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Single Dose Pharmacokinetic (PK) Study

Primary Purpose

Angelman Syndrome, Fragile X Syndrome

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
OV101
Sponsored by
Ovid Therapeutics Inc.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Angelman Syndrome

Eligibility Criteria

13 Years - 17 Years (Child)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Ovid Therapeutics Investigative Site
  • Ovid Therapeutics Investigative Site

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Single-dose 5 mg OV101

Arm Description

Outcomes

Primary Outcome Measures

Measurement of maximum plasma concentration achieved following a single dose of OV101
Maximum plasma concentration (Cmax)
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)
Measurement of plasma half-life following a single dose of OV101
Plasma half-life (T1/2)
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)
Measurement of clearance following a single dose of OV101
Total body clearance (CL)
Measurement of the apparent volume of distribution following a single dose of OV101
Apparent volume of distribution during terminal phase (Vz)

Secondary Outcome Measures

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

Full Information

First Posted
April 6, 2017
Last Updated
November 29, 2017
Sponsor
Ovid Therapeutics Inc.
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1. Study Identification

Unique Protocol Identification Number
NCT03109756
Brief Title
Single Dose Pharmacokinetic (PK) Study
Official Title
A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome
Study Type
Interventional

2. Study Status

Record Verification Date
September 2017
Overall Recruitment Status
Completed
Study Start Date
April 3, 2017 (Actual)
Primary Completion Date
November 28, 2017 (Actual)
Study Completion Date
November 28, 2017 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Ovid Therapeutics Inc.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Angelman Syndrome, Fragile X Syndrome

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
12 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Single-dose 5 mg OV101
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
OV101
Other Intervention Name(s)
Gaboxadol
Intervention Description
Single-dose 5 mg OV101
Primary Outcome Measure Information:
Title
Measurement of maximum plasma concentration achieved following a single dose of OV101
Description
Maximum plasma concentration (Cmax)
Time Frame
10 hours
Title
Measurement of time of maximum plasma concentration following a single dose of OV101
Description
Time after administration of drug when maximum plasma concentration is reached (Tmax)
Time Frame
10 hours
Title
Measurement of plasma half-life following a single dose of OV101
Description
Plasma half-life (T1/2)
Time Frame
10 hours
Title
Measurement of the area under the plasma concentration versus time curve following a single dose of OV101
Description
Area under the curve from 0 to 10 hours (AUC 0-10)
Time Frame
10 hours
Title
Measurement of clearance following a single dose of OV101
Description
Total body clearance (CL)
Time Frame
10 hours
Title
Measurement of the apparent volume of distribution following a single dose of OV101
Description
Apparent volume of distribution during terminal phase (Vz)
Time Frame
10 hours
Secondary Outcome Measure Information:
Title
Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)
Time Frame
10 hours

10. Eligibility

Sex
All
Minimum Age & Unit of Time
13 Years
Maximum Age & Unit of Time
17 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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. Exclusion Criteria: 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
Facility Information:
Facility Name
Ovid Therapeutics Investigative Site
City
Chicago
State/Province
Illinois
ZIP/Postal Code
60612
Country
United States
Facility Name
Ovid Therapeutics Investigative Site
City
Nashville
State/Province
Tennessee
ZIP/Postal Code
37212
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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Single Dose Pharmacokinetic (PK) Study

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