Clinical trials: Rare Cancers
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)
Non-Small-Cell Lung Cancer, Metastatic Non-Small-Cell Lung Cancer
Study to Evaluate Efficacy and Safety of Firmonertinib Compared With Investigator's Choice of EGFR Inhibitor as First-Line Treatment in Participants Who Have Locally Advanced or Metastatic NSCLC With EGFR P-Loop and Alpha C-Helix Compressing (PACC) Uncommon Mutations
Global, Phase 3, randomized, multicenter, open-label study evaluating the efficacy and safety of firmonertinib at a dose level of 240 mg QD compared to investigator's choice of osimertinib (80 mg QD) or afatinib (40 mg QD) in…
- Conditions
- Non-Small-Cell Lung Cancer, Metastatic Non-Small-Cell Lung Cancer, Advanced Non-Small-Cell Lung Cancer, EGFR P-Loop and Alpha C-Helix Compressing
- Trial name
- NCT07185997
- Testing
- Firmonertinib, EGFR-TKI inhibitor based on investigator's choice
- Where
- 139 sites in United States, Australia and 15 more
- Volunteers
- 480 people
- Sponsor
- ArriVent BioPharma, Inc.
Rare Histiocytic Disorders (RHDs), Erdheim-Chester Disease (ECD)
International Rare Histiocytic Disorders Registry (IRHDR)
The rare histiocytic disorders (RHDs) are characterized by the infiltration of one or more organs by non-LCH histiocytes. They can range from localized disease that resolves spontaneously, to progressive disseminated forms that…
- Conditions
- Rare Histiocytic Disorders (RHDs), Erdheim-Chester Disease (ECD), Rosai-Dorfman Disease (RDD), Xanthogranuloma Family (XG)
- Trial name
- IRHDR (NCT02285582)
- Testing
- Registry study
- Where
- 15 sites in United States, Argentina and 7 more
- Volunteers
- 400 people
- Sponsor
- The Hospital for Sick Children
Nasopharyngeal Neoplasms
Safety and Efficacy of CC-486 in Previously Treated Patients With Locally Advanced or Metastatic Nasopharyngeal Carcinoma
The purpose of this study is to evaluate the safety and efficacy of CC-486 in previously treated patients with locally advanced or metastatic nasopharyngeal carcinoma having failed one to two previous regimens, including…
- Trial name
- NCT02269943
- Testing
- CC-486
- Where
- 25 sites in United States, Canada and 7 more
- Volunteers
- 36 people
- Sponsor
- Celgene
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
Non-small Cell Lung Cancer
A Study to Evaluate the Efficacy and Safety of Sutetinib Maleate Capsule in Locally Advanced or Metastatic NSCLC
The main objective of the study will be to evaluate the efficacy of sutetinib maleate capsules in participants with locally advanced or metastatic non-small cell lung cancer NSCLC (uncommon EGFR mutations only).
- Trial name
- NCT06010329
- Testing
- Sutetinib Maleate Capsule
- Where
- 9 sites in United States
- Volunteers
- 66 people
- Sponsor
- Teligene US
Inherited Disease, Rare Diseases
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less than 5:100,000) in adulthood through the systematic longitudinal…
- Conditions
- Inherited Disease, Rare Diseases, Metabolic Disease, Undiagnosed Disease
- Trial name
- NCT04880356
- Testing
- collection of data
- Where
- Italy
- Volunteers
- 100 people
- Sponsor
- Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta
Hemangioma
Randomized Study of Hormonal Regulation of Infantile Hemangioma
OBJECTIVES: I. Evaluate the clinical efficacy of leuprolide, a gonadotropin-releasing hormone agonist (GnRHa), in treating infants with vision-endangering or large, disfiguring hemangiomas. II.
- Trial name
- NCT00004436
- Testing
- leuprolide, prednisone
- Volunteers
- 30 people
- Sponsor
- FDA Office of Orphan Products Development
Neurofibromatosis 1, Noonan Syndrome
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
Background: RASopathies are a group of genetic diseases that affect a child s development. They cause physical, cognitive, and behavioral symptoms. Caring for a child with a RASopathy can be stressful.
- Conditions
- Neurofibromatosis 1, Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous Syndrome
- Trial name
- NCT05361811
- Testing
- Waitlist, ACT Intervention
- Where
- United States
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- National Cancer Institute (NCI)
Lung Cancer (NSCLC)
Perioperative Targeted Therapy in Resectable or Potentially Resectable Stage III Non-small Cell Lung Cancer With Rare Driver Mutations
This study evaluates the effectiveness and safety of targeted therapy given before and after surgery in patients with resectable or potentially resectable stage III non-small cell lung cancer with selected genetic alterations.
- Trial name
- NCT07829744
- Testing
- Surgical Resection
- Volunteers
- 190 people
- Sponsor
- Shanghai Zhongshan Hospital
Gynecologic Cancer, Skin Cancer
Stereotactic Body Radiotherapy in Patients With Rare Oligometastatic Cancers (OligoRARE)
This is a randomized open-label multicentre Phase III superiority study of the effect of adding SBRT to the standard of care treatment on overall survival in patients with rare oligometastatic cancers.
