Single Dose Pharmacokinetic (PK) Study

NCT03109756 | Completed Phase 1 FDA Drug

• 1 other identifier: OV101-16-001
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 2

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.

Conditions

Angelman Syndrome; Fragile X Syndrome

Outcome Measures

Primary Outcomes (6)

• 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 Outcomes (1)

• Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)

  10 hours

Study Arms (1)

Single-dose 5 mg OV101

EXPERIMENTAL

Drug: OV101

Interventions

OV101 DRUG

Single-dose 5 mg OV101

Also known as: Gaboxadol

Single-dose 5 mg OV101

Eligibility Criteria

• Age: 13 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• Adolescents with confirmed clinical and previous molecular diagnosis of FXS or AS, age between 13 to 17 years, inclusive.
• 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.
• Subjects must be receiving a stable dose of concomitant medications
• Subjects should be able to complete study assessments.
• 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.
• 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.

You may not qualify if:

• Inability to swallow a capsule.
• Poorly controlled seizures
• Clinically significant abnormal ECG at the time of screening.
• 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.
• Allergy to gaboxadol or any excipients
• Concomitant cardiovascular, respiratory, liver, renal, or hematologic diseases of a degree that would limit participation in the study
• History of suicidal behavior or considered a high suicidal risk by the investigator.
• Any medical, psychological, social disorder(s), or other conditions - including seizure disorder

Sponsors & Collaborators

• Healx AI: lead

Study Sites (2)

Ovid Therapeutics Investigative Site

Chicago, Illinois, 60612, United States

Location

Ovid Therapeutics Investigative Site

Nashville, Tennessee, 37212, United States

Location

MeSH Terms

Conditions

Angelman Syndrome; Fragile X Syndrome

Interventions

gaboxadol

Condition Hierarchy (Ancestors)

Movement Disorders; Central Nervous System Diseases; Nervous System Diseases; Abnormalities, Multiple; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Chromosome Disorders; Genetic Diseases, Inborn; Imprinting Disorders; X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Sex Chromosome Disorders; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 6, 2017
• First Posted: April 12, 2017
• Study Start: April 3, 2017
• Primary Completion: November 28, 2017
• Study Completion: November 28, 2017
• Last Updated: November 30, 2017

Record last verified: 2017-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)