Search for a trial

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…

A Randomized, Controlled, Open-label, Multicenter, Phase IIb
Safety and Efficacy Study of HGT-1410 (Recombinant Human
Heparan N Sulfatase) Administration via an Intrathecal Drug
Delivery Device in Pediatric Patients with Early Stage
Mucopolysaccharidosis Type IIIA Disease

The primary objective of this study is to assess the potential clinical efficacy of HGT-1410administered via a surgically implanted IDDD in patients with MPS IIIA. Efficacy will bemeasured as a meaningful amelioration in the progression of cognitive…

Effects of beef protein consumption on energy intake - The protein leverage hypothesis

To determine ad libitum daily energy intake in response to protein/carbohydrate and fat ratio over 12 consecutive days, and in relation to age, gender, BMI and FTO gene alleles.

A Randomized, Crossover, Pharmacokinetic and Pharmacodynamic Study to Determine the Safety and Efficacy of Cysteamine Bitartrate Delayed-release Capsules (RP103), Compared to Cystagon® in Patients with Nephropathic Cystinosis

The objective of this pivotal study is to assess the PK and PD as well as safety and tolerability of RP103 compared to Cystagon® in patients with nephropathic cystinosis. Results of this Phase 3 study will be used to support the registration…

A randomized, double-blind, placebo-controlled crossover study to assess the effect of 12-week fibre supplementation on mixed-meal challenge response in adults

Primary ObjectiveTo relate microbiome modulation by dietary fibre supplementation, as measured by 16S rRNA sequencing, to changes in response to a mixed-meal metabolic challenge (PhenFlex) as measured by composite biomarker score.Secondary…

A Global, Phase 1/2, Open-Label, Dose Optimization Study to Evaluate the Safety, Tolerability, Pharmacodynamics, and Pharmacokinetics of mRNA-3705 in Participants with Isolated Methylmalonic Acidemia Due to Methylmalonyl-CoA Mutase Deficiency

This study has been transitioned to CTIS with ID 2022-502492-32-00 check the CTIS register for the current data. PrimaryPart I: Evaluate the safety and tolerability of mRNA-3705 administered via IV infusion to participants with isolated MMA due to…

Physical Exercise Prehabilitation Program in mEtabolic suRgery

The aim of this study is to investigate whether prehabilitation could enhance preoperative physical condition and weight loss before bariatric surgery and induce a lifestyle behavior change.

An Open-Label Extension Study of the Safety of Relacorilant (CORT125134) in the Treatment of the Signs and Symptoms of Endogenous Cushing Syndrome

Primary Objectives: Safety Assessments: Effect of Administration of Relacorilant on: • Incidence of TEAEs (assessed monthly): TEAEs, SAEs, treatment-related TEAEs, TEAEs leading to early discontinuation of study treatment• Clinical laboratory tests…