Clinical trial NCT03109756: 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
Overall status: COMPLETED
Phase: PHASE1
Conditions: Angelman Syndrome
Fragile X Syndrome
Lead sponsor: Ovid Therapeutics Inc.
Sponsor class: INDUSTRY
First posted: 2017-04-12
Enrollment: 12
Sex: ALL
Minimum age (years): 13
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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

Primary outcomes:
measure: Measurement of maximum plasma concentration achieved following a single dose of OV101
timeFrame: 10 hours
measure: Measurement of time of maximum plasma concentration following a single dose of OV101
timeFrame: 10 hours
measure: Measurement of plasma half-life following a single dose of OV101
timeFrame: 10 hours
measure: Measurement of the area under the plasma concentration versus time curve following a single dose of OV101
timeFrame: 10 hours
measure: Measurement of clearance following a single dose of OV101
timeFrame: 10 hours
measure: Measurement of the apparent volume of distribution following a single dose of OV101
timeFrame: 10 hours
Secondary outcomes:
measure: Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)
timeFrame: 10 hours
Other outcomes:
Locations:
city: Chicago
country: United States
facility: Ovid Therapeutics Investigative Site
state: Illinois
city: Nashville
country: United States
facility: Ovid Therapeutics Investigative Site
state: Tennessee
Study officials:
References: