A study of Tinlarebant as treatment for Adolescent Stargardt Disease

• Research type

  Research Study

• Full 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

• IRAS ID

  306578

• Contact name

  Michel Michaelides

• Contact email

  michel.michaelides@ucl.ac.uk

• Sponsor organisation

  Belite Bio, Inc

• Eudract number

  2021-003253-36

• Clinicaltrials.gov Identifier

  NCT05244304

• Duration of Study in the UK

  3 years, 1 months, 7 days

• Research summary

  Stargardt Disease 1 (STGD1) is a rare inherited disorder of the eye that leads to progressive and irreversible sight loss, called macular degeneration. It is typically diagnosed in early childhood and adolescence. Currently there is no approved treatment for the condition.
  Participants with STGD1 have a gene mutation which leads to an excess accumulation of lipids that breakdown the light sensors within the retina. These lipids are wastes produced from retinol as by-products of the normal visual function.
  Retinol is a natural vitamin that can be obtained through the standard diet. When in the human body, it is transported to the eye via a protein called RBP4.
  The study drug, tinlarebant, has been developed to slow down the progression of STGD1 by reducing the amount of RBP4 in the blood and the supply of retinol into the eye. The reduction of retinol in the eye would in turn reduce the cytotoxic lipids within the retina.
  It is anticipated that early diagnosis and intervention of STGD1 would reduce the structural damage to the retina.
  Participants aged between 12 to 18 years old will be enrolled in this study. Participants will be randomly assigned to either receive 5mg of tinlarebant or matching placebo. The study drug will be taken orally once per day.
  The study consists of a 28-day screening period, a 24-month treatment period, and followed by a 28-day safety follow-up period. Each participant will take part of up to 26 months.
  Procedures involved include physical examinations, vital signs, blood and urine tests, eye tests, electrocardiograms, and quality of life questionnaires.
  The study is sponsored by Belite Bio, Inc. Approximately 60 participants will take part globally with 6 participants in the United Kingdom

• REC name

  Wales REC 5

• REC reference

  21/WA/0357

• Date of REC Opinion

  25 Nov 2021

• REC opinion

  Favourable Opinion