Drug Resistant EpilepsyPhase 1NCT06899230
Safety, Tolerability, and Pharmacokinetics (PK) of Single and Multiple Ascending Oral Doses of iQ-007
A Phase 1 study testing iQ-007 in people with Drug Resistant Epilepsy and Focal Seizure, run by iQure Australia Pty Ltd at 1 site in Australia.
Trial summary
This is a randomized, double-blind, placebo-controlled study conducted in healthy adult volunteers to assess the safety, tolerability and pharmacokinetics of iQ-007. iQ-007 may be indicated for use in patients with Focal Seizures and Drug-resistant Epilepsy (DRE). Read more about this study
- Who is it for?
- Ages 18 to 55 yearsHealthy volunteers can take part
- Conditions the study is for
- Drug Resistant Epilepsy, Focal Seizure
- What is being tested?
- iQ-007
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 72 participants
- Where
- 1 siteAustralia
- Study dates
- 8 Apr 2025to 29 Jan 2026
- Phase
- Phase 1
- Are there results?
- Not yet: the study is under way
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 55 years
- All sexes
- Healthy volunteers accepted
- Adult
Who may be able to join
- Must have given written informed consent before any study-related activities are performed and must be able to understand the full nature and purpose of the study, including possible risks and adverse effects.
- Adult males or females, 18 to 55 years of age (inclusive) at screening.
- Body mass index (BMI) greater than or equal to 18.0 and less than or equal to 32.0 kg/m2.
- Medically healthy (in the opinion of the PI), as determined by pre-study medical history, and without clinically significant (CS) abnormalities including the following:
- Physical examination without any clinically relevant findings.
- Systolic blood pressure in the range of 90 to 160 mmHg and diastolic blood pressure in the range of 50 to 95 mmHg after resting for 5 minutes in a supine or semi-supine position.
Show all 23Show fewer
- Pulse rate in the range of 45 to 100 bpm after 5 minutes resting in a supine or semi-supine position.
- Body temperature (tympanic), between 35.5°C and 37.7°C. If temperature above 36.6°C, any infective etiology must be ruled out by the PI.
- Electrocardiogram without CS abnormalities including QT interval corrected for Fredericia (QTcF) <450msec
- No CS findings in serum chemistry, hematology, coagulation, and urinalysis tests.
- Female volunteers:
- Must be of non-childbearing potential, i.e., surgically sterilized (hysterectomy, bilateral salpingectomy, bilateral oophorectomy) at least 6 weeks before the screening visit or postmenopausal (where postmenopausal is defined as no menses for 12 months without an alternative medical cause and a follicle-stimulating hormone [FSH] level consistent with postmenopausal status, per local laboratory guidelines), or
- If of childbearing potential, must:
- Have a negative pregnancy test at the screening visit and on admission to the study site on Day-1.
- Agree not to attempt to become pregnant or donate ova from signing the consent form until at least 90 days after the last dose of the study drug.
- Agree to use adequate contraception (defined as use of a condom by the male partner combined with use of a highly effective method of contraception) established at screening until at least 30 days after the last dose of the study drug, if not exclusively in a same-sex relationship, or in relationship without a risk of pregnancy (sterile partner), or abstinent as a committed lifestyle.
- Male volunteers, must:
- Agree not to donate sperm from signing the consent form until at least 90 days after the last dose of the study drug.
- If engaging in sexual intercourse with a female partner who could become pregnant, agree to use adequate contraception (defined as use of a condom combined with their female partner's use of a highly effective method of contraception) from signing the consent form until at least 90 days after the last dose of the study drug.
- If engaging in sexual intercourse with a partner who is not at risk of pregnancy (sterile partner), agree to use a condom from signing the consent form until at least 90 days after the last dose of the study drug.
- For SAD Cohort A4 only: Willing and able to adhere to standard high fat, high calorie meal.
- Have suitable venous access for blood sampling.
- Willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
Who cannot join
- Known hypersensitivity to any of the study drug ingredients.
- History of anaphylaxis or other significant allergy which, in the opinion of the PI, would interfere with the volunteer's ability to participate in the study.
- History or presence of CS cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal/bariatric modification, endocrine, immunologic, dermatologic, psychiatric, or neurological disease/disorder, including any acute illness, within the past 3 months determined by the PI to be clinically relevant.
- Note: Participants with history of resolved childhood asthma, psoriasis, atopic dermatitis, non-hospitalized depression, anxiety and/or migraine are permitted to be included in the study.
- History of surgery or hospitalization within 3 months prior to screening, or elective surgery planned during the study.
- Any history of malignant disease in the last 10 years (excludes surgically resected skin squamous cell or basal cell carcinoma).
Show all 23Show fewer
- Presence of clinically relevant immunosuppression from, but not limited to, immunodeficiency conditions such as common variable hypogammaglobulinemia.
- History of risk factors for torsade de pointes (including a family history of long QT syndrome or sudden cardiac death) or a known arrythmia.
- Presence or having sequelae of gastrointestinal, liver (including Gilbert's syndrome), kidney, or other conditions known to interfere with the absorption, distribution, metabolism, or excretion of drugs.
- Liver function test results elevated >1.5-fold above the ULN for gamma glutamyl transferase, bilirubin(total), ALP, AST or ALT. Volunteers with ALP and/or ALT/AST above the limits specified may be included, at the discretion of the PI (or delegate), if the levels are unaccompanied by clinical signs and are determined to be normal variants.
- Estimated creatinine clearance <60 mL/min using the Cockcroft-Gault formula or serum creatinine>1.2-fold above the ULN.
- A history of or positive test results for human immunodeficiency virus (HIV), hepatitis B surface antigen(HBsAg) or hepatitis C (HCV) at the screening visit. Note that a history of hepatitis C virus (HCV) is not exclusionary and if deemed cured by the PI, may be included on a case-by-case basis at the MM and Sponsor's discretion.
- Positive drugs of abuse test, cotinine test or alcohol breath test results at the screening visit and/or on admission to the study site on Day -1.
- Regular consumption of more than 10 standard alcoholic drinks/week and/or more than 4 standard alcoholic drinks on any one day, where 1 standard drink is 10 g of pure alcohol and is equivalent to 285 mL beer [4.9% Alc/Vol], 100 mL wine [12% Alc/Vol], or 30 mL spirit [40% Alc/Vol]).
- Volunteer smokes more than 5 cigarettes or equivalent nicotine-containing products per week, and/or the volunteer is unwilling to abstain from smoking or the use of nicotine-containing products for 72 hours prior to check-in on Day -1 and throughout the confinement period at the study site.
- Females who are breastfeeding or planning to breastfeed.
- Unable to swallow oral medication.
- Use of any prescription or over-the-counter medication (including herbal products, diet aids, vitamins, and hormone supplements) within 10 days or 5 half-lives of the medication (whichever is longer) prior to the first dose of study drug, except use of contraceptives, the use of paracetamol (up to 2 g per day) for over 3 consecutive days and the use of ibuprofen (up to 1.2 g per day) for over 3 consecutive days.
- Current infection that requires systemically absorbed antibiotic, antifungal, antiparasitic or antiviral medication within 10 days prior to first dose of study drug.
- Use of any vaccinations within 30 days prior to screening.
- Donation of blood or plasma within 30 days prior to first dose of study drug, or loss of whole blood of more than 500 mL within 30 days prior to first dose of study drug, or receipt of a blood transfusion within 1year of the first dose of study drug.
- Participation in any clinical study of an investigational drug or investigational device within 30 days or 5 half-lives of the investigational drug (whichever is longer) prior to screening.
- Any other condition or prior therapy that in the opinion of the PI (or delegate) would make the volunteer unsuitable for this study, including inability to cooperate fully with the requirements of the study protocol or likelihood of noncompliance with any study requirements.
