Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12625000452493
Ethics application status
Approved
Date submitted
9/04/2025
Date registered
14/05/2025
Date last updated
14/05/2025
Date data sharing statement initially provided
14/05/2025
Type of registration
Prospectively registered

Titles & IDs
Public title
A Phase 1 Randomized, Double-Blind, Placebo-Controlled Study With A Food Effect Assessment To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Single Ascending Doses And Multiple Ascending Doses Of PX578 In Healthy Adult Participants (Part C)
Scientific title
A Phase 1 Randomized, Double-Blind, Placebo-Controlled Study With A Food Effect Assessment To Evaluate The Safety, Tolerability, And Pharmacokinetics Of Single Ascending Doses And Multiple Ascending Doses Of PX578 In Healthy Adult Participants (Part C)
Secondary ID [1] 313865 0
PX578-001
Universal Trial Number (UTN)
Trial acronym
Linked study record
ACTRN12625000230459p is Parts A & B of a 3-part study. This registration is for Part C.

Health condition
Health condition(s) or problem(s) studied:
Mitochondrial disease 336531 0
Condition category
Condition code
Human Genetics and Inherited Disorders 333039 333039 0 0
Other human genetics and inherited disorders
Neurological 333040 333040 0 0
Other neurological disorders
Musculoskeletal 333041 333041 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Part C: Single dose, food effect, 2-way crossover design evaluation of PX578 administered in the fed and fasted state to healthy adult volunteers. This sub study (Part C) is part of a 3-part Phase 1 Healthy Volunteer study to assess the safety, tolerability and pharmacokinetics (PK) of PX578 in healthy participants. The Part A single ascending dose (SAD) and Part B multiple ascending dose (MAD) study has been registered separately.

The dose will be determined by the Safety Review Committee (SRC) based on safety, tolerability and PK data from previous cohorts in Part A and/or Part B up to a maximum dose of 1000 mg. Participants who have completed Part A or Part B will be eligible to enrol in Part C if they meet eligibility criteria (there must be at least 30 days between the last dose of study drug in Part A/Part B and Day 1 of Part C).

In Period 1 participants will be randomised to 1 of 2 open-label PX578 administration sequences, fasting or fed (standard high fat meal). On the morning of Day 1 each participant will receive a single dose of PX578. Participants randomised to Sequence 1 will be dosed fasted (required to fast for 10 hours pre-dose and 4 hours post-dose). Participants randomized to Sequence 2 will be dosed fed (participants will need to consume at least 75% of a standard high fat meal prior to receiving the dose)

In Period 2 participants will switch to the alternative fed/fasting condition to receive a single dose of PX578 following a washout of approximately 48 hours (actual duration will be determined based on data from Part A). The contents of the high fat meal will be dependent on individual dietary restrictions, participants need to consume at least 75% of the high fat meal. As per the protocol, participants dosed in a fed state will follow FDA guidance of a standardised high fat meal (see the FDA Guidance for Industry; Assessing the Effect of Food on Drugs in INDs and NDAs – Clinical Pharmacology Considerations [June 2022]/, accessed online at https://www.fda.gov/media/121313/download).

Participants will be dosed at the site and will receive study drug directly from the Investigator or designee, under medical supervision. Drug administration will be recorded and any discrepancies with the dosing regimen will be documented and explained in the eCRF and the source documents. The dose of study drug and study participant identification will be confirmed at the time of dosing by a member of the study site staff other than the person administering the study drug.
Intervention code [1] 330452 0
Treatment: Drugs
Comparator / control treatment
PX578 oral capsules dosed in a fasted state will serve as the control treatment.
Control group
Active

Outcomes
Primary outcome [1] 340581 0
To assess the effect of food on plasma PK profile of PX578 in healthy adult participants (Part C Periods 1 & 2)
Timepoint [1] 340581 0
Blood samples will be collected pre-dose Day 1 - 0.25, 0.5, 1. 2, 3, 4, 5, 6, 8. 12 and 16 hrs post-dose. Day 2 - 24, 30 and 36 hrs post-dose and Day 3 48 hrs post-dose.
Secondary outcome [1] 445461 0
To evaluate the presence and PK of potential PX578 metabolites in plasma samples from healthy adult participants
Timepoint [1] 445461 0
Blood samples will be collected pre-dose Day 1 - 0.25, 0.5, 1. 2, 3, 4, 5, 6, 8. 12 and 16 hrs post-dose. Day 2 - 24, 30 and 36 hrs post-dose and Day 3 48 hrs post-dose.

