Clinical trials: Rare CNS Tumor, open to volunteers
Brain (Nervous System) Cancers, Glioblastoma
Pharmacoscopy for Patients With Refractory Primary Brain Tumors
Advanced technology of ex vivo drug profiling referred to as pharmacoscopy may allow to identify novel drugs for the treatment of glioblastoma and other refractory brain tumors at an individual patient level.
- Conditions
- Brain (Nervous System) Cancers, Glioblastoma, Glioma, Ependymoma
- Trial name
- EViDENCE-BT (NCT06804655)
- Testing
- Pharmacoscopy 1.0
- Where
- 3 sites in Switzerland
- Volunteers
- 40 people
- Sponsor
- University of Zurich
Glioma, Glioneuronal Tumor
Observational Study for Assessing Treatment and Outcome of Patients With Primary Brain Tumours Using cIMPACT-NOW and 2021 WHO Classification
Every new classification depends on its prognostic power and on the type of treatment given. With the rapid evolution of diagnostic methods and the advance in new treatments, there is much less reliable information available on…
- Conditions
- Glioma, Glioneuronal Tumor, Choroid Plexus Tumor, Pineal Tumors
- Trial name
- NCT05259605
- Testing
- Observational
- Where
- 42 sites in Austria, Belgium and 10 more
- Volunteers
- 1,650 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Embryonal Tumor
Rare Embryonal Tumors of the Central Nervous System: International Registry
Central nervous system (CNS) tumors are the most common solid malignancies among children. Although some types of CNS tumors like medulloblastomas and low-grade gliomas are widespread and well-studied, there is a huge number of…
- Trial name
- NCT05711992
- Where
- 11 sites in Armenia, Canada and 7 more
- Volunteers
- 300 people
- Sponsor
- Immune Oncology Research Institute
Periodontal Disease, Periodontitis
Periodontal Disease in Rare Renal Disorders (PERIO-RA-RE)
This study aims to evaluate the burden and phenotypic spectrum of periodontal disease in patients with rare kidney disorders (such as Alport syndrome, Fabry disease, and tuberous sclerosis complex) and systemic lupus…
- Conditions
- Periodontal Disease, Periodontitis, CKD, Chronic Kidney Disease
- Trial name
- PERIO-RA-RE (NCT07575347)
- Testing
- Observational assessment
- Where
- Romania
- Volunteers
- 100 people
- Sponsor
- Stefan Lujinschi
Medulloblastoma Recurrent, Ependymoma Recurrent
Antiangiogenic Therapy for Children With Recurrent Medulloblastoma, Ependymoma, ATRT and Rare CNS Tumors
Patients with with recurrent or progressive medulloblastoma, ependymoma, atypical teratoid rhabdoid tumor (ATRT), and CNS tumors of various histologies have a very poor prognosis whether treated with conventional chemotherapy…
- Conditions
- Medulloblastoma Recurrent, Ependymoma Recurrent, ATRT Recurrent, Rare CNS Tumor Recurrent
- Trial name
- MEMMAT (NCT01356290)
- Testing
- Bevacizumab, Thalidomide, Celecoxib
- Where
- 22 sites in United States, Austria and 6 more
- Volunteers
- 232 people
- Sponsor
- Medical University of Vienna
Prader-Willi Syndrome, PWS-like Syndrome
GROWing Up With Rare GENEtic Syndromes
Introduction Rare complex syndromes Patients with complex genetic syndromes, by definition, have combined medical problems affecting multiple organ systems, and intellectual disability is often part of the syndrome.
- Conditions
- Prader-Willi Syndrome, PWS-like Syndrome, Silver Russel Syndrome, Congenital Hypopituitarism
- Trial name
- GROW UR GENES (NCT04463316)
- Testing
- Retrospective file studies
- Where
- Netherlands
- Volunteers
- 600 people
- Sponsor
- dr. Laura C. G. de Graaff-Herder
Rare CNS Tumor, FOXR2-activated
Ultra-Rapid Profiling and Drug Screening for Treatment Selection in Patients With Relapsed or Recurrent Rare Brain Tumors
The current study will use a new treatment approach based on the molecular characteristics of each participant's tumor. The study will test the feasibility in of performing real-time drug screening on tissue taken during surgery…
- Conditions
- Rare CNS Tumor, FOXR2-activated, BCOR ITD Sarcoma, CNS Sarcoma
- Trial name
- SUPERNOVA-RBT (NCT07851636)
- Testing
- Personalized Treatment Recommendation
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Adriana Fonseca
Brain Tumors
SIMPLIFication of Care Pathways for Patients With Rare Brain Tumors Through Artificial Intelligence
This study focuses on rare brain tumors, which are heterogeneous entities with different morphological, biological, and clinical characteristics.
- Trial name
- SIMPLIF-AI (NCT06878469)
- Testing
- RehaCom
- Where
- 2 sites in Italy
- Volunteers
- 200 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis
National Registry of Rare Kidney Diseases
The goal of this National Registry is to is to collect information from patients with rare kidney diseases, so that it that can be used for research.
