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
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|ClinicalTrials.gov Identifier: NCT05266014|
Recruitment Status : Active, not recruiting
First Posted : March 4, 2022
Last Update Posted : April 26, 2022
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 Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||13 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|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|
Daily, oral administration of one 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.
- 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.
- 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.
- Change in atrophic lesion size. [ Time Frame: From baseline to 24 months. ]
- Maximum Plasma Concentration (Cmax) of tinlarebant in plasma. [ Time Frame: Up to 24 months ]
- Time to Maximum Plasma Concentration (Tmax) of tinlarebant in plasma. [ Time Frame: Up to 24 months ]
- Half-life (t1/2) of tinlarebant in plasma. [ Time Frame: Up to 24 months ]
- Time to minimal plasma RBP4 level (Tmin) [ Time Frame: Up to 24 months ]
- Minimum concentration of RBP4 (Cmin) [ Time Frame: Up to 24 months ]
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
|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|
|National Taiwan University Hospital|
|Taipei, Taiwan, 100|