Eligibility
Key inclusion criteria
1. Must provide written informed consent before any study-related activities are carried out and must be able to understand the full nature and purpose of the trial. including possible risks and adverse effects.
2. Healthy male or female participant, 18-55 years of age at the time of signing informed consent.
3, Both male and female participants must agree to follow contraceptive requirements and gamete
donation restrictions during the Screening period and for 90 days following the last dose of study drug.
4. A body mass index (BMI) of between 18 and 32 kg/m2 at Screening with a minimum body weight of 45 kg.
5. Hematocrit or hemoglobin levels within normal limits for age and sex at Screening and on Day -1, prior to dose administration.
6. Be willing and able to comply with all study assessments and adhere to the protocol schedule, procedures, and restrictions
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. Clinically-significant medical history or ongoing chronic illness that, in the opinion of the study Investigator, would jeopardize the safety of the participant or compromise the integrity of the data derived from their participation in this study.
2. Clinically-significant infection of any kind requiring systemic antimicrobial therapy or hospitalization within 4 weeks prior to the first dose of study drug.
3. Clinically-significant abnormal findings at Screening on physical examination, ECG, or laboratory testing in the opinion of the Investigator.
4. OTcF > 470 ms (females) or > 450 ms (males) at Screening or Day -1 based on the mean of triplicate ECGs.
5. Liver test results during Screening or at check-in day (Day -1) that are above the upper limit of normal (ULN) for gamma-glutamyl transferase, bilirubin (total), aspartate aminotransferase (AST) or alanine aminotransferase (ALT), unless a diagnosis of Gilbert syndrome. For history of Gilbert syndrome, the limit is extended to 2-fold above the ULN.
6. Positive test results for hepatitis B surface antigen (HBsAg), hepatitis C (HCV) antibody or human immunodeficiency virus (HIV) antibody at Screening. Participants who are positive for HCV antibody but do not have active HCV (ie, test negative for HCV RNA on a polymerase chain reaction (PCRI test) are eligible for the study.
7, Estimated glomerular filtration rate (eGFR) <90 ml/min/1.73 m2 at Screening using the Chronic Kidney Disease Epidemiology Collaboration (CKD-EPI) 2009 formula.
8. History of active malignancy within 3 years prior to Screening, except basal cell carcinoma of the skin, curatively resected squamous cell carcinoma of the skin, cervical carcinoma in situ curatively treated or low-grade prostate adenocarcinoma for which appropriate management is observation alone.
9. History of surgery or hospitalization within 3 months prior to Screening, or surgery planned during the study.
10. History of hypersensitivity, allergic or anaphylactic reactions to the study drug ingredients or other therapeutic proteins.
11. History of drug or alcohol abuse (as defined by the Investigator) within 5 years prior to Screening, a positive urine drug or alcohol test at Screening or Day -1, or an unwillingness to abstain from drugs of abuse throughout the study, or from alcohol for 48 hours prior to Day -1 and for the duration of the study.
12. Participant smokes more than 5 cigarettes or equivalent nicotine-containing products per day, and/ or 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 study.
Note: 1 average cigar equals approximately 5 average cigarettes: 1 average pipe session equals approximately 5 average cigarettes: 1 average nicotine liquid vape session equals 1 average e-cigarette equals 1 average cigarette.
13. Has a positive urine screen for cotinine at Day -1.
14. Unwilling to abstain from caffeine containing products for 24 hours prior to Day -1 and for the duration of the study.
15. Unwilling to abstain from eating cruciferous vegetables, charcoal-grilled meats, and poppy seeds for 48 hours prior to Day -1 and for the duration of the study.
16. Unwilling to refrain from consumption of Seville oranges, grapefruit or grapefruit juice, (pomelos, exotic citrus fruits, grapefruit hybrids, or their juices) within 14 days of Day -1 and for the duration of the study.
17 Substance abuse, mental illness, or any reason that makes it unlikely in the judgment of the Investigator for the participant to be able to comply fully with study procedures.
18. Females who are pregnant (positive pregnancy test at Screening or during the study), lactating, or, if having reproductive potential. are considered potentially unreliable with respect to contraceptive practice.
19. Use of prescription medication (with the exception of oral contraceptives) within 7 days or 5 half-lives prior to initiation of study drug dosing (whichever is longer) and for the duration of the study.
20. Participant is using medications which are exogenous modulators of CYP pathways as identified in the Flockhart table (Flockhart Table)
21. Participant has taken over-the-counter medications including herbal/dietary supplements, protein powders/creatinine, or homeopathic preparations within 7 days prior to initiation of study drug dosing (within 28 days of Day -1 or 10 half lives, whichever is longer, for St John's Wort). Prophylactic doses of vitamin/mineral supplements and occasional paracetamol or ibuprofen are allowed.
22. 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 Day 1.
23. Administration of any live, attenuated vaccines within 30 days and all other vaccinations within 14 days prior to initiation of study drug dosing.
24. Participant has donated or lost greater than or equal to 400 ml blood in the 6 weeks prior to Day 1.
25. Participant is unable to swallow oral medication.
26. Participant has an inability to follow a standardized meal schedule and diet or inability to fast. as required by the study protocol.
NOTE: Potential study participants with abnormal laboratory values may be rescreened once for specific laboratory tests within the Screening period (up to 4 weeks prior to dosing) before being designated a Screen failure. Repeat values within the normal range must be confirmed at Day -1 for inclusion.

