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A Phase I/II study of Brigatinib in pediatric and young adult patients with ALK+ Anaplastic Large Cell Lymphoma, Inflammatory Myofibroblastic Tumors or other solid tumors

This study has been transitioned to CTIS with ID 2024-513412-10-00 check the CTIS register for the current data. Phase 1: • To determine the MTD/RP2D regimen of brigatinib monotherapy when administered in pediatric and AYA patients with ALK+ ALCL or…

Virtual Reality to Assess How Boys with Mild to Borderline Intellectual Disabilities Behave in Social Situations

The first aim is to investigate whether SIP indicators measured with VR are better predictors of aggression in real-life than SIP indicators measured with hypothetical vignettes in youth with MID-BIF. The second aim is to explore whether we can…

TEMOkids study (ORP-TMZ-I- b): A Population pharmacokinetic, acceptability and safety study for KIMOZO, a paediatric oral suspension of temozolomide

Primary objective:• Evaluate PK parameters of the oral suspension of temozolomide in the paediatric population aged 1 year and over.Secondary objectives:• Evaluate the safety of the oral suspension of temozolomide,• Evaluate the acceptability of the…

A multicenter open-label study to evaluate safety and dosimetry of Lutathera in adolescent patients with somatostatin receptor positive gastroenteropancreatic neuroendocrine (GEP-NET) tumors, pheochromocytoma and paragangliomas

Primary objectives:• Evaluate organ absorbed radiation doses from PRRT with Lutathera in adolescent patients with SSTR-positive GEP-NETs• Evaluate safety and tolerability of Lutathera in adolescents with SSTR-positive GEP-NETsSecondary objectives:•…

A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease

This study has been transitioned to CTIS with ID 2024-515202-84-00 check the CTIS register for the current data. Phase 2:Primary ObjectivesTo determine the recommended Phase3 dose of mitapivat by evaluating the effect of 2 dose levels of mitapivat…

A Phase 2 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, Tolerability, and Pharmacokinetics of NBI-921352 as Adjunctive Therapy in Subjects with hSCN8A Developmental and Epileptic Encephalopathy Syndrome (SCN8A-DEE)

Main objective: To assess the efficacy of NBI-921352 as adjunctive therapy on the frequency of countable motor seizuresSecondary Objectives • To evaluate the efficacy of NBI-921352 using the Clinical and Parent/Caregiver Global Impression of Change…

A phase 2, open-label, single-arm, cohort study to evaluate the safety, efficacy, and pharmacokinetics of sparsentan treatment in pediatric subjects with selected proteinuric glomerular diseases (EPPIK)

This study has been transitioned to CTIS with ID 2023-505497-14-00 check the CTIS register for the current data. Primary Objectives:• Evaluate the safety and tolerability of sparsentan oral suspension (Population 1 and Population 2) and tablets (…

A Phase 2, Open-Label, Multicenter Study to Evaluate the Safety and Efficacy of the Oral Pan-RAF Inhibitor DAY101 in Pediatric Patients with RAF-Altered, Recurrent or Progressive Low-Grade Glioma and Advanced Solid Tumors

This study has been transitioned to CTIS with ID 2024-510691-20-00 check the CTIS register for the current data. The primary objective of Arm 1 is to evaluate the overall response rate (ORR) as determined by an independent radiology review committee…

EPIK-P2: A Phase II double-blind study with an upfront, 16-week randomized, placebo-controlled period, to assess the efficacy, safety and pharmacokinetics of alpelisib (BYL719) in pediatric and adult patients with PIK3CA-related overgrowth spectrum (PROS)

This study has been transitioned to CTIS with ID 2023-508530-34-00 check the CTIS register for the current data. Primary objective:* To demonstrate the efficacy of alpelisib as measured by the proportion of participants randomized to alpelisib with…

A Phase 1/2 Study of the Highly Selective ROS1 Inhibitor NVL-520 in Patients with Advanced NSCLC and Other Solid Tumors (ARROS-1)

This study has been transitioned to CTIS with ID 2024-511793-71-00 check the CTIS register for the current data. Phase 1Primary Objective• To determine the RP2D and, if applicable, the maximum tolerated dose (MTD) of NVL-520 in patients with…

Open-label Uncontrolled Trial to Evaluate Pharmacokinetics, Pharmacodynamics, Safety, and Activity of Efgartigimod in Children From 2 to Less Than 18 Years of Age With Generalized Myasthenia Gravis

This study has been transitioned to CTIS with ID 2024-513854-31-00 check the CTIS register for the current data. To confirm an age-adjusted optimum dose of efgartigimod IV and provide (model-predicted) evidence for a treatment response.

A Phase 2 study to assess the efficacy and safety of 2 dosage regimens of oral fidrisertib (IPN60130) for the treatment of fibrodysplasia ossificans progressiva in male and female paediatric and adult participants

This study has been transitioned to CTIS with ID 2024-511469-13-00 check the CTIS register for the current data. Main objective:• Evaluate the efficacy of IPN60130 monotherapy compared with placebo recipients in inhibiting new HO volume in adult and…

Transvamix (10% THC / 5% CBD) to treat chronic pain in Epidermolysis Bullosa: An explorative randomized, placebo-controlled and double-blind intervention crossover study

The primary objective is to determine the effect of Transvamix on the pain quality item *unpleasantness*, relative to placebo. Secondary objectives include the assessment of general pain qualities, pain self-efficacy, general pain and pruritus…

Prospective validation and clinical evaluation of a new posaconazole dosing regimen for children and adolescents with cystic fibrosis and Aspergillus infection

- In silico definition of the most optimal posaconazole dose for children and adolescents with CF aged 8 to 17 years.- Assess the prevalence of Aspergillus infection in children and adolescents with CF aged 8 -17 years.- An intensive sampling…

A 2-Stage (Open-Label Followed by Randomized Double-Blind, Placebo-Controlled Stage), Phase 2 Trial of Setmelanotide in Patients with Specific Gene Variants in the Melanocortin-4 Receptor Pathway

Primary:• To evaluate the proportion of patients with obesity with genetic variants in a specific gene in the melanocortin-4 receptor (MC4R) pathway who achieve a clinically meaningful reduction in body weight in response to setmelanotide at the end…

TUMOR-AGNOSTIC PRECISION IMMUNOONCOLOGY AND SOMATIC TARGETING RATIONAL FOR YOU (TAPISTRY) PHASE II PLATFORM TRIAL

This study will evaluate the efficacy and safety of multiple therapies that are selected using somatic alterations and potential predictive biomarkers identified via NGS assays in patients with solid tumors:

Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability,
and Efficacy of EDIT-101 in Adult and Pediatric Participants with Leber Congenital Amaurosis
Type 10 (LCA10), with Centrosomal Protein 290 (CEP290)-Related Retinal Degeneration
Caused by a Compound Heterozygous or Homozygous Mutation Involving c.2991+1655A>G in
Intron 26 (IVS26) of the CEP290 Gene (*LCA10-IVS26*)

Primary-To evaluate the safety and tolerability of a single dose of EDIT-101 when administered to participants with LCA10-IVS26 mutationSecondary-To evaluate different doses of EDIT-101 for subsequent clinical evaluation-To evaluate the efficacy of…

A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients with Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation

This study has been transitioned to CTIS with ID 2024-513759-34-00 check the CTIS register for the current data. Primary ObjectivesPhase 2The primary objectives of Phase 2 are:• To evaluate short- and long-term safety and tolerability of SNDX 5613…