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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06899230




Registration number
NCT06899230
Ethics application status
Date submitted
13/03/2025
Date registered
27/03/2025

Titles & IDs
Public title
Safety, Tolerability, and Pharmacokinetics (PK) of Single and Multiple Ascending Oral Doses of IQ-007
Scientific 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
Secondary ID [1] 0 0
iQ-007-CL-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Drug Resistant Epilepsy 0 0
Focal Seizure 0 0
Condition category
Condition code
Neurological 0 0 0 0
Epilepsy
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - iQ-007
Treatment: Drugs - Placebo

Experimental: iQ-007 - 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.

Placebo comparator: Placebo - 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.


Treatment: Drugs: iQ-007
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).

Treatment: Drugs: Placebo
Oral capsules identical in appearance to drug and containing vehicle only (Gelucire/Capmul-M).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence, severity and relationship of treatment-emergent adverse events (TEAEs), serious adverse events (SAEs), and TEAEs.
Timepoint [1] 0 0
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.
Primary outcome [2] 0 0
Changes from baseline in Blood pressure measurements
Timepoint [2] 0 0
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.
Primary outcome [3] 0 0
Changes from baseline in electrocardiogram (ECG) parameters
Timepoint [3] 0 0
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.
Primary outcome [4] 0 0
Changes from baseline in clinical laboratory results
Timepoint [4] 0 0
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.
Primary outcome [5] 0 0
Changes from baseline in heart rate measurements
Timepoint [5] 0 0
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.
Primary outcome [6] 0 0
Changes from baseline in temperature measurements
Timepoint [6] 0 0
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.
Primary outcome [7] 0 0
Changes from baseline in ECG PR interval
Timepoint [7] 0 0
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.
Primary outcome [8] 0 0
Changes from baseline in ECG QRS duration
Timepoint [8] 0 0
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.
Primary outcome [9] 0 0
Changes from baseline in ECG QT interval
Timepoint [9] 0 0
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.
Primary outcome [10] 0 0
Changes from baseline in ECG QTcF
Timepoint [10] 0 0
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 outcome [1] 0 0
Single Ascending Dose: Plasma PK parameter - Maximum observed concentration (Cmax)
Timepoint [1] 0 0
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.
Secondary outcome [2] 0 0
Multiple Ascending Dose: Plasma PK parameters - Maximum observed concentration (Cmax)
Timepoint [2] 0 0
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
Secondary outcome [3] 0 0
Multiple ascending dose: Urine PK endpoints include • Cumulative amount of un-changed drug excreted inurine (Ae)
Timepoint [3] 0 0
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.
Secondary outcome [4] 0 0
Single Ascending Dose: Plasma PK parameter - Time to maximum observed concentration (Tmax)
Timepoint [4] 0 0
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.
Secondary outcome [5] 0 0
Single Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUClast)
Timepoint [5] 0 0
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.
Secondary outcome [6] 0 0
Single Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
Timepoint [6] 0 0
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.
Secondary outcome [7] 0 0
Single Ascending Dose: Plasma PK parameter - Apparent terminal elimination half-life (T1/2)
Timepoint [7] 0 0
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.
Secondary outcome [8] 0 0
Single Ascending Dose: Plasma PK parameter - Volume of distribution (Vz/F)
Timepoint [8] 0 0
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.
Secondary outcome [9] 0 0
Single Ascending Dose: Plasma PK parameter - Clearance (CL/F)
Timepoint [9] 0 0
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.
Secondary outcome [10] 0 0
Multiple Ascending Dose: Plasma PK parameters - Time to maximum observed concentration (Tmax)
Timepoint [10] 0 0
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
Secondary outcome [11] 0 0
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from 0 to time of last quantifiable concentration (AUClast)
Timepoint [11] 0 0
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
Secondary outcome [12] 0 0
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration versus time curve for the dosing interval (AUCtau)
Timepoint [12] 0 0
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
Secondary outcome [13] 0 0
Multiple Ascending Dose: Plasma PK parameter - Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)
Timepoint [13] 0 0
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
Secondary outcome [14] 0 0
Multiple Ascending Dose: Plasma PK parameter - Apparent terminal elimination half-life (T1/2)
Timepoint [14] 0 0
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
Secondary outcome [15] 0 0
Multiple Ascending Dose: Plasma PK parameter - Volume of distribution (Vz/F)
Timepoint [15] 0 0
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
Secondary outcome [16] 0 0
Multiple Ascending Dose: Plasma PK parameter - Clearance (CL/F)
Timepoint [16] 0 0
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
Secondary outcome [17] 0 0
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
Timepoint [17] 0 0
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
Secondary outcome [18] 0 0
Multiple ascending dose: Urine PK endpoints include • Fraction of the dose excreted in urine (Fe)
Timepoint [18] 0 0
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.
Secondary outcome [19] 0 0
Multiple ascending dose: Urine PK endpoints include • Renal clearance (CLr).
Timepoint [19] 0 0
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.

