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Trial record 2 of 3 for:    LBS-008

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

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 evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05266014
Recruitment Status : Active, not recruiting
First Posted : March 4, 2022
Last Update Posted : April 26, 2022
Sponsor:
Collaborator:
Belite Bio, Inc
Information provided by (Responsible Party):
RBP4 Pty Ltd

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

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Study Type : Interventional  (Clinical Trial)
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 : March 12, 2021
Estimated Primary Completion Date : August 22, 2023
Estimated Study Completion Date : August 22, 2023

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

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.





Primary Outcome Measures :
  1. To evaluate systemic and ocular safety and tolerability of tinlarebant. [ Time Frame: 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.

  2. The optimal dose for Phase 2. [ Time Frame: Up to 24 months ]
    To determine optimal dose of tinlarebant administered orally in adolescent patients with Stargardt Disease.


Secondary Outcome Measures :
  1. Change in atrophic lesion size. [ Time Frame: From baseline to 24 months. ]
  2. Maximum Plasma Concentration (Cmax) of tinlarebant in plasma. [ Time Frame: Up to 24 months ]
  3. Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma. [ Time Frame: Up to 24 months ]
  4. Half-life (t1/2) of tinlarebant in plasma. [ Time Frame: Up to 24 months ]
  5. Time to minimal plasma RBP4 level (Tmin) [ Time Frame: Up to 24 months ]
  6. Minimum concentration of RBP4 (Cmin) [ Time Frame: Up to 24 months ]


Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05266014


Locations
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Australia, New South Wales
Sydney Children's Hospitals Network
Westmead, New South Wales, Australia, 2145
Australia, Western Australia
Lions Eye Institute
Perth, Western Australia, Australia, 6009
Taiwan
National Taiwan University Hospital
Taipei, Taiwan, 100
Sponsors and Collaborators
RBP4 Pty Ltd
Belite Bio, Inc
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Responsible Party: RBP4 Pty Ltd
ClinicalTrials.gov Identifier: NCT05266014    
Other Study ID Numbers: LBS-008-CT02
First Posted: March 4, 2022    Key Record Dates
Last Update Posted: April 26, 2022
Last Verified: April 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Product Manufactured in and Exported from the U.S.: Yes
Additional relevant MeSH terms:
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Stargardt Disease
Macular Degeneration
Eye Diseases, Hereditary
Eye Diseases
Retinal Degeneration
Retinal Diseases
Genetic Diseases, Inborn