Clinical Trials on Orphan Diseases in United States

Total 148012 results

Pfizer

Recruiting

First-line Palbociclib and Endocrine Therapy for Patients With HR+/HER2- Advanced Breast Cancer in the Real-world Setting. (PERFORM)

Breast Neoplasms

Austria, Germany
AstraZeneca
Bristol-Myers Squibb; Hadassah Medical Organization; The TIMI Study Group

Completed

Multicenter Trial to Evaluate the Effect of Dapagliflozin on the Incidence of Cardiovascular Events (DECLARE-TIMI58)

Diabetes Mellitus, Non-Insulin-Dependent | High Risk for Cardiovascular Event

United States, Canada, France, Italy, Netherlands, Spain, Belgium, Thailand, Vietnam, Bulgaria, Korea, Republic of, Brazil, Philippines, Russian Federation, Turkey, Taiwan, Australia, Israel, South Africa, Germany, Japan, Czechia, H... and more
National Cancer Institute (NCI)
NRG Oncology

Completed

Veliparib, Pembrolizumab, and Combination Chemotherapy in Treating Patient With Locally Advanced Rectal Cancer

Rectal Adenocarcinoma | Stage III Rectal Cancer AJCC v7 | Stage II Rectal Cancer AJCC v7

United States, Puerto Rico
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Combination Chemotherapy With or Without Gemtuzumab in Treating Young Patients With Newly Diagnosed Acute Myeloid Leukemia

Leukemia

United States, Canada, Australia, Switzerland, New Zealand, Puerto Rico
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Surgery and Combination Chemotherapy in Treating Children With Extracranial Germ Cell Tumors

Childhood Teratoma | Stage II Malignant Testicular Germ Cell Tumor | Stage III Malignant Testicular Germ Cell Tumor | Childhood Embryonal Tumor | Childhood Extracranial Germ Cell Tumor | Childhood Extragonadal Germ Cell Tumor | Childhood Malignant Ovarian Germ Cell Tumor | Childhood Malignant Testicular... and other conditions

United States, Canada, Australia, New Zealand, Puerto Rico, Switzerland
Hoffmann-La Roche

Active, not recruiting

A Study Investigating the Outcomes and Safety of Atezolizumab Under Real-World Conditions in Patients Treated in Routine Clinical Practice (IMreal)

Small Cell Lung Cancer | Hepatocellular Carcinoma | Non-Small Cell Lung Cancer | Urothelial Carcinoma

Hungary, United Kingdom, Belgium, Croatia, France, Italy, Spain, United Arab Emirates, Argentina, Bulgaria, Poland, Philippines, Serbia, Netherlands, Romania, Colombia, Austria, Russian Federation, Singapore, Estonia, Egypt, India, ... and more
ECOG-ACRIN Cancer Research Group
National Cancer Institute (NCI)

Active, not recruiting

Cisplatin, Carboplatin and Etoposide or Temozolomide and Capecitabine in Treating Patients With Neuroendocrine Carcinoma of the Gastrointestinal Tract or Pancreas That Is Metastatic or Cannot Be Removed by Surgery

Gastric Neuroendocrine Carcinoma | Intestinal Neuroendocrine Carcinoma | Pancreatic Neuroendocrine Carcinoma

United States
Celgene

Active, not recruiting

Efficacy of Oral Azacitidine Plus Best Supportive Care as Maintenance Therapy in Subjects With Acute Myeloid Leukemia (AML) in Complete Remission (QUAZAR AML-001)

Leukemia, Myeloid, Acute

United States, Australia, Brazil, Canada, Czechia, Finland, France, Germany, Italy, Korea, Republic of, Mexico, Portugal, Russian Federation, Spain, United Kingdom, Belgium, Taiwan, Austria, Ireland, Israel, Lithuania, Poland, Turkey
Astellas Pharma Inc

