Prader-Willi SyndromePhase 2NCT02893618
A 5 Treatment Period Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR)
A Phase 2 study of Prader-Willi Syndrome, run by Essentialis, Inc..
Trial summary
Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food. Read more about this study
- Who is it for?
- Ages 18 to 65 yearsHealthy volunteers can take part
- Conditions the study is for
- Prader-Willi Syndrome
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 32 participants
- Where
- No sites listed yet
- Study dates
- Jul 2017to Dec 2017
- Phase
- Phase 2
- 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 18 to 65 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Ability to follow verbal and written instructions
- Informed consent form signed by the subject
- Completed screening within 7 days prior to dosing
- BMI between 18.5 and 35 kg/m2
- Generally healthy
- fasting glucose less than or equal to 100 mg/dL
- HbA1c less than or equal to 6%
Who cannot join
- Pregnancy or breast feeding
- absence of contraception
- administration of investigational drug within 1 month prior to screening
- anticipated requirement for prohibited medication (systemic corticosteroids or anti-diabetic medications)
- allergic reaction to or significant intolerance of diazoxide, thiazides or sulfonamides
- known type 1 or type 2 diabetes mellitus
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- congestive heart failure
- gastric bypass surgery
- history of drug or alcohol abuse
About this study
Official title: A 5 Treatment Period Crossover Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR)
Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food.
Read the detailed description
Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food, with a 10 Day washout period between treatments. There will be 8 treatment sequences with 4 subjects randomized to each.
- Conditions
- Prader-Willi Syndrome
- Being tested
- Diazoxide choline controlled-release tablet
- Medical subject headings
- Prader-Willi Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Diazoxide choline controlled-release tabletDrug
Also called DCCR
QD tablet formulation of choline salt of diazoxide
| Study group | Role | What they receive |
|---|---|---|
| DCCR 75 mg fasted | Experimental | Administered a single 75 mg dose of DCCR after an overnight fast followed by 4 hours of fasting Receives: Drug: Diazoxide choline controlled-release tablet |
| DCCR 150 mg fasted | Experimental | Administered a single 150 mg dose of DCCR after an overnight fast followed by 4 hours of fasting Receives: Drug: Diazoxide choline controlled-release tablet |
| DCCR 300 mg fasted | Experimental | Administered a single 300 mg dose of DCCR after an overnight fast followed by 4 hours of fasting Receives: Drug: Diazoxide choline controlled-release tablet |
| DCCR 450 mg fasted | Experimental | Administered a single 450 mg dose of DCCR after an overnight fast followed by 4 hours of fasting Receives: Drug: Diazoxide choline controlled-release tablet |
| DCCR 300 mg fed | Experimental | Administered a single 300 mg dose of DCCR after a standardized meal Receives: Drug: Diazoxide choline controlled-release tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Pharmacokinetic parameters: Cmax
Cmax derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours post-dose
Measured: up to 24 hours
Pharmacokinetic parameters: AUC0-24
AUC0-24 derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours
Measured: up to 24 hours
Secondary outcomes (2)
Pharmacokinetic parameters: CL/F
CL/F derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours
Measured: up to 24 hours
Pharmacokinetic parameters: Tmax
Tmax derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours
Measured: up to 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Sep 2016 (estimated)
- Study startJul 2017
- Primary completionOct 2017 (estimated)
- Study completionDec 2017 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Prader-Willi 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 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Enrolment
- 32 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Essentialis, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02893618
- Sponsor's protocol ID
- PK024
- Status last verified
- Sep 2016
- First submitted
- 30 Aug 2016
- Last updated
- 9 Sep 2016
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 9 Sep 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 5 Treatment Period Crossover Pharmacokinetic Study Evaluating Dose Proportionality and Food Effects of Diazoxide Choline Controlled-Release Tablet (DCCR)
- ClinicalTrials.gov ID
- NCT02893618
- Acronym / sponsor ID
- PK024
- Conditions
- Prader-Willi Syndrome
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 32 participants (estimated)
- Lead sponsor
- Essentialis, Inc.Industry (pharma, biotech, devices)
- Study start
- Jul 2017
- Primary completion
- Oct 2017 (estimated)
- Study completion
- Dec 2017 (estimated)
- First posted
- 8 Sep 2016 (estimated)
- 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.
Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food.
Detailed description
Open label, parallel-group, single site, 5 treatment-period study with 4 dose levels of DCCR, 1 of which is administered both with and without food, with a 10 Day washout period between treatments. There will be 8 treatment sequences with 4 subjects randomized to each.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Crossover
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| DCCR 75 mg fasted | Experimental | Drug: Diazoxide choline controlled-release tabletAdministered a single 75 mg dose of DCCR after an overnight fast followed by 4 hours of fasting |
| DCCR 150 mg fasted | Experimental | Drug: Diazoxide choline controlled-release tabletAdministered a single 150 mg dose of DCCR after an overnight fast followed by 4 hours of fasting |
| DCCR 300 mg fasted | Experimental | Drug: Diazoxide choline controlled-release tabletAdministered a single 300 mg dose of DCCR after an overnight fast followed by 4 hours of fasting |
| DCCR 450 mg fasted | Experimental | Drug: Diazoxide choline controlled-release tabletAdministered a single 450 mg dose of DCCR after an overnight fast followed by 4 hours of fasting |
| DCCR 300 mg fed | Experimental | Drug: Diazoxide choline controlled-release tabletAdministered a single 300 mg dose of DCCR after a standardized meal |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Ability to follow verbal and written instructions
- Informed consent form signed by the subject
- Completed screening within 7 days prior to dosing
- BMI between 18.5 and 35 kg/m2
- Generally healthy
Key exclusion criteria (9 listed)
- Pregnancy or breast feeding
- absence of contraception
- administration of investigational drug within 1 month prior to screening
- anticipated requirement for prohibited medication (systemic corticosteroids or anti-diabetic medications)
- allergic reaction to or significant intolerance of diazoxide, thiazides or sulfonamides
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 |
|---|---|---|
| Diazoxide choline controlled-release tabletAlso: DCCRQD tablet formulation of choline salt of diazoxide | Drug | DCCR 150 mg fasted, DCCR 300 mg fasted, DCCR 300 mg fed, DCCR 450 mg fasted, DCCR 75 mg fasted |
Primary outcome
Pharmacokinetic parameters: Cmax
Time frame: up to 24 hours
Cmax derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours post-dose
Pharmacokinetic parameters: AUC0-24
Time frame: up to 24 hours
AUC0-24 derived from PK samples taken at 0, 1, 3, 6, 9, 12, 15, 18, 21 and 24 hours
2 secondary outcomes
Pharmacokinetic parameters: CL/F
Time frame: up to 24 hours
Pharmacokinetic parameters: Tmax
Time frame: up to 24 hours
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 9 Sep 2016.
- Individual participant dataSharing planned: Undecided
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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.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study of Prader-Willi Syndrome, run by Essentialis, Inc..
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 7 inclusion and 9 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: status not confirmed, as of the sponsor's last update on 9 Sep 2016. Status can change between updates, so confirm with the study contact before making plans.
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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
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?
Pharmacokinetic parameters: Cmax (time frame: up to 24 hours). The study lists 2 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 9 Sep 2016.