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Trial registered on ANZCTR


Registration number
ACTRN12625000428460
Ethics application status
Approved
Date submitted
10/01/2025
Date registered
8/05/2025
Date last updated
8/05/2025
Date data sharing statement initially provided
8/05/2025
Type of registration
Retrospectively registered

Titles & IDs
Public title
An Ascending, Single and Multiple Dose(s), Double-Blind, Randomized, Placebo Controlled Study Assessing the Safety, Tolerability, and Pharmacokinetics of Intravenous OV350 in Healthy Male and Female Participants
Scientific title
An Ascending, Single and Multiple Dose(s), Double-Blind, Randomized, Placebo Controlled Study Assessing the Safety, Tolerability, and Pharmacokinetics of Intravenous OV350 in Healthy Male and Female Participants
Secondary ID [1] 313580 0
OV350-IV-24-001
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Parkinson's Disease 336109 0
Condition category
Condition code
Neurological 332663 332663 0 0
Dementias
Neurological 332662 332662 0 0
Parkinson's disease

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
The study will be a Phase 1, single-center, randomized, double-blind, placebo-controlled, inpatient study of the safety and tolerability of single and multiple ascending dose(s) of intravenous OV350 in healthy male and female participants.

The study is comprised of two parts: Part A, approximately 5 single ascending dose (SAD) administered as a 10-minute infusion starting at 50mg; and Part B, approximately 4 multiple ascending doses (MAD) administered once a day for 7 days. SAD and MAD cohorts can be conducted in parallel but dosing in a MAD cohort will only be initiated after the dose is cleared in a SAD cohort. The study sponsor, the study principal investigator (PI), medical monitor, and a pharmacokineticist will review data summaries from each cohort and recommend whether to proceed with dose escalation or MAD cohort dosing.

The study will assess the safety profile of intravenous OV350 on ECG parameters, VS, physical and neurological examinations, hematology and chemistry laboratory results and adverse events (AEs). In addition, neurophysiologic PD variables in the study are quantitative EEG, particularly the timing and extent of EEG changes in relation to the doses administered.

In each cohort, a sentinel group of 2 participants (1 active: 1 placebo) will be randomized and dosed ahead of the rest of the cohort. These 2 participants will be monitored for 2 days until the end of the in-patient portion of Part A (Day 3) and for 8 days for the in-patient portion of Part B (Day 9) before their available data will be reviewed by the Principal Investigator. The remaining 6 participants will be dosed once the Principal Investigator has concluded that it is safe to proceed with the rest of the cohort.

Participants involved in SAD cohorts will not be eligible for inclusion in MAD cohorts, ensuring unique enrollment across both segments.

Monitoring visits by the CRO to the study site will be made periodically during the study to ensure that all aspects of the protocol are followed.
Intervention code [1] 330174 0
Treatment: Drugs
Comparator / control treatment
Matching Placebo (Saline) intravenous infusion each dosing strength
Part A: single intravenous infusion administered with infusion rate determined by the previous cohort for later cohorts.
Part B: repeated administration for 7 days, given once daily as a single infusion with the infusion rate determined by the previous cohort for later cohorts.
Control group
Placebo

Outcomes
Primary outcome [1] 340434 0
To assess the safety and tolerability of multiple doses of intravenous OV350 in healthy male and female participants
Timepoint [1] 340434 0
Vital signs (blood pressure and Heart Rate - Screening, baseline, D1, -D9 and D14 post-dose, C-SSRS Screening, baseline, and D9 post-dose, 12-lead ECGs Screening, baseline, D1, D2, D3, D7, D8, D9 and D14 post-dose, Bloods- Haematology, Coagulation, Chemistry and Urine Analysis- Screening, baseline, D1, -D9 and D14 post-dose,
Primary outcome [2] 340205 0
To assess the safety and tolerability of single (Part A) dose of intravenous OV350 in healthy male and female participants
Timepoint [2] 340205 0
Vital signs (blood pressure and Heart Rate - Screening, baseline, D1, D2, D3, and D7 post-dose, C-SSRS Screening, baseline, D3, and D7 post-dose, 12-lead ECGs Screening, baseline, D1, D2, D3, and D7 post-dose, Bloods- Haematology, Coagulation, Chemistry and Urine Analysis- Screening, baseline, D1, D2, D3, and D7 post-dose,
Secondary outcome [1] 443289 0
To characterize the plasma and urinary PK of intravenous OV350 in healthy male and female participants after single (Part A) intravenous doses.
Timepoint [1] 443289 0
Blood PK: Time points are: baseline, 5 min, 10 min, 15 min, 20 min, 40 min, 1, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48 hours Urine PK: Timepoints are: pre-dose and [0-6], [>6-12] [>12-24] and [>24-48] hours post-dose
Secondary outcome [2] 444343 0
To characterize the plasma and urinary PK of intravenous OV350 in healthy male and female participants after multiple intravenous doses.
Timepoint [2] 444343 0
Blood PK: Time points to be collected on Day1 and Day 7 are: baseline, 5 min, 10 min, 15 min, 20 min, 40 min, 1, 2, 3, 4, 6, 8, 10, 12, 24, 36, 48 hours. Blood samples for D2-D5 will be collected just prior to infusion start Urine PK: Timepoints are: Day 1 pre-dose and [0-6], [>6-12] [>12-24] and [>24-48] hours post-dose and on Day 7 [0-6], [>6-12] [>12-24] and [>24-48] hours post-dose.