- Conditions
- Adenine Phosphoribosyltransferase Deficiency, AH Amyloidosis, AHL Amyloidosis, AL Amyloidosis
- Trial name
- RaDaR (NCT06065852)
- Where
- United Kingdom
- Volunteers
- 35,000 people
- Sponsor
- UK Kidney Association
Haematological Malignancy, Malignancy
DETERMINE Trial Treatment Arm 03: Entrectinib in Adult, Paediatric and Teenage/Young Adult Patients With ROS1 Gene Fusion-Positive Cancers.
This clinical trial is looking at a drug called entrectinib. Entrectinib is approved as standard of care treatment for adult patients with non-small cell lung cancer (NSCLC) which have a particular molecular alteration called…
- Conditions
- Haematological Malignancy, Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders
- Trial name
- DETERMINE (NCT05770544)
- Testing
- Entrectinib
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Astroblastoma, BCOR ITD Sarcoma
International Rare Brain Tumor Registry
The objective of the International Rare Brain Tumor Registry (IRBTR) is to better understand rare brain tumors through the collection of biospecimens and matched clinical data of children, adolescents, and young adult patients…
- Conditions
- Astroblastoma, BCOR ITD Sarcoma, CNS Sarcoma, Unclassified Tumor, Malignant
- Trial name
- IRBTR (NCT05697874)
- Where
- 13 sites in United States, Brazil and 2 more
- Volunteers
- 5,800 people
- Sponsor
- Children's National Research Institute
Rare Diseases, Amyloidosis
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD).
- Conditions
- Rare Diseases, Amyloidosis, Sarcoidosis, Phacomatosis
- Trial name
- NCT06573723
- Where
- Argentina
- Volunteers
- 380 people
- Sponsor
- Hospital Italiano de Buenos Aires
Congenital Portosystemic Shunt, CPSS (Congenital Portosystemic Shunt)
International Registry of Congenital Portosystemic Shunt (IRCPSS)
Congenital Portosystemic Shunt (CPSS) is a rare condition important by the multiplicity and severity of associated complications.
- Trial name
- IRCPSS (NCT06041906)
- Testing
- Shunt Closure
- Where
- 26 sites in United States, Australia and 14 more
- Volunteers
- 500 people
- Sponsor
- Prof. Valérie Mc Lin
Nervous System Diseases
Collection of Biological Samples From Patients With Rare Neurological Diseases
The aim of this project is to improve biological collections of patients presenting rare neurological disorders with known or suspected autoimmune origin.
- Trial name
- EXPLAINEUR (NCT04698421)
- Testing
- Blood collection on admission and longitudinally
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- University Hospital, Toulouse
Central Nervous System Neoplasms, Glioblastoma
Phase I Study of Oral ONC206 in Recurrent and Rare Primary Central Nervous System Neoplasms
The primary objective of this Phase 1, open-label, dose-escalation, and exploratory study is to evaluate the safety and tolerability profile (establish the maximum-tolerated dose) and evaluate the occurrence of dose-limiting…
- Conditions
- Central Nervous System Neoplasms, Glioblastoma, Gliosarcoma, Adult, Anaplastic Oligodendroglioma
- Trial name
- NCT04541082
- Testing
- ONC206
- Where
- United States
- Volunteers
- 102 people
- Sponsor
- Jazz Pharmaceuticals
Fragile X Syndrome, Angelman Syndrome
Parent and Infant Inter(X)Action Intervention (PIXI)
The objective is to develop and test, through an iterative process, an intervention to address and support the development of infants with a confirmed diagnosis of a neurogenetic disorder with associated developmental delays or…
- Conditions
- Fragile X Syndrome, Angelman Syndrome, Prader-Willi Syndrome, Dup15Q Syndrome
- Trial name
- NCT03836300
- Testing
- Parent-Infant Inter(X)action Intervention (PIXI)
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- RTI International
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
CADASIL, Cavernous Angioma
Biological Collection of the Rare Diseases of the Brain and Eye Vessels Cohort - 2
CERVCO is the French National Reference Centre for Rare Cerebrovascular and Retinal Diseases, accredited by the Ministry of Health since 2005.
- Conditions
- CADASIL, Cavernous Angioma, Cerebral Autosomal Dominant Arteriopathy With Subcortical Infarcts and Leukoencephalopathy, Small Vessel Disease
- Trial name
- B-MRVC2 (NCT07650110)
- Testing
- Biocollection, Neurological evaluation and Biocollection
- Volunteers
- 600 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Inherited Disease, Rare Diseases
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less than 5:100,000) in adulthood through the systematic longitudinal…
- Conditions
- Inherited Disease, Rare Diseases, Metabolic Disease, Undiagnosed Disease
- Trial name
- NCT04880356
- Testing
- collection of data
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta