Clinical trials: Multiple Primary Neoplasm
Hereditary Angioedema (HAE)
A European Post-Authorisation Observational Study Of Patients With Hereditary Angioedema
This observational (non-interventional) study is being conducted to characterize the safety and use of CINRYZE in routine clinical practice when administered for (1) routine prevention of angioedema attacks, (2) pre-procedure…
- Trial name
- NCT01541423
- Where
- 20 sites in Belgium, France and 4 more
- Volunteers
- 83 people
- Sponsor
- Shire
Primary Ciliary Dyskinesia (PCD)
Malnutrition and Sarcopenia Among Primary Ciliary Dyskinesia
The prevalence and causes of malnutrition and sarcopenia in children and adolescents with Primary ciliary dyskinesia (PCD) have not been sufficiently elucidated.
- Trial name
- Malnutrition (NCT07740538)
- Testing
- Physical assessments of individuals
- Where
- Turkey (Türkiye)
- Volunteers
- 15 people
- Sponsor
- Izmir Democracy University
Ciliary Motility Disorders, Primary Ciliary Dyskinesia
Nasal Nitric Oxide Across Mutations in Primary Ciliary Dyskinesia
Primary Ciliary Dyskinesia (PCD) is a rare genetic disorder characterized by dysfunction of motile cilia associated with recurrent infections of the airways, laterality defects (Situs inversus totalis in about 50% of cases) and…
- Trial name
- nNO_PCD (NCT04949308)
- Where
- Germany
- Volunteers
- 2,000 people
- Sponsor
- University Hospital Muenster
Chronic Myeloproliferative Disorders, Kidney Cancer
Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer, Metastatic Kidney Cancer, or Aplastic Anemia
RATIONALE: Giving low doses of chemotherapy before a donor stem cell transplant using stem cells that closely match the patient's stem cells, helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Kidney Cancer, Leukemia, Lymphoma
- Trial name
- NCT00295997
- Testing
- anti-thymocyte globulin, filgrastim, graft-versus-tumor induction therapy
- Where
- 2 sites in United States
- Volunteers
- 35 people
- Sponsor
- University of California, San Francisco
Pregnancy Related
Elafibranor Pregnancy Surveillance Program: A Study to Evaluate the Safety of Elafibranor During Pregnancy
The study will include participants who were exposed to at least one dose of elafibranor either within the three weeks before conception or at any time during pregnancy (based on estimated last menstrual period [LMP]).
- Trial name
- NCT07556913
- Testing
- Elafibranor
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Ipsen
Epilepsy, Dravet Syndrome
A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy, Safety, and Tolerability of TAK-935 (OV935) as an Adjunctive Therapy in Pediatric Participants With Developmental and/or Epileptic Encephalopathies
The purpose of this study is to investigate the effect on the frequency of all seizures (convulsive and drop) in participants treated with TAK-935 compared to placebo.
- Conditions
- Epilepsy, Dravet Syndrome, Lennox-Gastaut Syndrome
- Trial name
- ELEKTRA (NCT03650452)
- Testing
- TAK-935
- Where
- 44 sites in United States, Australia and 6 more
- Volunteers
- 141 people
- Sponsor
- Takeda
Acute Myeloid Leukemia, Acute Lymphocytic Leukemia
Myeloablative Umbilical Cord Blood Transplantation in Hematological Diseases
RATIONALE: Giving chemotherapy drugs, such as fludarabine and cyclophosphamide, and total-body irradiation before a donor umbilical cord blood stem cell transplant helps stop the growth of cancer cells and prepares the patient's…
- Conditions
- Acute Myeloid Leukemia, Acute Lymphocytic Leukemia, Chronic Myelogenous Leukemia, Myelofibrosis
- Trial name
- NCT00309842
- Testing
- filgrastim, cyclophosphamide, cyclosporine
- Where
- 2 sites in United States
- Volunteers
- 213 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Tuberous Sclerosis Complex, Epilepsy
Safety and Efficacy of AV078 in Participants With Tuberous Sclerosis Complex (TSC) Refractory Epilepsy (RESTOR-1)
This Phase 2, randomized, double-blind, placebo-controlled study will evaluate the safety, tolerability, and efficacy of oral AV078 in participants with refractory epilepsy due to tuberous sclerosis complex (TSC).
- Trial name
- RESTOR-1 (NCT07680322)
- Testing
- AV078
- Where
- Canada
- Volunteers
- 42 people
- Sponsor
- Aeovian Pharmaceuticals, Inc.
