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A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Adolescent Patients 12 to 17 Years of Age with Chronic Migraine - the REBUILD-2

This study has been transitioned to CTIS with ID 2023-505835-11-00 check the CTIS register for the current data. PrimaryTo demonstrate the superiority of galcanezumab versus placebo in the prevention of migraine in an adolescent population (12 to 17…

A Phase 3, Randomized, Double-blind, Placebo-controlled, Single-dose Study to Evaluate the Efficacy and Safety of Suvratoxumab in Mechanically Ventilated Adults and Adolescents for the Prevention of Nosocomial Pneumonia

Primary:- To evaluate the effect of suvratoxumab on reducing the incidence of nosocomial all-cause pneumonia.Secondary:- To evaluate the safety of a single IV dose of suvratoxumab.- To evaluate the effect of suvratoxumab on reducing the incidence of…

Effectiveness of L-serine dietary supplementation in children with a GRIN2B loss-of-function mutation: n-of-1 series

Assess the potential efficay of L-serine dietary supplementation in patients children with a GRIN2B LoS mutation.

A Randomized, Double-Blind, Placebo-Controlled Study of Galcanezumab in Patients 6 to 17 Years of Age with Episodic Migraine - the REBUILD-1

This study has been transitioned to CTIS with ID 2023-505836-36-00 check the CTIS register for the current data. Main Objective:To demonstrate the superiority of galcanezumab versus placebo in the prevention of migraine in (at least) 1 of the…

A Phase 3 Double Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ISIS 721744 in Patients with Hereditary Angioedema (HAE)

Primary objective: The primary objective of the study is to evaluate the clinical efficacy of ISIS 721744 in patients with hereditary angioedema (HAE).Secondary objective: Evaluate the effects of ISIS 721744 on the quality and pattern of HAE attacks…

An Open-Label Uncontrolled Multicenter Study to Evaluate the Pharmacokinetics, Pharmacodynamics, Safety and Activity of Nipocalimab in Children Aged 2 to less than 18 years with Generalized Myasthenia Gravis

This study has been transitioned to CTIS with ID 2022-502539-21-00 check the CTIS register for the current data. The purpose of this study is to determine the effect of nipocalimab on total serum immunoglobulin G (IgG) in pediatric participants 2 to…

A multicenter, randomized, double-blind, placebo-controlled study of enzastaurin for the prevention of arterial events in patients with vascular Ehlers-Danlos Syndrome (vEDS) confirmed with COL3A1 mutations, followed by an open label extension (OLE)

The aim of this study is to evaluate the efficacy, safety, and pharmacokinetics (PK) of enzastaurin in preventing new arterial events (rupture, dissection, pseudoaneurysm, carotid-cavernous sinus fistula, or aneurysm, fatal or not) leading to…

A Phase 3 Double-blind, Randomized, Placebo-controlled Study Evaluating the Efficacy and Safety of ELX/TEZ/IVA in Cystic Fibrosis Subjects 6 Years of Age and Older With a Non-F508del ELX/TEZ/IVA-responsive CFTR Mutation

To evaluate the efficacy and pharmacodynamics (PD) of ELX/TEZ/IVA

Long Term Follow Up Study for Participants of Kite Sponsored Interventional Studies Treated with Gene Modified Cells

This study has been transitioned to CTIS with ID 2023-507041-28-00 check the CTIS register for the current data. The primary objectives of this study are to:• Evaluate the incidence and severity of late-onset targeted adverse events (AEs)/serious…

A PHASE III, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED, MULTICENTER BASKET STUDY TO EVALUATE THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF SATRALIZUMAB IN PATIENTS WITH ANTI*N METHYL D ASPARTIC ACID RECEPTOR (NMDAR) OR ANTI*LEUCINE RICH GLIOMA INACTIVATED 1 (LGI1) ENCEPHALITIS

This study has been transitioned to CTIS with ID 2023-504226-18-00 check the CTIS register for the current data. This study will evaluate the efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab compared with placebo in each of…

A Phase III, Multicentre, Randomised, Double-Blind Study to Assess the Safety and Efficacy of
Emactuzumab vs. Placebo in Subjects with Tenosynovial Giant Cell Tumour.

