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Trial registered on ANZCTR


Registration number
ACTRN12621001019897
Ethics application status
Approved
Date submitted
23/06/2021
Date registered
4/08/2021
Date last updated
4/08/2021
Date data sharing statement initially provided
4/08/2021
Type of registration
Prospectively registered

Titles & IDs
Public title
R3R01 single dose study in healthy adults
Scientific title
An Exploratory Pharmacokinetic Study to Evaluate the Exposure of R3R01 after
Administration as New Polymorph in Healthy Subjects
Secondary ID [1] 304605 0
R3R01-PK-103
Universal Trial Number (UTN)
U1111-1266-8348
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alport syndrome 322515 0
Focal Segmental Glomerulosclerosis 322516 0
Condition category
Condition code
Renal and Urogenital 320152 320152 0 0
Other renal and urogenital disorders
Human Genetics and Inherited Disorders 320400 320400 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
R3R01 - Oral capsules administered once only at doses below:
Cohort A: 25 mg (1 tablet of 25 mg)
Cohort B: 100 mg (1 tablet of 100 mg)
Cohort C: 200 mg (2 tablets of 100 mg)
Cohorts will immediately follow to the next cohort once one cohort is complete. Each cohort is distinct and participants will be enrolled into only one of the above cohorts.
Tablets will given under direct supervision and mouth check ensure tablet is taken as directed.
Intervention code [1] 320953 0
Treatment: Drugs
Comparator / control treatment
No control group
Control group
Uncontrolled

Outcomes
Primary outcome [1] 328030 0
Plasma samples - To evaluate the Cmax, Tmax, t½, AUC0-24, AUClast, AUC truncated at 48 and 72 hrs(AUCt), CL/F and V/F of R3R01
Timepoint [1] 328030 0
PK samples will be taken pre-dose on Day 1 and at 2, 3, 6, 8, 10, 16 hours after dose. and once on the following days: 2, 3, 4, 5, 8, 15 and 84 post dose
Secondary outcome [1] 397267 0
Plasma samples - To evaluate the dose proportionality of R3R01 Cmax, Tmax, t½, AUC0-24, AUClast, AUC truncated at 48 and 72 hrs(AUCt), CL/F and V/F.
Timepoint [1] 397267 0
PK samples will be taken pre-dose on Day 1 and at 2, 3, 6, 8, 10, 16 hours after dose. and once on the following days: 2, 3, 4, 5, 8, 15 and 84 post dose,
Secondary outcome [2] 397268 0
To assess the safety and tolerability (review of occurrence of adverse events (by questioning of participants at each study visit), changes in physical examinations, vital signs (heart rate, temperature, blood pressure and respiratory rate), ECGs and clinical laboratory (hematology,biochemistry, urinalysis) parameters.) of a single dose of R3R01
Timepoint [2] 397268 0
Adverse events reviewed continually until Day 84, post dose, throughout the study,
Physical exams: Day -1, Day 15. Day 84 post dose
Vital signs: Day -1, Day 1, Day 2,Day 3, Day 4, Day 5, Day 8, Day 15, Day 84 post dose
ECGs Day -1, Day 1, Day 2, Day 8, Day 15, Day 84 post dose
Laboratory samples Day -1, Day 2, Day 8, Day 15, Day 84 post dose


Eligibility
Key inclusion criteria
-18 to 45 years of age
-A BMI between 18 to 32 kg/m2
-Participants of childbearing potential must agree use highly effective methods of contraception
Minimum age
18 Years
Maximum age
45 Years
Gender
Both males and females
Can healthy volunteers participate?
Yes
Key exclusion criteria
-Participation in an investigational drug/device study within 3 months
- A history of clinically significant gastro-intestinal, cardiovascular, musculoskeletal, endocrine, hematological, psychiatric, renal, hepatic, broncho-pulmonary or neurological conditions, allergic disease or lipid metabolism disorders.
- A history of clinically significant drug hypersensitivity.
-A known history of porphyria, myopathy, or an active liver disease.
-Total bilirubin exceeding 1.5-fold upper limit of normal.
-clinically significant symptoms of an infectious illness within four weeks of dosing or a history of recurrent infections.
-Positive urine, breath or blood test for drugs of abuse and alcohol
- Positive for hepatitis B, hepatitis C, or HIV 1 and 2
- Systolic blood pressure greater than 140 or less than 90 mm Hg, and diastolic blood pressure greater than 90 or less than 50 mm Hg and heart rate greater than 100 or less than 45 beats per minute.
-Personal or family history of congenital long QT syndrome.
- ECGs with QRS and/or T-wave judged by the investigator to be unfavorable
-Clinically significant abnormalities in laboratory test results
-Donation of blood over 450 mL within three months prior to screening.
- Smoker or non-smoker less than 6 months.
- Taking any drug or herbal medication that is an inhibitor or inducer of CYP450 within 4 weeks prior to the first dosing.
- Use of any prescription drug within 7 days of first dose of study drug
- Administration of live (attenuated) vaccines within 4 weeks before dosing

