Single Dose Pharmacokinetic (PK) Study

A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome

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.

Study Overview

• Status: Completed
• Conditions: Fragile X Syndrome; Angelman Syndrome
• Intervention / Treatment: Drug: OV101

• Study Type: Interventional
• Enrollment (Actual): 12
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Illinois
    • Chicago, Illinois, United States, 60612
      • Ovid Therapeutics Investigative Site
  • Tennessee
    • Nashville, Tennessee, United States, 37212
      • Ovid Therapeutics Investigative Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 13 years to 17 years (CHILD)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: TREATMENT
• Allocation: NA
• Interventional Model: SINGLE_GROUP
• Masking: NONE

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
EXPERIMENTAL: Single-dose 5 mg OV101	Drug: OV101; Single-dose 5 mg OV101; Other Names: Gaboxadol

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure	Time Frame
Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)	10 hours

Collaborators and Investigators

• Sponsor: Ovid Therapeutics Inc.

Study record dates

Study Major Dates

• Study Start (ACTUAL): April 3, 2017
• Primary Completion (ACTUAL): November 28, 2017
• Study Completion (ACTUAL): November 28, 2017

Study Registration Dates

• First Submitted: April 6, 2017
• First Submitted That Met QC Criteria: April 10, 2017
• First Posted (ACTUAL): April 12, 2017

Study Record Updates

• Last Update Posted (ACTUAL): November 30, 2017
• Last Update Submitted That Met QC Criteria: November 29, 2017
• Last Verified: September 1, 2017

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Central Nervous System Diseases; Nervous System Diseases; Neurologic Manifestations; Neurobehavioral Manifestations; Disease; Congenital Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Movement Disorders; Mental Retardation, X-Linked; Intellectual Disability; Heredodegenerative Disorders, Nervous System; Abnormalities, Multiple; Chromosome Disorders; Sex Chromosome Disorders; Syndrome; Fragile X Syndrome; Angelman Syndrome; Physiological Effects of Drugs; Neurotransmitter Agents; Molecular Mechanisms of Pharmacological Action; Peripheral Nervous System Agents; Analgesics; Sensory System Agents; GABA Agents; Anticonvulsants; GABA Agonists; Gaboxadol
• Other Study ID Numbers: OV101-16-001

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No