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A phase 2, double-blind, randomized, placebo-controlled pharmacokinetic trial in two parallel groups to investigate possible drug- drug interactions between stiripentol or valproate and GWP42003-P in patients with epilepsy.

Primary Objective: To determine whether GWP42003-P affects the pharmacokinetic (PK) profile of stiripentol (STP) or valproate (VPA).Secondary Objective: To assess the safety and tolerability of GWP42003-P in the presence of STP or VPA.To assess…

A Randomized, Double-Blind, Placebo-Controlled Phase 2 Study to Evaluate the Safety and Efficacy of Avacopan (CCX168) in Patients with C3 Glomerulopathy

The primary objective is to evaluate the efficacy of avacopan compared to placebo based on histologic changes in kidney biopsies taken before and during treatment.The secondary objectives of this study include evaluation of:1. The safety of avacopan…

A Phase 3 Double-blind, Randomized, Placebo-controlled, Multicenter Study of Voxelotor Administered Orally to Patients With Sickle Cell Disease

Primary ObjectiveThe primary objective is to assess the effect of vexelotor compared to placebo on improvement in hemoglobinSecondary ObjectivesThe secondary objectives are t o evaluate the effects of voxelotor compared to placebo on : - Clinical…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study To Investigate The Efficacy And Safety Of Mongersen (GED-0301) For The Treatment Of Adult and Adolescent Subjects With Active Crohn*s Disease

To evaluate the efficacy of GED-0301 at Week 12, administered as either a single 160 mg tablet or as four 40 mg tablets, compared with placebo on clinical activity in subjects with active CD

Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of 3 Doses of MOTREM in Patients with Septic Shock. A Randomised, Double-blind, Two-stage, Placebo Controlled Study.

The objectives of the study are to: -To evaluate the safety and tolerability of MOTREM in patients with septic shock. -To evaluate the effects of MOTREM exposure over up to 5 days in patients with septic shock -To evaluate the PK/PD and dose/PD…

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Induction Study of the Efficacy and Safety of Risankizumab in Subjects with Moderately to Severely Active Crohn's Disease

The objective is to evaluate the efficacy and safety of risankizumab versus placebo during induction therapy in subjects with moderately to severely active CD.

A Randomized, Double Blind, Placebo-Controlled, Study to Assess the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys with Duchenne Muscular Dystrophy.

Primary* To compare the efficacy of RO7239361 (BMS-986089) to placebo in ambulatory boys with Duchenne Muscular Dystrophy.Secondary* To compare the efficacy of RO7239361 (BMS-986089) to placebo using the following tests:- 4 stair climb velocity (…

A Phase 3 Randomized, Double-blind, Placebo-controlled, Parallel-group Efficacy and Safety Study of SHP647 as Induction Therapy in Subjects with Moderate to Severe Ulcerative Colitis (FIGARO UC 301)

Primary: To evaluate the efficacy of ontamalimab in inducing remission, based on composite score of patient reported symptoms and centrally read endoscopy, in subjects with moderate to severe ulcerative colitis (UC).Key Secondary:* To evaluate the…

A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study with an Open-Label Extension to Evaluate the Efficacy and Safety of Givosiran in Patients with Acute Hepatic Porphyrias (ENVISION)

Primary:Evaluate the effect of subcutaneous (SC) givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks requiring hospitalization, urgent healthcare visit, or intravenous (IV) hemin administration at home in patients with acute…

A Multicenter, Randomized, Double-Blind, Placebo Controlled Induction Study to Assess the Efficacy and Safety of Risankizumab in Subjects with Moderately to Severly Active Crohn's Disease Who Failed Prior Biologic Treatment

The objective is to evaluate the efficacy and safety of risankizumab versus placebo during induction therapy in subjects with moderately to severely active CD.