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)
Participants to be dosed in Part C will be assigned a randomization number prior to dose administration on Day 1 in accordance with the randomization schedule. The randomization schedules will be maintained under controlled access.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
The allocation to PX578 dosed under fed or fasted conditions will be performed using a block randomization algorithm and will be documented in the study randomization schedule.
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



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

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
1/07/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment outside Australia
Country [1] 26858 0
New Zealand
State/province [1] 26858 0
Christchurch

Funding & Sponsors
Funding source category [1] 318332 0
Commercial sector/Industry
Name [1] 318332 0
Pretzel Therapeutics Inc.
Country [1] 318332 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
Pretzel Therapeutics Inc.
Address
Country
United States of America
Secondary sponsor category [1] 320727 0
Commercial sector/Industry
Name [1] 320727 0
Avance Clinical Pty Ltd
Address [1] 320727 0
Country [1] 320727 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316968 0
Central Health and Disability Ethics Committee
Ethics committee address [1] 316968 0
Ethics committee country [1] 316968 0
New Zealand
Date submitted for ethics approval [1] 316968 0
13/12/2024
Approval date [1] 316968 0
03/03/2025
Ethics approval number [1] 316968 0

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

Contacts
Principal investigator
Name 139598 0
Dr Christopher Wynne
Address 139598 0
New Zealand Clinical Research, 3 Ferncroft Street, Grafton, Auckland 1010, New Zealand
Country 139598 0
New Zealand
Phone 139598 0
+64 272443963
Fax 139598 0
Email 139598 0
[email protected]
Contact person for public queries
Name 139599 0
Christopher Wynne
Address 139599 0
New Zealand Clinical Research, 3 Ferncroft Street, Grafton, Auckland 1010, New Zealand
Country 139599 0
New Zealand
Phone 139599 0
+64 272443963
Fax 139599 0
Email 139599 0
[email protected]
Contact person for scientific queries
Name 139600 0
Christopher Wynne
Address 139600 0
New Zealand Clinical Research, 3 Ferncroft Street, Grafton, Auckland 1010, New Zealand
Country 139600 0
New Zealand
Phone 139600 0
+64 272443963
Fax 139600 0
Email 139600 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

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.