Back to results

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

Primary Purpose

Stargardt Disease

Status

Active

Phase

Phase 1

Locations

International

Study Type

Interventional

Intervention

tinlarebant

About this trial

This is an interventional treatment trial for Stargardt Disease

Eligibility Criteria

12 Years - 18 Years (Child, Adult)All SexesDoes not accept 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.

Sites / Locations

Sydney Children's Hospitals Network
Lions Eye Institute
National Taiwan University Hospital

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

tinlarebant

Arm Description

Daily, oral administration of one tinlarebant.

Outcomes

Primary Outcome Measures

To evaluate systemic and ocular safety and tolerability of tinlarebant.

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.

To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.

Secondary Outcome Measures

Change in atrophic lesion size.

Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.

Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.

Half-life (t1/2) of tinlarebant in plasma.

Time to minimal plasma RBP4 level (Tmin)

Minimum concentration of RBP4 (Cmin)

Full Information

NCT ID

NCT05266014

First Posted

January 26, 2022

Last Updated

April 25, 2022

Sponsor

RBP4 Pty Ltd

Collaborators

Belite Bio, Inc

1. Study Identification

Unique Protocol Identification Number

NCT05266014

Brief Title

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

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2022

Overall Recruitment Status

Active, not recruiting

Study Start Date

March 12, 2021 (Actual)

Primary Completion Date

August 22, 2023 (Anticipated)

Study Completion Date

August 22, 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

RBP4 Pty Ltd

Collaborators

Belite Bio, Inc

4. Oversight

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

Data Monitoring Committee

Yes

5. 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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Stargardt Disease

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

13 (Actual)

8. Arms, Groups, and Interventions

Arm Title

tinlarebant

Arm Type

Experimental

Arm Description

Daily, oral administration of one tinlarebant.

Intervention Type

Drug

Intervention Name(s)

tinlarebant

Intervention Description

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.

Primary Outcome Measure Information:

Title

To evaluate systemic and ocular safety and tolerability of tinlarebant.

Description

To evaluate safety and tolerability of daily dosing of tinlarebant assessed by incidence and/or severity of ocular and non-ocular adverse events.

Time Frame

From baseline to 24 months

Title

The optimal dose for Phase 2.

Description

To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.

Time Frame

Up to 24 months

Secondary Outcome Measure Information:

Title

Change in atrophic lesion size.

Time Frame

From baseline to 24 months.

Title

Maximum Plasma Concentration (Cmax) of tinlarebant in plasma.

Time Frame

Up to 24 months

Title

Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma.

Time Frame

Up to 24 months

Title

Half-life (t1/2) of tinlarebant in plasma.

Time Frame

Up to 24 months

Title

Time to minimal plasma RBP4 level (Tmin)

Time Frame

Up to 24 months

Title

Minimum concentration of RBP4 (Cmin)

Time Frame

Up to 24 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Maximum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

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.

Facility Information:

Facility Name

Sydney Children's Hospitals Network

City

Westmead

State/Province

New South Wales

ZIP/Postal Code

2145

Country

Australia

Facility Name

Lions Eye Institute

City

Perth

State/Province

Western Australia

ZIP/Postal Code

6009

Country

Australia

Facility Name

National Taiwan University Hospital

City

Taipei

ZIP/Postal Code

100

Country

Taiwan

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

We'll reach out to this number within 24 hrs