Clinical Trials Logo

Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT03633058
Other study ID # Ket-101-RSRT
Secondary ID
Status Completed
Phase Phase 2
First received
Last updated
Start date March 12, 2019
Est. completion date November 22, 2021

Study information

Verified date May 2024
Source Rett Syndrome Research Trust
Contact n/a
Is FDA regulated No
Health authority
Study type Interventional

Clinical Trial 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.


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.


Recruitment information / eligibility

Status Completed
Enrollment 23
Est. completion date November 22, 2021
Est. primary completion date November 8, 2021
Accepts healthy volunteers No
Gender Female
Age group 6 Years to 12 Years
Eligibility 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.

Study Design


Related Conditions & MeSH terms


Intervention

Drug:
Ketamine
oral ketamine dosed twice daily for 5 days

Locations

Country Name City State
United States Children's Hospital Colorado Aurora Colorado
United States University of Alabama Birmingham School of Medicine Birmingham Alabama
United States Boston Children's Hospital Boston Massachusetts
United States Rush University Medical Center Chicago Illinois
United States Texas Children's Hospital Houston Texas
United States Vanderbilt University Medical Center Nashville Tennessee
United States Children's Hospital of Philadelphia Philadelphia Pennsylvania

Sponsors (2)

Lead Sponsor Collaborator
Rett Syndrome Research Trust Vanderbilt University Medical Center

