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A multi-center, randomized, double-blind, placebo controlled study of ligelizumab (QGE031) in the treatment of Chronic Inducible Urticaria (CINDU) in adolescents and adults inadequately controlled by H1-antihistamines

The purpose of this study is to establish efficacy and safety of ligelizumab (QGE031) versus placebo in participants with chronic inducible urticaria who remain symptomatic despite treatment with H1 antihistamine.

DAVINCY trial: optimal Duration of (fos)aprepitant prophylaxis for nausea and Vomiting INduced by ChemotherapY in children: a double-blind placebo-controlled crossover randomized phase III trial*

This study has been transitioned to CTIS with ID 2024-517846-32-00 check the CTIS register for the current data. To evaluate the effect of prolonged duration of (fos)aprepitant prophylaxis on the prevention of delayed CINV (complete remission in the…

A randomised, double-blind, placebo-controlled, Phase III trial to determine the efficacy and safety of inhaled SNG001 for the treatment of patients hospitalised due to moderate COVID-19

Primary Objective:To evaluate recovery in patients with moderate COVID-19 after administration of SNG001 compared to placebo.Secondary Objectives:a. To evaluate the efficacy of SNG001 compared to placebo in patients with moderate COVID-19, using a…

A LONG-TERM, OPEN-LABEL FOLLOW-UP STUDY OF TOFACITINIB FOR TREATMENT OF JUVENILE IDIOPATHIC ARTHRITIS (JIA)

Primary* The primary objective of this study is to determine the long term safety and tolerability of tofacitinib for treatment of the signs and symptoms of JIA.Secondary* The secondary objective of this study is to evaluate the persistence of…

A 52 week, multi-center, randomized, double-blind placebo-controlled study to assess the clinical efficacy and safety of ligelizumab (QGE031) in decreasing the sensitivity to peanuts in patients with peanut allergy

The purpose of this Phase 3 study is to evaluate the safety and clinical efficacy of ligelizumab 240 mg and 120 mg given subcutaneously (s.c.) every 4 weeks (q4w) to ensure protection against allergic reaction by decreasing the sensitivity to oral…

ABROCITINIB EXPANDED ACCESS PROTOCOL FOR THE TREATMENT OF ADOLESCENTS AND ADULTS WITH MODERATE TO SEVERE ATOPIC DERMATITIS

Primary:To provide access to abrocitinib to adolescent and adult patients with or without background topical therapy who have inadequate treatment options due to inadequate response or intolerance to available approved medicated topical and systemic…

OPEN-LABEL INDUCTION AND MAINTENANCE STUDY OF ORAL CP-690,550 (TOFACITINIB) IN CHILDREN WITH MODERATELY TO SEVERELY ACTIVE ULCERATIVE COLITIS

This study has been transitioned to CTIS with ID 2023-509694-22-00 check the CTIS register for the current data. Primary objective: • To evaluate the efficacy of tofacitinib based on remission in pediatric participants with moderately to severely…

Peppermint Oil for the treatment of Irritable Bowel Syndrome or Functional Abdominal Pain in Children:
the MINT study

The aim of the Mint study is to investigate the effectiveness of peppermint oil capsules compared to placebo capsules in reducing abdominal pain intensity in these children. In addition, we investigate the effect of peppermint oil capsules compared…

Phase 3 Randomized, Controlled Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

Primary:To assess the effect of bilateral treatment with AAV5-hRKp.RPGR on functional vision as measured by vision-guided mobility assessment.Secondary:To assess changes after treatment administration in retinal function, functional vision, visual…

A Prospective, Open-label, Multicenter, Randomized Study to Evaluate the Benefits and Risks of
Conversion of Existing Adolescent Renal Allograft Recipients Aged 12 to Less Than 18 Years of
Age to a Belatacept-based Immunosuppressive Regimen as Compared to Continuation of a
Calcineurin Inhibitor-based Regimen, and Their Adherence to Immunosuppressive Medications

This study has been transitioned to CTIS with ID 2022-501677-39-00 check the CTIS register for the current data. Evaluation of patient and functional graft survival of adolescent renal allograft recipients converted from CNI to belatacept-based…

A randomized phase 3 trial of fludarabine/cytarabine/gemtuzumab ozogamicin with or without venetoclax in children with relapsed AML

