Search for a trial

An Open-label, Multicenter, Phase 2 Study Evaluating the Efficacy and Safety of Daratumumab in Pediatric and Young Adult Subjects >=1 and <=30 Years of Age With Relapsed/Refractory Precursor B-cell or T-cell Acute Lymphoblastic Leukemia or Lymphoblastic Lymphoma

The primary objective is to evaluate the efficacy of daratumumab in addition to standard chemotherapy in relapsed/refractory B-cell acute lymphoblastic leukemia (ALL) and T-cell ALL as measured by the complete response (CR) rate.The secondary…

An Open-label, Randomized, Multi-center, Parallel Group, Two-arm Study to Assess the Safety, Overall Tolerability, and Antiviral Activity of Brincidofovir versus Standard of Care for Treatment of Adenovirus Infections in High-risk Pediatric Allogeneic Hematopoietic Cell Transplant Recipients

The primary objective of this study is to compare the safety, overall tolerability, and virologic response of BCV vs. SoC (i.e., investigator-assigned therapy) for the treatment of AdV infection in high-risk pediatric allogeneic HCT recipients. A…

Providing optimal exposure to youth with anxiety disorders: effectiveness and working mechanisms

The goal of the study is to examine whether optimal exposure is more effective than CBT as usual in youth with anxiety disorders. The secondary objective is to investigate whether decreases in harm expectancies are predictive of treatment outcome,…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Efficacy and Safety Study of Palovarotene in Subjects with Multiple Osteochondromas

Primary Objective• To evaluate the efficacy of two dosage regimens of palovarotene compared with placebo in preventing new osteochondromas (OCs) in subjects with multiple osteochondromas (MO) due to exostosin 1 (Ext1) or exostosin 2 (Ext2) mutations…

An Open-Label Phase 2 Proof-of-Concept Study in Patients with C3 Glomerulopathy (C3G) or Immune-Complex Membranoproliferative Glomerulonephritis (IC-MPGN) Treated with ACH-0144471.

See protocol P5:The primary objective of this study is to evaluate the efficacy of 12 months of oral ACH-0144471 in participants with C3G or ICMPGNbased on histologic scoring and proteinuriaThe secondary objectives of this study are: * To evaluate…

An open-label single-arm Phase IIb study of F901318 as treatment of invasive fungal infections due to Lomentospora prolificans, Scedosporium spp., Aspergillus spp., and other resistant fungi in patients lacking suitable alternative treatment options.

Primary:• Describe the Data Review Committee (DRC)-adjudicated efficacy of F901318 as treatment for infections due to resistant fungi in patients lacking suitable alternative treatment options.Secondary:• Describe the safety of F901318 as treatment…

A Phase 1/2, Multicenter, Dose-Escalating Study To Evaluate the Safety,
Pharmacokinetics, Pharmacodynamics, and Efficacy Of Quizartinib
Administered in Combination with Re-Induction Chemotherapy, and as a
Single-Agent Continuation Therapy, in Pediatric Relapsed/Refractory AML
Subjects Aged 1 Month to <18 Years (and Young Adults Aged up to 21 Years)
with FLT3-ITD mutations

This study has been transitioned to CTIS with ID 2023-510009-16-00 check the CTIS register for the current data. Primary Objectives:• Phase 1 Only: To determine the recommended Phase 2 dose(RP2D) of quizartinib, in combination with chemotherapy,…

International multicenter, open-label, phase 2 study to treat molecular relapse of pediatric acute myeloid leukemia with azacitidine

• Primary Objective: To evaluate the effect of azacitidine treatment in AML subjects at molecular relapse after CR1 with regard to molecular response prior to further treatment (reinduction / HSCT)• Secondary Objectives:o To assess safety of…

An open-label study to investigate the safety, tolerability, and Pharmacokinetics/Pharmacodynamics of risdiplam (RO7034067) in adult and pediatric patients with spinal muscular atrophy

This study has been transitioned to CTIS with ID 2023-506739-14-00 check the CTIS register for the current data. Primary ObjectivesThe primary objectives of this study are as follows:• To evaluate the safety and tolerability of risdiplam.• To…

A pilot, open-label, single arm, multicenter study to evaluate safety, tolerability, pharmacokinetics and efficacy of intravenous administrations of emapalumab, an anti-interferon gamma (anti-IFN&gamma;) monoclonal antibody, in patients with systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still*s Disease (AOSD) developing Macrophage Activation Syndrome/ secondary HLH (MAS/sHLH)

