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68Ga-SATO in paediatric neuroblastoma patient; exploratory, safety, non-randomized, open label, comparative study - GAP NBL study*

This study has been transitioned to CTIS with ID 2024-513843-10-00 check the CTIS register for the current data. Primary objective: Assess the short term safety and tolerability of 68Ga-SATO in pediatric patients with NBLSecondary objectives: -…

Clinical safety study on 5-Aminolevulinic acid (5-ALA) in children and adolescents with brain tumors

This study has been transitioned to CTIS with ID 2024-517575-20-00 check the CTIS register for the current data. This study proposes a safety study in children in which tumors are operated on using fluorescence-guided resection which are similar to…

A two-cohort, open-label, single arm, multicenter study to evaluate efficacy, safety and
tolerability, pharmacokinetics and pharmacodynamics, of emapalumab in children and
adults with macrophage activation syndrome (MAS) in Still*s disease (including systemic
juvenile idiopathic arthritis and Adult onset Still*s disease) or with MAS in Systemic lupus
erythematous

This study has been transitioned to CTIS with ID 2024-516153-52-00 check the CTIS register for the current data. Primary objective: To demonstrate efficacy of emapalumab in the treatment of patients in:• Cohort 1: Macrophage activation syndrome (MAS…

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.

Effectivity and Working Mechanisms of Schema Therapy for patients with a Treatment Resistant Depression

Main research question:Does group-based schema therapy result in a reduction of depressive symptoms when comparing symptom change during the treatment to patients* baseline symptom level?Sub research questions:Does group-based schema therapy result…

A phase II trial of [177]Lutetium-DOTATATE in children with primary refractory or relapsed high-risk neuroblastoma

This study has been transitioned to CTIS with ID 2023-503684-42-00 check the CTIS register for the current data. Main Study Objective• To confirm the dose and assess response to single agent 177Lutetium-DOTATATE treatmentin patients with relapsed or…

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…

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 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)…

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).

An Open-Label Study to Evaluate the Pharmacokinetics, Pharmacodynamics, and Safety of Bempedoic
Acid in Pediatric Patients (6 to 17 Years of Age) with Heterozygous Familial Hypercholesterolemia

This study has been transitioned to CTIS with ID 2024-515864-30-00 check the CTIS register for the current data. Primary Objectives:• To assess the pharmacokinetics (PK) of bempedoic acid (ETC-1002 and ESP15228) in pediatric patients (6 to 17 years…

A PROSPECTIVE, RANDOMIZED, OPEN-LABEL PHASE 2 STUDY TO EVALUATE THE SUPERIORITY OF INOTUZUMAB OZOGAMICIN MONOTHERAPY VERSUS ALLR3 FOR INDUCTION TREATMENT OF CHILDHOOD HIGH RISK FIRST RELAPSE B-CELL PRECURSOR ACUTE LYMPHOBLASTIC LEUKAEMIA

This study has been transitioned to CTIS with ID 2023-509810-13-00 check the CTIS register for the current data. Primary Objectives Efficacy: To demonstrate the superiority of InO monotherapy vs ALLR3 induction in paediatric participants between 1…

An Open-Label, Single-Arm, Phase 2 Study to Evaluate the Safety, Pharmacokinetics, and Biologic Activity of Pegcetacoplan in Pediatric Patients with Paroxysmal Nocturnal Hemoglobinuria

The objectives of the study are to assess safety and tolerability, pharmacokinetics (PK), and biological activity (including efficacy and pharmacodynamics [PD]) of multiple subcutaneous doses of pegcetacoplan in pediatric patients with paroxysmal…

A multi-center, open-label study to determine the dose and safety of oral asciminib in pediatric patients with Philadelphia chromosome positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with one or more tyrosine kinase inhibitors.

This study has been transitioned to CTIS with ID 2023-508129-28-00 check the CTIS register for the current data. The primary objective of this study is to characterize the pharmacokinetic (PK) profile of asciminib in pediatric patients, with the…

PHASE 1/2 STUDY TO EVALUATE PALBOCICLIB (IBRANCE® ) IN
COMBINATION WITH IRINOTECAN AND TEMOZOLOMIDE AND/OR IN COMBINATION WITH TOPOTECAN AND CYCLOPHOSPHAMIDE IN
PEDIATRIC PATIENTS WITH RECURRENT OR REFRACTORY SOLID
TUMORS

Primary: To compare the efficacy of palbociclib in combination with TMZ and IRN vs TMZ and IRN chemotherapy alone in the treatment of children, adolescents, and young adults with recurrent or refractory EWS.Secundary: To further compare the efficacy…

Phase II trial of nivolumab for pediatric and adult relapsing/refractory ALK+ anaplastic large cell lymphoma, for evaluation of response in patients with progressive disease (Cohort 1) or as consolidative immunotherapy in patients in complete remission after relapse (Cohort 2)

Primary objectivesCohort 1 Estimate the efficacy of nivolumab treatment in patients with relapsed/refractory ALK+ ALCL in terms of best objective response rate within thefirst 24 weeksCohort 2 Estimate the efficacy of nivolumab treatment as…

An open-label, first-in-human, dose-escalation/expansion study of SAR443579 administered as single agent by intravenous infusion in adult and pediatric participants with relapsed or refractory acute myeloid leukemia (R/R AML), B-cell acute lymphoblastic leukemia (B-ALL), high risk-myelodysplasia (HR-MDS) or blastic plasmacytoid dendritic cell neoplasm (BPDCN)

This study has been transitioned to CTIS with ID 2023-508357-58-00 check the CTIS register for the current data. The aim of the escalation portion of this study, in which SAR443579 is administered for the first time in humans, is to establish the…

A phase 2/3 Adaptive, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of VX-147 in Adult and Pediatric Subjects With APOL1-mediated Proteinuric Kidney Disease

• To evaluate the efficacy of VX-147 to reduce proteinuria• To evaluate the efficacy of VX 147 on renal function as measured by eGFR slope