Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Phase 3 Study to Evaluate the Efficacy and Safety of OV101 in Pediatric Individuals With Angelman Syndrome (NEPTUNE)

Phase 3

Completed

Conditions: Angelman Syndroom
10010335

Registration Number: NL-OMON49584

Lead Sponsor: Ovid Therapeutics Inc.

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 12

Inclusion Criteria

1. Is male or female and 2 to 12 years old (inclusive) at the time of informed
consent.
2. Has a diagnosis of AS with molecular confirmation
3. Has a CGI-S-AS score of 3 or more.
4. Meets the following age-appropriate body weight criterion:
a) Subjects 2 to 3 years old must have a minimum body weight of 9 kg.
b) Subjects 4 years and older must be between 17 kg and 64 kg (inclusive).
5. Has a legally acceptable representative (LAR)/caregiver capable of providing
written informed consent and able to attend all scheduled study visits, oversee
the administration of study drug, and provide feedback regarding the subject*s
symptoms and performance as described in the protocol.
6. Provides assent to the protocol. Subjects providing assent must do so at the
same visit as LAR/caregiver written informed consent is provided.
7. Can swallow study drug capsules with water or ingest the contents of study
drug capsules after sprinkling the contents of each capsule onto up to 1
teaspoon of low fat semiliquid food.
8. If a subject is currently receiving a regimen of concomitant medications
such as antiepileptic medication, gabapentin, clonidine, trazodone, melatonin,
or a special diet regimen, that subject*s regimen is stable for at least 4
weeks before Day 1 (first day of study drug administration) and will be
maintained throughout the duration of the study (in the judgment of the
investigator).
9. If a subject is a sibling in a family with multiple children diagnosed with
AS, then only one of the siblings may enroll in study. The eldest eligible
subject should be enrolled (investigator discretion may be used to enroll a
younger sibling instead).
10. Has LAR(s)/caregiver(s) who agree not to post any of the subject*s personal
medical data or information related to the study on any website, message
board(s), online support group(s), or social media site (eg, Facebook,
Instagram, Twitter, etc.) until notified that the study is completed.
11. Female subjects who are of child-bearing potential (defined as having
experienced their first menarche) must agree to use either a highly effective
or acceptable form of birth control during the study and for 30 days following
the last dose of the study.

Exclusion Criteria

1. Has a circumstance or concomitant disease (eg, gastrointestinal, renal,
hepatic, endocrine, respiratory, or cardiovascular system disease), 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 the opinion of the investigator.
2. Has poorly controlled seizures defined as any of the following:
a. Weekly seizures of any frequency with a duration more than 3 minutes.
b. Weekly seizures occurring more than 3 times per week, each with a duration
of less than 3 minutes.
c. Investigator assessment.
3. Has any of the following laboratory abnormalities: total bilirubin >1.5 ×
upper limit of normal (ULN), unless known Gilbert*s syndrome; alanine
aminotransferase or aspartate aminotransferase >2.5 × ULN; serum creatinine
>1.2 × ULN; absolute neutrophil count <1.5 × 109/L; platelets <80 × 109/L;
hemoglobin <80 g/L; or thyroid-stimulating-hormone >1.25 × ULN or <0.8 × lower
limit of normal. Retesting of clinical laboratory parameters may be allowed
after consultation with the medical monitor or designee.
4. Use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone,
barbiturates, or ramelteon for sleep, or minocycline or levodopa within the 4
weeks prior to Day 1 or during the study. Benzodiazepines administered as
needed for situational anxiety related to occasional procedures or events are
permitted, and benzodiazepines are also permitted for seizure control.
5. Cannot tolerate wearing the actigraph for at least 7 consecutive days
(including 2 consecutive weekend days) during the 28-day screening period of
the study, after 3 attempts.
6. Is at risk of harming self and/or others (based on investigator assessment).
7. Has enrolled in any clinical study or used any investigational agent or
device, or has participated in any investigational procedure, within the 30
days before screening or does so concurrently with this study.
8. Is allergic to OV101 or any excipients of study drug.
9. The subject or LAR/caregiver is unable to comply with study requirements
(based on investigator assessment).
10. Is a family member of the investigator and/or study site staff.

Study & Design

Study Type: Interventional

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Efficacy assessments will include the CGI I-AS; the CGI S-AS; the VABS 3; the<br /><br>CSHQ; the Peds QL (Cerebral Palsy, Multidimensional Fatigue, and Family Impact<br /><br>modules); actigraphy; and sleep diary. </p><br>

Secondary Outcome Measures