Completed

Study to Test the Long Term Safety and Efficacy of the Beta-3 Agonist Mirabegron (YM178) in Patients With Symptoms of Overactive Bladder (TAURUS)

Urinary Bladder, Overactive

Canada, Poland, Belgium, Spain, Sweden, United States, Romania, Italy, France, Austria, United Kingdom, Germany, Hungary, Australia, Denmark, Netherlands, Switzerland, Lithuania, Russian Federation, Latvia, South Africa, Slovakia, F... and more
Hoffmann-La Roche

Active, not recruiting

A Study to Investigate Atezolizumab Subcutaneous in Patients With Previously Treated Locally Advanced or Metastatic Non-Small Cell Lung Cancer

Non-Small Cell Lung Cancer

Spain, Argentina, China, France, Turkey, Brazil, Korea, Republic of, Mexico, Chile, Guatemala, Peru, Thailand, Costa Rica, United Kingdom, New Zealand, Hungary, Latvia, Russian Federation, South Africa, Ukraine, Italy, Greece, Poland
Myovant Sciences GmbH

Completed

A Study to Evaluate the Safety and Efficacy of Relugolix in Men With Advanced Prostate Cancer (HERO)

Prostate Cancer

United States, Germany, Spain, New Zealand, Brazil, Belgium, Australia, Austria, Canada, China, Denmark, Finland, France, Italy, Japan, Korea, Republic of, Netherlands, Poland, Slovakia, Sweden, Taiwan, United Kingdom
National Cancer Institute (NCI)

Completed

Sorafenib With Either Temsirolimus or Tipifarnib in Treating Patients With Stage IV Malignant Melanoma That Cannot Be Removed By Surgery

Recurrent Melanoma | Stage IV Melanoma

United States
Alliance for Clinical Trials in Oncology
National Cancer Institute (NCI)

Active, not recruiting

A Clinical Study Evaluating The Benefit of Adding Rucaparib to Enzalutamide for Men With Metastatic Prostate Cancer That Has Become Resistant To Testosterone-Deprivation Therapy (CASPAR)

Castration-Resistant Prostate Carcinoma | Stage IV Prostate Cancer AJCC v8 | Stage IVA Prostate Cancer AJCC v8 | Stage IVB Prostate Cancer AJCC v8 | Metastatic Prostate Adenocarcinoma

United States
National Cancer Institute (NCI)

Recruiting

Adding an Immunotherapy Drug, MEDI4736 (Durvalumab), to the Usual Chemotherapy Treatment (Paclitaxel, Cyclophosphamide, and Doxorubicin) for Stage II-III Breast Cancer

Anatomic Stage II Breast Cancer AJCC v8 | Anatomic Stage III Breast Cancer AJCC v8 | Hormone Receptor-Positive Breast Carcinoma | HER2-Negative Breast Carcinoma

United States
Bayer

Terminated

A Study to Learn How Well the Study Treatment Asundexian Works and How Safe it is Compared to Apixaban to Prevent Stroke or Systemic Embolism in People With Irregular and Often Rapid Heartbeat (Atrial Fibrillation), and at Risk for Stroke (OCEANIC-AF)

Atrial Fibrillation | Prevention of Stroke or Systemic Embolism

Canada, United States, Israel, Korea, Republic of, China, Taiwan, Belgium, Australia, Singapore, Malaysia, Slovakia, United Kingdom, Czechia, Latvia, Japan, Brazil, Lithuania, Germany, France, Sweden, Turkey, Switzerland, Estonia, Bul... and more
Eli Lilly and Company

Recruiting

The Effect of Retatrutide Once Weekly on Cardiovascular Outcomes and Renal Function in Adults Living With Obesity (TRIUMPH-OUTCOMES)

Overweight and Obesity

United States, Australia, Italy, Hungary, Spain, Canada, Germany, Argentina, Belgium, Czechia, Greece, Israel, Austria, Brazil, Mexico, Netherlands, Romania, India, United Kingdom, Slovakia, Poland, Turkey, Puerto Rico, France, Denmark
National Cancer Institute (NCI)

