Angelman SyndromePhase 1NCT03109756
Single Dose Pharmacokinetic (PK) Study
A Phase 1 study testing OV101 in people with Angelman Syndrome and Fragile X Syndrome, run by Ovid Therapeutics Inc. at 2 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 13 to 17 years
- Conditions the study is for
- Angelman Syndrome, Fragile X Syndrome
- What is being tested?
- OV101
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 2 sitesUnited States
- Study dates
- 3 Apr 2017to 28 Nov 2017
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 13 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Ovid Therapeutics Investigative Site
Chicago, Illinois, 60612
Ovid Therapeutics Investigative Site
Nashville, Tennessee, 37212
About this study
Official title: A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome
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
- Being tested
- OV101
- Medical subject headings
- Angelman Syndrome, Fragile X Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OV101Drug
Also called Gaboxadol
Single-dose 5 mg OV101
| Study group | Role | What they receive |
|---|---|---|
| Single-dose 5 mg OV101 | Experimental | Not described Receives: Drug: OV101 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of maximum plasma concentration achieved following a single dose of OV101
Maximum plasma concentration (Cmax)
Measured: 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)
Measured: 10 hours
Measurement of plasma half-life following a single dose of OV101
Plasma half-life (T1/2)
Measured: 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)
Measured: 10 hours
Measurement of clearance following a single dose of OV101
Total body clearance (CL)
Measured: 10 hours
Measurement of the apparent volume of distribution following a single dose of OV101
Apparent volume of distribution during terminal phase (Vz)
Measured: 10 hours
Secondary outcomes (1)
Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)
Measured: 10 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted12 Apr 2017
- Study start3 Apr 2017
- Primary completion28 Nov 2017
- Study completion28 Nov 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Angelman SyndromeStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ovid Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03109756
- Sponsor's protocol ID
- OV101-16-001
- Status last verified
- Sep 2017
- First submitted
- 6 Apr 2017
- Last updated
- 30 Nov 2017
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Nov 2017. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Single Dose PK Study in Adolescent Subjects With Fragile X Syndrome or Angelman Syndrome
- ClinicalTrials.gov ID
- NCT03109756
- Acronym / sponsor ID
- OV101-16-001
- Conditions
- Angelman Syndrome, Fragile X Syndrome
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 12 participants (actual)
- Lead sponsor
- Ovid Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 3 Apr 2017 (actual)
- Primary completion
- 28 Nov 2017 (actual)
- Study completion
- 28 Nov 2017 (actual)
- First posted
- 12 Apr 2017 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Single-dose 5 mg OV101 | Experimental | Drug: OV101 |
Who may qualify
- Ages
- 13 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (6 listed)
- 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.
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OV101Also: GaboxadolSingle-dose 5 mg OV101 | Drug | Single-dose 5 mg OV101 |
Primary outcome
Measurement of maximum plasma concentration achieved following a single dose of OV101
Time frame: 10 hours
Maximum plasma concentration (Cmax)
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)
Measurement of plasma half-life following a single dose of OV101
Time frame: 10 hours
Plasma half-life (T1/2)
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)
Measurement of clearance following a single dose of OV101
Time frame: 10 hours
Total body clearance (CL)
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)
1 secondary outcome
Safety parameters, adverse events, absolute values and changes over time of hematology, clinical chemistry, urinalysis, vital signs, electrocardiogram (ECG)
Time frame: 10 hours
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Nov 2017.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing OV101 in people with Angelman Syndrome and Fragile X Syndrome, run by Ovid Therapeutics Inc. at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 30 Nov 2017. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurement of maximum plasma concentration achieved following a single dose of OV101 (time frame: 10 hours). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 30 Nov 2017.