Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000473460
Ethics application status
Approved
Date submitted
30/04/2025
Date registered
16/05/2025
Date last updated
16/05/2025
Date data sharing statement initially provided
16/05/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
A clinical study to compare the safety and tolerability of two formulations of a new drug in Healthy Participants for Parkinson's disease.
Scientific title
A Phase 1, Open-Label, Randomized, 2-Way Crossover Study to Evaluate the Relative Bioavailability of Two GT-02287 Oral Suspension Formulations in Healthy Participants
Secondary ID [1] 314236 0
GANX-001-V104
Universal Trial Number (UTN)
Trial acronym
Linked study record
This study is a follow-up study with a new IP formulation to check bioavailability against the IP tested in ACTRN12623000860662 in healthy volunteers.

Health condition
Health condition(s) or problem(s) studied:
Parkinson's Disease 337144 0
Condition category
Condition code
Neurological 333564 333564 0 0
Parkinson's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
This is a Phase 1, single center, open-label, randomized, single-dose, 2-period, crossover study in healthy adult participants comparing 2 formulations of GT-02287 for oral use.

In this study, all participants will receive GT-02287 on two occasions, one week apart, in fasting condition. On one occasion, participants will receive GT-02287 as an oral suspension of powder mixed in a Vitamin E derivative and water (reference formulation), and on the other occasion participants will receive GT-02287 as an oral suspension of nanocrystal granules mixed in water (Test formulation). The sequence in which the participants will receive the two GT-02287 formulations is assigned through a randomisation list.

Participants will provide informed consent and undergo screening for the study no more than 28 days prior to Day 1. Eligible participants will check in to the clinical research unit (CRU) on Day -1, the day prior to first dosing. All participants will attend a Follow-up Visit, scheduled 7-13 days after their last dose of GT-02287, or an early termination visit.

- Period 1: Study participants will be randomly assigned to receive on Day 1 a single dose of 10 mg/kg GT-02287 as an oral suspension of powder mixed in a Vitamin E derivative and water or as an oral suspension of nanocrystal granules mixed in water.

- Period 2 will begin on Day 7, the day prior to dosing, when participants will undergo Period 2 baseline assessments. On Day 8, they will receive a single dose of the other formulation of GT-02287.

Participants will be confined in the CRU from Day -1 to Day 12. A 7-day washout between each dose administration will ensure that GT-02287 is not measurable at baseline prior to dosing in Period 2.

GT-02287 formulations will be administered in a fasted state and study participants will fast overnight for at least 10 hours before their dose in the morning of the two dosing days, followed by an additional 4-hour postdose fast. Water will be allowed as often as desired except for the intervals from 1 hour before dosing to 1 hour after dosing.

Individual doses will be provided for participants by CRU staff on the day of administration in a suitable container. All participants will receive GT-02287 formulations at the study site under the surveillance of appropriate study personnel.
Intervention code [1] 330864 0
Treatment: Drugs
Comparator / control treatment
The comparator / control arm will be the reference formulation.

The reference formulation will be provided as a micronized powder for oral suspension in vehicle (VIT-E TPGS 2.5% w/w in water) as a single dose of 10 mg/kg GT-02287. This will be provided for participants by CRU staff on the day of administration as oral suspension in 100 mL amber glass bottles with a polypropylene top seal and screw cap.
Control group
Active

