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An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome (ELARA)

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ClinicalTrials.gov Identifier: NCT03882918
Recruitment Status : Recruiting
First Posted : March 20, 2019
Last Update Posted : March 28, 2019
Sponsor:
Information provided by (Responsible Party):
Ovid Therapeutics Inc.

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.

Condition or disease Intervention/treatment Phase
Angelman Syndrome Drug: OV101 Phase 2

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.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 82 participants
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: An Open-Label Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of OV101 in Individuals With Angelman Syndrome
Actual Study Start Date : February 25, 2019
Estimated Primary Completion Date : July 2020
Estimated Study Completion Date : September 2020

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: OV101
once daily at bedtime (gaboxadol)
Drug: OV101
Each subject will be titrated to his or her maximal tolerated daily dose, up to a maximum daily dose of 15 mg at bedtime.
Other Name: gaboxadol




Primary Outcome Measures :
  1. Incidence of adverse events in active treatment group [ Time Frame: Change from baseline to Week 52 ]
    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.



Information from the National Library of Medicine

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Ages Eligible for Study:   13 Years to 49 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

Each subject must meet all the following criteria to be enrolled in this study:

  1. Has completed the OV101-15-001 or OV101-16-001 study up to the EOS.
  2. 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.
  3. Has a previous diagnosis of AS with molecular confirmation from the OV101-15-001 or OV101-16-001 study.
  4. 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.
  5. 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.
  6. 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.
  7. 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.
  8. Agrees to remain sexually abstinent from the first day of screening until 30 days after the last dose of study treatment.
  9. 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:

  1. Discontinued from the OV101-15-001 or OV101-16-001 study due to safety reasons causally related to OV101.
  2. 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.
  3. 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.
  4. 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.
  5. 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.
  6. Has a history of suicidal behavior or is considered by the investigator to be at increased risk of suicide.
  7. Has any condition or circumstance that, in the opinion of the investigator, makes the subject unsuitable for enrollment.
  8. 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.
  9. Is a family member of the investigator or of study site staff.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03882918


Locations
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United States, Arizona
Ovid Therapeutics Investigative Site Recruiting
Phoenix, Arizona, United States, 85006
Contact: Melmed    480-443-4018    raun.melmed@melmedcenter.com   
United States, Pennsylvania
Ovid Therapeutics Investigative Site Recruiting
Media, Pennsylvania, United States, 19063
Contact: Hatti    610-891-9699    shatti@suburbanresearch.com   
Sponsors and Collaborators
Ovid Therapeutics Inc.

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Responsible Party: Ovid Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT03882918     History of Changes
Other Study ID Numbers: OV101-18-002
First Posted: March 20, 2019    Key Record Dates
Last Update Posted: March 28, 2019
Last Verified: March 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Angelman Syndrome
Syndrome
Disease
Pathologic Processes
Movement Disorders
Central Nervous System Diseases
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn
Gaboxadol
Analgesics
Sensory System Agents
Peripheral Nervous System Agents
Physiological Effects of Drugs
Anticonvulsants
GABA Agonists
GABA Agents
Neurotransmitter Agents
Molecular Mechanisms of Pharmacological Action