An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome (ELARA)
Primary Purpose
Angelman Syndrome
Status
Terminated
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
OV101
Sponsored by
About this trial
This is an interventional treatment trial for Angelman Syndrome
Eligibility Criteria
Inclusion Criteria:
Each subject must meet all the following criteria to be enrolled in this study:
- Has completed the OV101-15-001 or OV101-16-001 study up to the EOS.
- Is male or female and 13 to 49 years old (inclusive) at the time of inclusion in the OV101-15-001 or OV101-16-001 study.
- Has a previous diagnosis of AS with molecular confirmation from the OV101-15-001 or OV101-16-001 study.
- Has an 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.
- Provides assent to the protocol (to the extent possible and in accordance with local institutional review board (IRB) 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.
- Can swallow study drug capsules or ingest the contents of study drug capsules after sprinkling the capsule contents onto 1 spoon of applesauce or low-fat yogurt.
- Is currently receiving a stable dose of concomitant medications such as anti-epileptic medication, gabapentin, clonidine, trazadone, melatonin, and special diets for at least 4 weeks prior to Baseline.
- Agrees to remain sexually abstinent from the first day of screening until 30 days after the last dose of study treatment.
- 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 or social media site (eg, Facebook, Instagram, Twitter) until notified that the study is completed.
Exclusion Criteria:
Subjects meeting any of the following criteria will be excluded from the study:
- Discontinued from the OV101-15-001 or OV101-16-001 study due to safety reasons causally related to OV101.
- Has a concomitant disease (eg, 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 poorly controlled seizures defined as > 3 seizures lasting < 3 minutes per week or > 1 seizure episode lasting more than 3 minutes per week or as per medical monitor judgment.
- Has clinically significant clinical laboratory abnormalities or vital signs at the time of screening (eg, alanine aminotransferase or aspartate aminotransferase > 2.5 × upper limit of normal; total bilirubin or creatinine > 1.5 × upper limit of normal). Retesting of clinical laboratory parameters may be allowed after consultation with the medical monitor or designee.
- Current use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone, barbiturates, or ramelteon for sleep within the 4 weeks prior to Day 1. Benzodiazepines administered for situational anxiety related to occasional procedures or events are permitted.
- Has a history of suicidal behavior or is considered by the investigator to be at increased risk of suicide.
- Has any condition or circumstance that, in the opinion of the investigator, makes the subject unsuitable for enrollment.
- Has enrolled in any clinical trial 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.
- Is a family member of the investigator or of study site staff.
Sites / Locations
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
- Ovid Therapeutics Investigative Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
OV101
Arm Description
once daily at bedtime (gaboxadol)
Outcomes
Primary Outcome Measures
Incidence of adverse events in active treatment group
Safety assessments related to the primary study objective of evaluating safety and tolerability of OV101 will include frequency, severity, and causality of AEs (including SAEs and AEs leading to study discontinuation), clinical assessment of suicidality, vital sign measurements, physical examinations, and EEG evaluations.
Secondary Outcome Measures
Full Information
NCT ID
NCT03882918
First Posted
March 18, 2019
Last Updated
January 20, 2022
Sponsor
Ovid Therapeutics Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03882918
Brief Title
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
Acronym
ELARA
Official Title
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
Study Type
Interventional
2. Study Status
Record Verification Date
January 2022
Overall Recruitment Status
Terminated
Why Stopped
Company decision
Study Start Date
January 31, 2019 (Actual)
Primary Completion Date
June 4, 2021 (Actual)
Study Completion Date
June 30, 2021 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ovid Therapeutics Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
5. Study Description
Brief Summary
This open-label study (OV101-18-002) will evaluate the long-term (52 weeks) safety of OV101 in subjects with AS and provide additional OV101 treatment to those subjects who completed Study OV101-15-001 (NCT02996305). Subjects with AS who completed the pharmacokinetic Study OV101-16-001 (NCT03109756) will also be permitted to participate, provided they meet all entry criteria.
