Clinical trials: Rare Cancer
Acute Disseminated Encephalomyelitis, Devic's Syndrome
Phase III Randomized, Double-Blind, Sham-Controlled Study of Plasma Exchange for Acute Severe Attacks of Inflammatory Demyelinating Disease Refractory to Intravenous Methylprednisolone
OBJECTIVES: I. Evaluate the effectiveness of plasma exchange in the treatment of acute severe attacks of inflammatory demyelinating disease in patients who have failed intravenous steroid therapy.
- Conditions
- Acute Disseminated Encephalomyelitis, Devic's Syndrome, Marburg's Variant of Multiple Sclerosis, Balo's Concentric Sclerosis
- Trial name
- NCT00004645
- Testing
- Plasma exchange
- Volunteers
- 22 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Cancer
Imatinib Mesylate And Cyclophosphamide In Metronomic Administration: Dose Escalation Study Of Imatinib Mesylate
The purpose of this study is to determine the maximum tolerated dose of imatinib mesylate, given in association with a fixed dose of cyclophosphamide (50 mg bid).
- Trial name
- PALANGI3 (NCT01046487)
- Testing
- Imatinib mesylate, Cyclophosphamide (Dosing level 1 ), Imatinib mesylate, Cyclophosphamide (Dosing level 2), Imatinib mesylate, Cyclophosphamide (Dosing level 3)
- Where
- 3 sites in France
- Volunteers
- 26 people
- Sponsor
- Centre Oscar Lambret
Rare Gynecological Tumors, Multi-omics Analysis
Prospective Multi-omics Observational Study of Gynecological Rare Pathological Tumors
The goal of this observational study is to draw the characteristic maps of genomics, transcriptome, proteomics, metabolomics, etc. in 600 patients with gynecological rare pathological types of malignant tumors.
- Trial name
- NCT05985941
- Volunteers
- 600 people
- Sponsor
- RenJi Hospital
Lymphangioleiomyomatosis
Safety and Durability of Sirolimus for Treatment of LAM
The MIDAS study aims to follow male and female LAM patients who are currently taking, have previously failed or been intolerant of, or may (at some time in the future) take mTOR inhibitors (sirolimus or everolimus) as part of…
- Trial name
- MIDAS (NCT02432560)
- Testing
- Sirolimus, Everolimus
- Where
- 20 sites in United States
- Volunteers
- 600 people
- Sponsor
- University of Cincinnati
Rare Diseases, Generalized Myasthenia Gravis (gMG)
Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia
This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes among adults with acetylcholine receptor (AChR)…
- Trial name
- NCT07247279
- Where
- 7 sites in Russia
- Volunteers
- 450 people
- Sponsor
- AstraZeneca
Cancer
Efficacy and Safety of the Combination Therapy of Dabrafenib and Trametinib in Subjects With BRAF V600E- Mutated Rare Cancers
This was a Phase II, open-label, non-randomized, multi-center study of oral dabrafenib in combination with oral trametinib in subjects with rare cancers harboring the BRAF V600E mutation including anaplastic thyroid cancer (ATC)…
- Trial name
- NCT02034110
- Testing
- Dabrafenib, Trametinib
- Where
- 41 sites in United States, Austria and 12 more
- Volunteers
- 206 people
- Sponsor
- Novartis Pharmaceuticals
Acinar Cell Carcinoma, Adenoid Cystic Carcinoma
Nivolumab and Ipilimumab in Treating Patients With Rare Tumors
This phase II trial studies nivolumab and ipilimumab in treating patients with rare tumors. Immunotherapy with monoclonal antibodies, such as nivolumab and ipilimumab, may help the body's immune system attack the cancer, and may…
- Conditions
- Acinar Cell Carcinoma, Adenoid Cystic Carcinoma, Adrenal Cortical Carcinoma, Adrenal Gland Pheochromocytoma
- Trial name
- NCT02834013
- Testing
- Biospecimen Collection, Computed Tomography, Echocardiography Test
- Where
- 1,007 sites in United States, Guam
- Volunteers
- 798 people
- Sponsor
- National Cancer Institute (NCI)
Inherited Hematological Diseases, Rare Diseases
Longitudinal Studies of Patient With FPDMM
Background: Genes tell the body and its cells how to work. Familial platelet disease (FPD) or FPD with associated malignancies (FPDMM) is caused by a variant in the gene RUNX1.
- Conditions
- Inherited Hematological Diseases, Rare Diseases, FPDMM
- Trial name
- NCT03854318
- Where
- United States
- Volunteers
- 1,000 people, healthy volunteers welcome
- Sponsor
- National Human Genome Research Institute (NHGRI)
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
Advanced Malignant Solid Neoplasm, Carcinoma of Unknown Primary
Pembrolizumab in Treating Patients With Rare Tumors That Cannot Be Removed by Surgery or Are Metastatic
This phase II trial studies how well pembrolizumab works in treating patients with rare tumors that cannot be removed by surgery or have spread to other parts of the body.