Recruiting

Testing the Addition of Nivolumab to Standard Treatment for Patients With Metastatic or Unresectable Colorectal Cancer That Have a BRAF Mutation

Metastatic Colon Adenocarcinoma | Metastatic Rectal Adenocarcinoma | Stage III Colon Cancer AJCC v8 | Stage III Rectal Cancer AJCC v8 | Stage IV Colon Cancer AJCC v8 | Stage IV Rectal Cancer AJCC v8 | Unresectable Colon Adenocarcinoma | Unresectable Rectal Adenocarcinoma

United States, Puerto Rico
Midnight Pharma, LLC

Completed

Almorexant (ACT 078573) in Adult Subjects With Chronic Primary Insomnia (RESTORA 1)

Primary Insomnia

Spain, Germany, Belgium, France, United Kingdom, Australia, Austria, Bulgaria, Czech Republic, Denmark, Finland, Hungary, Israel, Italy, Poland, Slovakia, South Africa, Sweden, Switzerland, Ukraine
Alliance for Clinical Trials in Oncology
Bristol-Myers Squibb; National Cancer Institute (NCI)

Completed

Dasatinib or Placebo, Radiation Therapy, and Temozolomide in Treating Patients With Newly Diagnosed Glioblastoma Multiforme

Brain and Central Nervous System Tumors

United States
National Cancer Institute (NCI)

Active, not recruiting

Testing the Addition of Nivolumab to Chemotherapy in Treatment of Soft Tissue Sarcoma

Skin Angiosarcoma | Skin Radiation-Related Angiosarcoma | Visceral Angiosarcoma

United States, Guam
NRG Oncology
National Cancer Institute (NCI)

Recruiting

Testing the Addition of High Dose, Targeted Radiation to the Usual Treatment for Locally-Advanced Inoperable Non-small Cell Lung Cancer

Stage III Lung Cancer AJCC v8 | Stage IIB Lung Cancer AJCC v8 | Locally Advanced Lung Non-Small Cell Carcinoma

United States
Sesen Bio, Inc.

Terminated

Study of Proxinium Plus Best Supportive Care Versus Best Supportive Care for Patients With Advanced Head and Neck Cancer

Head and Neck Cancer | Head and Neck Neoplasms | Carcinoma, Squamous Cell | Neoplasms, Squamous Cell | Mouth Neoplasms | Carcinoma, Squamous Cell of Head and Neck

Spain, Germany, Israel, Italy, Poland, United Kingdom, India, Argentina, France, Hungary, Serbia, Croatia, Ukraine, Brazil, Russian Federation, Mexico, Romania, Slovakia
BeiGene

Completed

Study of BGB-A317 in Participants With Previously Treated Unresectable HCC

Hepatocellular Carcinoma (HCC)

Spain, China, Taiwan, Germany, Italy, United Kingdom, France, Poland
Fresenius Kabi
IQVIA Commercial GmbH & Co. OHG

Completed

Observational Cohort Study on the Use of Parenteral Nutrition and Clinical Outcomes in Adult Critically Ill Patients

Critical Illness

France, Austria, Spain, Belgium, Germany, Hungary, United Kingdom, Poland, Italy, Czechia, Sweden
Boehringer Ingelheim

Recruiting

A Study to Test the Effect of Survodutide (BI 456906) on Cardiovascular Safety in People With Overweight or Obesity (SYNCHRONIZE™ - CVOT)

Obesity

United States, Korea, Republic of, Belgium, Australia, United Kingdom, Mexico, Spain, Germany, Taiwan, Netherlands, Brazil, Hungary, Denmark, Canada, Poland, Austria, China, Finland, Greece, Italy, Hong Kong, Czechia, Portugal, Saudi... and more
Daiichi Sankyo
AstraZeneca