Eligibility
Key inclusion criteria
1. 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.
2. Adult males or females, 18 to 55 years of age (inclusive) at screening.
3. Body mass index (BMI) greater than or equal to 18.0 and less than or equal to 32.0 kg/m2.
4. Medically healthy (in the opinion of the PI), as determined by pre-study medical history, and without clinically significant (CS) abnormalities including the following:

1. Physical examination without any clinically relevant findings.
2. 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.
3. Pulse rate in the range of 45 to 100 bpm after 5 minutes resting in a supine or semi-supine position.
4. 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.
5. Electrocardiogram without CS abnormalities including QT interval corrected for Fredericia (QTcF) <450msec
6. No CS findings in serum chemistry, hematology, coagulation, and urinalysis tests.
5. Female volunteers:

1. 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
2. If of childbearing potential, must:

i. Have a negative pregnancy test at the screening visit and on admission to the study site on Day-1.

ii. 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.

iii. 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.
6. Male volunteers, must:

1. Agree not to donate sperm from signing the consent form until at least 90 days after the last dose of the study drug.
2. 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.
3. 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.
7. For SAD Cohort A4 only: Willing and able to adhere to standard high fat, high calorie meal.
8. Have suitable venous access for blood sampling.
9. Willing and able to comply with all study assessments and adhere to the protocol schedule and restrictions.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Known hypersensitivity to any of the study drug ingredients.
2. 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.
3. 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.
4. History of surgery or hospitalization within 3 months prior to screening, or elective surgery planned during the study.
5. Any history of malignant disease in the last 10 years (excludes surgically resected skin squamous cell or basal cell carcinoma).
6. Presence of clinically relevant immunosuppression from, but not limited to, immunodeficiency conditions such as common variable hypogammaglobulinemia.
7. History of risk factors for torsade de pointes (including a family history of long QT syndrome or sudden cardiac death) or a known arrythmia.
8. 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.
9. 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.
10. Estimated creatinine clearance <60 mL/min using the Cockcroft-Gault formula or serum creatinine>1.2-fold above the ULN.
11. 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.
12. 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.
13. 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]).
14. 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.
15. Females who are breastfeeding or planning to breastfeed.
16. Unable to swallow oral medication.
17. 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.
18. Current infection that requires systemically absorbed antibiotic, antifungal, antiparasitic or antiviral medication within 10 days prior to first dose of study drug.
19. Use of any vaccinations within 30 days prior to screening.
20. 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.
21. 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.
22. 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.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Intervention assignment
Other
Other design features
Phase
Phase 1
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
24/04/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
31/10/2025
Actual
Sample size
Target
72
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 0 0
CMAX Clinical Research Pty Ltd - Adelaide
Recruitment postcode(s) [1] 0 0
5000 - Adelaide

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
iQure Australia Pty Ltd
Address
Country
Other collaborator category [1] 0 0
Commercial sector/industry
Name [1] 0 0
Avance Clinical Pty Ltd.
Address [1] 0 0
Country [1] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Henk de Wilde
Address 0 0
iQure Australia Pty Ltd
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Mohamed Bakra, Dr
Address 0 0
Country 0 0
Phone 0 0
+61 0424 428 642
Fax 0 0
Email 0 0
[email protected]
Contact person for scientific queries

Data sharing statement


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06899230