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Treatment of Ectopic Mineralization in Pseudoxanthoma Elasticum.

The main objective is to determine if bisphosphonate therapy with etidronate leads to stabilization or attenuation of ongoing calcification in the leg arteries as quantified by 18F-sodium fluoride(18F-NaF) PET-CT imaging in patients with PXE.…

Multicenter, double-blind, placebo-controlled, randomized, prospective study of bosentan as adjunctive therapy to inhaled nitric oxide in the management of persistent pulmonary hypertension of the newborn (PPHN)

To assess the efficacy of bosentan in neonates with persistent pulmonary hypertension of the newborn (PPHN) who are in need of continued inhaled nitric oxide (iNO) after at least 4 hours of continuous iNO treatment and to evaluate the…

Phase 2a, Double-Blind, Placebo-Controlled, 2-Period, 2-Treatment, Crossover Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Multiple Oral Doses of XPF-001 (400 mg bid) in Patients with Inherited Erythromelalgia

• To evaluate the safety and efficacy of multiple doses of XPF-001 (400 mg bid) for relief of pain in patients with IEM.• To evaluate the efficacy of multiple doses of XPF-001 (400 mg bid) for relief of vasomotor signs in patient with IEM.• To…

International randomized double blind clinical study evaluating the efficacy and safety of clopidogrel 0.2mg/kg once daily versus placebo in neonates and infants with cyanotic congenital heart disease palliated with a systemic-to-pulmonary artery shunt (e.g. modified Blalock Taussig shunt)

Primary:To evaluate the efficacy of 0.2 mg/kg/day of clopidogrel versus placebo for the reduction of all-cause mortality and shunt-related morbidity in neonates or infants with cyanotic congenital heart disease palliated with a systemic-to-pulmonary…

A Phase 2/3, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of GS-6615 on Exercise Capacity in Subjects with Symptomatic Hypertrophic Cardiomyopathy

The primary objective of this study is to:* Evaluate the effect of GS-6615 on exercise capacity, as measured by Peak VO2 achieved during cardiopulmonary exercise testing (CPET), in subjects with symptomatic hypertrophiccardiomyopathy (HCM).The…

A multi-center, double-blind, randomized, placebo-controlled, parallel-group, Phase 3 study to evaluate the effects of macitentan on exercise capacity in subjects with Eisenmenger Syndrome.

To demonstrate that macitentan improves exercise capacity in comparison with placebo in subjects with Eisenmenger Syndrome (ES).

CeRebrUm and CardIac protection with Allopurinol in neonates with critical congenital heart disease requiring cardiac surgery with cardiopulmonary bypass

This study has been transitioned to CTIS with ID 2024-513041-37-00 check the CTIS register for the current data. Primary objective of this study is to significantly reduce relevant (moderate/severe) parenchymatous brain injury on postoperative MRI…

Two part (double-blind) inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2] randomized multicentre study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with homozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-13)

Study CKJX839C12302 (ORION-13) is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (aged 12 to <18 years) with HoFH and LDL-C >130 mg/dL (3.4 mmol/L). The use of inclisiran…

Two part (double-blind inclisiran versus placebo [Year 1] followed by open-label inclisiran [Year 2]) randomized multicenter study to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (12 to less than 18 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol (ORION-16)

Study CKJX839C12301 (ORION-16) is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents (aged 12 to <18 years) with HeFH and LDL-C >130 mg/dL (3.4 mmol/L). The use of inclisiran…

A randomised, placebo controlled, double blind, multicentre proof of concept study to assess the safety and efficacy of two doses of VAD044 in patients with hereditary haemorrhagic telangiectasia (HHT)

PART I:Primary:• To investigate the safety and tolerability of two doses of VAD044 administered daily for up to 12 weeks in HHT patients.Secondary:• To assess the effects of two doses of VAD044 for up to 12 weeks of treatment on:o Epistaxis:•…

Nitric Oxide during Cardio Pulmonary Bypass during surgery for congenital heart defects: A Randomised Controlled Trial.

Primary objective of this trial is to investigate in a double blind randomized controlled trial in children undergoing open heart surgery if NO exposure during CPB reduces the postoperative duration of invasive mechanical ventilation (defined as…

A Randomized, Double blind, Placebo controlled Clinical Study to Evaluate
Mavacamten (MYK-461) in Adults with Symptomatic Obstructive
Hypertrophic Cardiomyopathy

Primary Objective* To compare the effect of a 30-week course of mavacamten with placebo on clinical response comprising of exercise capacity and clinical symptoms in participants with symptomatic obstructive hypertrophic cardiomyopathy (oHCM)…

A placebo-controlled, double-blind, randomized trial to evaluate the effect of 300 mg of inclisiran sodium given as subcutaneous injections in subjects with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C)

Primary :The primary objective is to evaluate the effect of inclisiran treatment on:* LDL-C levels at Day 510.* Time adjusted percent change in LDL-C levels from baseline after Day 90 up to Day 540 levelsSecondary:The secondary objectives are to…

A Randomized, Double-blind, Placebo-controlled Clinical Study to Evaluate Mavacamten in Adults with Symptomatic Non-obstructive Hypertrophic Cardiomyopathy

This study has been transitioned to CTIS with ID 2023-506352-24-00 check the CTIS register for the current data. Primary Objectives: • To assess the efficacy of a 48-week course of mavacamten compared to placebo on patient- reported health status (…

Morphine intravenous vs. paracetamol intravenous after cardiac surgery in neonates and infants.

The aim of the study is to test the hypothesis that intermittent intravenous paracetamol administration in children after cardiac surgery will result in a reduction of at least 30% of the cumulative morphine requirement.

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Effect of Praluent on Neurocognitive Function in Patients with Heterozygous Familial Hypercholesterolemia or with Non-Familial Hypercholesterolemia at High and Very High Cardiovascular Risk

The primary objective of the study is to evaluate neurocognitive function with use of Praluent after 96 weeks of treatment versus placebo.

Phase III, multi-center, randomized, 48 weeks, double-blind, parallel-group, placebo-controlled study to evaluate efficacy and safety of CER-001 on vessel wall area in patients with genetically definded familial primary hypoalphalipoproteinemia and receiving background optimized lipid therapy, with optional open-label safety extension

The primary objectives of the study are:• To evaluate the effect of 24 weeks treatment with CER-001 on carotid Mean Vessel Wall Area (MVWA) as compared to placebo using 3T magnetic resonance imaging (3T-MRI);• To evaluate the safety and tolerability…