Rationale and design of two trials assessing efficacy, safety and tolerability of Inclisiran in adolescents with homozygous and heterozygous familial hypercholesterolaemia

Abstract

Methods: ORION-13 and ORION-16 are both two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran, Figure 1) multicentre trials including adolescents aged 12 to <18 years diagnosed with familial hypercholesterolaemia. ORION-13 will include approximately 12 participants diagnosed with homozygous familial hypercholesterolaemia and ORION-16 will include approximately 150 participants diagnosed with heterozygous familial hypercholesterolaemia. The primary endpoint is the percentage change in LDL-C from baseline to day 330. Other efficacy and safety endpoints include: changes in other lipid parameters and adverse events, and also exploratory endpoints, such as individual responsiveness of the participants and change in LDL-C according to the type of underlying causal mutation.Results: Not applicable yetConclusions: Not applicable yet Methods: ORION-13 and ORION-16 are both two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran, Figure 1) multicentre trials including adolescents aged 12 to <18 years diagnosed with familial hypercholesterolaemia. ORION-13 will include approximately 12 participants diagnosed with homozygous familial hypercholesterolaemia and ORION-16 will include approximately 150 participants diagnosed with heterozygous familial hypercholesterolaemia. The primary endpoint is the percentage change in LDL-C from baseline to day 330. Other efficacy and safety endpoints include: changes in other lipid parameters and adverse events, and also exploratory endpoints, such as individual responsiveness of the participants and change in LDL-C according to the type of underlying causal mutation. Results: Not applicable yet Conclusions: Not applicable yet