Low-Dose Ketamine in Children With ADNP Syndrome (NCT04388774) | Clinical Trial Compass
CompletedPhase 1/2
Low-Dose Ketamine in Children With ADNP Syndrome
United States10 participantsStarted 2020-08-19
Plain-language summary
This is a Phase 2A, single dose, open-label study to evaluate the safety, tolerability, and efficacy of a low-dose, 40-minute infusion into the veins (intravenous infusion or "IV") of ketamine in children with ADNP syndrome (Activity-Dependent Neuroprotective Protein). The study team will enroll 10 participants, ages 5 to 12, at Mount Sinai. The study participation is expected to last 4 weeks and will include 5 scheduled clinic visits in order to complete safety monitoring, clinical assessments, and biomarker collection. At the conclusion of this study, the study team expects to demonstrate the safety and tolerability of low-dose ketamine in children with ADNP syndrome. Additionally, the study team anticipates identifying meaningful signals of efficacy in clinical outcome measures using RNA and DNA sequencing to analyze ADNP protein expression and DNA methylation profiles, a natural process by which methyl groups are added to the DNA to change its activity, in order to assess sensitivity to change with low-dose ketamine treatment and inform future phase 3 studies. Ketamine is not currently approved by the Food and Drug Administration to treat this syndrome, but it is approved for use in children in other situations, for example in anesthesia.
Who can participate
Age range5 Years – 12 Years
SexALL
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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 s…