Outcomes
Primary outcome [1] 341169 0
To assess the relative bioavailability (BA) between 2 formulations of GT-02287 (Test and Reference) after a single dose in healthy adult participants.
Timepoint [1] 341169 0
Blood sampling for PK of GT-02287 at the following timepoints of both periods: Day 1 and Day 8 pre-dose, and at 0.25, 0.5, 0.75, 1, 1.5, 2, 4, 6, and 12 hours post-dose); Day 2 and Day 9 at 24 hours post-dose; Day 3 and Day 10 at 48 and 60 hours post-dose; and Day 4 and Day 11 at 84 hours post-dose. Plasma PK parameters of GT-02287 will be calculated for Period 1 and 2, including: Cmax, AUCinf, AUClast, tmax, t1/2, CL/F, Vz/F, and other parameters, as appropriate.
Secondary outcome [1] 446612 0
To evaluate the PK of single oral doses of 2 formulations of GT-02287 in plasma in healthy adult participants.
Timepoint [1] 446612 0
Blood sampling for PK of GT-02287 at the following timepoints of both periods: Day 1 and Day 8 pre-dose, and at 0.25, 0.5, 0.75, 1, 1.5, 2, 4, 6, and 12 hours post-dose); Day 2 and Day 9 at 24 hours post-dose; Day 3 and Day 10 at 48 and 60 hours post-dose; and Day 4 and Day 11 at 84 hours post-dose. Plasma PK parameters of GT-02287 will be calculated for Period 1 and 2, including: Cmax, AUCinf, AUClast, tmax, t1/2, CL/F, Vz/F, and other parameters, as appropriate.
Secondary outcome [2] 446613 0
To compare the safety and tolerability between the Test formulation and the Reference formulation in healthy adult participants.
Timepoint [2] 446613 0
- Clinical laboratory evaluations (hematology, coagulation, serum chemistries, and urinalysis): They will be evaluated at Screening, Day-1, Day 2, Day 5, Day 7, Day 9, Day 12, ET (if applicable) and Follow up. - Full physical examination, including neurological examination, to be performed at Screening and at follow-up (and ET, if applicable). Brief symptom-directed physical examinations may be performed during the study on an as-needed basis. - Vital signs will be measured at screening, on Day -1, Day 1 and Day 8 within 1 hour pre-dose, and thereafter paired with PK samples, i.e., at 0.25, 0.5, 0.75, 1, 1.5, 2, 4, 6, and 12 hours post-dose; on Day 2 and Day 9 at 24 hours post-dose; Day 3 and Day 10 at 48 and 60 hours post-dose; and Day 4 and Day 11 at 84 hours post-dose. - 12-lead electrocardiograms (ECGs) will be taken at Screening, Day -1, Day 1, Day 5, Day 7, Day 8, Day 12 and ET (if applicable). - Columbia Suicide Severity Rating Scale (C-SSRS) will be collected at Screening, Day -1, Day 2, Day 7, Day 9 and ET (if applicable).