Recruiting

Trastuzumab Deruxtecan for Subjects With HER2-Positive Gastric Cancer or Gastro-Esophageal Junction Adenocarcinoma After Progression on or After a Trastuzumab-Containing Regimen (DESTINY-Gastric04)

Gastroesophageal Junction Adenocarcinoma | Gastric Cancer, Adenocarcinoma

Hong Kong, Korea, Republic of, France, United Kingdom, Spain, China, Ireland, Brazil, Japan, Singapore, Belgium, Israel, Russian Federation, Argentina, Chile, Hungary, Poland, Portugal, Taiwan, Turkey, Ukraine, Germany, Romania, Italy
National Cancer Institute (NCI)
Canadian Cancer Trials Group

Active, not recruiting

Nivolumab in Treating Patients With Localized Kidney Cancer Undergoing Nephrectomy (PROSPER)

Metastatic Renal Cell Carcinoma | Sarcomatoid Renal Cell Carcinoma | Stage III Renal Cell Cancer AJCC v7 | Unclassified Renal Cell Carcinoma | Stage II Renal Cell Cancer AJCC v7

United States, Canada, Israel
Children's Oncology Group
National Cancer Institute (NCI)

Completed

Combination Chemotherapy and Peripheral Stem Cell Transplantation in Treating Patients With Neuroblastoma

Neuroblastoma

United States, Canada
AstraZeneca

Completed

A Study Comparing Cardiovascular Effects of Ticagrelor Versus Placebo in Patients With Type 2 Diabetes Mellitus (THEMIS)

Diabetes Mellitus, Type 2

United States, Austria, Belgium, Canada, Denmark, France, Germany, Italy, Netherlands, Spain, United Kingdom, Czechia, Hungary, Thailand, Turkey, Vietnam, Korea, Republic of, Brazil, Peru, Philippines, Poland, China, Japan, Taiwan, Hong... and more
Children's Oncology Group

Recruiting

A Study to See if Memantine Protects the Brain During Radiation Therapy Treatment for Primary Central Nervous System Tumors

Central Nervous System Carcinoma

United States, Puerto Rico
Eli Lilly and Company

Completed

Open-Label Study of Intramuscular Olanzapine Depot in Patients With Schizophrenia or Schizoaffective Disorder

Schizoaffective Disorder | Schizophrenic Disorders

United States, France, Italy, Belgium, Mexico, Russian Federation, Puerto Rico, Taiwan, Australia, Romania, Croatia, Germany, Poland, Spain, Argentina, Brazil, Portugal, Slovakia, South Africa, Israel, Hungary, Austria, Czech Republic, Netherlan... and more
National Cancer Institute (NCI)
NRG Oncology

Recruiting

Testing the Addition of Radiation Therapy to the Usual Immune Therapy Treatment (Atezolizumab) for Extensive Stage Small Cell Lung Cancer, The RAPTOR Trial

Extensive Stage Lung Small Cell Carcinoma

United States
Eli Lilly and Company

Recruiting

A Study of Imlunestrant Versus Standard Endocrine Therapy in Participants With Early Breast Cancer (EMBER-4)

Breast Neoplasms

China, Japan, United States, Australia, Taiwan, Belgium, Canada, United Kingdom, Spain, France, Argentina, Ireland, Netherlands, Turkey, Czechia, Korea, Republic of, Finland, Hungary, Italy, Mexico, Brazil, Germany, India, Austria, Greece, Roman... and more
Incyte Corporation

Recruiting

A Study to Evaluate the Efficacy and Safety of Pemigatinib Versus Chemotherapy in Unresectable or Metastatic Cholangiocarcinoma (FIGHT-302)

Metastatic Cholangiocarcinoma | Unresectable Cholangiocarcinoma

United States, Canada, Belgium, France, Germany, Italy, China, Spain, Japan, United Kingdom, Netherlands, Switzerland, Austria, Denmark, Finland, Ireland, Israel, Norway, Sweden
Boehringer Ingelheim
Uppsala University; Population Health Research Institute