Trial locations and contacts
1 site in 1 country.
Australia1 site
CMAX Clinical Research Pty Ltd
Adelaide, South Australia, 5000
About this study
Official title: A Phase 1, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability and Pharmacokinetics of Oral iQ-007 in Healthy Male and Female Adult Participants
This is a randomized, double-blind, placebo-controlled study conducted in healthy adult volunteers to assess the safety, tolerability and pharmacokinetics of iQ-007. iQ-007 may be indicated for use in patients with Focal Seizures and Drug-resistant Epilepsy (DRE).
Read the detailed description
This is a randomized, double-blind, placebo-controlled study conducted in healthy adult volunteers. The study will be conducted in 2 parts: a single ascending dose (SAD) component and a multiple ascending dose (MAD) component.
Part A (SAD) will involve the administration of a single oral dose of iQ-007 or placebo capsules on Day 1 in Cohorts Al, A2, A3, A5 and A6. Participants in Cohort A4, will be required to fast overnight (minimum of 8 hours) prior to eating a high fat breakfast 30 minutes prior to a single oral administration of iQ-007 or placebo capsules on Day 1.
Part B (MAD) will involve the administration of multiple twice daily oral doses of iQ-007 or placebo capsules on Days 1 to 14 (total of 27 doses) in cohorts B1, B2, B3.
- Conditions
- Drug Resistant Epilepsy, Focal Seizure
- Being tested
- iQ-007, Placebo
- Medical subject headings
- Drug Resistant Epilepsy, Seizures
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
iQ-007Drug
Highly selective, orally bioavailable, positive allosteric modulator (PAM) of the excitatory amino acid transporter-2 (EAAT2) and its rodent homologue (glutamate transporter-1[GLT-1).
PlaceboDrug
Oral capsules identical in appearance to drug and containing vehicle only (Gelucire/Capmul-M).
| Study group | Role | What they receive |
|---|---|---|
| iQ-007 | Experimental | Part A (SAD): a total of 48 healthy volunteers are planned to be enrolled across 6 SAD cohorts and will involve the administration of a single oral dose of iQ-007 (in the range of 200 mg up to 2800 mg) or placebo capsules on Day 1.
Part B (MAD): a total of 24 healthy volunteers are planned to be enrolled across 3 MAD cohorts and will involve the administration of multiple twice daily oral doses of iQ-007 (in the range of 600 mg up to 2400mg total per day) or placebo capsules on Days 1 to 14. Receives: Drug: iQ-007 |
| Placebo | Placebo comparator | Part A (SAD): a total of 48 healthy volunteers are planned to be enrolled across 6 SAD cohorts and will involve the administration of a single oral dose of iQ-007 (in the range of 200 mg up to 2800 mg) or placebo capsules on Day 1.
Part B (MAD): a total of 24 healthy volunteers are planned to be enrolled across 3 MAD cohorts and will involve the administration of multiple twice daily oral doses of iQ-007 (in the range of 600 mg up to 2400mg total per day) or placebo capsules on Days 1 to 14. Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence, severity and relationship of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and TEAEs.
