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A Phase I/II Open Label Study in MPS IIIB Subjects to Investigate the Safety, Biodistribution, Pharmacokinetics, and Pharmacodynamics/Efficacy of SBC-103 Administered Intravenously

To evaluate the safety and tolerability of IV administration of SBC-103 in subjects with MPS lllB, Sanfilippo B.

Open-Label Extension Study of EFC12492, R727-CL-1112, EFC12732 and LTS11717 Studies to Assess the Long-Term Safety and Efficacy of Alirocumab in Patients with Heterozygous Familial Hypercholesterolemia

Primary objective* To assess the long-term safety of alirocumab when added to currently available lipid-modifying drug therapy in patients with heterozygous familial hypercholesterolemia (heFH) who have completed one of the following studies:…

An Open-Label Extension of Study HGT-SAN-093 Evaluating the Safety and Efficacy Study of HGT-1410 (Recombinant Human Heparan N Sulfatase) Administration via an Intrathecal Drug Delivery Device in Pediatric Patients with Mucopolysaccharidosis Type IIIA Disease.

Primary Objective(s): To evaluate long-term safety in patients with mucopolysaccharidosis type IIIA disease (MPS IIIA or Sanfilippo Type A) who received HGT-1410Secondary Objective(s):To evaluate:• The long-term cognitive function as measured by the…

A Multicenter, Open-Label Study of Sebelipase Alfa in Patients with Lysosomal Acid Lipase deficiency

The primary objective of this study is to evaluate the safety of sebelipase alfa in a more broad population of subjects with LAL Deficiency than have been previously studied.The secondary objectives of this study are (1) to evaluate effects of…

Modifying Orphan Disease Evaluation (MODE) Study: A multicenter, open-label study of the
effects of CER-001 on plaque volume in subjects with Homozygous Familial
Hypercholesterolemia

The purpose of the study is to assess the effects of CER-001, given by intravenous infusion with regards to plaque burden of the carotid arteries and the descending thoracic aorta using 3T magnetic resonance imaging (3TMRI).The objective of the…

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 (…

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 Multicenter, Open-label Study to Assess the Long-term Safety,
Tolerability, and Efficacy of AMG 145 on LDL-C in Subjects With Severe Familial
Hypercholesterolemia

Primary Objective: To characterize the safety and tolerability of long-term administration ofAMG 145 among subjects with severe familial hypercholesterolemia

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…

Cholic acid treatment in Peroxisomal Biogenesis Disorders (Zellweger spectrum): biochemical and clinical effects.

To investigate the effect of cholic acid supplementation on the clinical and biochemical parameters of Zellweger spectrum disorder

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 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…

An Efficacy and 2-Year Safety Study of Open-label Rosuvastatin in Children and Adolescents (aged from 6 to less than 18 years) with Familial Hypercholesterolaemia

Primary Objectives * To assess the efficacy of rosuvastatin in paediatric patients with familial hypercholesterolaemia. * To establish long-term safety, tolerability and efficacy of rosuvastatin in paediatric patients with familial…

The Pharmacokinetics of an oral uracil dose in patients with colorectal carcinoma.

To compare the pharmacokinetic profile of uracil in cancer patients and healthy volunteers

A Phase III, randomised, double blind, placebo controlled, parallel group study, to evaluate the safety and efficacy of subcutaneous implants of afamelanotide (16 mg) in patients suffering from polymorphic light eruption (PLE).

Primary Objective - To determine if afamelanotide can reduce the severity of PLE related pruritis.Secondary Objectives- To determine if afamelanotide can reduce the frequency of PLE episodes;- To determine if afamelanotide can reduce the duration of…

A Phase III, Multicentre, Double-Blind, Randomised, Placebo-Controlled Study to Confirm the Safety and Efficacy of Subcutaneous Bioresorbable Afamelanotide Implants in Patients with Erythropoietic Protoporphyria (EPP).

Primary objective: - To determine whether afamelanotide can reduce the severity of phototoxic reactions in patients with EPPSecondary objectives:- To determine whether afamelanotide can reduce the number of phototoxic reactions in patients with EPP…

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…

CER-001 Infusions in Subjects with Familial HDL-c Deficiency

Evaluate the effects of CER-001 - adminstered as iv infusion - on plaque size/burden in arteries, plaque inflammation in the artery wall and the rate of cholesterol transport in the body