Search for a trial

A phase 3 open-label study of PTC923 (Sepiapterin) in Phenylketonuria

This study has been transitioned to CTIS with ID 2023-509229-31-00 check the CTIS register for the current data. Primary:• To evaluate the long-term safety of PTC923 in subjects with phenylketonuria (PKU)• To evaluate changes from baseline in…

PNOC022: A Combination Therapy Trial using an Adaptive Platform Design for Children and Young Adults with Diffuse Midline Gliomas (DMGs) including Diffuse Intrinsic Pontine Gliomas (DIPGs) at Initial Diagnosis, Post-Radiation Therapy and at Time of Progression

This study has been transitioned to CTIS with ID 2023-507598-16-00 check the CTIS register for the current data. Primary objectives:Cohorts 1 and 2Maintenance Arm(s) Combinations:-To assess efficacy of combination therapy with ONC201 and novel agent…

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to
Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase
Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515024-37-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in…

Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study

This study has been transitioned to CTIS with ID 2024-516124-34-00 check the CTIS register for the current data. Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3)…

An open-label, single arm, multicenter extension study to evaluate long-term safety and tolerability of inclisiran in participants with heterozygous or homozygous familial hypercholesterolemia who have completed the adolescent ORION-16 or ORION-13 studies (VICTORION-PEDS-OLE)

This study has been transitioned to CTIS with ID 2023-507278-41-00 check the CTIS register for the current data. The primary objective of the study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH

Phase 2 Open-label Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Sotatercept (MK-7962) in Children from 1 to Less Than 18 Years of Age With PAH on Standard of Care

This study has been transitioned to CTIS with ID 2023-504861-22-00 check the CTIS register for the current data. This study will assess the safety and tolerability of sotatercept in pediatric participants with PAH WHO Group 1 who receive PAH…

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…

MyKids: Molecular Identification and Characterization of non-Rhabdomyosarcoma Soft Tissue Sarcoma in Kids, Adolescents and Young Adults: an EpSSG NRSTS study

The MyKids study is a biological study towards the molecular profiling of pediatric NRSTS, the development of NRSTS tumoroid cultures, the establishment of liquid biopsy biomarkers and the molecular profiling of post-treatment tissue samples. The…

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…

Navigating uncertainty in gender incongruence and differences in sex development (DSD)/intersex conditions

The project aims are to 1) provide an integrative understanding and conceptualization of uncertainty in transgender and DSD/intersex care, and to 2) support children/adolescents, parents and healthcare professionals in recognizing, discussing and…

Metabolomics in Dravet syndrome

Primary Objectives: 1. To assess the metabolic profile of patients with Dravet syndrome and identify metabolic variations that contribute to the pathophysiology of seizures and developmental delaySecondary Objectives:2. To assess the association…

Autonomous Blood Drawing Optimization and Performance Testing

This study has several objectives, in different (subsequent) study phases:• A1. Optimize and validate the technology of the VD to reach non-inferiority• B1. Demonstrate non-inferior performance and safety of the VD, for CE marking• B2. Demonstrate…

BRAINmodel: Clinical characterization and understanding of excitation/inhibition ratio homeostasis in children with genetic syndromes caused by chromatin- or synapse regulatory factors.

In this study we aim to further characterize E/I balance (at different translational levels), and its relation to metabolic- and immune processes, clinical profiles, behavior and cognition

A Phase 1/2 Dose Escalation and Dose Expansion Study of ZN-c3 in Combination with Gemcitabine in Adult and Pediatric Subjects with Relapsed or Refractory Osteosarcoma.

Primary:Phase 1: • To investigate the safety and tolerability, including identification of the maximum tolerated dose (MTD) and recommended Phase 2 dose (RP2D) of ZN-c3 in combination with gemcitabinePhase 2: • To evaluate the clinical activity of…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva (PROGRESS)

This study has been transitioned to CTIS with ID 2023-504129-38-00 check the CTIS register for the current data. Primary:To determine the efficacy of INCB000928 for the prevention of new HO in participants with FOP.Key Secondary:To further evaluate…

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)…

An Open-Label, Long Term Safety and Efficacy Study of Donidalorsen in the Prophylactic Treatment of Hereditary Angioedema (HAE)

This study has been transitioned to CTIS with ID 2023-509201-77-00 check the CTIS register for the current data. Primary ObjectiveTo evaluate the safety of long-term dosing with donidalorsen in patients with HAE. Secondary ObjectivesTo evaluate the…

A Phase 3b/4 Randomized, Open-label, Efficacy Assessor Blinded Study, Comparing the Safety and Assessor Blinded Efficacy of Upadacitinib to Dupilumab in Subjects with Moderate to Severe Atopic Dermatitis (Level-Up)

This study compares upadacitinib to dupilumab in adolescent and adult participants with moderate to severe AD who have inadequate response to systemic therapies. Adverse events and change in the disease activity will be assessed.

A Phase 2 Study to Evaluate the Efficacy and Safety of Belzutifan (MK-6482, formerly PT2977) Monotherapy in Participants with Advanced Pheochromocytoma/Paraganglioma (PPGL), Pancreatic Neuroendocrine Tumor (pNET), or von Hippel-Lindau (VHL) Disease-Associated Tumors, Advanced Gastrointestinal Stromal Tumor (wt GIST), or Advanced Solid Tumors With HIF-2α related Genetic Alterations

This study has been transitioned to CTIS with ID 2023-504853-11-00 check the CTIS register for the current data. To evaluate the ORR of belzutifan per RECIST 1.1 by blinded independent central review (BICR).

Difference in patient reported outcomes and patellar tracking before and after isolated MPFL reconstruction.

Evaluate pre- and postoperative reported outcomes in patients receiving a primary isolated soft-tissue loop MPFL reconstruction for the treatment of patellar instability. Additionally, it is aimed to investigate the difference in patella tracking…