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Other Continuous Biosensor Data 2 different non-invasive, wearable devices will be used in the study to determine changes in physiologic measures for the patient in the home environment. Biosensors will be worn continuously during the screening and treatment period to measure activity, sleep, position, heart rate, and breathing. 6-8 weeks
Other Clinical Global Impression of Improvement Clinicians will use a 7 point Likert Scale to rate change from baseline symptom severity by addressing the question, "Compared to the patient's condition of Rett syndrome prior to treatment initiation at baseline, the patient's current condition is: 1 very much improved, 2 much improved, 3 minimally improved, 4 no change, 5 minimally worse, 6 much worse, 7 very much worse. No change would be scored a 0, while improvement would be scored at -1, -2, or -3 and worsening would be scored +1, +2 or +3, depending on the degree of perceived change. Negative change indicates improvement while positive change indicates worsening 4 weeks
Other Motor Behavioral Assessment a 37-item questionnaire for clinicians to evaluate current behavioral/social, orofacial/respiratory, and motor/physical symptoms. Each item is rated either 0 (normal or never), 1 (mild or rare), 2 (moderate or occasional), 3 (marked or frequent), 4 (very severe or constant), where higher numbers indicate higher severity. Each subscale is summed for a subscale score, while the total score is a sum of the subscale scores. The behavioral/social subscale score may range from 0 to 64; the orofacial/respiratory subscale may range from 0 to 28, and motor/physical subscale may range from 0 to 56. Total score may range from 0 to 148. 4 weeks
Other Clinician Domain Likert Scale an 8 category (domain) questionnaire to assess current hand function, walking, verbal and non-verbal communication, comprehension, attention, behavior problems, and mood by considering the question, "Considering your experience with the patient at this visit, please rate the level of function in each category". Each domain will be rated on a 7 point Likert Scale where the clinician will select 1 of 7 choices: 1 normal, 2 borderline abnormal, 3 mildly abnormal, 4 moderately abnormal, 5 markedly abnormal, 6 severely abnormal, 7 extremely abnormal. Lower values indicate lesser severity while higher values indicate higher severity. 4 weeks
Other Rett Syndrome Behaviour Questionnaire a 45-item questionnaire for clinicians to evaluate current behavior and emotional features. Each item is rated either 0 (not true or not done), 1 (somewhat or sometimes true) and 2 (very true), where higher numbers indicate higher severity. Total score is summed and may range from 0 to 90. 4 weeks
Other Parent Domain Likert Scale a 9 category (domain) questionnaire to assess current hand function, walking, verbal and non-verbal communication, comprehension, attention, behavior problems, mood, and seizure activity, by considering the question, "Considering your experience with your child over the past 7 days, please rate your child's level of function in each category". Each domain will be rated on a 7 point Likert Scale where the parent/caregiver will select 1 of 7 choices: 1 normal, 2 borderline abnormal, 3 mildly abnormal, 4 moderately abnormal, 5 markedly abnormal, 6 severely abnormal, 7 extremely abnormal. Lower values indicate lesser severity while higher values indicate higher severity. 4 weeks
Other Children's Sleep Habits Questionnaire a 35-item questionnaire for parents to evaluate current sleep and common sleep problems. The parent/caregiver will rate each item as 1 (rarely), 2 (sometimes), or 3 (usually), where higher scores indicate higher severity. The parent/caregiver will also indicate if the item is a problem or not. A total score of the sum of each item and 8 subscale scores (bedtime resistance, sleep onset delay, sleep duration, sleep anxiety, night wakings, parasomnias, sleep disordered breathing, and daytime sleepiness) are possible. Total scores between 0 and 70 are possible, where higher scores indicate more sleep problems. 4 weeks
Other Rett Caregiver Burden Inventory Assessment a 26-item questionnaire for parents to evaluate the burden of care on their quality of life. The parent/caregiver will rate each item as 0 (never), 1 (rarely), 2 (sometimes), 3 (quite frequently), or 4 (nearly always) where higher scores indicate higher severity. Total scores between 0 and 104 are possible, where higher scores indicate more caregiver burden. 4 weeks
Other EEG Signature ketamine EEG alpha, beta, gamma, delta and theta waveform signatures compared to placebo to indicate target engagement Day 1, Day 15
Primary Dose-Limiting Adverse Events The Number of Participants with Treatment-emergent adverse events on ketamine compared to placebo will be summarized 6 weeks
See also
  Status Clinical Trial Phase
Completed NCT04988867 - An Open-Label Study of Trofinetide for the Treatment of Girls Two to Five Years of Age Who Have Rett Syndrome Phase 2/Phase 3
Recruiting NCT00069550 - Independent Studies of Dextromethorphan and of Donepezil Hydrochloride for Rett Syndrome Phase 3
Enrolling by invitation NCT06139172 - Promoting Prosocial Behavior in Syndromic Intellectual and Developmental Disabilities N/A
Not yet recruiting NCT04041713 - A Pilot Study of an Antioxidant Cocktail vs. Placebo in the Treatment of Children and Adolescents With Rett Syndrome Phase 2
Not yet recruiting NCT04014985 - Patients With RETT Syndrome N/A
Completed NCT02705677 - Biobanking of Rett Syndrome and Related Disorders
Terminated NCT02790034 - Evaluation of the Efficacy, Safety, and Tolerability of Sarizotan in Rett Syndrome With Respiratory Symptoms Phase 2/Phase 3
Enrolling by invitation NCT03655223 - Early Check: Expanded Screening in Newborns
Recruiting NCT05932589 - Neurophysiologic Biomarkers in Rett Syndrome
Recruiting NCT04463316 - GROWing Up With Rare GENEtic Syndromes
Completed NCT04776746 - Open-Label Extension Study of Trofinetide for Rett Syndrome Phase 3
Completed NCT04181723 - Study of Trofinetide for the Treatment of Girls and Women With Rett Syndrome (LAVENDERâ„¢) Phase 3
Enrolling by invitation NCT03836300 - Parent and Infant Inter(X)Action Intervention (PIXI) N/A
Completed NCT04514549 - ASSESSING EMERALD AND MC10 BIOSTAMP nPOINT BIOSENSORS FOR RETT SYNDROME
Completed NCT02738281 - Natural History of Rett Syndrome & Related Disorders
Terminated NCT02562820 - An Exploratory Trial of Ketamine for the Treatment of Rett Syndrome Phase 1
Completed NCT05687214 - Osteopathic Manipulative Treatment for Constipation in People With Rett Syndrome N/A
Recruiting NCT06199700 - Esketamine for the Treatment of Rett Syndrome Early Phase 1
Completed NCT03941444 - ANAVEX2-73 Study in Patients With Rett Syndrome Phase 3
Recruiting NCT06346106 - The Diagnostic Experience of Male Rett Syndrome