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A Placebo-controlled, Double-blind, Randomised, Parallel-group, Long-term Phase III Trial Assessing the Safety and Efficacy of 50 microgram and 100 mircrogram/day of eprotirome in Patients with Heterozygous Familial Hypercholesterolaemia who are on Optimal Standard of Care

The primary objective of this study is to compare the efficacy of eprotirome 50 microgram and eprotirome 100 micorgram versus placebo in terms of the percent change in LDL-C from baseline to Week 12 in HeFH patients with CAD, or who are at high risk…

A First-in-Human (FIH), Randomized, Dose-Escalation, Double-Blind, Placebo-Controlled Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of SPC5001 Administered to Healthy Subjects and Subjects with Familial Hypercholesterolemia (FH)

- To evaluate the safety and toleration of SPC5001 in healthy subjects and in subjects with FH.- To assess the lipid lowering effect of SPC5001 in healthy subjects and in subjects with FH.- To assess the pharmacokinetics of SPC5001 in healthy…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study
Confirming the Efficacy and Safety of Genz-112638 in Patients with Gaucher
Disease Type 1

The primary objective of this study is to confirm the efficacy and safety of Genz 112638 after 39 weeks of treatment in patients with Gaucher disease type 1.The secondary objective of this study is to determine the long term efficacy, safety, and…

A Double-blind, Randomized, Placebo-controlled, Multicenter Study to;Evaluate Tolerability and Efficacy of AMG 145 on LDL-C in Subjects with;Heterozygous Familial Hypercholesterolemia

Primary: To evaluate the effect of 12 weeks of subcutaneous (SC) AMG 145, compared with placebo, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in subjects with heterozygous familial hypercholesterolemia.Secondary…

A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to evaluate the Efficacy and Safety of SAR236553 (REGN727) in Patients With Heterozygous Familial Hypercholesterolemia and LDL-C higher or equal to 160mg/dL with Their Lipid-Modifying Therapy

The objective of the study is to assess the efficacy and safety of Alirocumab in patients with heterozygote familial hypercholesterolemia whose LDL-C level is higher than or equal to 160 mg/dL (4.14 mmol/L) on maximally tolerated statin therapy with…

A Randomized, Double-Blind, Placebo-Controlled, Parallel Group Study to Evaluate the Efficacy and Safety of SAR236553/REGN727/Alirocumab in Patients With Heterozygous Familial Hypercholesterolemia Not Adequately Controlled With Their Lipid-Modifying Therapy

The objective of the study is to assess the efficacy, tolerability and safety of SAR236553/Alirocumab when administered during 1.5years in patients with heterozygote familial hypercholesterolemia, who despite of lipid lowering therapy still have…

A Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study to Evaluate the Efficacy and Safety of REGN727/SAR236553 in Patients with Heterozygous Familial Hypercholesterolemia Not Adequately Controlled with Their Lipid-Modifying Therapy

The primary objective of the study is to demonstrate the reduction of LDL-C by REGN727 as add-on therapy to stable, maximally-tolerated dialy statin therapy with or without other LMT in comparison with placebo after 24 weeks of treatment in patients…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Followed by an Open-Label Continuation Period to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients with Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol.

The primary objective of this study is to determine whether mipomersen significantly reduces atherogenic lipid levels in patients with severe HeFH, defined as LDL-C levels equal to or above 5.18 mmol/L plus the presence of CHD/risk equivalents or…

A Randomized, Double-Blind, Double-Dummy, Active-Controlled Study to Evaluate the Efficacy and Safety of REGN727/SAR236553 in Patients with Primary Hypercholesterolemia Who are Intolerant to Statins

The primary objective of the study is to demonstrate the reduction of low-density lipoprotein (LDL) cholesterol (LDL-C) by REGN727 in comparison with ezetimibe (EZE) 10 mg PO QD after 24 weeks in patients with primary hypercholesterolemia (…

Effects of short term increase of phenylalanine levels
on neuropsychological functions and well-being
in adults with phenylketonuria: the *diet for life* study

To evaluate the effects of short term supplementation of Phe to levels comparable to levels observed in adult patients who fully discontinued their diet on neuropsychological functions and wellbeing of adult patients with PKU.