Eligibility
Key inclusion criteria
1. Participant must give written informed consent prior to participation in the study.
2. Participant agrees not to post any of the participant's personal or medical data or information related to the study on any website, message board(s), online groups, or social media website (eg, Facebook, Instagram, X etc.) until notified that the study is completed.
3. Participant must be willing and able to comply with the study procedures (including diet) and visit schedules and must be able to follow verbal and written instructions.
4. Male and female participants aged 18 to 55 years at the time of informed consent.
5. Weighs at least 50 kg and body mass index (BMI) greater than or equal to 18.0 and <35.0 kg/m2 at screening. For the last cohort in MAD (CSF lumbar puncture [LP]) BMI greater than or equal to 18.0 and <30.0 kg/m2 at screening.
6. Continuous nonsmoker (less than 5 nicotine containing products per week) for longer than 1 year, who has not used nicotine containing products (including vaping) for at least 7 days prior to the first dosing and will remain abstinent throughout the duration of the study. Urine cotinine test will be conducted on D-1 and may be repeated during the trial at the discretion of the PI.
7. Resting supine systolic blood pressure (SBP) 90 to 145 mmHg (inclusive) and diastolic blood pressure (DBP) no higher than 90 mmHg at Screening and Check-In (D-2 or D-1) (to be taken after about 10 minutes in supine position).
8. A 12-lead ECG with no clinically significant abnormalities as deemed by the investigator and Fredericia corrected QT interval (QTcF) interval less than or equal to 470 milliseconds (females) and less than or equal to 450 milliseconds (males) at Screening and Check-In (D-2 or D-1; to be taken after about 10 minutes in supine position).
9. Resting supine or seated pulse rate between 45 and 100 beats per minute at Screening and Check-In (D-2 or D-1).
10. Normal physical examination and laboratory investigations within the normal reference range, or if outside of the reference ranges, deemed not clinically significant in the opinion of the investigator.
11. Normal baseline EEG prior to dosing
12. Willing to abstain from illicit drugs, alcohol, and nicotine products/tobacco use during study participation.
13. Participant must be willing to stay within the clinical research unit for the duration of the in-patient study period and return for all additional study visits as specified by the protocol.
14. Female participants who are sexually active with the opposite sex and of childbearing potential (defined as first menarche through post-menopause or permanent sterilization) must agree to use a highly effective method of birth control from time of screening
15. Female participants not of childbearing potential due to surgical sterilization (at least six weeks after hysterectomy, bilateral salpingectomy, or bilateral oophorectomy) confirmed by medical history, or menopause.
16. Male participants (post-pubertal unless permanently sterilized by bilateral orchidectomy) must agree to use male contraception (condom) combined with the use of a highly effective method of contraception by the female partner, and/or male abstinence from time of screening and for 90 days following the last dose of study drug.
Minimum age
18 Years
Maximum age
55 Years
Sex
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
1. History or presence of gastritis, gastrointestinal tract disorder, gastric bypass surgery, or hepatic disorder or other clinical condition which, in the opinion of the Investigator or designee, may affect the absorption, distribution, metabolism, or elimination of study drug. Cholecystectomy and diagnosis of Gilberts syndrome is also exclusionary.
2. Significant history or clinical manifestation of any metabolic, allergic, dermatological, hepatic, renal, hematological, pulmonary, cardiovascular (including family history of heart attack, or long QT syndrome), gastrointestinal, neurological (including migraine and depression), respiratory (childhood asthma), endocrine, or psychiatric disorder, as determined by the Investigator (or designee).
3. Renal clearance defined as an average of <60ml/min using the Cockroft & Gault formula.
4. History or presence of alcohol or drug abuse within the past 2 years prior to Screening visit as determined by the Investigator (or designee). Drug and alcohol tests will be conducted on D-1 and may be repeated during the trial at the discretion of the PI.
5. History or presence of hypersensitivity or idiosyncratic reaction to the study drug or related compounds.
6. Risk of suicide according to the Investigator’s clinical judgment (eg, per C-SSRS) or has made a suicide attempt in the previous year prior to Screening visit.
7. Unable to refrain from or anticipate the use of:
a. Any (oral and non-oral) systemic drug, including prescription and nonprescription medications, herbal remedies, or vitamin supplements within 14 days prior to the first dosing and throughout the study, including the follow-up period. After the first dose of study drug, acetaminophen (up to 2 g per 24 hours) may be administered at the discretion of the Investigator or designee.
b. Alcohol, caffeinated and dietary products such as grapefruit, Seville oranges etc. .
c. Any product, remedy, supplement, or medication containing cannabidiol (CBD), Tetrahydrocannabinol (THC) or related compounds within 30 days prior to the first dosing and throughout the study, including the follow-up period.
8. Has been on a diet incompatible with the on-study diet, in the opinion of the Investigator or designee, within the 30 days prior to the first dosing and throughout the study.
9. Participation in another clinical study within 30 days prior to the first dosing. The 30-day window will be derived from the date of the last blood collection or dosing, whichever is later, in the previous study to D1 of the current study. If the previous investigational product has a long half-life, three months or five half-lives (whichever is longer) should have passed; participation in a clinical trial involving an investigational product or nonapproved use of a drug or device (other than the investigational product used in this study), or concurrently enrolled in any other type of medical research judged not to be scientifically or medically compatible with this study.
10. Receipt of vaccination (inclusive of influenza and SARS COV2) within 14 days of dosing (D1).
11. If male, the participant intends to donate sperm during the course of this study or for 90 days following the last dose of study drug.
12. If female, the participant is pregnant or intending to become pregnant before participating in this study, during the study, and within 1 month after last dose of the study drug; or intending to donate ova during such time period; or lactating.
13. Ovid employees or Investigator site personnel where the study is being conducted and/or their immediate family.
Note: immediate family is defined as a spouse, parent, child, or sibling, whether biological or legally adopted.
14. EEG abnormalities deemed exclusionary at the discretion of the PI. Participants who have Obstructive Sleep Apnea or any other conditions that can cause daytime somnolence, including individuals who work night shifts or who are otherwise are typically nocturnal. Any other contraindications for the EEG procedure per discretion of the PI.
15. Presence or evidence of recent sunburn, scar tissue, tattoos, or open sores that in the opinion of the PI may interfere with evaluation of the study drug administration site
16. Individuals who, in the opinion of the Investigator or designee, should not participate in this study.
17. For the last cohort in MAD, participants that experienced chronic headaches as these individuals are at higher risk due to CSF Lumbar Puncture procedure.

