This is a Dose-finding Study Followed by 2-year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

Sponsor

RBP4 Pty Ltd (Industry)

Overall Status

Active, not recruiting

CT.gov ID

NCT05266014

Collaborator

Belite Bio, Inc (Industry)

Enrollment

Locations

Arm

29.3

Anticipated Duration (Months)

4.3

Patients Per Site

0.1

Patients Per Site Per Month

Study Details

Study Description

Brief Summary

Stargardt disease 1 (STGD1) is the most prevalent form of juvenile macular degeneration. It is caused by a rare, inherited autosomal recessive trait, leading to severe and irreversible blindness by the first or second decade of life. Earlier onset of the disease is related to a rapid vision loss, while patients with a later onset tend to have a better prognosis.

This study will enrol subjects aged 12-18 years old with a confirmed clinical diagnosis of Stargardt disease type 1 (STGD1). This study will include 2 phases, the phase 1b portion is to determine the optimal dose for phase 2 based on the extent of retinol binding protein 4 (RBP4) reduction after 2 cycles of tinlarebant treatment. The phase 2 portion will evaluate the safety and efficacy of a single daily dose of tinlarebant over a 24-month treatment period.

Condition or Disease	Intervention/Treatment	Phase
Stargardt Disease	Drug: tinlarebant	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Actual Enrollment :

13 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

Official Title:

Phase 1/2, Open-Label, Dose-Finding Followed by 2-Year Extension Study to Evaluate Safety and Tolerability of Tinlarebant in Adolescent Subjects With Stargardt Disease

Actual Study Start Date :

Mar 12, 2021

Anticipated Primary Completion Date :

Aug 22, 2023

Anticipated Study Completion Date :

Aug 22, 2023

Arms and Interventions

Arm	Intervention/Treatment
Experimental: tinlarebant Daily, oral administration of one tinlarebant.	Drug: tinlarebant Phase 1b Portion: tinlarebant will be self-administered orally once daily for 2 cycles, 14 days per cycle. Phase 2 portion: tinlarebant will be self-administered orally once daily for 24 months.

Arm

Intervention/Treatment

Experimental: tinlarebant

Daily, oral administration of one tinlarebant.

Drug: tinlarebant

Phase 1b Portion: tinlarebant will be self-administered orally once daily for 2 cycles, 14 days per cycle. Phase 2 portion: tinlarebant will be self-administered orally once daily for 24 months.

Outcome Measures

Primary Outcome Measures

To evaluate systemic and ocular safety and tolerability of tinlarebant. [From baseline to 24 months]
To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.
The optimal dose for Phase 2. [Up to 24 months]
To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.

Secondary Outcome Measures

Change in atrophic lesion size. [From baseline to 24 months.]
Maximum Plasma Concentration (Cmax) of tinlarebant in plasma. [Up to 24 months]
Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma. [Up to 24 months]
Half-life (t1/2) of tinlarebant in plasma. [Up to 24 months]
Time to minimal plasma RBP4 level (Tmin) [Up to 24 months]
Minimum concentration of RBP4 (Cmin) [Up to 24 months]

Eligibility Criteria

Criteria

Ages Eligible for Study:

12 Years to 18 Years

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Major Inclusion Criteria:

Subject must have clinically diagnosed Stargardt disease with at least one mutation identified in the ABCA4 gene.

Major Exclusion Criteria:

Any ocular disease other than Stargardt disease at baseline that, in the opinion of the PI, would complicate assessment of a treatment effect.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Sydney Children's Hospitals Network	Westmead	New South Wales	Australia	2145
2	Lions Eye Institute	Perth	Western Australia	Australia	6009
3	National Taiwan University Hospital	Taipei		Taiwan	100

Sponsors and Collaborators

RBP4 Pty Ltd
Belite Bio, Inc

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

RBP4 Pty Ltd

ClinicalTrials.gov Identifier:

NCT05266014

Other Study ID Numbers:

LBS-008-CT02

First Posted:

Mar 4, 2022

Last Update Posted:

Apr 26, 2022

Last Verified:

Apr 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Yes

Studies a U.S. FDA-regulated Device Product:

Product Manufactured in and Exported from the U.S.:

Yes

Additional relevant MeSH terms:

Stargardt Disease

Macular Degeneration

Study Results

No Results Posted as of Apr 26, 2022