Single Dose Pharmacokinetic (PK) Study

• ClinicalTrials.gov Identifier: NCT03109756
• Recruitment Status: Completed
• First Posted: April 12, 2017
• Last Update Posted: November 30, 2017
• Sponsor: Ovid Therapeutics Inc.
• Information provided by (Responsible Party): Ovid Therapeutics Inc.

Study Description

Brief Summary:

The trial is a Phase 1 Single Dose PK Study in Adolescent Subjects with Fragile X syndrome (FXS) or Angelman syndrome (AS).

• The primary objective of the study is to evaluate the pharmacokinetics (PK) of OV101 following a single 5 mg dose of OV101 in adolescents with FXS or AS.
• Secondary objectives are to determine the safety and tolerability of a single 5 mg dose of OV101 in adolescents with FXS or AS.

Condition or disease	Intervention/treatment	Phase
Angelman Syndrome; Fragile X Syndrome	Drug: OV101	Phase 1

Study Design

• Study Type: Interventional (Clinical Trial)
• Actual Enrollment: 12 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome
• Actual Study Start Date: April 3, 2017
• Actual Primary Completion Date: November 28, 2017
• Actual Study Completion Date: November 28, 2017

Arms and Interventions

Arm	Intervention/treatment
Experimental: Single-dose 5 mg OV101	Drug: OV101; Single-dose 5 mg OV101; Other Name: Gaboxadol

Outcome Measures

Primary Outcome Measures :

1. Measurement of maximum plasma concentration achieved following a single dose of OV101 [ Time Frame: 10 hours ]
   Maximum plasma concentration (Cmax)
2. Measurement of time of maximum plasma concentration following a single dose of OV101 [ Time Frame: 10 hours ]
   Time after administration of drug when maximum plasma concentration is reached (Tmax)
3. Measurement of plasma half-life following a single dose of OV101 [ Time Frame: 10 hours ]
   Plasma half-life (T1/2)
4. Measurement of the area under the plasma concentration versus time curve following a single dose of OV101 [ Time Frame: 10 hours ]
   Area under the curve from 0 to 10 hours (AUC 0-10)
5. Measurement of clearance following a single dose of OV101 [ Time Frame: 10 hours ]
   Total body clearance (CL)
6. Measurement of the apparent volume of distribution following a single dose of OV101 [ Time Frame: 10 hours ]
   Apparent volume of distribution during terminal phase (Vz)

Secondary Outcome Measures :

1. Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG) [ Time Frame: 10 hours ]

Eligibility Criteria

• Ages Eligible for Study: 13 Years to 17 Years (Child)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

Criteria

Inclusion Criteria:

1. Adolescents with confirmed clinical and previous molecular diagnosis of FXS or AS, age between 13 to 17 years, inclusive.
2. Subjects must assent to participation in the study (if appropriate), have a parent or legal guardian/representative capable of providing informed consent on behalf of the subject, and commit to participate in all assessments described in the protocol.
3. Subjects must be receiving a stable dose of concomitant medications
4. Subjects should be able to complete study assessments.
5. Subjects who are non-sterile must agree to either remain completely abstinent or to use two effective contraceptive methods from screening until 7 days after the last dose of study treatment.
6. Subjects must have a parent or other reliable caregiver who agrees to accompany the subject at all study visits and provide information about the subject as required by the study protocol, and ensure compliance with the protocol.

Exclusion Criteria:

1. Inability to swallow a capsule.
2. Poorly controlled seizures
3. Clinically significant abnormal ECG at the time of screening.
4. Positive result on serum or urine pregnancy test for women of child-bearing potential (have experienced menarche) who are not using a dual method of contraception (e.g., condoms plus oral contraceptives), with abstinence being an accepted method.
5. Allergy to gaboxadol or any excipients
6. Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
7. History of suicidal behavior or considered a high suicidal risk by the investigator.
8. Any medical, psychological, social disorder(s), or other conditions - including seizure disorder

Contacts and Locations

Locations

United States, Illinois

Ovid Therapeutics Investigative Site

Chicago, Illinois, United States, 60612

United States, Tennessee

Ovid Therapeutics Investigative Site

Nashville, Tennessee, United States, 37212

Sponsors and Collaborators

Ovid Therapeutics Inc.

More Information

• Responsible Party: Ovid Therapeutics Inc.
• ClinicalTrials.gov Identifier: NCT03109756
• Other Study ID Numbers: OV101-16-001
• First Posted: April 12, 2017
• Last Update Posted: November 30, 2017
• Last Verified: September 2017

Individual Participant Data (IPD) Sharing Statement:

• Plan to Share IPD: No

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

Additional relevant MeSH terms:

Fragile X Syndrome; Angelman Syndrome; Syndrome; Disease; Pathologic Processes; Mental Retardation, X-Linked; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System; Movement Disorders; Central Nervous System Diseases; Abnormalities, Multiple; 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