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised 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)
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)
Pharmacokinetics
Statistical methods / analysis
No formal statistical sample size estimation has been performed. Based on experience from previous similar studies, the sample size for this study is considered sufficient to adequately assess the study endpoints and meet the study objectives.
Twelve (12) male and twelve (12) female subjects will be assigned to 3 treatment groups
of eight (8) subjects (4 males and 4 females).
This analysis set is a subset of the safety set and includes all subjects who completed the
study treatments without major protocol deviation/violation or event likely affecting PK.
All subjects who have taken at least one dose of R3R01 will be included in the safety set.
Subjects will be analysed according to the study treatment they actually received.
The following non-compartmental PK parameters will be calculated from the individual
plasma concentration-time data by treatment using Phoenix WinNonlin (linear trapezoidal
linear/log interpolation): AUC0-tlast, AUC0-t, AUC0-inf, Cmax, tmax, tlag, CL/F, VZ/F, gammaz, t1/2

Recruitment
Recruitment status
Not yet recruiting
Date of first participant enrolment
Anticipated
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
Sample size
Target
Accrual to date
Final
Recruitment outside Australia
Country [1] 23838 0
New Zealand
State/province [1] 23838 0
Christchurch

Funding & Sponsors
Funding source category [1] 308967 0
Commercial sector/Industry
Name [1] 308967 0
River 3 Renal Corp
Address [1] 308967 0
One Rockefeller Plaza
New York, NY 10020
USA
Country [1] 308967 0
United States of America
Primary sponsor type
Commercial sector/Industry
Name
River 3 Renal Corp
Address
One Rockefeller Plaza
New York, NY 10020
USA
Country
United States of America
Secondary sponsor category [1] 309901 0
None
Name [1] 309901 0
Address [1] 309901 0
Country [1] 309901 0

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 308855 0
Northern A Health & Disability Ethics Committee
Ethics committee address [1] 308855 0
133 Molesworth Street
PO Box 5013
Wellington 6011
Ethics committee country [1] 308855 0
New Zealand
Date submitted for ethics approval [1] 308855 0
21/06/2021
Approval date [1] 308855 0
02/08/2021
Ethics approval number [1] 308855 0

Summary
Brief summary
The study will review the safety, tolerability, and PK of 3 different single, oral doses of R3R01 across 3 cohorts. It is envisaged that 24 (12 men and 12 women)participants will be recruited in the study.
The following doses will be used in this study:
Cohort A - R3R01 25 mg once
Cohort B - R3R01 100 mg once
Cohort C - R3R01 200 mg once.
8 participants will be enrolled in each cohort (4 men:4 Women).
In each cohort, R3R01 will be administered on Day 1. Participants will be admitted to the clinic on the day prior to dosing and remain in the clinic until discharged on the morning of Day 2 following completion of all scheduled study procedures and assessments.
Safety will be assessed through the reporting of AEs, vital signs measurements, ECGs and clinical laboratory results.
The study will be 15 weeks (from screening through study completion) for each enrolled subject as follows:
• Screening: Up to 3 weeks.
• Dosing period: 1 day.
• In-house stay: 3 days (Day -1 to Day 2 in the morning)
• Follow up: day 84 (+/- 4 days)
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 112130 0
Dr Chris Wynne
Address 112130 0
New Zealand Clinical Research Christchurch
264 Antigua Street
Christchurch 8140
Country 112130 0
New Zealand
Phone 112130 0
+6433729477
Fax 112130 0
Email 112130 0
chris.wynne@nzcr.co.nz
Contact person for public queries
Name 112131 0
Dr Chris Wynne
Address 112131 0
New Zealand Clinical Research Christchurch
264 Antigua Street
Christchurch 8140
Country 112131 0
New Zealand
Phone 112131 0
+6433729477
Fax 112131 0
Email 112131 0
chris.wynne@nzcr.co.nz
Contact person for scientific queries
Name 112132 0
Dr Chris Wynne
Address 112132 0
New Zealand Clinical Research Christchurch
264 Antigua Street
Christchurch 8140
Country 112132 0
New Zealand
Phone 112132 0
+6433729477
Fax 112132 0
Email 112132 0
chris.wynne@nzcr.co.nz

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 other documents available
Summary results
No Results