Clinical trials: Rare Cancer
Appendix Cancer, Pseudomyxoma Peritonei
The Appendix Cancer PMP Research Foundation Natural History Study/Patient Registry
ACPMP's first-ever patient-powered global patient registry/natural history study collects patient and caregiver information and data to fuel clinical trials and drive breakthroughs for appendix cancer and PMP.
- Trial name
- NCT07769346
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Appendix Cancer Pseudomyxoma Peritonei Research Foundation
Tuberous Sclerosis, Epilepsy
Clinical Trial Data Set Re-use With Statistical Methodologies Tailored for Clinical Trials in Rare Diseases
Tuberous sclerosis complex (TSC), affecting 1 in 6.000 live births, is characterized by the development of multisystem tumors. Seizures are frequent up to 80% of individuals.
- Trial name
- EPISTOPIDEAL (NCT05199402)
- Where
- France
- Volunteers
- 10 people
- Sponsor
- Imagine Institute
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
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
Genes, Sporadic Chordoma
Genetic Clues to Chordoma Etiology: A Protocol to Identify Sporadic Chordoma Patients for Studies of Cancer-Susceptibility Genes
Background: Chordoma is a rare, slow growing, often fatal bone cancer derived from remnants of the embryonic notochord. It occurs mostly in the axial skeleton (skull base, vertebrae, sacrum and coccyx), is more frequent in males…
- Trial name
- NCT01200680
- Where
- United States
- Volunteers
- 188 people
- Sponsor
- National Cancer Institute (NCI)
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
Haematological Malignancy, Colorectal Neoplasms
DETERMINE Trial Treatment Arm 04: Trastuzumab in Combination With Pertuzumab in Adult, Paediatric and Teenage/Young Adult Patients With Cancers With HER2 Amplification or Activating Mutations
This clinical trial is looking at a combination of drugs called trastuzumab and pertuzumab. This combination of drugs is approved together as standard of care treatment for adult patients with breast cancer (often with other…
- Conditions
- Haematological Malignancy, Colorectal Neoplasms, Urinary Bladder Neoplasm, Gallbladder Neoplasms
- Trial name
- DETERMINE (NCT05786716)
- Testing
- Trastuzumab, Pertuzumab
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Sarcoma, Malignant Mesenchymoma
The Sarcoma Biology and Outcome Project
SarcBOP - An interdisciplinary and translational registry SarcBOP aims to establish a database that integrates every aspect possibly relevant to sarcoma treatment and research.
- Conditions
- Sarcoma, Malignant Mesenchymoma, Sarcoma of Bone and Connective Tissue
- Trial name
- SarcBOP (NCT04758325)
- Where
- Germany
- Volunteers
- 3,000 people
- Sponsor
- Prof. Dr. Richard F Schlenk
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
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
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
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
Intrapericardial Teratoma Tumor, Tumors
Rare Tumor Case Review
The purpose of this study is to do a literature review and combine all of the cases of the intrapericardial teratoma tumor and see if some conclusions can be made about this rare tumor in children.
- Trial name
- NCT00229879
- Where
- United States
- Sponsor
- Children's Healthcare of Atlanta
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
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
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
Cardiovascular Diseases, Endocrinology
BEhavioral and Adherence Model for Improving Quality, Health Outcomes and Cost-Effectiveness of healthcaRe
Lack of adherence to treatment is a widespread issue worldwide, which leads to higher healthcare utilisation rates and even premature death.
- Conditions
- Cardiovascular Diseases, Endocrinology, Inmunology, Neurology
- Trial name
- BEAMER (NCT06856902)
- Testing
- B-COMPASS implementation
- Where
- 10 sites in Germany, Italy and 6 more
- Volunteers
- 6,190 people
- Sponsor
- Technical University of Madrid
Adnexal Tumor of Skin, Merkel Cell Carcinoma
French National Database of Rare Dermatological Cancers
Merkel cell carcinoma, advanced basal cell carcinoma requiring systemic treatment, and cutaneous adnexal carcinomas are 3 types of rare skin cancers for which much remains unknown in terms of natural behavior, prognosis…
- Conditions
- Adnexal Tumor of Skin, Merkel Cell Carcinoma, Advanced Basal Cell Carcinoma Requiring Systemic Treatment
- Trial name
- CARADERM (NCT03210935)
- Where
- 34 sites in France
- Volunteers
- 9,000 people
- Sponsor
- University Hospital, Lille
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
All Tumor Types
SPECTA: Screening Cancer Patients for Efficient Clinical Trial Access
SPECTA is a quality assured platform for collecting clinicopathologically annotated biological material, imaging data, operative images, environmental assessment, questionnaires as well as patient-reported outcomes from cancer…
- Trial name
- SPECTA (NCT02834884)
- Testing
- RP-1828 IMMUcan, RP-1759 AYA/TYA, RP-1843 Arcagen
- Where
- 136 sites in Austria, Belgium and 18 more
- Volunteers
- 4,975 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC