Dravet SyndromePhase 1NCT04069689
Study of Safety and Pharmacokinetics of Oral Doses of EPX-100 in Healthy Subjects.
A Phase 1 study testing EPX-100 (Clemizole Hydrochloride) in people with Dravet Syndrome, run by Epygenix at 1 site in United States.
Trial summary
This is a placebo-controlled, double-blind, 2-period study in 3 sequential groups of 8 healthy subjects each. The safety and pharmacokinetics of escalating single and multiple oral doses of EPX-100 will be assessed in fasting healthy subjects and following a high-fat meal. Read more about this study
- Who is it for?
- Ages 18 to 50 yearsHealthy volunteers can take part
- Conditions the study is for
- Dravet Syndrome
- What is being tested?
- EPX-100 (Clemizole Hydrochloride)
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 24 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Aug 2019to 27 Nov 2019
- 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 18 to 50 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Signed informed consent prior to any study-related procedures
- Male or female subjects 18 to 50 years of age inclusive
- Subject's body mass index (BMI) is ≤ 30 kg/m2
- Female subjects of childbearing potential must not be pregnant or lactating with a negative serum human chorionic gonadotropin (HCG) pregnancy test result at Screening, Day -1, or Day 19.
- Female subjects of childbearing potential and male subjects must use an adequate method of contraception from Screening until completion of the study. Acceptable methods of contraception are barrier methods (male condom, female condom, diaphragm, cervical cap, spermicide, or intrauterine device [IUD]), surgical sterility (documented doctor's report of vasectomy, hysterectomy, and/or bilateral oophorectomy), oral hormonal contraceptives, hormonal IUD, and/or postmenopausal status (defined as at least 1 year without menses as demonstrated by medical history or subject report).
- Subject is in good health as determined by vital signs, medical history, physical exam, and safety laboratory analyses at Screening and during the study.
Who cannot join
- Subject has used an investigational product within 30 days prior to enrollment or during the study.
- Subject has used prescription or non-prescription drugs (including vitamins, minerals, and herbal/plant-derived preparations) within 2 weeks of enrollment (excluding oral hormonal contraceptives, hormonal IUD, hormone replacement therapy, and acetaminophen) unless deemed acceptable by the Investigator in consultation with the Sponsor.
- Subject has a positive drug and/or alcohol test at Screening, Day -1, or Day 19.
- Subject has a history of drug or alcohol abuse within 2 years before Screening.
- Subject is unable to abstain from ingesting alcohol, caffeine, grapefruit or grapefruit juice, pomelo or pomelo juice, or Seville oranges or Seville orange juice for 72 hours prior to dosing and throughout the dosing periods.
- Concurrent use of substances, including drugs, known to interfere with EPX-100, including moderate or severe inducers or inhibitors of CYP3A4 and CYP2D6.
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- The subject has a clinically significant history of endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major diseases or malignancy.
- Subject has evidence of any of the following cardiac conduction abnormalities:
- QTcF interval >430 msec for males and >450 msec for females
- PR interval ⩾ 200 msec
- Evidence of second- or third-degree atrioventricular block (AVB)
- Electrocardiographic evidence of complete left bundle branch block (LBBB), complete right bundle branch block (RBBB), or incomplete LBBB
- Intraventricular conduction delay with QRS duration >120 msec
- Heart rate <40 bpm
- Pathological Q waves (defined as >40 msec or depth >0.4-0.5 mV)
- Evidence of ventricular pre-excitation.
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Phase I, Placebo-Controlled, Double-Blind, 2-Period Study to Assess Safety and Pharmacokinetics of Escalating Single and Multiple Oral Doses of EPX-100 in Fasting Healthy Subjects and Following a High-Fat Meal
This is a placebo-controlled, double-blind, 2-period study in 3 sequential groups of 8 healthy subjects each. The safety and pharmacokinetics of escalating single and multiple oral doses of EPX-100 will be assessed in fasting healthy subjects and following a high-fat meal.
Read the detailed description
This is a placebo-controlled, double-blind, 2-period study in 3 sequential groups of 8 healthy subjects each. Subjects will be admitted on two occasions to the clinical research center: Day -1 for 14 days and discharged on Day 13 and then re-admitted on Day 19 for 3 days and discharged on Day 21. Subjects will fast after midnight on the day of each admission.
On Day 1 of study of the low-dose group (cohort 1), subjects will be randomized to a single dose of 20 mg EPX-100 (N=6) or placebo (N=2) in the morning and then remain fasting for 4 hours after dosing. Safety will be assessed and blood samples will be obtained to calculate PK at the following time points: 0, 0.5, 1, 2, 4, 6, 8, 12, 18, and 24 hours following the first dose of EPX-100 or placebo. The subjects will remain in the study research center for daily 8 AM (± 2 hours) blood samples for 5 consecutive days (Days 3 - 7; one blood sample per day). On Days 8 - 11, subjects will be administered 20 mg EPX-100 or placebo twice daily (BID) at least one hour prior to the morning meal and at least 2 hours after the evening meal (approximately 12 hours apart). A single dose of 20 mg EPX-100 or placebo will be administered on Day 12 in the fasting state and subjects will remain fasting for 4 hours after dosing. Blood samples will be drawn at the following time points: 0, 0.5, 1, 2, 4, 6, 8, 12, 18, and 24 hours to determine multiple-dose PK. After a washout period of at least one week following the last dose of EPX-100 or placebo, subjects will return to the clinical research center on Day 19 and safety will be assessed. On Day 20, subjects will ingest a high-fat morning meal over 30 minutes; thereafter, the subject will receive a single dose of 20 mg EPX- 100 or placebo at 30 minutes after the start of the meal. Blood samples will be drawn at the following time points: 0, 0.5, 1, 2, 4, 6, 8, 12, 18, and 24 hours after the administration of study drug to determine the PK of EPX-100 in the fed state. Once the 20 mg dose level of EPX-100 is evaluated and the Safety Review Committee (SRC) determines it is safe to escalate to the next dose level, subsequent groups of 8 subjects each will be administered 40 mg (cohort 2) and 80 mg (cohort 3) (N=6 active drug, N=2 matching placebo) EPX-100 and follow the same study procedures as the low-dose group (cohort 1).
Throughout the study period, subjects will undergo cardiac assessments, safety assessments, and PK sampling.
- Conditions
- Dravet Syndrome
- Being tested
- EPX-100 (Clemizole Hydrochloride), Placebos
- Keywords
- Clemizole
- Medical subject headings
- Epilepsies, Myoclonic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
EPX-100 (Clemizole Hydrochloride)Drug
Also called Clemizole Hydrochloride, Clemizole HCL
EPX-100 (Clemizole Hydrochloride)
PlacebosDrug
Placebo to match EPX-100
| Study group | Role | What they receive |
|---|---|---|
| EPX-100 | Experimental | Single and multiple doses of 20, 40, 80mg of EPX-100 (Clemizole Hydrochloride) Receives: Drug: EPX-100 (Clemizole Hydrochloride) |
| Placebo | Placebo comparator | Single and multiple doses of 20, 40, 80mg of placebo Receives: Drug: Placebos |
What is being measured
The primary outcome is the main question the study is designed to answer.
Treatment-emergent adverse events (TEAEs)
To evaluate the safety of single and multiple escalating doses of oral EPX-100 in healthy subjects.
Measured: 21 days
Serial ECGs - QTcF Interval
To evaluate the safety of single and multiple escalating doses of oral EPX-100 in healthy subjects.
Measured: 21 days
Physical Examinations Including Actual Body Weight
To evaluate the safety of single and multiple escalating doses of oral EPX-100 in healthy subjects.