A 2-Part, Phase 2/3 Study to Assess the Safety, Tolerability and Efficacy of
AMG 145 in Subjects With Homozygous Familial Hypercholesterolemia
Part A - Open-label, Single-arm, Multicenter Pilot Study to Evaluate Safety,
Tolerability, and Efficacy of AMG 145 in Subjects With Homozygous Familial
Hypercholesterolemia
Part B - Double-blind, Randomized, Placebo-controlled, Multicenter Study to
Evaluate Safety, Tolerability and Efficacy of AMG 145 in Subjects

Primary ObjectivePart A: To characterize the effect of 12 weeks of subcutaneous (SC) AMG 145 on percentchange from baseline in low-density lipoprotein cholesterol (LDL-C) in subjects with homozygousfamilial hypercholesterolemiaPart B: To evaluate…

A Double-blind, Randomized, Placebo-controlled, Multicenter Study to Evaluate Safety, Tolerability and Efficacy of AMG 145 on LDL-C in Subjects with Heterozygous Familial Hypercholesterolemia (study 20110117)

Primary: To evaluate the effect of 12 weeks of subcutaneous (SC) AMG 145 every-2-weeks (Q2W) and every-4-weeks (Q4W), compared with placebo, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in subjects with heterozygous…

A Phase 2 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate the Efficacy, Safety, and Tolerability of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria

Primary:To assess the efficacy of DCR-PHXC in reducing urinary oxalate burden in patients with PH (types 1 and 2)Key Secondary:To assess the efficacy of DCR-PHXC in reducing urinary oxalate burden over time in patients with PHSecondary:1. To…

A Phase 1 Placebo-Controlled, Double-Blind, Multicenter Study to Evaluate
the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of a
Single Dose of DCR-PHXC in Patients with Primary Hyperoxaluria Type 3

Primary objective:To evaluate the safety and tolerability of a single dose of DCR-PHXC in patients with PH3Secondary objectives:To characterize the plasma PK of a single dose of DCR-PHXC in patients with PH3To assess the efficacy of a single dose of…

Double-blind, Randomized, Multicenter, Placebo-controlled, Parallel Group Study to Characterize the Efficacy, Safety, and Tolerability of 24 Weeks of Evolocumab for Low Density Lipoprotein-cholesterol (LDL-C) Reduction, as Add-On to Diet and Lipid-Lowering Therapy, in Pediatric Subjects 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH)

To evaluate the effect of 24 weeks of subcutaneous (SC) evolocumab compared with placebo, when added to standard of care, on percent change from baseline in low-density lipoprotein cholesterol (LDL-C) in pediatric subjects 10 to 17 years of age with…

A phase III double-blind, randomized study to evaluate the long-term
efficacy and safety of Oxabact® in patients with primary hyperoxaluria

Primary:- To evaluate the efficacy of Oxabact® following 52 weeks treatment in patients with maintained kidney function but below the lower limit of the normal range (eGFR < 90 ml/min/1.73 m2) and a total plasma oxalate concentration &gt…

A Phase 2b/3 study to evaluate the safety, tolerability, and effects of livoletide (AZP-531), an unacylated ghrelin analog, on food-related behaviors in patients with Prader-Willi syndrome

This Phase 2b/3 double-blind, placebo-controlled study will evaluate the safety, tolerability, and effects of livoletide on food-related behaviors in patients with Prader-Willi Syndrome (PWS).

A Randomized, Double blind, Active Control Study of the Safety and Efficacy of PRX-102 compared to Agalsidase Beta on Renal Function in Patients with Fabry Disease Previously Treated With Agalsidase Beta

To evaluate the safety and efficacy of PRX-102 compared to agalsidase beta in Fabry disease patients with impaired renal function.

Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series

This study has been transitioned to CTIS with ID 2024-514012-28-00 check the CTIS register for the current data. Assess the neurological efficacy of ambroxol in adults and children with GD3.

Effectiveness of methylphenidate in adults with phenylketonuria and attention-deficit/hyperactivity disorder: An N-of-1 series

Primary Objective: To determine the effectiveness of methylphenidate in reduction of ADHD symptomatology, operationalized by personalized goals that are important to the patient and its environment, in individuals with late-diagnosed PKU. Secondary…