Eligibility
Key inclusion criteria
A participant will be eligible for study participation if they meet all of the following criteria:
1. Participant must be able and willing to provide written informed consent and be willing to comply with the requirements and restrictions of the study.
2. Participant is of any sex, superior or equal to 18 and inferior or equal to 65 years of age at screening.
3. Participant has a body mass index superior or equal to 18 and inferior or equal to 35 kg/m2 at Screening.
4. Participant is not pregnant and not lactating.
5. If participant is either of childbearing potential or produces potentially viable sperm, participant must agree to use 2 forms of contraception (barrier method and a second highly effective form of birth control/contraception, as per definitions below) if engaging in potentially reproductive sexual intercourse (with a partner who produces potentially viable
sperm or is of childbearing potential, respectively) from Screening until at least 30 or 90 days respectively following final study dose administration:
• Barrier method: condom, AND
• Highly effective form of birth control/contraception: established use of oral, injected, intravaginal, or implanted hormonal contraception (combined or progestogen-only, associated with inhibition of ovulation), intrauterine device, intrauterine hormone-releasing system, tubal ligation, sterilized male partner, or true abstinent lifestyle
Note: A participant or partner is considered to be of childbearing potential if they are postmenarchal and premenopausal, unless surgically sterile (permanent sterilization methods include hysterectomy, bilateral salpingectomy and bilateral oophorectomy) or
physiologically incapable of becoming pregnant. A postmenopausal state is defined as no menses for at least 1 year without an alternative medical cause and a serum follicle-stimulating hormone (FSH) concentration superior or equal to 40 IU/L.
6. Participant must agree to not donate ova or sperm from Screening until at least 30 or 90 days respectively following final study dose administration.
7. Participant must agree to not participate in another investigational study while taking part in this study.
Minimum age
18 Years
Maximum age
65 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
A participant will be excluded from the study if they meet any of the following criteria:
1. Participant has a suspected hypersensitivity to GT-02287 or excipients.
2. Participant has aspartate transaminase (AST), alanine transaminase (ALT), alkaline phosphatase (ALP), ?-glutamyl transferase (GGT) or total bilirubin superior to 1.5 × ULN at Screening. If a participant is initially excluded due to this criterion, the assessment may be repeated once at the investigator’s discretion prior to Day -1.
3. Participant has any clinically significant history of allergies (including drug allergies, allergic eczema, or anaphylactic reactions, but excluding untreated allergic rhinitis or rhinoconjunctivitis caused by seasonal or house dust mite allergy).
4. Participant has a history of suicidal behavior or suicide attempts (an answer of “yes” to any lifetime suicidal behaviors on the C-SSRS), has had any active suicidal ideation within the last 3 months before Screening (an answer of “yes” to categories 4 or 5 of the C-SSRS), or who is at significant risk to commit suicide, as judged by the Investigator using the C-SSRS at Screening.
5. Participant has or had an active febrile illness or a symptomatic viral, bacterial (including upper respiratory infection), or fungal (non-cutaneous) infection within 2 weeks before Day -1.
6. Participant has any clinically significant abnormality following the investigator’s review of the physical examination, ECG, vital signs, or clinical laboratory tests at Screening or on Day -1. If a participant is initially excluded due to this criterion, the assessment may be repeated once at the investigator’s discretion prior to Day -1.
7. Participant has the below mean heart rate, systolic blood pressure (SBP), or diastolic blood pressure (DBP) when measured at rest in triplicate at Screening or Day -1:
• Mean heart rate inferior to 40 or superior to 100 beats per minute.
• Mean SBP superior to 160 mmHg.
• Mean DBP superior 95 mmHg.
If the mean pulse, mean SBP, or mean DBP is out of the range specified above, 1 additional triplicate measurement may be taken at Screening or Day -1. If this triplicate also gives an abnormal result, the participant should be excluded.
8. Participant has a mean QT interval corrected using Fridericia’s Formula (QTcF) of superior to 450 msec (for males) and superior to 470 msec (for females) at Screening or Day -1. If the mean QTcF exceeds the limits above, 1 additional triplicate ECG can be taken. If this triplicate also gives an abnormal result, the participant should be excluded.
9. Participant has a history of unexplained syncope, cardiac arrest, unexplained cardiac arrhythmias or torsade de pointes, structural heart disease, or a family history of long QT syndrome.
10. Participant has a history of seizures, head trauma, or loss of consciousness.
11. Participant uses any prescribed or nonprescribed drugs (including hormone replacement therapy, natural and herbal remedies, e.g., St. John’s Wort) within 2 weeks or 5 half-lives (whichever is longer) before Day -1. Vitamins and dietary supplements are prohibited within 7 days prior to Day -1. Paracetamol at doses up to 2 g/day and hormonal birth control/contraception are allowed.
12. Participant has used cigarettes (or equivalent nicotine-containing product) within 4 weeks prior to Screening or has a positive urine cotinine test at Screening or on Day -1. If a participant is initially excluded due to this criterion, the assessment may be repeated once at the investigator’s discretion prior to Day -1.
13. Participant has a history of consuming more than 14 units of alcoholic beverages per week (note: 1 unit = 250 mL of beer, 100 mL of wine, or 30 mL of spirits/hard liquor) within 6 months prior to Screening, has a history of alcoholism within 2 years prior to Screening, or has a positive alcohol breath test at Screening or Day -1. If a participant has a positive
alcohol breath test at Screening, the assessment may be repeated once at the investigator’s discretion prior to Day -1. A positive test on Day 1 is exclusionary.
14. Participant has excessive caffeine consumption (defined as superior to 4 cups per day of caffeinated coffee, tea, soda, energy drinks and/or other beverages containing caffeine, or equivalent in chocolate, supplements, or other sources of caffeine) within 2 weeks prior to Day -1.
15. Participant is unable to refrain from consumption of grapefruit, grapefruit juice, Seville oranges, pomegranate, tangelo, star fruit, or pomelo fruit within 7 days prior to Day -1 and for the duration of the study.
16. Participant has used amphetamines, barbiturates, benzodiazepine, methamphetamine, phencyclidine, methadone, tricyclic antidepressants, cannabinoids, cocaine, or opiates (drugs of abuse) within 3 months before Day -1 or has a positive urine drug screen at Screening or Day -1. If a participant has a positive urine drug screen at Screening, the assessment may be repeated once at the investigator’s discretion prior to Day -1.
17. Participant has used medications known to interfere with the CYP3A4 and/or P-gp metabolic pathways during the 3 months prior to Day -1.
18. Participant has used dietary supplements or herbal remedies known to interfere with the CYP3A4 and/or P-gp metabolic pathways during the 4 weeks prior to Day -1.
19. Participant has had any significant blood loss (superior or equal to 450 mL), donated 1 unit (superior or equal to 450 mL) of blood, or received a transfusion of any blood or blood products within 60 days before Day -1, or has donated plasma within 7 days before Day -1.
20. Participant has a positive serology test for hepatitis B surface antigen (HBsAg), hepatitis C virus antibodies (anti-HCV), or human immunodeficiency virus 1 and 2 antibodies (anti- HIV-1, anti-HIV-2).
21. Participant has a history of malabsorption, inflammatory bowel disease, pancreatitis, irritable bowel disease, cholecystectomy, Gilbert’s Syndrome, or any other clinically relevant disorder which may impact the absorption, metabolism, or elimination of
GT-02287.
22. Participant has any history or evidence of any clinically significant cardiovascular, gastrointestinal, endocrinologic, hematologic, hepatic, immunologic, metabolic, urologic, pulmonary, neurologic, dermatologic, psychiatric, renal, and/or other major disease or malignancy, as judged by the investigator or designee. A history of childhood asthma that is resolved (in the opinion of the investigator) is not exclusionary.
23. Participant has current or recent history (within the past 6 months) of recurrent headache (e.g., tension-type, cluster, or migraine with a frequency of superior or equal to 2 episodes per month and requiring treatment), which, in the opinion of the investigator, precludes participation in the study.
24. Participant has a history of severe depression, or mild/moderate depression lasting more than or equal to 3 months, requiring hospitalization and/or pharmacological therapy, or recent history (within the past 6 months of any other psychiatric condition which, in the opinion of the investigator, precludes participation in the study.
25. Participant’s estimated glomerular filtration rate (eGFR) (calculated based on the modified Cockcroft-Gault formula) is inferior to 60 mL/min/1.73 m2.
26. Participant has participated in any interventional clinical study or has been treated with any investigational drugs (including GT-02287) within 1 month or 5 half-lives, whichever is longer, before Screening. Previous participation in a GT-02287 clinical study prior to this period is not exclusionary.
27. Participant is an employee of the Gain Therapeutics group, or any vendor involved in the study.