Completed

Randomized Evaluation of Long Term Anticoagulant Therapy (RE-LY) With Dabigatran Etexilate

Stroke | Atrial Fibrillation

United States, Argentina, Australia, Austria, Belgium, Brazil, Bulgaria, Canada, China, Colombia, Czechia, Denmark, Finland, France, Germany, Greece, Hong Kong, Hungary, India, Israel, Italy, Japan, Korea, Republic of, Malaysia, Mexico, N... and more
SWOG Cancer Research Network
National Cancer Institute (NCI); Janssen, LP

Recruiting

S1803, Lenalidomide +/- Daratumumab/rHuPh20 as Post-ASCT Maintenance for MM w/MRD to Direct Therapy Duration (DRAMMATIC)

Multiple Myeloma

United States, Canada
Children's Oncology Group
National Cancer Institute (NCI); University College, London; Medical Research...

Completed

Combination Chemotherapy, PEG-Interferon Alfa-2b, and Surgery in Treating Patients With Osteosarcoma (EURAMOS-1)

Metastatic Osteosarcoma | Localized Osteosarcoma

United States, Canada, Australia, New Zealand, Puerto Rico, Switzerland
Bayer

Active, not recruiting

A Study to Learn More About How Well Elinzanetant Works and How Safe it is Compared to Placebo for the Treatment of Hot Flashes Caused by Anti-cancer Therapy in Women With, or at High Risk for Developing Hormone-receptor Positive Breast Cancer (OASIS-4)

Hot Flashes | Vasomotor Symptoms Caused by Adjuvant Endocrine Therapy in Women With, or at High Risk for Developing Hormone-receptor Positive Breast Cancer

Spain, France, Belgium, United Kingdom, Hungary, Israel, Italy, Ireland, Germany, Portugal, Finland, Poland, Romania, Canada, Austria, Kazakhstan
Radiation Therapy Oncology Group
National Cancer Institute (NCI)

Withdrawn

Combination Chemotherapy, Radiation Therapy, and Surgery in Treating Patients With Primary or Recurrent Sarcoma

Recurrent Adult Soft Tissue Sarcoma | Stage III Adult Soft Tissue Sarcoma | Stage IVA Adult Soft Tissue Sarcoma | Stage IIB Adult Soft Tissue Sarcoma | Stage IIC Adult Soft Tissue Sarcoma

United States, Canada
AbbVie

Active, not recruiting

A Study Evaluating Safety and Efficacy of Venetoclax in Combination With Azacitidine Versus Standard of Care After Allogeneic Stem Cell Transplantation (SCT) in Participants With Acute Myeloid Leukemia (AML) (VIALE-T)

Cancer | Acute Myeloid Leukemia (AML)

United States, Australia, Brazil, Canada, China, Czechia, France, Germany, Greece, Hungary, Israel, Italy, Japan, Korea, Republic of, Spain, Switzerland, Taiwan, United Kingdom
National Cancer Institute (NCI)

Active, not recruiting

Combination Chemotherapy With or Without Atezolizumab in Treating Patients With Stage III Colon Cancer and Deficient DNA Mismatch Repair

Lynch Syndrome | Stage III Colon Cancer AJCC v8 | Colon Adenocarcinoma | DNA Repair Disorder

United States, Puerto Rico, Germany
National Cancer Institute (NCI)
NRG Oncology

Active, not recruiting

Pegylated Liposomal Doxorubicin Hydrochloride With Atezolizumab and/or Bevacizumab in Treating Patients With Recurrent Ovarian, Fallopian Tube, or Primary Peritoneal Cancer

Recurrent Fallopian Tube Carcinoma | Recurrent Ovarian Carcinoma | Recurrent Primary Peritoneal Carcinoma | Ovarian Seromucinous Carcinoma | Recurrent Platinum-Resistant Ovarian Carcinoma | Primary Peritoneal High Grade Serous Adenocarcinoma | Ovarian High Grade Serous Adenocarcinoma | Fallopian... and other conditions

