Search for a trial

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…

Additional effect of hydrolysed collagen/vitamin C in exercise treatment for Patellar Tendinopathy (Jumper*s knee); a randomized controlled trial

The primary aim of this RCT is to evaluate whether the use of oral supplementation of hydrolysed collagen/vitamin C in addition to usual care (education, load management and PTLE) is superior to usual care and placebo on VISA-P score after 24 weeks…

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…

Effect of a probiotic strain on immune response to influenza vaccination

To investigate the effect of Bifidobacterium lactis CECT 8145 BPL1® on the seroconversion rate after influenza vaccination in healthy adults, compared to placebo treatment.

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…

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

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…

Reduction of blood loss in pediatric osteotomies around the hip
- A randomized placebo-controlled trial with tranexamic acid -

This study has been transitioned to CTIS with ID 2024-516324-34-01 check the CTIS register for the current data. To evaluate (reduction of) intraoperative blood-loss with and without pre-operative TXA administration, in children undergoing a…

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 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…

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.

A Randomized, Double-Blind, Placebo-Controlled, Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Ambulatory and Non-Ambulatory Participants with Spinal Muscular Atrophy with Open-Label Extension (RESILIENT)

This study has been transitioned to CTIS with ID 2024-511852-42-00 check the CTIS register for the current data. Primary Objective:To evaluate the efficacy of taldefgrobep alfa in participants who are already taking a stable dose of nusinersen or…

A Phase 3, Randomized, Double-Blind, Placebo-Controlled Trial: Multiple Independent Sub-studies of Setmelanotide in Patients with POMC/PCSK1, LEPR, NCOA1 (SRC1) or SH2B1 Gene Variants in the Melanocortin-4 Receptor Pathway

This study has been transitioned to CTIS with ID 2023-507634-24-00 check the CTIS register for the current data. To evaluate the efficacy of setmelanotide on changes in body weight.

A Phase 3, Multicenter, Randomized, Double-blind, Placebo-Controlled Study to
Evaluate the Efficacy and Safety of Mitapivat in Pediatric Subjects With Pyruvate Kinase
Deficiency Who Are Regularly Transfused, Followed by a 5-Year Open-label Extension Period

This study has been transitioned to CTIS with ID 2024-515024-37-00 check the CTIS register for the current data. To determine the efficacy of treatment with mitapivat compared with placebo, as assessed by the reduction in transfusion burden in…

Withdrawal of Tiratricol Treatment in Males with Monocarboxylate Transporter 8 Deficiency (MCT8 Deficiency): A Double-blind, Randomized, Placebo-controlled Study

This study has been transitioned to CTIS with ID 2024-516124-34-00 check the CTIS register for the current data. Primary Objective:• To evaluate the effects of withdrawal of tiratricol treatment (placebo group) on serum total triiodothyronine (T3)…

A Randomized, Blinded, Placebo-Controlled, Phase 1 Single Ascending Dose Study in Healthy Adult Male Volunteers and an Open-Label Multiple Ascending Dose Study in Pediatric SMA Participants Previously Treated with Onasemnogene Abeparvovec (Zolgensma*) to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB115

This study has been transitioned to CTIS with ID 2023-505643-39-00 check the CTIS register for the current data. Part A: PrimaryTo evaluate the safety and tolerability of single ascending dose of BIIB115 administered via IT bolus injection to…