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Trial record 3 of 3 for:    LBS-008

Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects Lesion(s) in Adolescent Subjects With STGD1

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. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05244304
Recruitment Status : Recruiting
First Posted : February 17, 2022
Last Update Posted : August 24, 2022
Sponsor:
Information provided by (Responsible Party):
Belite Bio, Inc

Brief Summary:
The primary objective of this trial is to assesses the efficacy of tinlarebant in slowing the rate of growth of atrophic lesion(s) in adolescent subjects with STGD1

Condition or disease Intervention/treatment Phase
Stargardt Disease 1 Drug: Tinlarebant Drug: Placebo Phase 3

Detailed Description:
Approximately 60 subjects will be enrolled in this study. Subjects will be assigned to study drug (tinlarebant 5 mg/placebo) with treatment period of upto 24 months with 28 days of follow-up.

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 60 participants
Allocation: Randomized
Intervention Model: Single Group Assignment
Masking: Double (Participant, Investigator)
Primary Purpose: Treatment
Official Title: Phase 3, Multicenter, Randomized, Double-Masked, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Tinlarebant in the Treatment of Stargardt Disease in Adolescent Subjects
Actual Study Start Date : March 28, 2022
Estimated Primary Completion Date : December 31, 2024
Estimated Study Completion Date : February 15, 2025

Resource links provided by the National Library of Medicine


Arm Intervention/treatment
Experimental: Tinlarebant
5 mg tablet taken orally once a day
Drug: Tinlarebant
Tinlarebant drug substance is a white to off-white substance and is dispensed as a tablet for oral administration.

Placebo Comparator: Placebo
Placebo tablets for tinlarebant 5 mg are prepared similarly but use microcrystalline cellulose, NF, in place of the active drug substance and will be identical in size and appearance.
Drug: Placebo
Not active drug




Primary Outcome Measures :
  1. To measure change in atrophic lesion size (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline [ Time Frame: Baseline thru month 24 ]

Secondary Outcome Measures :
  1. To measure the change in retinal thickness assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline [ Time Frame: Baseline thru month 24 ]
  2. To measure the change in retinal morphology assessed by spectral-domain optical coherence tomography (SD-OCT) from baseline [ Time Frame: Baseline thru month 24 ]
  3. To measure change in BCVA (Best Corrected Visual Acuity) score measured by the EDTRS method from baseline [ Time Frame: Baseline thru month 24 ]
  4. To measure change in plasma concentration of RBP4 levels (μM) from baseline [ Time Frame: Baseline thru month 24 ]
  5. The correlation between change in plasma RBP4 level and the rate of lesion size growth (definitely decreased autofluorescence, DDAF) by fundus autofluorescence (FAF) photography from baseline [ Time Frame: Baseline thru month 24 ]
  6. To assess the systemic and ocular safety and tolerability of tinlarebant [ Time Frame: Baseline thru month 24 ]
    Frequency, duration, and severity of AEs


Other Outcome Measures:
  1. To measure change in total decreased autofluorescence (DAF) by FAF photography from baseline [ Time Frame: Baseline thru month 24 ]
  2. To measure change in questionably decreased autofluorescence (QDAF) by FAF photography from baseline [ Time Frame: Baseline thru month 24 ]
  3. To measure change in quantitative autofluorescence (qAF) level from baseline [ Time Frame: Baseline thru month 24 ]


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

Inclusion Criteria:

  • Male or female subjects 12 to 18 years old, inclusive.
  • Subject must have clinically diagnosed STGD1 (Stargardt disease 1) with at least 1 mutation identified in the ABCA4 gene.
  • Subject must have a defined aggregate atrophic lesion size within 3 disc areas (7.62 mm2), as imaged by FAF in the study eye Subjects must have a BCVA of 20/200 or better for the study eye based on ETDRS letter score
  • Subject and their parent(s) or legal guardian are willing to provide their consent on an Institutional Review Board (IRB)/Independent Ethics Committee (IEC)/Human Research Ethics Committee (HREC)-approved informed consent form (ICF) prior to participating in any study-related procedures.
  • Subject agrees to comply with all protocol requirements.

Exclusion Criteria:

  • Any ocular disease other than Stargardt (STGD1) at baseline that, in the opinion of the investigator, would complicate assessment of a treatment effect.
  • History of ocular surgery in the study eye in the last 3 months.
  • Investigational drug use of any kind in the last 3 months or within 5 half-lives of the investigational drug, whichever is shorter.
  • Any prior gene therapy.
  • Vitamin A (retinol) deficiency as defined as a retinol serum level less than 20 mcg/dL (=0.7 μmol/L).

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): NCT05244304


Contacts
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Contact: Belitebio - Clinical Trial +886 972 080 097 clinicaltrial@belitebio.com

Locations
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Sponsors and Collaborators
Belite Bio, Inc
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Responsible Party: Belite Bio, Inc
ClinicalTrials.gov Identifier: NCT05244304    
Other Study ID Numbers: LBS-008-CT03
First Posted: February 17, 2022    Key Record Dates
Last Update Posted: August 24, 2022
Last Verified: August 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
Additional relevant MeSH terms:
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Stargardt Disease
Macular Degeneration
Eye Diseases, Hereditary
Eye Diseases
Retinal Degeneration
Retinal Diseases
Genetic Diseases, Inborn