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A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Fixed-Dose, Multicenter Study to Examine the Efficacy and Safety of ZX008 in Subjects with CDKL5 Deficiency Disorder Followed by an Open-Label Extension

This study has been transitioned to CTIS with ID 2023-506269-78-00 check the CTIS register for the current data. Objectives for Part 1: Primary (Efficacy): To demonstrate that the efficacy of fenfluramine (ZX008) 0.8 mg/kg/day is superior to placebo…

Phenotyping and classifying of childhood and adult asthma exacerbations: towards future personalised treatment. An observational multicentre study.

The primary objective of the study is to determine the relationship between exacerbation treatment response at day 7 and the phenotypical characteristics of asthma exacerbations. Secondary objectives are 1) developing a prediction model based on…

Effect of an immune-supportive diet on gut permeability and allergic symptoms in children with peanut and/or nut allergy.

Therefore, the purpose of our study is to study, in peanut and nut allergic children:1. the relationship between gut permeability and threshold levels to peanut or nuts;2. the effect of an immune-supportive diet on gut permeability, coexisting…

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…

ACT your way: A transdiagnostic intervention for multiple disorders in transitional-age youth

The primary aim is to evaluate the effectiveness of ACT your way, by comparing ACT your way to treatment as usual (TAU) in TAY. The secondary aim is to evaluate the cost-effectiveness of ACT your way, by comparing ACT your way to TAU in TAY. The…

An Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Efficacy and Pharmacokinetics of Multiple Doses of ELA026 in Participants with Secondary Hemophagocytic Lymphohistiocytosis (sHLH).

Primary Objectives:• To determine the safety of ELA026 administered IV and SC to participants with sHLH.• To identify the RP3D and schedule for ELA026.Secondary Objectives:• To characterize the pharmacokinetic (PK) profile of ELA026 administered IV…

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…

Pharmacokinetic-guided dosing of emicizumab in congenital haemophilia A patients - The DosEmi study

This study has been transitioned to CTIS with ID 2024-515528-35-00 check the CTIS register for the current data. Primary objectiveTo determine whether individualized pharmacokinetic (PK)-guided dosing of emicizumab is non-inferior to conventional…

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 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…

Phase IIIb, open-label, single-arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101 administered intrathecally (1.2 x 1014 vector genomes) to participants 2 < 18 years of age with spinal muscular atrophy (SMA) who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®)

The purpose of this study is to characterize the safety and tolerability of OAV101 IT in participants who have discontinued treatment with nusinersen (Spinraza®) or risdiplam (Evrysdi®). The data from this study will expand on the data generated…

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

Optimising surgical anastomosis in ileocolic resection for Crohn's disease to reduce recurrent disease: A Randomized controlled trial comparing hand-sewn (END-TO-END or Kono-S) to stapled anastomosis (END2END study)

To understand if handsewn (end to end and Kono S side to side) anastomoses is superior to side to side stapled anastomosis after ileocolic resection for Crohn's disease with respect to endoscopic recurrence, gastrointestinal function and costs.

A PHASE 2, RANDOMIZED, OPEN-LABEL STUDY OF ENCORAFENIB AND CETUXIMAB PLUS
PEMBROLIZUMAB VERSUS PEMBROLIZUMAB ALONE IN PARTICIPANTS WITH PREVIOUSLY UNTREATED BRAF V600E-MUTANT, MSI-H/DMMR METASTATIC COLORECTAL CANCER

This study has been transitioned to CTIS with ID 2024-512119-34-00 check the CTIS register for the current data. Comparing the efficacy of encorafenib and cetuximab plus pembrolizumab (triplet group [group A]) vs. pembrolizumab (control group [group…

An open-label, single-center, exploratory study of the safety and efficacy of avalglucosidase alfa in patients with non-classic Pompe disease aged >= 5 years.

This study has been transitioned to CTIS with ID 2024-518215-18-00 check the CTIS register for the current data. To explore safety, tolerability and efficacy of avalglucosidase alfa in previously treated alglucosidase alfa late-onset Pompe disease…