An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals with Angelman Syndrome (ELARA)
- Conditions
- Angelman SyndromeMedDRA version: 20.0Level: PTClassification code 10049004Term: Angelman's syndromeSystem Organ Class: 10010331 - Congenital, familial and genetic disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2019-004478-24-NL
- Lead Sponsor
- Ovid Therapeutics Inc.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- All
- Target Recruitment
- 200
Study enrollment criteria:
• Has completed the OV101-15-001 or OV101-16-001 study up to the end of study (EOS), or
• Has completed the OV101-19-001 (EudraCT # 2019-002907-17) study up to the end of treatment (EOT), or
• Is a sibling of a subject with AS who has completed OV101-15-001 , OV101 16 001, or OV101-19-001 (EudraCT # 2019-002907-17)
1. Has a previous diagnosis of AS with molecular confirmation.
2. Is at least 2 years old and has a body weight of at least 9 kg.
3. 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.
4. 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.
5. 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.
6. 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).
7. 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.
8. 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. Highly effective contraceptive methods are as follows:
• Combined (estrogen- and progestogen-containing) hormonal contraception associated with inhibition of ovulation: Oral, Intravaginal, Transdermal
• Progestogen-only hormonal contraception associated with inhibition of ovulation: Oral, Injectable, Implantable, Intrauterine device
• Intrauterine hormone-releasing system
• Bilateral tubal occlusion
• Vasectomized partner
• Sexual abstinence
Acceptable birth control methods that result in a failure rate of more than 1% per year include:
• Progestogen-only oral hormonal contraception, where inhibition of ovulation is not the primary mode of action
• Male or female condom with or without spermicide*
• Cap, diaphragm, or sponge with spermicide*
*A combination of male condom with either cap, diaphragm, or sponge with spermicide (double barrier methods) are also considered acceptable, but not highly effective, birth control methods.
Are the trial subjects under 18? yes
Number of subjects for this age range: 200
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 10
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Subjects meeting any of the following criteria will be excluded from the study:
1. Discontinued from the OV101-15-001, OV101-16-001, or OV101-19-001 (EudraCT # 2019-002907-17) 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 clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method