Detailed Description
This will be an open-label, long-term safety study for evaluation of further treatment with OV101 in subjects with AS who have completed previous Ovid studies (OV101-15-001 or OV101-16-001). There will be no placebo treatment. As this study will enroll subjects who have completed previous AS studies for different periods of time before entering this study, subjects will be required to complete screening and baseline visits before receiving OV101 under this protocol.The secondary objective of this study is to evaluate the long-term efficacy of OV101 treatment assessed by changes in behavior, sleep, and functioning in individuals with AS.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angelman Syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
125 (Actual)
8. Arms, Groups, and Interventions
Arm Title
OV101
Arm Type
Experimental
Arm Description
once daily at bedtime (gaboxadol)
Intervention Type
Drug
Intervention Name(s)
OV101
Other Intervention Name(s)
gaboxadol
Intervention Description
Each subject will be titrated to his or her maximal tolerated daily dose, up to a maximum daily dose of 15 mg at bedtime.
Primary Outcome Measure Information:
Title
Incidence of adverse events in active treatment group
Description
Safety assessments related to the primary study objective of evaluating safety and tolerability of OV101 will include frequency, severity, and causality of AEs (including SAEs and AEs leading to study discontinuation), clinical assessment of suicidality, vital sign measurements, physical examinations, and EEG evaluations.
Time Frame
Change from baseline to Week 52
10. Eligibility
Sex
All
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
49 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Each subject must meet all the following criteria to be enrolled in this study:
Has completed the OV101-15-001 or OV101-16-001 study up to the EOS.
Is male or female and 13 to 49 years old (inclusive) at the time of inclusion in the OV101-15-001 or OV101-16-001 study.
Has a previous diagnosis of AS with molecular confirmation from the OV101-15-001 or OV101-16-001 study.
Has an 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.
Provides assent to the protocol (to the extent possible and in accordance with local institutional review board (IRB) 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.
Can swallow study drug capsules or ingest the contents of study drug capsules after sprinkling the capsule contents onto 1 spoon of applesauce or low-fat yogurt.
Is currently receiving a stable dose of concomitant medications such as anti-epileptic medication, gabapentin, clonidine, trazadone, melatonin, and special diets for at least 4 weeks prior to Baseline.
Agrees to remain sexually abstinent from the first day of screening until 30 days after the last dose of study treatment.
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 or social media site (eg, Facebook, Instagram, Twitter) until notified that the study is completed.
Exclusion Criteria:
Subjects meeting any of the following criteria will be excluded from the study:
Discontinued from the OV101-15-001 or OV101-16-001 study due to safety reasons causally related to OV101.
Has a concomitant disease (eg, 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 poorly controlled seizures defined as > 3 seizures lasting < 3 minutes per week or > 1 seizure episode lasting more than 3 minutes per week or as per medical monitor judgment.
Has clinically significant clinical laboratory abnormalities or vital signs at the time of screening (eg, alanine aminotransferase or aspartate aminotransferase > 2.5 × upper limit of normal; total bilirubin or creatinine > 1.5 × upper limit of normal). Retesting of clinical laboratory parameters may be allowed after consultation with the medical monitor or designee.
Current use of benzodiazepines, zolpidem, zaleplon, zopiclone, eszopiclone, barbiturates, or ramelteon for sleep within the 4 weeks prior to Day 1. Benzodiazepines administered for situational anxiety related to occasional procedures or events are permitted.
Has a history of suicidal behavior or is considered by the investigator to be at increased risk of suicide.
Has any condition or circumstance that, in the opinion of the investigator, makes the subject unsuitable for enrollment.
Has enrolled in any clinical trial 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.
Is a family member of the investigator or of study site staff.
Facility Information:
Facility Name
Ovid Therapeutics Investigative Site
City
Phoenix
State/Province
Arizona
ZIP/Postal Code
85006
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
San Diego
State/Province
California
ZIP/Postal Code
32123
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Atlanta
State/Province
Georgia
ZIP/Postal Code
30322
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Lexington
State/Province
Massachusetts
ZIP/Postal Code
02421
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Media
State/Province
Pennsylvania
ZIP/Postal Code
19063
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Tacoma
State/Province
Washington
ZIP/Postal Code
98405
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Ramat Gan
ZIP/Postal Code
52621
Country
Israel
12. IPD Sharing Statement
Plan to Share IPD
No
Citations:
PubMed Identifier
33395098
Citation
Heussler HS. Emerging Therapies and challenges for individuals with Angelman syndrome. Curr Opin Psychiatry. 2021 Mar 1;34(2):123-128. doi: 10.1097/YCO.0000000000000674. Erratum In: Curr Opin Psychiatry. 2021 Sep 1;34(5):514.
Results Reference
derived
Learn more about this trial
An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
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