Measured: 21 days
Secondary outcomes (2)
Plasma Concentrations of EPX-100 in Fasting State
Determine the pharmacokinetic (PK) profile of single and multiples doses of 20 mg, 40 mg, and 80 mg twice daily of EPX-100.
Measured: 13 days
Plasma Concentration of EPX-100 following a High-Fat Meal
Determine the PK profile of a single dose of EPX-100 in the fasting state compared with after a high-fat meal.
Measured: 24 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Aug 2019
- Study start29 Aug 2019
- Primary completion27 Nov 2019
- Study completion27 Nov 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Dravet 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
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 24 participants (actual)
Sponsor and investigators
- Lead sponsor
- Epygenix
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hahn-Jun Lee, Ph.D., Study director, Epygenix Therapeutics, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04069689
- Sponsor's protocol ID
- EPX-100-002
- Status last verified
- Sep 2024
- First submitted
- 22 Aug 2019
- Last updated
- 19 Sep 2024
- 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 19 Sep 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Placebo-Controlled, Double-Blind, 2-Period Study to Assess Safety and Pharmacokinetics of Escalating Single and Multiple Oral Doses of EPX-100 in Fasting Healthy Subjects and Following a High-Fat Meal
- ClinicalTrials.gov ID
- NCT04069689
- Acronym / sponsor ID
- EPX-100-002
- Conditions
- Dravet Syndrome
- Keywords
- Clemizole
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 24 participants (actual)
- Lead sponsor
- EpygenixIndustry (pharma, biotech, devices)
- Study start
- 29 Aug 2019 (actual)
- Primary completion
- 27 Nov 2019 (actual)
- Study completion
- 27 Nov 2019 (actual)
- First posted
- 28 Aug 2019 (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.
This is a placebo-controlled, double-blind, 2-period study in 3 sequential groups of 8 healthy subjects each. The safety and pharmacokinetics of escalating single and multiple oral doses of EPX-100 will be assessed in fasting healthy subjects and following a high-fat meal.
Detailed description
This is a placebo-controlled, double-blind, 2-period study in 3 sequential groups of 8 healthy subjects each. Subjects will be admitted on two occasions to the clinical research center: Day -1 for 14 days and discharged on Day 13 and then re-admitted on Day 19 for 3 days and discharged on Day 21. Subjects will fast after midnight on the day of each admission.
On Day 1 of study of the low-dose group (cohort 1), subjects will be randomized to a single dose of 20 mg EPX-100 (N=6) or placebo (N=2) in the morning and then remain fasting for 4 hours after dosing. Safety will be assessed and blood samples will be obtained to calculate PK at the following time points: 0, 0.5, 1, 2, 4, 6, 8, 12, 18, and 24 hours following the first dose of EPX-100 or placebo. The subjects will remain in the study research center for daily 8 AM (± 2 hours) blood samples for 5 consecutive days (Days 3 - 7; one blood sample per day). On Days 8 - 11, subjects will be administered 20 mg EPX-100 or placebo twice daily (BID) at least one hour prior to the morning meal and at least 2 hours after the evening meal (approximately 12 hours apart). A single dose of 20 mg EPX-100 or placebo will be administered on Day 12 in the fasting state and subjects will remain fasting for 4 hours after dosing. Blood samples will be drawn at the following time points: 0, 0.5, 1, 2, 4, 6, 8, 12, 18, and 24 hours to determine multiple-dose PK. After a washout period of at least one week following the last dose of EPX-100 or placebo, subjects will return to the clinical research center on Day 19 and safety will be assessed. On Day 20, subjects will ingest a high-fat morning meal over 30 minutes; thereafter, the subject will receive a single dose of 20 mg EPX- 100 or placebo at 30 minutes after the start of the meal. Blood samples will be drawn at the following time points: 0, 0.5, 1, 2, 4, 6, 8, 12, 18, and 24 hours after the administration of study drug to determine the PK of EPX-100 in the fed state. Once the 20 mg dose level of EPX-100 is evaluated and the Safety Review Committee (SRC) determines it is safe to escalate to the next dose level, subsequent groups of 8 subjects each will be administered 40 mg (cohort 2) and 80 mg (cohort 3) (N=6 active drug, N=2 matching placebo) EPX-100 and follow the same study procedures as the low-dose group (cohort 1).