Adverse event (AE) - graded using the categories Mild, Moderate or Severe and will be classified as normal, abnormal not clinically significant (NCS), or abnormal CS.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in Blood pressure measurements
Vital signs assessed as normal, abnormal not clinically significant (NCS), or abnormal clinically significant (CS) with Blood pressure assessed using digital sphygmomanometer
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in electrocardiogram (ECG) parameters
Twelve-lead Electrocardiogram's (ECG's) including the measurement of ventricular heart rate [HR] will be conducted in triplicate.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in clinical laboratory results
Clinical laboratory results assessed using blood and urine samples and will be classified as normal, abnormal not clinically significant (NCS), or abnormal CS.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in heart rate measurements
Vital signs assessed as normal, abnormal not clinically significant (NCS), or abnormal CS with heart rate and respiratory rate assessed using pulse oximeter
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in temperature measurements
Vital signs assessed as normal, abnormal not clinically significant (NCS), or abnormal CS with temperature assessed using digital thermometer.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in ECG PR interval
Twelve-lead Electrocardiogram's (ECG's) including the measurement of PR interval will be conducted in triplicate.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in ECG QRS duration
Twelve-lead Electrocardiogram's (ECG's) including the measurement of QRS duration will be conducted in triplicate.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in ECG QT interval
Twelve-lead Electrocardiogram's (ECG's) including the measurement of QT interval will be conducted in triplicate.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Changes from baseline in ECG QTcF
Twelve-lead Electrocardiogram's (ECG's) including the measurement of QTcF will be conducted in triplicate.
Measured: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Secondary outcomes (19)
Single Ascending Dose: Plasma PK parameter - Maximum observed concentration (Cmax)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Multiple Ascending Dose: Plasma PK parameters - Maximum observed concentration (Cmax)
Blood plasma samples will be assessed
Measured: pre-dose Day 1 then 0.5, 1, 2, 3, 4, 6, 8, & 12 hrs post-dose, pre-dose on Days 2 - 13, pre-dose Day 14 then 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hrs post-dose, Day 15 24 hrs post-dose, Day 16 48 hrs post-dose, Day 17 72 hrs post-dose & Day 18 96 hrs post-dose
Multiple ascending dose: Urine PK endpoints include • Cumulative amount of un-changed drug excreted inurine (Ae)
Urine samples will be assessed
Measured: Pre-dose Day 1 - up to 12 hours post AM (first) dose, Day 14 and Day 15, up to 12 hours post AM (final) dose, 12 to 24 hours post AM (final) dose, Day 16, 24 to 48 hours post-dose, Day 17, 48 to 72 hours post-dose and Day 18, 72 to 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Time to maximum observed concentration (Tmax)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUClast)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Apparent terminal elimination half-life (T1/2)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Volume of distribution (Vz/F)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Clearance (CL/F)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Multiple Ascending Dose: Plasma PK parameters - Time to maximum observed concentration (Tmax)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUClast)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration versus time curve for the dosing interval (AUCtau)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Apparent terminal elimination half-life (T1/2)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Volume of distribution (Vz/F)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Clearance (CL/F)
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Accumulation ratio for Maximum observed concentration (Cmax) and Area under the concentration versus time curve for the dosing interval (AUCtau) using the first and last dose
Blood plasma samples will be assessed
Measured: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple ascending dose: Urine PK endpoints include • Fraction of the dose excreted in urine (Fe)
Urine samples will be assessed
Measured: Pre-dose Day 1 - up to 12 hours post AM (first) dose, Day 14 and Day 15, up to 12 hours post AM (final) dose, 12 to 24 hours post AM (final) dose, Day 16, 24 to 48 hours post-dose, Day 17, 48 to 72 hours post-dose and Day 18, 72 to 96 hours post-dose.
Multiple ascending dose: Urine PK endpoints include • Renal clearance (CLr).
Urine samples will be assessed
Measured: Pre-dose Day 1 - up to 12 hours post AM (first) dose, Day 14 and Day 15, up to 12 hours post AM (final) dose, 12 to 24 hours post AM (final) dose, Day 16, 24 to 48 hours post-dose, Day 17, 48 to 72 hours post-dose and Day 18, 72 to 96 hours post-dose.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Mar 2025
- Study start8 Apr 2025
- Primary completion29 Jan 2026 (estimated)
- Study completion29 Jan 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Drug Resistant EpilepsyStudies 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
- Sequential
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 72 participants (estimated)
Sponsor and investigators
- Lead sponsor
- iQure Australia Pty Ltd
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Avance Clinical Pty Ltd.