Randomized, Double-Blind, Phase 3B Trial to Evaluate the Safety and Efficacy of 2 Treatment Regimens of Aztreonam 75 mg Powder and Solvent for Nebulizer Solution / Aztreonam for Inhalation Solution (AZLI) in Pediatric Subjects with Cystic Fibrosis (CF) and New Onset Respiratory Tract Pseudomonas aeruginosa (PA) Infection/Colonization

To evaluate the safety and efficacy of a 14-day course vs a 28 day course of AZLI 75 mg three times a day (TID) in subjects with new onset PA respiratory tract colonization/infection as determined by PA eradication over a 28-day post treatment…

A Phase 3 Randomized, Double-blind, Placebo controlled, Parallel group Efficacy and Safety Study of SHP647 as Maintenance Therapy in Subjects With Moderate to Severe Ulcerative Colitis (FIGARO UC 303)

Primary: To evaluate the efficacy of ontamalimab as maintenance treatment of remission, based on composite score of patient reported symptoms and centrally read endoscopy, in subjects with moderate to severe ulcerative colitis (UC).Key Secondary:*…

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…

An International, Multicenter, Randomized, Double-Blind, Parallel-Group Phase 2 Study Evaluating the Safety and Efficacy of Diacerein 1% Ointment Topical Formulation in Subjects with Epidermolysis Bullosa Simplex (EBS) [DELIVERS Study]

To compare the efficacy of Diacerein 1% Ointment to Control Ointment when applied once-daily in subjects with EBS.To compare the effects of Diacerein 1% Ointment to Control Ointment in subjects with EBS in:* Safety and tolerability* Pruritus * Pain…

Reducing Aggressive behaviour among People with an Intellectual Disability through Supplementation of Vitamins, Minerals and n-3 Fatty Acids

The objective of this study is to test whether supplementation of vitamins, minerals and n-3 fatty acids may reduce aggressive behavior in people with intellectual disabilities. The research is relevant for the practice of social work. The…

An Open Label, 1-Year Trial, including a Double-Blind Placebo-Controlled Withdrawal Period, of Setmelanotide (RM-493), a Melanocortin 4 Receptor (MC4R) Agonist, in Early Onset Leptin Receptor (LEPR) Deficiency Obesity due to Bi-Allelic Loss-of-Function LEPR Genetic mutations

The main purpose of this trial is to find out if an investigational drug called setmelanotide (RM-493), which mimics MSH, can help control body weight. The efficacy of setmelanotide will be compared to the efficacy of a placebo during 4 to 8 weeks.

A double-blind, randomized, placebo-controlled study to investigate the efficacy and safety of cannabidiol (GWP42003-P, CBD) as add-on therapy in patients with tuberous sclerosis complex who experience inadequately-controlled seizures

Primary Objective Blinded Phase:• To evaluate the efficacy of GWP42003-P as add-on therapy in reducing the frequency of seizures when compared with placebo in patients with TSC.Open-label Extension:• To evaluate via the adverse events (AE) profile…

CANGLIA: Endocannabinoid control of microglia activation as a new therapeutic target in the treatment of schizophrenia

The primary objective of this study is to compare microglia activation as measured with proton Magnetic Resonance Spectroscopy (1H-MRS) between recent-onset schizophrenia patients who are randomised to CBD and those randomised to placebo. Secondary…

Randomized, double-blind, placebo controlled, multi-centre superiority phase II study to evaluate the safety, pharmacokinetic, efficacy of gabapentin liquid formulation as add-on to morphine in children from 3 months to less than 18 years of age experiencing severe chronic neuropathic or mixed pain.

The primary objective of this study is to assess the efficacy of gabapentin as add-on to morphine for the treatment of severe chronic neuropathic or mixed pain in children from 3 months to less than 18 years of age. Secondary objectives 1. To assess…

A three-part randomized, double-blind, placebo-controlled study to investigate the efficacy and safety of secukinumab treatment in Juvenile Idiopathic arthritis subtypes of psoriatic and enthesitis-related arthritis

The purpose of the study is to investigate the use of secukinumab (AIN457) treat ment in children from 2 to18 years of age with either active Enthesitis -Related Arthritis (ERA) or Juvenile Psoriatic Arthritis (JPsA) subtypes of Juvenile Idiopathic…