Clinical trials: Rare Cancer
Stenosis, Histiocytoma
Genetic Study of Patients and Families With Diaphyseal Medullary Stenosis With Malignant Fibrous Histiocytoma of the Bone
OBJECTIVES: I. Identify and characterize the gene causing diaphyseal medullary stenosis with malignant fibrous histiocytoma of the bone. II. Determine the clinical manifestations of this disease in these patients.
- Trial name
- NCT00007046
- Where
- United States
- Volunteers
- healthy volunteers welcome
- Sponsor
- National Center for Research Resources (NCRR)
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
Ewing Sarcoma, Ewing Family of Tumors
A Trial For Participants With Ewing's Sarcoma Treated With Vigil in Combination With Irinotecan and Temozolomide
The goal of this clinical trial was to compare participants with first relapse or refractory Ewing's sarcoma when treated with investigational product (Vigil) in addition to the standard treatment of irinotecan and temozolomide…
- Conditions
- Ewing Sarcoma, Ewing Family of Tumors, Ewing's Tumor Metastatic, Ewing's Sarcoma Metastatic
- Trial name
- VITA (NCT03495921)
- Testing
- Vigil, Irinotecan, Temozolomide
- Where
- 15 sites in United States
- Volunteers
- 32 people
- Sponsor
- Gradalis, Inc.
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
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
Rheumatoid Arthritis, Spondyloarthritis
Pregnancy and Medically Assisted Conception in Rare Diseases
Rare diseases frequently affect women of childbearing age. Pregnancy in these women has become less rare, but remains associated with high levels of complications.
- Conditions
- Rheumatoid Arthritis, Spondyloarthritis, Psoriatic Arthritis, Systemic Lupus Erythematosus
- Trial name
- EGR2 (NCT02450396)
- Where
- France
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Pediatric Solid Tumor, Pediatric Lymphoma
Dose Escalation Study of CLR 131 in Pediatric Relapsed/Refractory Malignant Tumors Including Neuroblastoma and Sarcomas
The study evaluates CLR 131 in children, adolescents, and young adults with relapsed or refractory malignant solid tumors and lymphoma and recurrent or refractory malignant brain tumors for which there are no standard treatment…
- Conditions
- Pediatric Solid Tumor, Pediatric Lymphoma, Pediatric Brain Tumor, DIPG
- Trial name
- CLOVER-2 (NCT03478462)
- Testing
- CLR 131
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 30 people
- Sponsor
- Cellectar Biosciences, Inc.
Metabolic Disease, Obesity
Characterization of Patients With Uncommon Presentations and/or Uncommon Diseases Associated With the Cardiovascular System
Background: Researchers are interested in studying individuals who have known or suspected metabolic, inflammatory or genetic diseases that may put them at a high risk for heart diseases or diseases of their blood vessels.
- Conditions
- Metabolic Disease, Obesity, Li-Fraumeni Syndrome, Cardiomyopathy
- Trial name
- NCT01143454
- Testing
- NSR IDE-MRI: Research pulse sequences
- Where
- 3 sites in United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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)
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
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
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
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
Genetic Predisposition
Initiative for Clinical Long-read Sequencing
The study aims to comprehensively introduce Long-read Genome sequencing (LR-GS) based genetic testing into clinical routine. In order to demonstrate the superiority of untargeted LR-GS over Short-read Genome sequencing (SR-GS) to…
- Trial name
- IonGER (NCT06060184)
- Testing
- Next-Generation Sequencing (NGS)
- Where
- Germany
- Volunteers
- 500 people
- Sponsor
- University Hospital Tuebingen
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
Acanthosis Nigricans, Polycystic Ovary Syndrome
Phase II Study of the Effect of Leuprolide Acetate and Spironolactone on Insulin Resistance in Hyperandrogenic Women With Polycystic Ovarian Disease or Hyperandrogenism Insulin Resistance Acanthosis Nigricans Syndrome
OBJECTIVES: I. Evaluate insulin resistance in thin and obese hyperandrogenic women with polycystic ovarian disease or hyperandrogenism insulin resistance acanthosis nigricans syndrome and in thin and obese controls, using an…
- Trial name
- NCT00004311
- Testing
- leuprolide acetate, spironolactone
- Sponsor
- National Center for Research Resources (NCRR)
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
Advanced Rare Tumours
Durvalumab and Tremelimumab in Patients With Advanced Rare Tumours
The standard or usual treatment for this disease may be chemotherapy or other types of treatment to slow the spread of the disease and relieve some symptoms of this cancer.
- Trial name
- NCT02879162
- Testing
- Durvalumab, Tremelimumab
- Where
- 13 sites in Canada
- Volunteers
- 140 people
- Sponsor
- Canadian Cancer Trials Group
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)