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)
Based on the randomization schedule, the unblinded pharmacy staff will prepare sealed opaque code-break envelopes containing the treatment assignment information. If needed for intentional unblinding for safety reasons, the code-break envelope will be used in the event of an emergency if the pharmacy staff is not available.
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Treatment is randomized 1 to 1 (OV350 : placebo) in the sentinel group, and 5 to 1 (OV350 : placebo) in the rest of the cohort. A randomization schedule will be provided from the unblinded biostatistician
Masking / blinding
Who is / are masked / blinded?



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

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
7/03/2025
Date of last participant enrolment
Anticipated
30/09/2025
Actual
Date of last data collection
Anticipated
31/10/2025
Actual
Sample size
Target
72
Accrual to date
6
Final
Recruitment in Australia
Recruitment state(s)
SA

Funding & Sponsors
Funding source category [1] 318046 0
Commercial sector/Industry
Name [1] 318046 0
Ovid Therapeutics Australia Pty Ltd
Country [1] 318046 0
Australia
Primary sponsor type
Commercial sector/Industry
Name
Ovid Therapeutics Australia Pty Ltd
Address
Country
Australia
Secondary sponsor category [1] 320405 0
None
Name [1] 320405 0
Address [1] 320405 0
Country [1] 320405 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316701 0
Bellberry Human Research Ethics Committee B
Ethics committee address [1] 316701 0
Ethics committee country [1] 316701 0
Australia
Date submitted for ethics approval [1] 316701 0
13/12/2024
Approval date [1] 316701 0
28/01/2025
Ethics approval number [1] 316701 0

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

Contacts
Principal investigator
Name 138718 0
Dr Michele De Sciscio
Address 138718 0
CMAX Clinical Research Pty Ltd, Level 5 18a North Terrace Adelaide SA 5000
Country 138718 0
Australia
Phone 138718 0
+61 422447902
Fax 138718 0
Email 138718 0
[email protected]
Contact person for public queries
Name 138719 0
Matthew Jung
Address 138719 0
Ovid Therapeutics, 441 9th Avenue, NY, NY 10001
Country 138719 0
United States of America
Phone 138719 0
+1 646 875 4179
Fax 138719 0
Email 138719 0
[email protected]
Contact person for scientific queries
Name 138720 0
Julia Tsai, PhD
Address 138720 0
Ovid Therapeutics, 441 9th Avenue, NY, NY 10001
Country 138720 0
United States of America
Phone 138720 0
+1 203 623 1996
Fax 138720 0
Email 138720 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.