- Conditions
- Gynecologic Cancer, Skin Cancer, Head and Neck Cancer, Sarcoma
- Trial name
- OligoRARE (NCT04498767)
- Testing
- Stereotactic body radiotherapy, Palliative RT
- Where
- 13 sites in Belgium, France and 5 more
- Volunteers
- 200 people
- Sponsor
- European Organisation for Research and Treatment of Cancer - EORTC
Rare Malignant Neoplasm
Marker Assisted Selective ThErapy in Rare Cancers: Knowledge Database Establishing registrY Asia
This is a registry study that aims to collect patients' data with advanced-stage rare cancer in Asia-Pacific region. Data includes clinical information, details of treatment, prognosis, pathological diagnosis and genetic…
- Trial name
- MASTERKEY ASIA (NCT05217407)
- Testing
- Genomic sequence
- Where
- 18 sites in Japan, Malaysia and 5 more
- Volunteers
- 1,000 people
- Sponsor
- National Cancer Center, Japan
HER2-positive Breast Cancer, HER2 Gene Mutation
Study of A166 in Patients With Relapsed/Refractory Cancers Expressing HER2 Antigen or Having Amplified HER2 Gene
Open-label, Phase I-II, first-in-human (FIH) study for A166 monotherapy in HER2-expressing or amplified patients who progressed on or did not respond to available standard therapies.
- Conditions
- HER2-positive Breast Cancer, HER2 Gene Mutation, HER-2 Gene Amplification, HER2 Positive Gastric Cancer
- Trial name
- NCT03602079
- Testing
- A166
- Where
- 10 sites in United States
- Volunteers
- 49 people
- Sponsor
- Klus Pharma Inc.
Multiple Osteochondroma, Osteogenesis Imperfecta
Resilience and Coping in a Rare Skeletal Disease Population to Face Coronavirus (COVID-19) Outbreak Distress: a Longitudinal Study
In the COVID-19 outbreak context, people living with rare diseases have been highly troubled with anxiety, loneliness, and depression.
- Conditions
- Multiple Osteochondroma, Osteogenesis Imperfecta, Ollier Disease, Maffucci Syndrome
- Trial name
- RELOAD (NCT04844697)
- Where
- Italy
- Volunteers
- 15 people
- Sponsor
- Istituto Ortopedico Rizzoli
Haematological Malignancy, Malignant Neoplasm
DETERMINE Trial Treatment Arm 07: Dabrafenib in Combination With Trametinib in Adult, Paediatric and Teenage/Young Adult Patients With BRAF V600 Mutation-Positive Cancers.
This clinical trial is looking at two drugs called dabrafenib and trametinib. Dabrafenib and trametinib are approved as standard of care treatment for adult patients with melanoma (a type of skin cancer) or lung cancer and in…
- Conditions
- Haematological Malignancy, Malignant Neoplasm, Lymphoproliferative Disorders, Neoplasms by Histologic Type
- Trial name
- DETERMINE (NCT07440290)
- Testing
- Dabrafenib, Trametinib
- Where
- 27 sites in United Kingdom
- Volunteers
- 30 people
- Sponsor
- Cancer Research UK
Amyloidosis
Study of High-Dose Melphalan and Autologous Stem Cell Transplantation in Patients With Primary Light Chain Amyloidosis
OBJECTIVES: I. Determine the response, disease-free survival, and overall survival of patients with primary light chain amyloidosis treated with high-dose melphalan and autologous stem cell transplantation. II.
- Trial name
- NCT00017680
- Testing
- Melphalan, Autologous Stem Cell Transplantation
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- Herbert Irving Comprehensive Cancer Center
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
Rare Diseases, Genetic Predisposition to Disease
Genome-based Management of Patients in Precision Medicine (Ge-Med) Towards a Genomic Health Program
The GE-MED APPROACH project will enroll patients (n = appr. 12.000) with unclear molecular cause of the disease, suspected genetic cause of the disease without detailed molecular analysis like Whole Exome Sequencing (WES).
- Trial name
- GE-MED (NCT04760522)
- Testing
- WGS Diagnostic: Blood take for genetic diagnostic
- Where
- Germany
- Volunteers
- 12,000 people
- Sponsor
- University Hospital Tuebingen
Refractory Soft Tissue Sarcomas
Cabozantinib for Adults With Advanced Soft Tissue Sarcoma
Background: Cabozantinib is a cancer treatment drug that blocks the growth of new blood vessels in tumors. It can also block a chemical on tumor cells that allows the cells to grow.
- Trial name
- NCT01755195
- Testing
- Cabozantinib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Locally Advanced Undifferentiated Pleomorphic Sarcoma, Metastatic Undifferentiated Pleomorphic Sarcoma
Sintilimab for the Treatment of Locally Advanced, Metastatic, Recurrent, or Unresectable Undifferentiated Pleomorphic Sarcoma, SiARa Cancer Study
This phase II trial investigates the effects of sintilimab in treating patients with undifferentiated pleomorphic sarcoma that has spread to nearby tissue or lymph nodes (locally advanced), spread to other places in the body…
- Conditions
- Locally Advanced Undifferentiated Pleomorphic Sarcoma, Metastatic Undifferentiated Pleomorphic Sarcoma, Recurrent Undifferentiated Pleomorphic Sarcoma, Unresectable Undifferentiated Pleomorphic Sarcoma
- Trial name
- NCT05017103
- Testing
- Sintilimab
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- M.D. Anderson Cancer Center