Rare Diseases Clinical Trial
Official title:
Rare Tumours in Children and Adolescents (STEP 2.0) - Register for the Documentation of Rare Tumours in Children and Adolescents
The aim of the STEP registry is to collect and evaluate experience and data on the diagnosis and treatment of rare childhood tumors in order to use the knowledge gained to improve the treatment prospects for our patients. The rarity of a disease should not be a disadvantage for the young patients.
The objective of the STEP registry is to optimise the diagnosis and treatment of patients with rare tumour diseases in childhood and adolescence. Therefore, a continuous prospective collection of clinical data on rare paediatric tumours is conducted to improve the understanding of these tumours. Beyond analysis of clinical data, further scientific research on the biological and molecular genetic characteristics of these tumours is performed. These data and a close collaboration with international partners, especially the European EXPeRT group, enable the improvement of treatment recommendations for these tumours along with establishment a global interdisciplinary network of rare tumour specialists. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT05794217 -
A Multi-Site Leukopak Repository Providing Annotated Biospecimens for Approved Investigator-Directed Biomedical Research Initiatives
|
||
Completed |
NCT03680365 -
Your Voice; Impact of Duchenne Muscular Dystrophy (DMD) on the Lives of Families
|
||
Completed |
NCT03290469 -
NICUSeq: A Trial to Evaluate the Clinical Utility of Human Whole Genome Sequencing (WGS) Compared to Standard of Care in Acute Care Neonates and Infants
|
N/A | |
Not yet recruiting |
NCT05955794 -
Vocal Pattern Assessment as a New Key to Identifying Rare Syndromes
|
N/A | |
Recruiting |
NCT04429750 -
Intact Cord Resuscitation in CDH
|
N/A | |
Not yet recruiting |
NCT04152876 -
Functional Genomics of Rare Genetic Diseases: Realization of Innovative Tools With High Diagnostic Power
|
||
Recruiting |
NCT03683966 -
MigALastat Therapy Adherence Among FABRY Patients: A Prospective Multicentral Observational Study
|
||
Not yet recruiting |
NCT04319796 -
European Registry on Rare Neurological Diseases
|
||
Completed |
NCT02736565 -
Pbi-shRNAâ„¢ EWS/FLI1 Type 1 LPX in Subjects With Advanced Ewing's Sarcoma
|
Phase 1 | |
Completed |
NCT05070988 -
Oral Health Related Quality of Life of Patients With Rare Diseases: a Qualitative Approach
|
||
Completed |
NCT03563677 -
Dual Guidance Structure for Evaluation of Patients With Unclear Diagnosis in Centers for Rare Diseases
|
N/A | |
Recruiting |
NCT05499091 -
Functional Study to Indentify Genetic Etiology of Rare Diseases - ORIGIN
|
N/A | |
Completed |
NCT03954652 -
Whole Genome Trio Sequencing as a Standard Routine Test in Patients With Rare Diseases - "GENOME FIRST APPROACH"
|
N/A | |
Recruiting |
NCT05703893 -
Investigation of the Neurovegetative Pattern in Patients With Thoracic Aortic Aneurysms (TAA)
|
||
Enrolling by invitation |
NCT04703179 -
Rare and Undiagnosed Disease Research Biorepository
|
||
Recruiting |
NCT04024774 -
Diagnostic Research in Patients With Rare Diseases -Solving the Unsolved Rare Diseases
|
||
Recruiting |
NCT06343558 -
Gait and Balance Impairment in Rare and Very Rare Neurological Diseases
|
||
Not yet recruiting |
NCT06412718 -
Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies
|
||
Enrolling by invitation |
NCT04654000 -
Rheopheresis as Adjuvant Treatment of Calciphylaxis
|
N/A | |
Recruiting |
NCT04651439 -
Severe Bullous Drug Eruption and Filgrastim
|
Phase 2/Phase 3 |