Register a trial

Please note the ANZCTR will be unattended from Friday 20 December 2024 for the holidays. The Registry will re-open on Tuesday 7 January 2025. Submissions and updates will not be processed during that time.

Registering a new trial?

To achieve prospective registration, we recommend submitting your trial for registration at the same time as ethics submission.

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this information for consumers
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06732180




Registration number
NCT06732180
Ethics application status
Date submitted
9/12/2024
Date registered
13/12/2024

Titles & IDs
Public title
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Parkinson's Disease
Scientific title
An Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of GT-02287 in Participants with Parkinson's Disease with or Without a Pathogenic GBA1 Mutation
Secondary ID [1] 0 0
GANX-001-V102
Universal Trial Number (UTN)
Trial acronym
Linked study record

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

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - GT-02287

Experimental: GT-02287. All participants receive once daily GT-02287 administration (13.5 mg/kg/day) -


Treatment: Drugs: GT-02287
Dose of 13.5 mg/kg/day (plus/minus 2 mg/kg/day based on body weight) to be administered orally once a day for 90 days. Dosage form: powder in sachet (200 mg of GT-02287 per sachet) for reconstitution with a suspending agent
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence, nature, relationship to investigational product (IP), and severity of adverse events (AEs)
Timepoint [1] 0 0
From first dose to Day 105
Primary outcome [2] 0 0
Incidence of clinically significant findings for clinical laboratory evaluations, physical and neurological examinations, body weight , vital signs measurements, 12-lead o 12-lead electrocardiograms (ECGs), Columbia Suicide Severity Rating Scale (C-SSRS)
Timepoint [2] 0 0
From first dose to Day 105
Secondary outcome [1] 0 0
Maximum Plasma Concentration [Cmax]
Timepoint [1] 0 0
Day 1 and Day 90
Secondary outcome [2] 0 0
Area under the curve [AUC]
Timepoint [2] 0 0
Day 1 and Day 90
Secondary outcome [3] 0 0
Time to reach Cmax [Tmax]
Timepoint [3] 0 0
Day 1 and Day 90
Secondary outcome [4] 0 0
Elimination half life [T1/2]
Timepoint [4] 0 0
Day 90- Day 93
Secondary outcome [5] 0 0
Apparent Clearance (CL/F)
Timepoint [5] 0 0
Day 90- Day 93
Secondary outcome [6] 0 0
Apparent Volume of Distribution (Vz/F)
Timepoint [6] 0 0
Day1 and Day 90- Day 93

Eligibility
Key inclusion criteria
* Able and willing to provide written informed consent and be willing to comply with the requirements and restrictions of the study
* Any sex, =30 and =85 years of age
* Diagnosis of PD based on MDS criteria
* Within 7 years of PD diagnosis
* Body mass index of =18 and =40 kg/m2, and a body weight =45 kg and =120 kg
* Willing to provide a blood sample for PD-related genetic testing
* Hoehn & Yahr 1-3, inclusive
* No severe motor fluctuations or disabling dyskinesias based on the investigator's clinical assessment
* Naïve to pharmacological treatment for PD or on stable PD medication for =3 months prior to Screening, including =4 weeks at the same dose(s) immediately before Screening
* Not pregnant or breastfeeding
* 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 defined in the protocol) if engaging in potentially reproductive intercourse (with a partner who produces potentially viable sperm or is of childbearing potential, respectively)
* Agreeing to not participate in another investigational study while taking part in this study
* For participants with known GBA1 mutations, presence of a GBA1 mutation that has been associated with an increased risk of PD
Minimum age
30 Years
Maximum age
85 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Other neurological disorders, including but not limited to Alzheimer's disease, amyotrophic lateral sclerosis, frontotemporal dementia, progressive supranuclear palsy, corticobasal syndrome, Huntington's disease, multiple system atrophy, dementia with Lewy bodies, secondary (e.g. drug-induced) parkinsonism, multiple sclerosis, or epilepsy
* A history of Gaucher disease or homozygous for a GBA1 pathogenic variant known to be associated with GD or compound heterozygous for 2 alleles that are known to be associated with GD.
* Known PD-associated LRRK2 pathogenic variant or other PD-associated genetic mutations other than GBA1
* Dementia or a moderate cognitive impairment (score =17 on the Montreal Cognitive Assessment)
* Hypersensitivity to GT-02287 or any of its excipients
* Concomitant medications metabolized primarily by cytochrome P450 3A4 (CYP3A4) that have a narrow therapeutic window, concomitant medications that are substrates of breast cancer resistance protein and/or P-glycoprotein and that have a narrow therapeutic window, concomitant medications that are potent inhibitors or inducers of CYP3A4
* Use of dopamine antagonists (antipsychotics) or anticholinergic medications
* Concomitant disease including, but not limited to cardiovascular conditions, diabetes, autoimmune disease, cancer, active infectious disease, psychotic disorders and symptoms, depressive symptoms, drug and/or alcohol misuse as defined in the protocol
* Malabsorption or relevant disorder which may impact the absorption of GT-02287
* Clinically significant abnormalities in laboratory test
* Contraindications to lumbar puncture (LP)
* Blood donation >500 mL within 3 months
* Unable to comply with restrictions on food products, smoking, and /or alcohol use as defined in protocol
* participation in any interventional clinical study within 3 months or 5 half-lives, whichever is longer, prior to Screening

Study design
Purpose of the study
Treatment
Allocation to intervention
Not applicable
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
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
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
15/01/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
30/11/2025
Actual
Sample size
Target
20
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
NSW,QLD,SA,VIC
Recruitment hospital [1] 0 0
St Vincent's Hospital Sydney - Darlinghurst
Recruitment hospital [2] 0 0
Southern Neurology - Kogarah
Recruitment hospital [3] 0 0
Westmead Hospital - Westmead
Recruitment hospital [4] 0 0
Princess Alexandra Hospital - Woolloongabba
Recruitment hospital [5] 0 0
CMAX - Adelaide
Recruitment hospital [6] 0 0
Alfred Health - Melbourne
Recruitment hospital [7] 0 0
Royal Melbourne Hospital - Parkville
Recruitment postcode(s) [1] 0 0
- Darlinghurst
Recruitment postcode(s) [2] 0 0
- Kogarah
Recruitment postcode(s) [3] 0 0
- Westmead
Recruitment postcode(s) [4] 0 0
- Woolloongabba
Recruitment postcode(s) [5] 0 0
- Adelaide
Recruitment postcode(s) [6] 0 0
- Melbourne
Recruitment postcode(s) [7] 0 0
- Parkville

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Gain Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

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

Contacts
Principal investigator
Name 0 0
Chief Medical Officer
Address 0 0
Gain Therapeutics, Inc.
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Gain Therapeutics Clinical Operations
Address 0 0
Country 0 0
Phone 0 0
+41919211131
Fax 0 0
Email 0 0
info@gaintherapeutics.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


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/NCT06732180