Primary:To estimate the treatment effect of emactuzumab on objective response rate (ORR) by 6 months from initiation of therapy in the blinded phase compared to placeboSecondary:the effect of emactuzumab on clinical outcome assessments (COAs) for:o…

Multicentre prospective trial for extracranial malignant germ cell tumours including a randomized comparison of Carboplatin and Cisplatin

This study has been transitioned to CTIS with ID 2023-507582-25-00 check the CTIS register for the current data. PRIMARY OBJECTIVESThe primary objective of MAKEI V is to assess in a randomized comparison whether the efficacy of Carboplatin (600 mg/…

Phase 3, Double-Blind, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Apitegromab (SRK-015) in Patients with Later-Onset Spinal Muscular Atrophy Receiving Background Nusinersen or Risdiplam Therapy

Primary-Assess the efficacy of apitegromab compared with placebo using the HFMSE in patients 2 through 12 years oldKey secondary-Assess the efficacy of apitegromab compared with placebo based on the number of patients with clinical improvement in…

A Phase 3, 52-week, open-label, single arm study to investigate the efficacy and safety of mepolizumab SC in participants aged 6 to 17 years with hypereosinophilic syndrome.

This study has been transitioned to CTIS with ID 2023-510110-36-00 check the CTIS register for the current data. Primary objective: To evaluate the efficacy of mepolizumab SC given every 4 weeks in participants aged 6 to 17 years with HESSecondary…

A phase 3 study of PTC923 in subjects with phenylketonuria

Primary:• To evaluate the efficacy of PTC923 in reducing blood phenylalanine (Phe) levels in subjects with phenylketonuria (PKU) as measured by mean change in blood Phe levels from baseline to Weeks 5 and 6 (ie, the average of each respective…

A Phase 3 Open-label Study Evaluating the Long-term Safety and Efficacy of Elexacaftor/Tezacaftor/Ivacaftor in Cystic Fibrosis Subjects With Non-F508del CFTR Genotypes

This study has been transitioned to CTIS with ID 2024-515637-14-00 check the CTIS register for the current data. To evaluate the long-term safety and tolerability of elexacaftor(ELX)/tezacaftor (TEZ)/ivacaftor (IVA)

A Prospective, Open-label Pilot Study to Evaluate Effector mechanisms of Hyperbaric Oxygen Therapy in Patients with Moderate-to-Severe Ulcerative Colitis: The PARADOX study

This study has been transitioned to CTIS with ID 2024-515278-28-00 check the CTIS register for the current data. To analyze the molecular effects and dose-response relationship of HBOT in patients with moderate-to-severe UC refractory to medical…

Effectiveness of ambroxol in children and adults with Gaucher disease 3: n-of-1 series

This study has been transitioned to CTIS with ID 2024-514012-28-00 check the CTIS register for the current data. Assess the neurological efficacy of ambroxol in adults and children with GD3.

International cooperative prospective study for children and adolescents with standard risk ALK-positive anaplastic large cell lymphoma (ALCL) estimating the efficacy of Vinblastine.

This study has been transitioned to CTIS with ID 2022-501454-11-00 check the CTIS register for the current data. PRIMARY OBJECTIVE:- To show that it is possible to cure at least 75% of patients belonging to the SR group with Vinblastine-monotherapy…

A Double-Masked, Randomized, Controlled, Multiple-Dose Study to Evaluate the Efficacy, Safety and Tolerability of QR-421a in Subjects with Retinitis Pigmentosa (RP) due to Mutations in Exon 13 of the USH2A Gene with Advanced Vision Loss

Primary • To evaluate the efficacy of QR-421aSecondary• To evaluate the safety and tolerability of QR-421a • To evaluate changes in Patient-Reported Outcome (PRO) measures in subjects treated with QR-421a• To evaluate systemic exposure of QR-421a