Low-Dose Ketamine in Children With ADNP Syndrome | NCT04388774

NCT04388774

Protocol Section

Identification Module

Results Section

Participant Flow Module

Annotation Section

No data available

No data is available for this block.

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module

Status Module

Description Module

Label	Type	Description	Intervention Names
Ketamine	Experimental	Total dose administration or 0.5 mg/kg of ketamine	Drug: Ketamine

Name	Type	Description	Arm Group Labels	Other Names
Ketamine	Drug	A single 40-minute intravenous infusion	Ketamine

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Number of Participants With an Adverse Event	Number of Participants with an adverse event as defined by the Systematic Longitudinal Assessment of Adverse Events (SLAES) which is a comprehensive form that assesses medical and behavioral conditions that were present at screening and/or baseline. Conditions are considered treatment emergent if their severity increased significantly after the participant had taken at least one dose of the study treatment. Treatment emergent adverse events will be tracked considered in the adverse event safety analysis. Severity of adverse events are categorized as mild, moderate, severe, life-threatening, or resulting in death and the treating physician indicates if the adverse event was related or unrelated to study drug.	Week 4

Secondary Outcomes

Measure	Description	Time Frame
Aberrant Behavior Checklist	Changes in scale at week 1 compared to baseline. Aberrant Behavior Checklist is a behavior rating scale for the assessment of treatment effects. Each item is scored as 0 (never a problem), 1 (slight problem), 2 (moderately serious problem), or 3 (severe problem). Subscales from 0-45 for Irritability; 0-48 for Social Withdrawal and Hyperactivity; from 0-21 for Stereotypy; and 0-12 for In Speech , with total scale from 0 to 48, with higher score indicating worse health outcomes.

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

5 to 12 years old (inclusive) at the time of informed consent;
Has a diagnosis of ADNP syndrome, confirmed by genetic testing prior to subject randomization;
Has a Clinical Global Impression-Severity score of 4 (moderately ill) or greater at screening;
Any concomitant medication, including anti-epileptic and/or behavioral medications, supplements, and special diets, must be at a stable dose for at least 4 weeks before;
Has an English-speaking caregiver capable of providing informed consent and able to attend all scheduled study visits, oversee the administration of study drug, and provide feedback regarding the subject's behavior and other symptoms as described in the protocol;
Provide assent to the protocol (when applicable);
Has a caregiver who will agree not to post any of the subject's personal medical data related to the study or information related to the study on any website or social media site (e.g., Facebook and Twitter) until they have been notified that the study is completed.
Age-specific blood pressure parameters for inclusion in the study will be based on established guidelines.

Exclusion Criteria:

Has a concomitant disease (e.g., gastrointestinal, renal, hepatic, endocrine, respiratory, or cardiovascular system disease) or condition or any clinically significant finding at screening that could interfere with the conduct of the study or that would pose an unacceptable risk to the subject in this study;
Has clinically significant lab abnormalities or vital signs at the time of screening (e.g., alanine aminotransferase or aspartate aminotransferase >2.5 × upper limit of normal; total bilirubin or creatinine >1.5 × upper limit of normal). Re-testing of safety labs is allowed;
Hypertension that is not well controlled (systolic BP >130-140 mm Hg or diastolic BP >85-95 mm Hg depending on age);
A blood pressure reading over 160/90 or two separate readings over 140/90 at screening or baseline visits;
Thyroid impairment, as reflected by a TSH > 4.2 mU/L;
Cardiac disease, as reflected by an EKG that is abnormal and of concern for cardiac disease;
Has had changes in his/her medication regimen within the previous month;
Has a history of uncontrollable seizure disorder or seizure episodes within 1 month of screening;
Has a history of suicidal behavior or considered by the investigator to be at high risk of suicide;
Has a current or past history of psychotic symptoms;
Has enrolled in any clinical trial or used of any investigational agent, device, and/or investigational procedure within the 30 days before screening or does so concurrently with this study.

Name	Affiliation	Role
Alexander Kolevzon, MD	Icahn School of Medicine at Mount Sinai	Principal Investigator

Facility	Status	City	State	ZIP	Country	Contacts
Icahn School of Medicine at Mount Sinai		New York	New York	10029	United States

ID	Title	Description
FG000	Ketamine	Single-dose (0.5 mg/kg) infused intravenously over 40 minutes

ID	Title	Description
BG000	Ketamine	Single-dose (0.5 mg/kg) infused intravenously over 40 minutes

Units	Counts
Participants	BG000

Title	Description	Population Description	Parameter Type	Dispersion Type	Unit of Measure	Calculate Percentage	Denominator Units Selected	Denominators	Classes
Age, Continuous			Mean

Outcome Measures Module

Outcome Measures

Type

Title

Description

Population Description

Reporting Status

Anticipated Posting Date

Parameter Type

Dispersion Type

Unit of Measure

Calculate Percentage

Time Frame

Units Analyzed

Denominator Units Selected

Arm/Group Information

Denominators

Classes

Analyses

Primary

Number of Participants With an Adverse Event

Number of Participants with an adverse event as defined by the Systematic Longitudinal Assessment of Adverse Events (SLAES) which is a comprehensive form that assesses medical and behavioral conditions that were present at screening and/or baseline. Conditions are considered treatment emergent if their severity increased significantly after the participant had taken at least one dose of the study treatment. Treatment emergent adverse events will be tracked considered in the adverse event safety analysis. Severity of adverse events are categorized as mild, moderate, severe, life-threatening, or resulting in death and the treating physician indicates if the adverse event was related or unrelated to study drug.

Posted

Count of Participants

Participants

Week 4

ID	Title	Description
OG000	Ketamine	Single-dose (0.5 mg/kg) infused intravenously over 40 minutes

Adverse Events Module

Arm/Groups

ID	Title	Description	Deaths (Affected)	Deaths (At Risk)	Serious Events (Affected)	Serious Events (At Risk)	Other Events (Affected)	Other Events (At Risk)
EG000	Ketamine	Single-dose (0.5 mg/kg) infused intravenously over 40 minutes	0

ID	Term
D025063	Chromosome Disorders
D002869	Chromosome Aberrations

ID	Term
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities
D030342	Genetic Diseases, Inborn
D010335	Pathologic Processes

ID	Term
D003510	Cyclohexanes
D003516	Cycloparaffins
D006840	Hydrocarbons, Alicyclic
D006844	Hydrocarbons, Cyclic