ANZCTR - Registration

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 registered on ANZCTR

Registration number

ACTRN12618001823268

Ethics application status

Approved

Date submitted

3/11/2018

Date registered

9/11/2018

Date last updated

3/05/2023

Date data sharing statement initially provided

9/11/2018

Type of registration

Prospectively registered

Titles & IDs

Public title

Safety and tolerability Phase I study of LBS-008 in healthy adult subjects after single and multiple doses

Scientific title

A Phase 1, Randomized, Double-Blind, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of LBS-008 in Healthy Adult Subjects

Secondary ID [1]

LBS-008-CT01

Universal Trial Number (UTN)

Trial acronym

Linked study record

Health condition

Health condition(s) or problem(s) studied:

Dry age-related macular degeneration

Condition category

Condition code

Eye

Normal eye development and function

Intervention/exposure

Study type

Interventional

Description of intervention(s) / exposure

The Single Ascending Dose (SAD) portion will have up to 5 cohorts and up to a total of 40 subjects (8 subjects per cohort). The starting dose of LBS-008 will be 50 mg and the planned doses for subsequent cohorts are 100, 200, 400 mg. An additional cohort at 25 mg dose level is determined to further evaluate the safety, PK and PD profile of LBS-008.

The Multiple Ascending Dose (MAD) portion will start after the completion of Cohort 4 of SAD and will have up to 4 cohorts and up to a total of 32 subjects (8 subjects per cohort). The starting dose of LBS-008 will be 10 mg and the planned doses for subsequent cohorts are 25, 5 and 12 mg. The final doses are determined based on the outcome of SAD portion.

Route of administration: Oral
Duration: SAD on Day 1 and MAD from day 1 to day 14

The strategies used to monitor adherence to the study drug will be: administration under direct medical supervision, mouth inspection for checking the ingestion, appropriate record of the dosing information, counts of the number of capsules.

Intervention code [1]

Treatment: Drugs

Comparator / control treatment

Placebo (microcrystalline cellulose capsule) controlled treatment.

Control group

Placebo

Outcomes

Primary outcome [1]

Evaluate the safety and tolerability to LBS-008, access by adverse events, clinical laboratory test result, vital sign measurement, physical examination and 12-lead ECG result.

Timepoint [1]

Monitored daily through Day 8 for SAD; Monitored daily through Day 21 for MAD.

Primary outcome [2]

To evaluate the ocular safety by access the slit lamp biomicroscopy, dilated ophthalmoscopy, and intraocular pressure, visual acuity and color vision result.

Timepoint [2]

Monitored daily through day 2 and day 8 of SAD; Monitored on Day 2, 7, 16 and 21 of MAD.

Primary outcome [3]

Pharmacokinetics (PK) parameters (AUC0-t, AUC0-inf, Cmax, Tmax, lambda z, t1/2, CL/F, and Vz/F) of administration of LBS-008 subjects.

Timepoint [3]

Blood PK will be collected at Day 1, 2, 3, 4, 5, 6 and 8 after IP administration of SAD. Blood PK will be collected at Day 1, 2, 3, 4, 7, 11, 14, 15, 16, 17, 18, 19, 21 and 28 for MAD after IP administration.

Secondary outcome [1]

1. To determine the effects of LBS-008 on plasma levels of RBP4, a PD marker.

Timepoint [1]

Blood collection of RBP4 for SAD will be on Day 1, 2, 3, 4, 5, 6 and 8 after IP administration and on day 1, 2, 3, 4, 7, 11, 14, 15, 16, 17, 18, 19, 21 and 28 for MAD after IP administration.

Eligibility

Key inclusion criteria

1.The subject is male or female, 18 to 65 years of age, inclusive, at screening.
2. The subject voluntarily consents to participate in this study and provides written informed consent before the start of any study-specific procedures.
3. The subject is willing and able to remain in the study unit for the entire duration of the confinement period and return for outpatient visits.
4. Female subjects must be of nonchildbearing potential (defined as surgically sterile [i.e., had a bilateral tubal ligation, hysterectomy, or bilateral oophorectomy at least 6 months before the dose of study drug] or postmenopausal for at least 1 year before study drug administration confirmed by FSH test at screening; FSH level >40 mIU/mL). Female subjects may also be considered of non-childbearing if they have a confirmed medical condition which would deem the subject as infertile. E.g. MRKH Syndrome (Mullerian Agenesis) or another applicable condition.
5. Male subjects must be surgically sterile (i.e., vasectomy) for at least 3 months before screening; or remain abstinent or agree to use a highly effective form of contraception when sexually active with a female partner for 90 days after study drug administration. Highly effective contraception requires use of a condom and appropriate contraceptive measures for your female partner (i.e. oral, injected or implanted hormonal methods, or placement of an intrauterine device or intrauterine system). This requirement does not apply to subjects in a same sex relationship and female partners of non-childbearing potential.
6. The subject has a body mass index (BMI) of 18 to 30 kg/m2, inclusive, and weighs 50 to 100 kg (110 to 220 pounds), inclusive, at screening and check-in.
7. The subject is considered to be in stable health by the investigator.
8. The subject agrees to comply with all protocol requirements.