This study has been transitioned to CTIS with ID 2023-510160-12-00 check the CTIS register for the current data. To assess if venetoclax combined with FLA+GO (fludarabine, high-dose cytarabine, and gemtuzumab ozogamicin) will improve overall…

A Phase 3, Open-label, Long-term Safety Study of Oral Linaclotide Administered to Pediatric Participants with Functional Constipation (FC) or Irritable Bowel Syndrome with Constipation (IBS-C)

The objective of this study is to assess the long-term safety of linaclotide in pediatric participants with FC (total exposure with linaclotide for 24 weeks) or IBS-C (total exposure with linaclotide for 52 weeks) who have completed study…

A Multi-Center, Randomized, Double-Blind, Placebo-Controlled Phase III Study to Assess the Efficacy, Safety, and Tolerability of PXT3003 in Charcot-Marie-Tooth type 1A (CMT1A)

Primary:To evaluate the efficacy of treatment with PXT3003 (a fixed-dose combination of (RS)-baclofen, naltrexone hydrochloride, and D-sorbitol) compared to placebo in subjects with CMT1A.Secondary: To evaluate the safety and tolerability of PXT3003…

A 6-month multicenter, randomized, double-blind, placebo-controlled study to evaluate the
efficacy, safety and PK/PD of an age-and body weight-adjusted oral finerenone regimen, in
addition to an ACEI or ARB, for the treatment of children, 6 months to <18 years of age, with
chronic kidney disease and proteinuria

This study has been transitioned to CTIS with ID 2023-504884-17-00 check the CTIS register for the current data. The main goal of this study is to find out whether finerenone in combination with either an ACE inhibitor or an ARB can reduce the…

AN INTERNATIONAL PROSPECTIVE TRIAL ON HIGHRISK MEDULLOBLASTOMA IN PATIENTS OLDER THAN 3 YEARS

This study has been transitioned to CTIS with ID 2024-510578-25-00 check the CTIS register for the current data. Primary objectives:- To evaluate whether the outcome in children, young people and adults with HR-MB is improved over standard therapy i…

A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study of Adeno-Associated
Virus Serotype 8 (AAV8)-Mediated Gene Transfer of Human Ornithine Transcarbamylase
(OTC) in Patients with Late-Onset OTC Deficiency

This study has been transitioned to CTIS with ID 2024-514337-38-00 check the CTIS register for the current data. PrimaryTo evaluate the efficacy of DTX301 on the improvement of OTC function by maintaining safe plasma ammonia levelsSecondaryTo…

A Phase 3 open-label, multicenter study of the long-term safety and efficacy of intravenous recombinant coagulation factor VIII Fc-von Willebrand factor-XTEN fusion protein (rFVIIIFc-VWF-XTEN; BIVV001) in Previously Treated Patients with severe hemophilia A

This study has been transitioned to CTIS with ID 2023-508929-27-00 check the CTIS register for the current data. -To evaluate the long-term safety of BIVV001 in previously treated patients with hemophilia A.-To evaluate the efficacy of BIVV001 as a…

An Open-label Study to Evaluate the Long-term Safety and Efficacy of CSL312 (Garadacimab) in the Prophylactic Treatment of Hereditary Angioedema

This study has been transitioned to CTIS with ID 2024-510777-18-00 check the CTIS register for the current data. The primary objective of the study is to evaluate the long-term safety of SC administration of CSL312 in the prophylactic treatment of…

Phase 3, Multicenter, Double-blind, Randomized, Placebo-controlled, Parallel Group Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Apremilast in Children From 5 to Less Than 18 Years of Age With Active Juvenile Psoriatic Arthritis (PEAPOD)

This study has been transitioned to CTIS with ID 2023-503435-17-00 check the CTIS register for the current data. Estimate the efficacy of apremilast compared with placebo in the treatment of Juvenile Psoriatic Arthritis (JPsA) in pediatric subjects…

Phase 3 Follow-up Study of AAV5-hRKp.RPGR for the Treatment of X-linked Retinitis Pigmentosa Associated with Variants in the RPGR gene

This study has been transitioned to CTIS with ID 2024-511411-25-00 check the CTIS register for the current data. Main objective:To assess the long-term safety and tolerability of AAV5-hRKp.RPGR in individuals with RPGR-XLRPTo assess the long-term…