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MyopiaX Treatment for the Reduction of Myopia Progression in Children and Adolescents: Safety and Efficacy Investigation

Primary objectiveTo evaluate the effects of treatment with MyopiaX on the rate of myopia progression in children and adolescents as reflected in spherical equivalent refraction and axial length changes at 6months relative to baseline.Secondary…

A phase 3 study of PTC923 in subjects with phenylketonuria

Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…

Prospective clinical trial to evaluate the efficacy of acetazolamide for the treatment of cystoid macular edema in inherited retinal dystrophies: the CAR trial

Our study objective is to evaluate the efficacy of acetazolamide for the treatment of cystoid macula edema in inherited retinal dystrophies in anticipation of future clinical trials.

A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Patients 6 to 17 Years of Age with Episodic Migraine - the REBUILD-1

This study has been transitioned to CTIS with ID 2023-505836-36-00 check the CTIS register for the current data. Main Objective:To demonstrate the superiority of galcanezumab versus placebo in the prevention of migraine in (at least) 1 of the…

A Phase 3 Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE)

Primary objective: The primary objective of the study is to evaluate the clinical efficacy of ISIS 721744 in patients with hereditary angioedema (HAE).Secondary objective: Evaluate the effects of ISIS 721744 on the quality and pattern of HAE attacks…

A Double-Blind, Placebo-Controlled, Multicenter Study With an Open-Label Extension to Evaluate the Efficacy and Safety of SRP-4045 and SRP-4053 in Patients With Duchenne Muscular Dystrophy

Primary Objective:Double-blind period : Evaluate the effect of SRP-4045 and SRP-4053 (combined-active group) compared to placebo on ambulation, endurance, and muscle function, as measured by the 6MWTSecondary Objectives:- Double-blind period:…

A multicenter, randomized, double-blind, placebo-controlled study of enzastaurin for the prevention of arterial events in patients with vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 mutations, followed by an open label extension (OLE)

The aim of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of enzastaurin in preventing new arterial events (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) leading to…

A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA

Effectiveness of a Brief Intensive Trauma Treatment for adolescents with (subclinical) PTSD: a multi-center randomized controlled trial

The aim of the study is:1) To test the effectiveness and cost-effectiveness of KIT for adolescents with (s)PTSD and comorbid symptoms. 2) To compare the outcomes from the current study with outcomes we have collected in two previous randomized…

A Phase 2/3, Multicenter, Double-Blind, Randomized Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult Participants With Neuronopathic or Non-Neuronopathic Mucopolysaccharidosis Type II

This study has been transitioned to CTIS with ID 2024-510990-21-00 check the CTIS register for the current data. The co-primary objectives are: To evaluate the CNS activity of DNL310 vs idursulfase as measured by the cerebrospinal fluid (CSF)…

A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Adolescent Patients 12 to 17 Years of Age with Chronic Migraine - the REBUILD-2

This study has been transitioned to CTIS with ID 2023-505835-11-00 check the CTIS register for the current data. PrimaryTo demonstrate the superiority of galcanezumab versus placebo in the prevention of migraine in an adolescent population (12 to 17…

Reduction of blood loss in pediatric osteotomies around the hip
- A randomized placebo-controlled trial with tranexamic acid -

This study has been transitioned to CTIS with ID 2024-516324-34-01 check the CTIS register for the current data. To evaluate (reduction of) intraoperative blood-loss with and without pre-operative TXA administration, in children undergoing a…

A Phase 3, Randomized, Double-blind, Placebo-controlled, Single-dose Study to Evaluate the Efficacy and Safety of Suvratoxumab in Mechanically Ventilated Adults and Adolescents for the Prevention of Nosocomial Pneumonia

Primary:- To evaluate the effect of suvratoxumab on reducing the incidence of nosocomial all-cause pneumonia.Secondary:- To evaluate the safety of a single IV dose of suvratoxumab.- To evaluate the effect of suvratoxumab on reducing the incidence of…

The effectiveness of blended Forensic Ambulant Systemic Treatment (FASTb): A randomized controlled trial comparing blended and regular FAST

The main goal of the current study is to investigate whether FASTb is equally effective as FASTr.

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…

A Phase III, Multicentre, Randomised, Double-Blind Study to Assess the Safety and Efficacy of
Emactuzumab vs. Placebo in Subjects with Tenosynovial Giant Cell Tumour.

Primary:To estimate the treatment effect of emactuzumab on objective response rate (ORR) by 6 months from initiation of therapy in the blinded phase compared to placeboSecondary:the effect of emactuzumab on clinical outcome assessments (COAs) for:o…

Multicentre prospective trial for extracranial malignant germ cell tumours including a randomized comparison of Carboplatin and Cisplatin

This study has been transitioned to CTIS with ID 2023-507582-25-00 check the CTIS register for the current data. PRIMARY OBJECTIVESThe primary objective of MAKEI V is to assess in a randomized comparison whether the efficacy of Carboplatin (600 mg/…

A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma*) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115

This study has been transitioned to CTIS with ID 2023-505643-39-00 check the CTIS register for the current data. Part A: PrimaryTo evaluate the safety and tolerability of single ascending dose of BIIB115 administered via IT bolus injection to…

A PHASE 3 TRIAL OF FIANLIMAB (REGN3767, ANTI-LAG-3) + CEMIPLIMAB VERSUS PEMBROLIZUMAB IN PATIENTS WITH PREVIOUSLY UNTREATED UNRESECTABLE LOCALLY ADVANCED OR METASTATIC MELANOMA

This study has been transitioned to CTIS with ID 2023-505772-30-00 check the CTIS register for the current data. From protocol amendment 3 27JUn2022, Clinical Study Protocol Synopsis, page 1Primary Objective• To demonstrate superiority of fianlimab…

Optimal inter stimulus interval in spinal mTc-MEP monitoring. A multicenter study.

The aim of this research proposal is to examine the effect of different ISIs on mTc-MEPs.Primary research question: What is the effect of different interstimulus intervals (ISIs) on mTc-MEP thresholds, peak-to-peak amplitudes, and area under the…