- Responsible party
- Sponsor
- Investigators
- Henk de Wilde, Study director, iQure Australia Pty Ltd
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06899230
- Sponsor's protocol ID
- iQ-007-CL-001
- Status last verified
- Dec 2025
- First submitted
- 13 Mar 2025
- Last updated
- 29 Dec 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Randomized, Double-blind, Placebo-controlled, Single and Multiple Ascending Dose Study to Assess the Safety, Tolerability and Pharmacokinetics of Oral iQ-007 in Healthy Male and Female Adult Participants
- ClinicalTrials.gov ID
- NCT06899230
- Acronym / sponsor ID
- iQ-007-CL-001
- Conditions
- Drug Resistant Epilepsy, Focal Seizure
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 72 participants (estimated)
- Lead sponsor
- iQure Australia Pty LtdIndustry (pharma, biotech, devices)
- Collaborators
- Avance Clinical Pty Ltd.
- Study start
- 8 Apr 2025 (actual)
- Primary completion
- 29 Jan 2026 (estimated)
- Study completion
- 29 Jan 2026 (estimated)
- First posted
- 27 Mar 2025 (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 randomized, double-blind, placebo-controlled study conducted in healthy adult volunteers to assess the safety, tolerability and pharmacokinetics of iQ-007. iQ-007 may be indicated for use in patients with Focal Seizures and Drug-resistant Epilepsy (DRE).
Detailed description
This is a randomized, double-blind, placebo-controlled study conducted in healthy adult volunteers. The study will be conducted in 2 parts: a single ascending dose (SAD) component and a multiple ascending dose (MAD) component.
Part A (SAD) will involve the administration of a single oral dose of iQ-007 or placebo capsules on Day 1 in Cohorts Al, A2, A3, A5 and A6. Participants in Cohort A4, will be required to fast overnight (minimum of 8 hours) prior to eating a high fat breakfast 30 minutes prior to a single oral administration of iQ-007 or placebo capsules on Day 1.
Part B (MAD) will involve the administration of multiple twice daily oral doses of iQ-007 or placebo capsules on Days 1 to 14 (total of 27 doses) in cohorts B1, B2, B3.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Sequential
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| iQ-007 | Experimental | Drug: iQ-007Part A (SAD): a total of 48 healthy volunteers are planned to be enrolled across 6 SAD cohorts and will involve the administration of a single oral dose of iQ-007 (in the range of 200 mg up to 2800 mg) or placebo capsules on Day 1. Part B (MAD): a total of 24 healthy volunteers are planned to be enrolled across 3 MAD cohorts and will involve the administration of multiple twice daily oral doses of iQ-007 (in the range of 600 mg up to 2400mg total per day) or placebo capsules on Days 1 to 14. |
| Placebo | Placebo comparator | Drug: PlaceboPart A (SAD): a total of 48 healthy volunteers are planned to be enrolled across 6 SAD cohorts and will involve the administration of a single oral dose of iQ-007 (in the range of 200 mg up to 2800 mg) or placebo capsules on Day 1. Part B (MAD): a total of 24 healthy volunteers are planned to be enrolled across 3 MAD cohorts and will involve the administration of multiple twice daily oral doses of iQ-007 (in the range of 600 mg up to 2400mg total per day) or placebo capsules on Days 1 to 14. |
Who may qualify
- Ages
- 18 to 55 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult
Key inclusion criteria (23 listed)
- Must have given written informed consent before any study-related activities are performed and must be able to understand the full nature and purpose of the study, including possible risks and adverse effects.
- Adult males or females, 18 to 55 years of age (inclusive) at screening.
- Body mass index (BMI) greater than or equal to 18.0 and less than or equal to 32.0 kg/m2.
- Medically healthy (in the opinion of the PI), as determined by pre-study medical history, and without clinically significant (CS) abnormalities including the following:
- Female volunteers:
Key exclusion criteria (23 listed)
- Known hypersensitivity to any of the study drug ingredients.
