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A Phase 3 Multicenter, Multi-dose, Open-label Maintenance Study to Investigate the Long-term Safety and Efficacy of ZS (Sodium Zirconium Cyclosilicate), an Oral Sorbent, in Subjects with Hyperkalemia

Primary objective of this trial is to generate open-label, long-term (up to 12 months) safety and tolerability data for ZS in subjects with hyperkalaemia (S-K * 5.1 mmol/L)Secondary objectives:* To evaluate the portion of ZS-treated subjects in whom…

An open label, long term, safety and tolerability extension to a randomized, double-blind, placebo controlled study of LCQ908 in subjects with Familial Chylomicronemia Syndrome

The purpose of this study is to determine long-term safety and tolerability, and continued efficacy in lowering triglycerides of LCQ908 in subjects FCS.

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:…

A Long-Term, Open-Label, Safety and Efficacy Study of Cysteamine Bitartrate Delayed-release Capsules (RP103) in Patients with Cystinosis.

The objective of this pivotal study is to assess the long-term safety, tolerability, pharmacokinetics and pharmacodynamics of RP103 in pediatric and adult patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support…

Using D-Galactose as a food supplement in Congenital Disorders of Glycosylation (CDG)

The goal of this study is to better characterize the metabolic alterations and sugar structure alterations (glycosylation abnormalities) in patients diagnosed with Congenital Disorders of Glycosylation.

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…

A Randomized, Multicenter, Multinational, Phase 3B, Open-Label, Parallel-Group Study of Fabrazyme (agalsidase beta) in Treatment-Naive Male Pediatric Patients with Fabry Disease Without Severe Symptoms

The objectives of this open-label study are to evaluate the efficacy (GL-3 clearance), pharmacokinetics (PK), and safety parameters (including immunogenicity) for 2 alternative dose regimens of Fabrazyme (0.5 mg/kg every 2 weeks [q2w] and 1.0 mg/kg…

A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome)

To evaluate the long-term safety and efficacy of BMN 110 administration at 2.0 mg/kg/qw and 2.0 mg/kg/qow in patients with MPS IVA.

Evaluation of Insuman Implantable 400 IU/ml in patients with Type 1 diabetes treated with the Medtronic MiniMed Implantable Pump System using Insuplant 400 IU/mL

(1) To generate new clinical efficacy and safety data to support the registration of Insuman Implantable 400 IU/ml,(2) To ensure the continuation of the treatment of the patients already implanted and avoid pump explantation, up to the approval of…

An Open-label (OL) Extension Study evaluating the Long-Term Safety of OxabactTM in Subjects with Primary Hyperoxaluria (PH) who participated in the Double-Blind (DB) Efficacy Study

Primary Objective:To evaluate the Safety of OxabactTM with continued exposure.Secondary Objectives:To obtain additional efficacy data with up to 48 weeks continuous exposure to Oxabact*.

A randomized, double-blind trial comparing the efficacy and safety of a fixed combination of fenofibrate and metformin vs rosiglitazone in patients with type 2 diabetes mellitus and dyslipidemia

Primary objective : Under conditions of first-line drug treatment in antidiabetic drug naïve/drug free patients (no antidiabetic drug treatment in the last 6 months), with type 2 diabetes mellitus and dyslipidemia, to show that : - the efficacy of a…

The effect of L-arginine on brown adipose tissue metabolism in South Asian and white Caucasian subjects

In this study, we will investigate the influence of the amino acid L-arginine, which enhances release of nitric oxide in the body, on BAT activity and metabolism in healthy subjects with overweight. This will increase understanding on the effect of…

A Randomized, Double-blind, Placebo-controlled, Multi-center, Cross-over Study of Rosuvastatin in Children and Adolescents (aged 6 to <18 years) with Homozygous Familial Hypercholesterolemia (HoFH)

To assess the efficacy of rosuvastatin 20 mg on low-density lipoprotein cholesterol (LDL-C), compared to placebo, after 6 weeks of treatment in pediatric patients with HoFH.

A Multicenter, Open-label Extension (OLE) Study to Assess the Long-term Safety and Efficacy of Evolocumab

To characterize the safety and tolerability of long-term administration of evolocumab in subjects with known coronary artery disease andhypercholesterolemia.

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…

The effect of bile acid binding with colesevelam on postprandial glucose concentrations in patients after RYGB and cholecystectomy : a meal test study

Objective: Primary : To compare the difference in glucose nadir during the MMT on and off pre-treatment with colesevelam in RYGB-CCx patients with a hypoglycaemia (RYGB-CCx-hypo) in the first MMT Secondary:To evaluate the difference in number of…

A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia

PrimaryThe primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).The primary objective for Part B of the study is to demonstrate a…

A long term extension trial of the Phase III lipid-lowering trials to assess the effect of long term dosing of inclisiran given as subcutaneous injections in subjects with high cardiovascular risk and elevated LDL-C (ORION-8)

The primary objectives are to evaluate:-The effect of inclisiran treatment on the proportion of subjects achieving prespecified low densitylipoprotein cholesterol (LDL-C) targets at end of study (EOS)-The safety and tolerability profile of long term…

An Open-Label Roll-Over Study to Evaluate the Long-Term Safety and Efficacy of DCR-PHXC Solution for Injection (subcutaneous use) in Patients with Primary Hyperoxaluria

This study has been transitioned to CTIS with ID 2024-512260-54-00 check the CTIS register for the current data. PrimaryTo evaluate the effect of DCR PHXC on estimated glomerular filtration rate (eGFR) in participants with PH1Secondary:To identify…

An open-label study to evaluate the long-term safety and efficacy of evinacumab in patients with homozygous familial hypercholesterolemia

The primary objectives of the study are- To evaluate the long-term safety and tolerability of evinacumab 15 mg/kg intravenous (IV) administered every 4 weeks (Q4W) in patients with homozygous familial hypercholesterolemia (HoFH).- To evaluate the…