Throughout the study period, subjects will undergo cardiac assessments, safety assessments, and PK sampling.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| EPX-100 | Experimental | Drug: EPX-100 (Clemizole Hydrochloride)Single and multiple doses of 20, 40, 80mg of EPX-100 (Clemizole Hydrochloride) |
| Placebo | Placebo comparator | Drug: PlacebosSingle and multiple doses of 20, 40, 80mg of placebo |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (6 listed)
- Signed informed consent prior to any study-related procedures
- Male or female subjects 18 to 50 years of age inclusive
- Subject's body mass index (BMI) is ≤ 30 kg/m2
- Female subjects of childbearing potential must not be pregnant or lactating with a negative serum human chorionic gonadotropin (HCG) pregnancy test result at Screening, Day -1, or Day 19.
- Female subjects of childbearing potential and male subjects must use an adequate method of contraception from Screening until completion of the study. Acceptable methods of contraception are barrier methods (male condom, female condom, diaphragm, cervical cap, spermicide, or intrauterine device [IUD]), surgical sterility (documented doctor's report of vasectomy, hysterectomy, and/or bilateral oophorectomy), oral hormonal contraceptives, hormonal IUD, and/or postmenopausal status (defined as at least 1 year without menses as demonstrated by medical history or subject report).
Key exclusion criteria (16 listed)
- Subject has used an investigational product within 30 days prior to enrollment or during the study.
- Subject has used prescription or non-prescription drugs (including vitamins, minerals, and herbal/plant-derived preparations) within 2 weeks of enrollment (excluding oral hormonal contraceptives, hormonal IUD, hormone replacement therapy, and acetaminophen) unless deemed acceptable by the Investigator in consultation with the Sponsor.
- Subject has a positive drug and/or alcohol test at Screening, Day -1, or Day 19.
- Subject has a history of drug or alcohol abuse within 2 years before Screening.
- Subject is unable to abstain from ingesting alcohol, caffeine, grapefruit or grapefruit juice, pomelo or pomelo juice, or Seville oranges or Seville orange juice for 72 hours prior to dosing and throughout the dosing periods.
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 |
|---|---|---|
| EPX-100 (Clemizole Hydrochloride)Also: Clemizole Hydrochloride, Clemizole HCLEPX-100 (Clemizole Hydrochloride) | Drug | EPX-100 |
| PlacebosPlacebo to match EPX-100 | Drug | Placebo |
Primary outcome
Treatment-emergent adverse events (TEAEs)
Time frame: 21 days
To evaluate the safety of single and multiple escalating doses of oral EPX-100 in healthy subjects.
Serial ECGs - QTcF Interval
Time frame: 21 days
To evaluate the safety of single and multiple escalating doses of oral EPX-100 in healthy subjects.
Physical Examinations Including Actual Body Weight
Time frame: 21 days
To evaluate the safety of single and multiple escalating doses of oral EPX-100 in healthy subjects.
2 secondary outcomes
Plasma Concentrations of EPX-100 in Fasting State
Time frame: 13 days
Plasma Concentration of EPX-100 following a High-Fat Meal
Time frame: 24 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 19 Sep 2024.
- 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.
Hahn-Jun Lee, Ph.D. study director
Epygenix Therapeutics, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing EPX-100 (Clemizole Hydrochloride) in people with Dravet Syndrome, run by Epygenix at 1 site 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 16 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 19 Sep 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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. Some participants in this study may receive a placebo. 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?
Treatment-emergent adverse events (TEAEs) (time frame: 21 days). The study lists 3 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 19 Sep 2024.