The aim of this study is to investigate how safe, effective, and well-tolerated multiple infusions of the experimental study treatment, emapalumab (the study medication) are in controlling this disease, as well as to check the concentrations of…

A Multicenter, Randomized, Double-Blind, Placebo-Controlled Phase 2 Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis

Primary efficacy objectives:To evaluate the efficacy of lenabasum 20 mg twice per day (BID) compared to placebo in the treatment of cystic fibrosis (CF) by assessing the rate of pulmonary exacerbations (PEx) using primary definition of PEx.Secondary…

A Phase 1/2 Study of Oral LOXO-292 in Patients with Advanced Solid Tumors, Including RET Fusion-Positive Solid Tumors, Medullary Thyroid Cancer, and Other Tumors with RET Activation (LIBRETTO-001)

Primary objective:To determine the MTD/recommended dose for further study of oral LOXO-292 in patients with advanced solid tumors, including RET-fusion NSCLC, MTC, and other tumors with increased RET activity.Secondary objectives:* To determine the…

Phase II open-label global study to evaluate the effect of dabrafenib in combination with trametinib in childeren and adolescent patients with BRAF V600 mutation positive low grade glioma (LGG) or relapsed or refractory High Grade Glioma (HGG)

This study aims to demonstrate the effectiveness of dabrafenib with trametinib in pediatric patients with BRAF V600 mutant relapsed refractory HGG.This study aims to demonstrate the effectiveness of dabrafenib with trametinib compared to…

A Phase 2, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Dose-Ranging Study to Evaluate the Safety and Efficacy of Eluxadoline in Pediatric Participants (Age 6 to 17 Years) with Irritable Bowel Syndrome with Diarrhea (IBS-D)

The primary objectives of this study are:- To explore the therapeutic effect of eluxadoline in treating IBS-D in pediatric patients 6-17 years of age.- To evaluate the pharmacokinetics (PK) of eluxadoline in pediatric patients with IBS-D.- To…

Pharmacokinetic evaluation and tolerability of dry powder tobramycin via the Cyclops® in children with cystic fibrosis

The main objectives are to investigate the pharmacokinetic properties of DP tobramycin via the Cyclops® at different dosages in children with CF, together with the local tolerability.

A multicentre study for the long-term follow-up of HLH patients who received treatment with NI-0501, an anti-interferon gamma monoclonal antibody

Study Objectives:* To monitor the long-term safety profile of NI-0501* To assess HLH patients* survival after NI-0501 treatment* To assess duration of response to NI-0501 treatment (i.e. maintenance of HLH control)* To assess post-HSCT outcome…

A Phase 1/2, open-label, single arm, multicohort, multicenter trial to evaluate the safety and efficacy of JCAR017 in pediatric subjects with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL) and B-cell non-Hodgkin lymphoma (B-NHL) (TRANSCEND PEDALL)

Phase 1:To identify the recommended Phase 2 dose (RP2D) of JCAR017 in pediatric subjects withCD19+ r/r B-ALL.Phase 2:To evaluate the following efficacy endpoints of the JCAR017 RP2D identified in Phase 1, in the following three diseasecohorts:•…

A Phase IIb, 2-Arm, Randomized, Double-blind, Placebo-Controlled, Multicentre Study to Optimize Diamyd® Therapy Administered into Lymph Nodes Combined with Oral Vitamin D to Investigate the Impact on the Progression of Type 1 diabetes

Primary objective:The primary objective is to evaluate the efficacy of Diamyd, administered into lymph nodes in combination with an oral vitamin D regimen, compared to placebo in terms of preserving endogenous insulin secretion as measured by C-…

LONG-TERM FOLLOW-UP PROTOCOL FOR SUBJECTS TREATED WITH GENE-MODIFIED T CELLS

This study has been transitioned to CTIS with ID 2023-504201-36-00 check the CTIS register for the current data. Per Health Authority guidelines for gene therapy medicinal products that utilize integrating vectors (eg, lentiviral vectors), long-term…

A Phase 1/2 Study of the Oral TRK Inhibitor larotrectinib in Pediatric patients with Advanced Solid or Primary Central Nervous System Tumors

This study has been transitioned to CTIS with ID 2022-502668-20-00 check the CTIS register for the current data. PHASE 1 OBJECTIVES:Primary:To determine the safety of oral larotrectinib , including dose-limiting toxicity (DLT), in pediatric patients…