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A Phase 4 Randomised, Double-Blind, Placebo-Controlled, Parallel-Group, Multi-Centre Study of Colesevelam as Add-on Therapy in Patients with Familial Hypercholesterolaemia.

This study is being conducted in patients with familial hypercholesterolaemia (FH) to:1. Assess the efficacy of colesevelam added to a maximal tolerated and stable regimen of statin and ezetimibe in further decreasing the low-density lipoprotein (…

Young adult Prader-Willi (YAP) Syndrome Study: Effects of growth hormone after final height: A clinical care to the optimal dosage of growth hormone in young adults with PWS.

To evaluate the effects of GH, after final height is reached, on weight, body composition, psychosocial functioning, carbohydrate metabolism and serum lipids. To find an optimal dose growth hormone for adults with PWS. We aimed to stabilize the body…

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated
Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase
(OTC) in Patients with Late-Onset OTC Deficiency

This study has been transitioned to CTIS with ID 2024-514337-38-00 check the CTIS register for the current data. PrimaryTo evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecondaryTo…

Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study

This study has been transitioned to CTIS with ID 2024-516124-34-00 check the CTIS register for the current data. Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3)…