Clinical trials: Rare Cancer
Malignant Solid Tumors, Other Neoplasms Solid Tumors
Natural History and Biospecimen Acquisition for Children and Adults With Rare Solid Tumors
Background: Approximately 150 cases of cancer per one million per year are considered rare cancers. While all tumors originate from genetic changes, a small percentage of these tumors are familial.
- Conditions
- Malignant Solid Tumors, Other Neoplasms Solid Tumors, Pediatric Solid Tumor, Refractory Solid Tumors
- Trial name
- NCT03739827
- Where
- 3 sites in United States
- Volunteers
- 10,000 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Acute Undifferentiated Leukemia, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative
Collecting and Storing Malignant, Borderline Malignant Neoplasms, and Related Samples From Young Patients With Cancer
This study is collecting and storing malignant, borderline malignant neoplasms, and related biological samples from young patients with cancer.
- Conditions
- Acute Undifferentiated Leukemia, Atypical Chronic Myeloid Leukemia, BCR-ABL1 Negative, Childhood Acute Lymphoblastic Leukemia, Childhood Acute Myeloid Leukemia
- Trial name
- NCT00898079
- Testing
- Cytology Specimen Collection Procedure
- Where
- 192 sites in United States, Australia and 4 more
- Volunteers
- 2,545 people
- Sponsor
- Children's Oncology Group
Rare Diseases
Rare Tumours in Children and Adolescents (STEP)
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.
- Trial name
- STEP 2 0 (NCT05773651)
- Testing
- Data collection
- Where
- Germany
- Volunteers
- 10,000 people
- Sponsor
- University Hospital Tuebingen
Chordoma
Children and Adults With Chordoma
Background: Chordoma is a rare type of bone cancer. It occurs in the skull base or spine. Researchers want to study people with chordoma in different ways.
- Trial name
- NCT03910465
- Where
- United States
- Volunteers
- 300 people
- Sponsor
- National Cancer Institute (NCI)
Hereditary Multiple Osteochondromas
Functional and Morphological Characterization of Multiple Osteochondromas Disorder
The main purpose of the study is the characterization of functional and morphological alterations due to multiple osteochondromas in a pediatric and adult population through the identification of relevant anthropometric and…
- Trial name
- MAMBO (NCT06703736)
- Where
- Italy
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- Istituto Ortopedico Rizzoli
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
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
Malignancy, Malignant Neoplasm
DETERMINE Trial Treatment Arm 02: Atezolizumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With High Tumour Mutational Burden (TMB) or Microsatellite Instability-high (MSI-high) or Proven Constitutional Mismatch Repair Deficiency (CMMRD) Disposition
This clinical trial is looking at a drug called atezolizumab. Atezolizumab is approved as standard of care treatment for adult patients with urothelial cancer, non-small cell lung cancer, extensive-stage small cell lung cancer…
- Conditions
- Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders, Neoplasms by Histologic Type
- Trial name
- DETERMINE (NCT05770102)
- Testing
- Atezolizumab
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
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
Cancer, Oncology
Longitudinal Registry Including Patients Treated With Heavy Particles
The purpose of this registry is to collect retrospective and prospective standardized data of patients treated with particle therapy, either with protons or carbon ions, at the National Center for Oncological Hadrontherapy (CNAO)…
- Conditions
- Cancer, Oncology, Radiation Exposure, Radiotherapy Side Effect
- Trial name
- NCT05203250
- Testing
- Heavy-ion therapy (hadrontherapy)
- Where
- Italy
- Volunteers
- 3,000 people
- Sponsor
- CNAO National Center of Oncological Hadrontherapy
Rare Malignant Neoplasm
Precision Cancer Therapy in Rare Cancers
Some rare cancers are hard-to-treat and patients have a poor prognosis. It is known that some of these patients have targetable molecular alterations, and some benefit from targeted drugs.
- Trial name
- MATRIX-Rare (NCT06119789)
- Testing
- Imatinib, Trametinib, Dabrafenib
- Where
- Norway
- Volunteers
- 96 people
- Sponsor
- Oslo University Hospital
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
Cancer
Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors
This study aims to determine the efficacy of daily sirolimus and celecoxib, with low dose etoposide alternating with cyclophosphamide for pediatric participants with relapsed or refractory tumors.
- Trial name
- AflacST1502 (NCT02574728)
- Testing
- Sirolimus, Celecoxib, Etoposide
- Where
- 6 sites in United States
- Volunteers
- 46 people
- Sponsor
- Emory University
Neurofibromatosis 2
Phase II Study of the Multichannel Auditory Brain Stem Implant for Deafness Following Surgery for Neurofibromatosis 2
OBJECTIVES: I. Define the tonotopocity of multichannel brain stem stimulation and use this information to better program the auditory brain stem implant for an individual. II.
- Trial name
- NCT00004437
- Testing
- Multichannel Auditory Brain Stem Implant
- Sponsor
- FDA Office of Orphan Products Development
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
Lung Neoplasms
Efficacy of EGFR TKIs in Patients With Rare EGFR-mutated NSCLC
Lung cancer is the leading cause of cancer-related death worldwide and is well known to remain a major health problem. Non-small-cell lung cancer (NSCLC) constitutes more than 80% of all the cases of lung cancer.
- Trial name
- NCT01775943
- Where
- South Korea
- Volunteers
- 90 people
- Sponsor
- Ulsan University Hospital
Very Rare Tumors, Very Rare Cancers
NCI Childhood Cancer Data Initiative (CCDI) Led Pediatric, Adolescent, and Young Adult Rare Cancer Registry for Very Rare Solid Tumors
Background: All childhood cancers are rare, but some are called very rare. Very rare cancers are diagnosed in 2 or fewer out of 1 million people each year.
- Conditions
- Very Rare Tumors, Very Rare Cancers, Other Solid Tumors, Solid Tumor
- Trial name
- NCT07489378
- Testing
- Natural history study of individuals with very rare tumors
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- National Cancer Institute (NCI)
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
Gynecologic Cancer
Study on Gynecological Rare Cancers
This is a prospective, observational, multicenter study on gynecological rare tumors.
- Trial name
- MITO9B (NCT05733806)
- Testing
- chemotherapy, surgery
- Where
- 2 sites in Italy
- Volunteers
- 500 people
- Sponsor
- National Cancer Institute, Naples
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)