- History of anaphylaxis or other significant allergy which, in the opinion of the PI, would interfere with the volunteer's ability to participate in the study.
- History or presence of CS cardiovascular, pulmonary, hepatic, renal, hematological, gastrointestinal/bariatric modification, endocrine, immunologic, dermatologic, psychiatric, or neurological disease/disorder, including any acute illness, within the past 3 months determined by the PI to be clinically relevant.
- Note: Participants with history of resolved childhood asthma, psoriasis, atopic dermatitis, non-hospitalized depression, anxiety and/or migraine are permitted to be included in the study.
- History of surgery or hospitalization within 3 months prior to screening, or elective surgery planned during the study.
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 |
|---|---|---|
| iQ-007Highly selective, orally bioavailable, positive allosteric modulator (PAM) of the excitatory amino acid transporter-2 (EAAT2) and its rodent homologue (glutamate transporter-1[GLT-1). | Drug | iQ-007 |
| PlaceboOral capsules identical in appearance to drug and containing vehicle only (Gelucire/Capmul-M). | Drug | Placebo |
Primary outcome
Incidence, severity and relationship of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and TEAEs.
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Adverse event (AE) - graded using the categories Mild, Moderate or Severe and will be classified as normal, abnormal not clinically significant (NCS), or abnormal CS.
Changes from baseline in Blood pressure measurements
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Vital signs assessed as normal, abnormal not clinically significant (NCS), or abnormal clinically significant (CS) with Blood pressure assessed using digital sphygmomanometer
Changes from baseline in electrocardiogram (ECG) parameters
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Twelve-lead Electrocardiogram's (ECG's) including the measurement of ventricular heart rate [HR] will be conducted in triplicate.
Changes from baseline in clinical laboratory results
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Clinical laboratory results assessed using blood and urine samples and will be classified as normal, abnormal not clinically significant (NCS), or abnormal CS.
Changes from baseline in heart rate measurements
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Vital signs assessed as normal, abnormal not clinically significant (NCS), or abnormal CS with heart rate and respiratory rate assessed using pulse oximeter
Changes from baseline in temperature measurements
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Vital signs assessed as normal, abnormal not clinically significant (NCS), or abnormal CS with temperature assessed using digital thermometer.
Changes from baseline in ECG PR interval
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Twelve-lead Electrocardiogram's (ECG's) including the measurement of PR interval will be conducted in triplicate.
Changes from baseline in ECG QRS duration
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Twelve-lead Electrocardiogram's (ECG's) including the measurement of QRS duration will be conducted in triplicate.
Changes from baseline in ECG QT interval
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Twelve-lead Electrocardiogram's (ECG's) including the measurement of QT interval will be conducted in triplicate.
Changes from baseline in ECG QTcF
Time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.
Twelve-lead Electrocardiogram's (ECG's) including the measurement of QTcF will be conducted in triplicate.
19 secondary outcomes
Single Ascending Dose: Plasma PK parameter - Maximum observed concentration (Cmax)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Multiple Ascending Dose: Plasma PK parameters - Maximum observed concentration (Cmax)
Time frame: pre-dose Day 1 then 0.5, 1, 2, 3, 4, 6, 8, & 12 hrs post-dose, pre-dose on Days 2 - 13, pre-dose Day 14 then 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hrs post-dose, Day 15 24 hrs post-dose, Day 16 48 hrs post-dose, Day 17 72 hrs post-dose & Day 18 96 hrs post-dose
Multiple ascending dose: Urine PK endpoints include • Cumulative amount of un-changed drug excreted inurine (Ae)
Time frame: Pre-dose Day 1 - up to 12 hours post AM (first) dose, Day 14 and Day 15, up to 12 hours post AM (final) dose, 12 to 24 hours post AM (final) dose, Day 16, 24 to 48 hours post-dose, Day 17, 48 to 72 hours post-dose and Day 18, 72 to 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Time to maximum observed concentration (Tmax)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUClast)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Apparent terminal elimination half-life (T1/2)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Volume of distribution (Vz/F)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Single Ascending Dose: Plasma PK parameter - Clearance (CL/F)
Time frame: Pre-dose Day 1, then 0.5, 1, 2, 3, 4, 6, 8, 12 and 16 hours post-dose, Day 2, 24 hours post-dose, Day 3, 48 hours post-dose, Day 4, 72 hours post-dose and Day 5, 96 hours post-dose.