United States, Puerto Rico
Eli Lilly and Company

Available

Named Patient Use Program to Provide Abemaciclib (LY2835219) for the Treatment of Metastatic Breast Cancer

Metastatic Breast Cancer

Australia, Canada, Egypt, India, Italy, Korea, Republic of, Malaysia, Singapore, South Africa, Spain, Switzerland, Taiwan, Thailand, United Kingdom
National Cancer Institute (NCI)

Completed

Gemcitabine Hydrochloride and Cisplatin With or Without Bevacizumab in Treating Patients With Advanced Urinary Tract Cancer

Metastatic Prostate Carcinoma | Metastatic Bladder Urothelial Carcinoma | Metastatic Renal Pelvis Urothelial Carcinoma | Metastatic Ureter Urothelial Carcinoma | Metastatic Urethral Urothelial Carcinoma | Metastatic Urothelial Carcinoma | Stage IV Bladder Urothelial Carcinoma AJCC v7 | Stage IV Renal... and other conditions

United States, Puerto Rico
Eli Lilly and Company
NSABP Foundation Inc

Active, not recruiting

Endocrine Therapy With or Without Abemaciclib (LY2835219) Following Surgery in Participants With Breast Cancer (monarchE)

Breast Cancer

United States, Spain, China, Germany, Canada, Taiwan, United Kingdom, Singapore, Italy, Australia, Japan, Hungary, Russian Federation, India, Belgium, New Zealand, Austria, Saudi Arabia, France, Czechia, Korea, Republic of, Finland, Argentin... and more
Antisoma Research

Unknown

Phase III Randomized Study of Amonafide (AS1413) and Cytarabine Versus Daunorubicin and Cytarabine in Patients With Secondary Acute Myeloid Leukemia (AML)- the ACCEDE Study

Secondary Acute Myeloid Leukemia (Secondary AML, sAML)

Korea, Republic of, United States, Spain, United Kingdom, Australia, Austria, Taiwan, Israel, Italy, Belgium, Germany, France, Romania, Estonia, Canada, Ukraine, Ecuador, Hungary, Poland, Chile, Argentina, Czech Republic, Russian Federation
National Cancer Institute (NCI)
Eastern Cooperative Oncology Group

Completed

Comparison of Combination Chemotherapy Regimens With or Without Cetuximab in Treating Patients Who Have Undergone Surgery For Stage III Colon Cancer

Stage III Colon Cancer | Adenocarcinoma of the Colon

United States, Canada, Puerto Rico
AstraZeneca

Completed

[SOCRATES -Acute Stroke Or Transient IsChaemic Attack TReated With Aspirin or Ticagrelor and Patient OutcomES] (SOCRATES)

Acute Ischaemic Stroke | Transient Ischaemic Attack

Sweden, United States, Australia, Brazil, Bulgaria, France, Italy, Korea, Republic of, Mexico, Peru, Poland, Russian Federation, Spain, Turkey, Vietnam, Germany, Philippines, China, Hungary, Romania, Ukraine, Hong Kong, Argentina, Bel... and more
Southwest Oncology Group
National Cancer Institute (NCI); Eastern Cooperative Oncology Group; Radiation... and other collaborators

Completed

S9900: Surgery With or Without Combination Chemotherapy in Treating Patients With Non-small Cell Lung Cancer

Lung Cancer

United States, Canada
NRG Oncology
National Cancer Institute (NCI)

Recruiting

Observation or Radiation Therapy in Treating Patients With Newly Diagnosed Grade II Meningioma That Has Been Completely Removed by Surgery

Intracranial Meningioma | Grade II Meningioma

Canada, United States, Japan, Saudi Arabia, India

Clinical Trials on Orphan Diseases in United States

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Egypt

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations