

oral ketamine dosed twice daily for 5 days

Intervention Type: DRUG

Other Intervention Names

Ketalar

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.

• Minimum Eligible Age: 6 Years
• Maximum Eligible Age: 12 Years
• Eligible Sex: FEMALE
• Accepts Healthy Volunteers: No

Sponsors

Vanderbilt University Medical Center

OTHER

Sponsor Role: collaborator

Rett Syndrome Research Trust

OTHER

Sponsor Role: lead

Responsible Party

Jeffrey Neul

Director, Vanderbilt Kennedy Center, Annette Schaffer Eskind Chair, Professor of Pediatrics, Pharmacology, and Special Education

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jeffrey Neul, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Vanderbilt University Medical Center

Jana von Hehn, PhD

Role: STUDY_DIRECTOR

Rett Syndrome Research Trust

Locations

University of Alabama Birmingham School of Medicine

Birmingham, Alabama, United States

Children's Hospital Colorado

Aurora, Colorado, United States

Rush University Medical Center

Chicago, Illinois, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Children's Hospital of Philadelphia

Philadelphia, Pennsylvania, United States

Vanderbilt University Medical Center

Nashville, Tennessee, United States

Texas Children's Hospital

Houston, Texas, United States

Countries

United States

References

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.

Reference Type: DERIVED

PMID: 39856538 (View on PubMed)

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

Ket-101-RSRT

Identifier Type: -

Identifier Source: org_study_id