Multiple Ascending Dose: Plasma PK parameters - Time to maximum observed concentration (Tmax)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUClast)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration versus time curve for the dosing interval (AUCtau)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Apparent terminal elimination half-life (T1/2)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Volume of distribution (Vz/F)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Clearance (CL/F)
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple Ascending Dose: Plasma PK parameter - Accumulation ratio for Maximum observed concentration (Cmax) and Area under the concentration versus time curve for the dosing interval (AUCtau) using the first and last dose
Time frame: Pre-dose Day 1 0.5, 1, 2, 3, 4, 6, 8, & 12 hours post-dose, pre-dose Days 2 - 13, pre-dose Day 14 0.5, 1, 2, 3, 4, 6, 8, 12 & 16 hours post-dose, Day 15 24 hours post-dose, Day 16 48 hours post-dose, Day 17 72 hours post-dose, Day 18 96 hours post-dose
Multiple ascending dose: Urine PK endpoints include • Fraction of the dose excreted in urine (Fe)
Time frame: Pre-dose Day 1 - up to 12 hours post AM (first) dose, Day 14 and Day 15, up to 12 hours post AM (final) dose, 12 to 24 hours post AM (final) dose, Day 16, 24 to 48 hours post-dose, Day 17, 48 to 72 hours post-dose and Day 18, 72 to 96 hours post-dose.
Multiple ascending dose: Urine PK endpoints include • Renal clearance (CLr).
Time frame: Pre-dose Day 1 - up to 12 hours post AM (first) dose, Day 14 and Day 15, up to 12 hours post AM (final) dose, 12 to 24 hours post AM (final) dose, Day 16, 24 to 48 hours post-dose, Day 17, 48 to 72 hours post-dose and Day 18, 72 to 96 hours post-dose.
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Dec 2025.
- Individual participant dataSharing planned: No
Related trials for Drug Resistant Epilepsy
Not yet recruiting
Clinical Trial of SEEG for Drug-resistant Epilepsy
Drug-Resistant Epilepsy
Recruiting
Identification of Biomarkers for Vagus Nerve Stimulation in Patients With Refractory Epilepsy
Refractory Epilepsy
RecruitingPhase 1
A Phase I Clinical Trial of UX-GIP001 in the Treatment of Drug-Resistant Unilateral Mesial Temporal Lobe Epilepsy
Epilepsy
Not yet recruiting
Electrophysiological Dose-Response to Anterior Thalamic Deep Brain Stimulation in Drug-Resistant Focal Epilepsy
Drug Resistant Epilepsy, Focal Epilepsy
Recruiting
Effect of Blue Light on Vagus Nerve Stimulation in Patients With Refractory Epilepsy
Refractory Epilepsy
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.
Henk de Wilde study director
iQure Australia Pty Ltd
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 iQ-007 in people with Drug Resistant Epilepsy and Focal Seizure, run by iQure Australia Pty Ltd at 1 site in Australia.
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 23 inclusion and 23 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: under way, not looking for participants, as of the sponsor's last update on 29 Dec 2025. 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 Australia. 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?
This study is still under way. Results are not available in the source record yet.
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?
Incidence, severity and relationship of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and TEAEs. (time frame: From baseline to the end of observation on Day 8 for Single ascending dose. From baseline to the end of observation on Day 21 for Multiple ascending dose.). The study lists 10 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 29 Dec 2025.