- Conditions
- Advanced Malignant Solid Neoplasm, Carcinoma of Unknown Primary, Metastatic Adrenal Gland Pheochromocytoma, Metastatic Kidney Medullary Carcinoma
- Trial name
- NCT02721732
- Testing
- Laboratory Biomarker Analysis, Pembrolizumab, Questionnaire Administration
- Where
- United States
- Volunteers
- 157 people
- Sponsor
- M.D. Anderson Cancer Center
Small Cell Carcinoma of the Bladder, Small Cell Carcinoma of the Urinary Tract
Sacituzumab Govitecan With or Without Atezolizumab Immunotherapy in Rare Genitourinary Tumors (SMART) Such as High Grade Neuroendocrine Carcinomas, Adenocarcinoma, and Squamous Cell Bladder/Urinary Tract Cancer, Renal Medullary Carcinoma and Penile C...
Background: Rare tumors of the genitourinary (GU) tract can appear in the kidney, bladder, ureters, and penis. Rare tumors are difficult to study because there are not enough people to conduct large trials for new treatments.
- Conditions
- Small Cell Carcinoma of the Bladder, Small Cell Carcinoma of the Urinary Tract, Squamous Cell Carcinoma of the Bladder, Squamous Cell Carcinoma of the Urinary Tract
- Trial name
- NCT06161532
- Testing
- Sacituzumab govitecan, Atezolizumab
- Where
- United States
- Volunteers
- 60 people
- Sponsor
- National Cancer Institute (NCI)
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
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
Malignant Solid Neoplasm
Testing the Combination of Anti-Cancer Drugs Talazoparib and Temozolomide in Patients With Advanced Stage Rare Cancers, RARE 2 Trial
This phase II trial tests whether combination of talazoparib and temozolomide works to shrink tumors in patients with rare cancer that may have spread from where it first started to nearby tissue, lymph nodes, or distant parts of…
- Trial name
- NCT05142241
- Testing
- Biopsy Procedure, Biospecimen Collection, Computed Tomography
- Where
- 2 sites in United States
- Volunteers
- 14 people
- Sponsor
- National Cancer Institute (NCI)
Adenocarcinoma of the Bladder, Squamous Cell Carcinoma of the Bladder
Enfortumab Vedotin With or Without Pembrolizumab in Rare Genitourinary Tumors (E-VIRTUE)
Background: Many cancers of the testicles and urinary tract are rare diseases; these are diseases that affect less than 200,000 people in the United States. It can be hard to study treatments for these diseases.
- Conditions
- Adenocarcinoma of the Bladder, Squamous Cell Carcinoma of the Bladder, Testicular Germ Cell Tumors
- Trial name
- NCT06041503
- Testing
- Pembrolizumab, Enfortumab vedotin
- Where
- United States
- Volunteers
- 68 people
- Sponsor
- National Cancer Institute (NCI)
Hyperinsulinism, Polycystic Ovary Syndrome
Randomized Study of the Effect of Decreased Hyperinsulinemia on the Ovulatory Response to Clomiphene Citrate in Obese Women With Polycystic Ovary Syndrome
OBJECTIVES: I. Determine whether reduction of serum insulin levels by metformin increases ovulatory response to clomiphene or spontaneous ovulation in obese women with polycystic ovary syndrome.
- Trial name
- NCT00005654
- Testing
- clomiphene citrate, metformin
- Volunteers
- 88 people
- Sponsor
- National Center for Research Resources (NCRR)
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
Neuroblastoma, Nephroblastoma
Psychosocial Situation of Children With Rare Solid Abdominal Tumors and Their Families
Families of children with rare diseases (i.e., not more than 5 out of 10.000 people are affected) are often highly burdened with fears, insecurities and concerns regarding the affected child and his/her siblings.
- Conditions
- Neuroblastoma, Nephroblastoma, Hepatoblastoma
- Trial name
- PsyRareCare (NCT05245123)
- Where
- 2 sites in Germany
- Volunteers
- 100 people
- Sponsor
- Universitätsklinikum Hamburg-Eppendorf
Head and Neck Cancer
Camrelizumab With Famitinib for Patients With Rare Head and Neck Malignancies
This is a prospective, multicenter, multi-cohort, Phase II clinical trial enrolling patients with unresectable, recurrent, or metastatic soft tissue sarcoma, malignant melanoma, adenoid cystic carcinoma, or salivary gland…
- Trial name
- NCT07662681
- Testing
- camrelizumab with famitinib
- Where
- China
- Volunteers
- 40 people
- Sponsor
- Shanghai Ninth People's Hospital Affiliated to Shanghai Jiao Tong University
Polycystic Ovary Syndrome, Hyperinsulinism
Randomized Study of Decreased Hyperinsulinemia on the Ovulatory Response to Clomiphene Citrate in Women With Polycystic Ovary Syndrome
OBJECTIVES: I. Determine whether reduction of serum insulin levels by metformin increases ovulatory response to clomiphene citrate in women with polycystic ovary syndrome.
- Trial name
- NCT00005104
- Testing
- clomiphene citrate, metformin
- Where
- 6 sites in United States, Italy
- Volunteers
- 99 people
- Sponsor
- National Center for Research Resources (NCRR)