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)
Allocation is not concealed
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
The randomization schedule will be computer generated using a permuted block algorithm and will randomly allocate IP to randomization numbers. The randomization numbers will be assigned to participants just before being dosed fo the first time in the study.

In this crossover study, participants who meet study entry criteria will be randomized to Sequence A or Sequence B in a 1:1 ratio upon admission to the CRU:
• Sequence A: Test formulation (Period 1), Reference formulation (Period 2)
• Sequence B: Reference formulation (Period 1), Test formulation (Period 2)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Crossover
Other design features
Phase
Phase 1
Type of endpoint/s
Bio-availability
Statistical methods / analysis

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
16/06/2025
Actual
Date of last participant enrolment
Anticipated
17/07/2025
Actual
Date of last data collection
Anticipated
31/07/2025
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA
Recruitment hospital [1] 27802 0
CMAX Clinical Research Pty Ltd - Adelaide
Recruitment postcode(s) [1] 43994 0
5000 - Adelaide

Funding & Sponsors
Funding source category [1] 318753 0
Commercial sector/Industry
Name [1] 318753 0
Gain Therapeutics, Inc.
Country [1] 318753 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Gain Therapeutics, Australia Pty Ltd (local Sponsor)
Address
Country
Australia
Secondary sponsor category [1] 321193 0
None
Name [1] 321193 0
Address [1] 321193 0
Country [1] 321193 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 317367 0
Bellberry Human Research Ethics Committee A
Ethics committee address [1] 317367 0
Ethics committee country [1] 317367 0
Australia
Date submitted for ethics approval [1] 317367 0
02/04/2025
Approval date [1] 317367 0
09/05/2025
Ethics approval number [1] 317367 0

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

Contacts
Principal investigator
Name 140826 0
Dr Michele de Sciscio
Address 140826 0
CMAX Clinical Research Ground Floor, 21-24 North Terrace Adelaide, SA 5000
Country 140826 0
Australia
Phone 140826 0
+61 422 447 902
Fax 140826 0
Email 140826 0
[email protected]
Contact person for public queries
Name 140827 0
Michele de Sciscio
Address 140827 0
CMAX Clinical Research Ground Floor, 21-24 North Terrace Adelaide, SA 5000
Country 140827 0
Australia
Phone 140827 0
+61 422 447 902
Fax 140827 0
Email 140827 0
[email protected]
Contact person for scientific queries
Name 140828 0
Michele de Sciscio
Address 140828 0
CMAX Clinical Research Ground Floor, 21-24 North Terrace Adelaide, SA 5000
Country 140828 0
Australia
Phone 140828 0
+61 422 447 902
Fax 140828 0
Email 140828 0
[email protected]

Data sharing statement
Will the study consider sharing individual participant data?
No


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

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No documents have been uploaded by study researchers.

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