Clinical trials: Rare CNS Tumor
Anaplastic Astrocytoma, Anaplastic Oligodendroglioma
Phase II Study of Carmustine, Streptozocin, and Mercaptopurine for Refractory or Recurrent Brain Neoplasms
OBJECTIVES: I. Assess the clinical response of patients with refractory or recurrent brain neoplasms treated with carmustine, streptozocin, and mercaptopurine.
- Conditions
- Anaplastic Astrocytoma, Anaplastic Oligodendroglioma, Mixed Gliomas, Glioblastoma Multiforme
- Trial name
- NCT00004688
- Testing
- carmustine, mercaptopurine, streptozocin
- Volunteers
- 35 people
- Sponsor
- Emory University
Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder
The Longitudinal CONQUER Study of Rare Neuroimmunologic Disorders
This study seeks to determine the biologic causes of inflammation in patients with Transverse Myelitis (TM) Neuromyelitis Optica Spectrum Disorder (NMOSD) and related conditions.
- Conditions
- Neuromyelitis Optica, Neuromyelitis Optica Spectrum Disorder, Transverse Myelitis, Optic Neuritis
- Trial name
- NCT01623076
- Where
- United States
- Volunteers
- 150 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
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
Medulloblastoma
A Clinical and Molecular Risk-Directed Therapy for Newly Diagnosed Medulloblastoma
Historically, medulloblastoma treatment has been determined by the amount of leftover disease present after surgery, also known as clinical risk (standard vs. high risk).
- Trial name
- NCT01878617
- Testing
- Craniospinal Irradiation with boost to the primary tumor site, Cyclophosphamide, Cisplatin
- Where
- 23 sites in United States, Australia and 2 more
- Volunteers
- 660 people
- Sponsor
- St. Jude Children's Research Hospital
Adult Immunoblastic Lymphoma, Central Nervous System Non-Hodgkin Lymphoma
Collecting and Storing Tissue Samples From Patients With Rare or Cutaneous Non-Hodgkin Lymphoma
This research study is collecting and storing tissue samples from patients with rare or cutaneous non-Hodgkin lymphoma. Collecting and storing samples of tissue from patients with cancer to test in the laboratory may help the…
- Conditions
- Adult Immunoblastic Lymphoma, Central Nervous System Non-Hodgkin Lymphoma, Childhood Immunoblastic Lymphoma, Grade 1 Follicular Lymphoma
- Trial name
- NCT01000753
- Testing
- Cytology Specimen Collection Procedure
- Where
- 130 sites in United States, Australia and 2 more
- Volunteers
- 174 people
- Sponsor
- Children's Oncology Group
Anaplastic Astrocytoma, Anaplastic Ependymoma
SJDAWN: St. Jude Children's Research Hospital Phase 1 Study Evaluating Molecularly-Driven Doublet Therapies for Children and Young Adults With Recurrent Brain Tumors
Approximately 90% of children with malignant brain tumors that have recurred or relapsed after receiving conventional therapy will die of disease.
- Conditions
- Anaplastic Astrocytoma, Anaplastic Ependymoma, Anaplastic Ganglioglioma, Anaplastic Meningioma
- Trial name
- NCT03434262
- Testing
- Gemcitabine, ribociclib, sonidegib
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- St. Jude Children's Research Hospital
Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma
Cabozantinib-S-Malate in Treating Younger Patients With Recurrent, Refractory, or Newly Diagnosed Sarcomas, Wilms Tumor, or Other Rare Tumors
This phase II trial studies how well cabozantinib-s-malate works in treating younger patients with sarcomas, Wilms tumor, or other rare tumors that have come back, do not respond to therapy, or are newly diagnosed.
- Conditions
- Adrenal Cortical Carcinoma, Alveolar Soft Part Sarcoma, Central Nervous System Neoplasm, Childhood Clear Cell Sarcoma of Soft Tissue
- Trial name
- NCT02867592
- Testing
- Cabozantinib, Cabozantinib S-malate, Pharmacological Study
- Where
- 142 sites in United States
- Volunteers
- 109 people
- Sponsor
- National Cancer Institute (NCI)
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, international patient registry for all rare diseases.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- 2 sites in United States, Australia
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
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
Wilms Tumor, Fibrosarcoma
Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.
- Conditions
- Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer
- Trial name
- NCT00141765
- Testing
- Myeloablative Chemotherapy, Stem Cell Rescue
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Michigan Rogel Cancer Center
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
Brain Neoplasms
Phase II Randomized Study of Stereotactic Radiosurgery Plus Fractionated Whole-Brain Radiotherapy Vs Fractionated Whole-Brain Radiotherapy Alone for Multiple Primary or Metastatic Brain Tumors
OBJECTIVES: I. Evaluate whether stereotactic radiosurgery provides local control at multiple sites in patients with primary or metastatic brain tumors, controlled systemic disease, and preserved neurologic function. II.
- Trial name
- NCT00004659
- Testing
- irradiation, radiosurgery
- Volunteers
- 40 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Ewing's Sarcoma, Leukemia
Evaluation, Treatment, and Natural History of Children and Young Adults With Cancer or Rare Diseases
This protocol is designed to evaluate children with cancer who appear to be probable candidates for future protocol entry or have disease manifestations that are of unique scientific importance or educational value.
- Conditions
- Ewing's Sarcoma, Leukemia, Lymphoma, Brain Tumors
- Trial name
- NCT00001686
- Where
- United States
- Volunteers
- 482 people
- Sponsor
- National Cancer Institute (NCI)
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
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
Neuroblastoma, Medulloblastoma
Molecular-Guided Therapy for Relapsed and Refractory Childhood Cancer
The purpose of this study is to test the feasibility (ability to be done) of experimental technologies to determine a tumor's molecular makeup (gene expression profile) and mutations.
- Conditions
- Neuroblastoma, Medulloblastoma, Brain Tumors, Rare Tumors
- Trial name
- NCT01802567
- Testing
- Guided Therapy- Pediatric Gene Analysis Platform
- Where
- 13 sites in United States
- Volunteers
- 52 people
- Sponsor
- Giselle Sholler
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
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
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
Sarcoma, Ovarian Neoplasm
Secured Access to Pembrolizumab for Patients With Selected Rare Cancer Types
This is a Phase 2, non-randomised, open-label, multicentric study to investigate the efficacy and safety of pembrolizumab monotherapy in 7 cohorts of patients with specific rare cancers who have unresectable locally advanced or…
- Conditions
- Sarcoma, Ovarian Neoplasm, Central Nervous System Neoplasm, Thyroid Neoplasm
- Trial name
- AcSé (NCT03012620)
- Testing
- Pembrolizumab
- Where
- France
- Volunteers
- 334 people
- Sponsor
- UNICANCER