An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA)
- Conditions
- Angelman Syndroom10010335
- Registration Number
- NL-OMON49727
- Lead Sponsor
- Ovid Therapeutics Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 4
1. Ovid study enrollment criteria:
• Has completed the OV101-19-001 study up to the end of treatment (EOT), or
• Is a sibling of a subject with AS who has completed OV101-19-001.
2. Has a previous diagnosis of AS with molecular confirmation.
3. Is at least 2 years old and has a body weight of at least 9 kg.
4. Has a legally acceptable representative (LAR)/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
symptoms and performance as described in the protocol.
5. Provides assent to the protocol (to the extent possible and in accordance
with local institutional review board and regulatory requirements) and has an
LAR/caregiver who will provide written informed consent. Subjects providing
assent must do so at the same visit as LAR/caregiver written informed consent
is provided.
6. Can swallow study drug capsules with water or ingest the contents of study
drug capsules after sprinkling the capsule contents onto up to 1 teaspoon of
low-fat semiliquid food.
7. If a subject is currently receiving a regimen of concomitant medications
such as antiepileptic medication, gabapentin, clonidine, trazodone, melatonin,
and/or a special dietary 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).
8. Has LAR/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,
online support group, or social media site (eg, Facebook, Instagram, Twitter)
until notified that the study is completed.
9. Female subjects who are of childbearing 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 drug.
1. Discontinued from the OV101-19-001 study due to safety reasons causally
related to OV101.
2. Has a circumstance, condition, concomitant disease (eg, gastrointestinal,
renal, hepatic, endocrine, respiratory, or cardiovascular system disease), or
any clinically significant finding that could interfere with the conduct of the
study or that would pose an unacceptable risk to the subject in this study.
3. Has poorly controlled seizures defined as any of the following:
• Weekly seizures of any frequency with a duration of more than 3 minutes each
• Weekly seizures occurring more than 3 times per week, each with a duration of
less than 3 minutes
• Investigator assessment
4. 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.
5. 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 or regularly scheduled for indications other than insomnia and
benzodiazepines for seizure control are permitted.
6. Is at risk of harming self and/or others (based on investigator assessment).
7. With the exception of an Ovid study of OV101, has enrolled in any clinical
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).
Study & Design
- Study Type
- Interventional
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>Analysis sets include the enrolled analysis set (subjects enrolled in the<br /><br>extension study), the safety analysis set (all subjects who receive at least 1<br /><br>dose of study drug), and the full-analysis set (all subjects who receive at<br /><br>least 1 dose of study drug and have at least 1 postbaseline efficacy<br /><br>assessment).<br /><br>Descriptive statistics (number of subjects, mean, SD, median, minimum, and<br /><br>maximum) will be presented for continuous variables and frequency and<br /><br>percentage will be presented for categorical and ordinal variables. If there<br /><br>are missing values, the number missing will be presented, but without a<br /><br>percentage. All data collected will be included in by-subject data listings.<br /><br>Two sided 95% CIs will be provided where appropriate. Graphical displays will<br /><br>be utilized to investigate trends over time overall and by relevant subgroups<br /><br>as needed. </p><br>
- Secondary Outcome Measures
Name Time Method