Minimum age

18 Years

Maximum age

65 Years

Sex

Both males and females

Can healthy volunteers participate?

Yes

Key exclusion criteria

1.Any significant acute or chronic medical illness including history or presence of clinically significant cardiovascular, pulmonary, hepatic, renal, hematologic, gastrointestinal, endocrine, immunologic, dermatologic, neurologic, oncologic, or psychiatric disease.
2. Vitamin A deficiency.
3. Any recent viral or bacterial infection.
4. Participated in any clinical study in last 6 weeks.
5. History of significant drug allergy
6. History of significant vision, ocular or retinal disorder.
7. Recent surgery, blood transfusion, drug or alcohol abuse and use of tobacco or nicotine containing products in past month.
8. Evidence of organ dysfunction or any clinically significant deviation from normal in physical examination, vital signs, ECGs, or clinical laboratory determinations Other protocol-defined inclusion/exclusion criteria could apply.

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)

Methods used to generate the sequence in which subjects will be randomised (sequence generation)

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

Completed

Date of first participant enrolment

Anticipated

15/11/2018

Actual

15/11/2018

Date of last participant enrolment

Anticipated

Actual

20/08/2019

Date of last data collection

Anticipated

31/10/2019

Actual

16/09/2019

Sample size

Target

Accrual to date

Final

Recruitment in Australia

Recruitment state(s)

Recruitment hospital [1]

Linear Clinical Research - Nedlands

Recruitment postcode(s) [1]

6009 - Nedlands

Funding & Sponsors

Funding source category [1]

Commercial sector/Industry

Name [1]

RBP4 Pty Ltd

Address [1]

58 Gipps Street, Collingwood Victoria 3066, Australia

Country [1]

Australia

Primary sponsor type

Commercial sector/Industry

Name

RBP4 Pty Ltd

Address

58 Gipps Street, Collingwood Victoria 3066, Australia

Country

Australia

Secondary sponsor category [1]

None

Name [1]

Address [1]

Country [1]

Other collaborator category [1]

Commercial sector/Industry

Name [1]

Belite Bio, Inc

Address [1]

Ugland House
Grand Cayman, KY1-1104
Cayman Islands

Country [1]

United Kingdom

Ethics approval

Ethics application status

Approved

Ethics committee name [1]

Bellberry Human Research Ethics Committee H [EC00459]

Ethics committee address [1]

129 Glen Osmond Rd Eastwood SA 5063

Ethics committee country [1]

Australia

Date submitted for ethics approval [1]

Approval date [1]

24/09/2018

Ethics approval number [1]

Summary

Brief summary

A double-blind, placebo-controlled, single ascending dose (SAD) study is planned to assess safety, pharmacokinetics (PK), and pharmacodynamics of LBS-008 in healthy adult volunteers. Healthy male or female adults with no significant ocular abnormalities will be enrolled. The plan is to enroll 40 subjects, in five cohorts of eight subjects each; additional cohorts (eight subjects per cohort) may be enrolled if it is deemed appropriate by the sponsor to repeat a dose level or to study another dose level. Within each cohort, six subjects will be randomized to receive active drug and two subjects will receive placebo. Each subject will participate in only one dose level. Subjects will receive single ascending doses of 50, 100, 200, 400, and 25 mg LBS-008 administered as 25 or 200 mg capsules, or an equivalent number of placebo capsules. Each dose cohort will be separated into two groups; a sentinel group of two subjects (one active and one placebo) will be dosed at least 24 hours before the remaining six subjects (five active and one placebo).

The MAD portion will start after the completion of Cohort 4 of SAD and will have up to 4 cohorts and up to a total of 32 subjects (8 subjects per cohort). The starting dose of LBS-008 will be 10 mg and the planned doses for subsequent cohorts are 25, 5 and 12 mg. The final doses are determined based on the outcome of SAD portion.

Trial website

Trial related presentations / publications

Public notes

Contacts

Principal investigator

Name

Dr Lara Hatchuel

Address

QEII Medical Centre, First Floor, B Block Hospital Avenue, Nedlands, Western Australia, 6009, Australia

Country

Australia

Phone

+61 0424686547

Fax

lhatchuel@linear.org.au

Contact person for public queries

Name

Lara Hatchuel

Address

QEII Medical Centre, First Floor, B Block Hospital Avenue, Nedlands, Western Australia, 6009, Australia

Country

Australia

Phone

+61 0424686547

Fax

lhatchuel@linear.org.au

Contact person for scientific queries

Name

Yvonne Chen

Address

RBP4 Pty Ltd
58 Gipps Street, Collingwood Victoria 3066, Australia

Country

Australia

Phone

+61 3 94197607

Fax

yvonnechen@linbioscience.com

Data sharing statement

Will individual participant data (IPD) for this trial be available (including data dictionaries)?

No/undecided IPD sharing reason/comment

IPD will not be available.

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

Source	Title	Year of Publication	DOI
Embase	Stargardt disease and progress in therapeutic strategies.	2022	https://dx.doi.org/10.1080/13816810.2021.1966053

N.B. These documents automatically identified may not have been verified by the study sponsor.

Trial Review

Registering a new trial?

Documents added manually

Documents added automatically