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Single Dose Pharmacokinetic (PK) Study

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

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 Type : Interventional  (Clinical Trial)
Actual Enrollment : 12 participants
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


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




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 ]


Information from the National Library of Medicine

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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

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): NCT03109756


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.

Responsible Party: Ovid Therapeutics Inc.
ClinicalTrials.gov Identifier: NCT03109756     History of Changes
Other Study ID Numbers: OV101-16-001
First Posted: April 12, 2017    Key Record Dates
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:
Syndrome
Fragile X Syndrome
Angelman 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