Single Dose Pharmacokinetic (PK) Study

Fragile X Syndrome Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03109756
• Other study ID #: OV101-16-001
• Status: Completed
• Phase: Phase 1
• First received: April 6, 2017
• Last updated: November 29, 2017
• Start date: April 3, 2017
• Est. completion date: November 28, 2017

Study information

• Verified date: September 2017
• Source: Ovid Therapeutics Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 12
• Est. completion date: November 28, 2017
• Est. primary completion date: November 28, 2017
• Accepts healthy volunteers: No
• Gender: All
• Age group: 13 Years to 17 Years

Eligibility

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

Related Conditions & MeSH terms

• Angelman Syndrome
• Fragile X Syndrome
• Syndrome

Intervention

Drug:
• OV101
Single-dose 5 mg OV101

Locations

Country	Name	City	State
United States	Ovid Therapeutics Investigative Site	Chicago	Illinois
United States	Ovid Therapeutics Investigative Site	Nashville	Tennessee

Sponsors (1)

Lead Sponsor	Collaborator
Ovid Therapeutics Inc.

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Measurement of maximum plasma concentration achieved following a single dose of OV101	Maximum plasma concentration (Cmax)	10 hours
Primary	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
Primary	Measurement of plasma half-life following a single dose of OV101	Plasma half-life (T1/2)	10 hours
Primary	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
Primary	Measurement of clearance following a single dose of OV101	Total body clearance (CL)	10 hours
Primary	Measurement of the apparent volume of distribution following a single dose of OV101	Apparent volume of distribution during terminal phase (Vz)	10 hours
Secondary	Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)		10 hours