Invasive Aspergillosis
A Study To Evaluate The Safety Of Voriconazole As Treatment Of Invasive Aspergillosis (Fungal Infection) And Other Rare Molds In Children
The purpose of this study is to evaluate the safety profile of voriconazole (an antifungal drug) when used in children who have invasive aspergillosis (IA) and other rare systemic fungal infections.
- Trial name
- NCT00836875
- Testing
- Voriconazole
- Where
- 25 sites in United States, Canada and 6 more
- Volunteers
- 31 people
- Sponsor
- Pfizer
Chronic Myeloproliferative Disorders, Leukemia
Reduced-Intensity Busulfan and Fludarabine With or Without Antithymocyte Globulin Followed by Donor Stem Cell Transplant in Treating Patients With Hematologic Cancer or Other Disease
RATIONALE: Giving low doses of chemotherapy, such as busulfan and fludarabine, before a donor stem cell transplant helps stop the growth of cancer and abnormal cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00448201
- Testing
- anti-thymocyte globulin, sargramostim, therapeutic allogeneic lymphocytes
- Where
- United States
- Volunteers
- 71 people
- Sponsor
- UNC Lineberger Comprehensive Cancer Center
Parkinson Disease, Multiple System Atrophy
Facilitating Diagnostics and Prognostics of Parkinsonian Syndromes Using Neuroimaging
The goals of this study are: 1) to identify biomarkers using neuroimaging that are associated with progression rate using statistical methods, and 2) to identify biomarkers that are associated with the differential diagnosis of…
- Conditions
- Parkinson Disease, Multiple System Atrophy, Progressive Supranuclear Palsy
- Trial name
- NCT03872102
- Where
- United States
- Volunteers
- 90 people, healthy volunteers welcome
- Sponsor
- University of Texas Southwestern Medical Center
Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission
Fludarabine Phosphate, Cyclophosphamide, Total Body Irradiation, and Donor Stem Cell Transplant in Treating Patients With Blood Cancer
This phase II trial studies how well fludarabine phosphate, cyclophosphamide, total body irradiation, and donor stem cell transplant work in treating patients with blood cancer.
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, BCR-ABL1 Positive, Acute Leukemia in Remission, Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia
- Trial name
- NCT03333486
- Testing
- Cyclophosphamide, Fludarabine Phosphate, Laboratory Biomarker Analysis
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Roswell Park Cancer Institute
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
Osteoarthritis, Arthritis
AMIStem Primary Hip System Prospective Post-marketing Multi-centre Surveillance Study
This is a multicentre post-marketing prospective study to evaluate performance and clinical outcomes of AMIIStem primary hip system.
- Conditions
- Osteoarthritis, Arthritis, Avascular Necrosis, Fracture of the Femoral Neck or Head
- Trial name
- NCT01107340
- Testing
- AMIStem Hip System
- Where
- 2 sites in Switzerland, United Kingdom
- Volunteers
- 500 people
- Sponsor
- Medacta International SA
Childhood Obesity
NET-Works:Community Preschooler Obesity Prevention
The goal of the Minnesota center is to integrate primary care, home, and community-based intervention strategies to promote sustained patterns of change in food intake, physical activity, and body weight among low income…
- Trial name
- NET-Works (NCT01606891)
- Testing
- multi-component/setting parent-targeted intervention
- Where
- United States
- Volunteers
- 534 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Multiple Endocrine Neoplasia
Studies of Inherited Diseases of Metabolism
Diseases of mineral metabolism such as familial multiple endocrine neoplasia type 1 (FMEN1), familial hypocaliuric hypercalcemia (FHH), familial hyperparathyroidism (FH), and pseudohypoparathyroidism (PHP) are known as hereditary…
- Trial name
- NCT00001345
- Where
- United States
- Volunteers
- 969 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Tuberous Sclerosis, Neurofibromatoses
Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC)…
- Conditions
- Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
- Trial name
- NCT01031901
- Testing
- Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Neurodegenerative Diseases, Alzheimer Disease
Sociodemographic Factors and Criminal Behaviour Preceding Neurodegenerative Disease - Retrospective Register Study
In this retrospective register study, clinically classified individuals with neurodegenerative disease from the years 2010-2021 will be verified from the clinical records from KUH and Oulu University Hospital (OUH).
- Conditions
- Neurodegenerative Diseases, Alzheimer Disease, Frontotemporal Dementia, Lewy Body Disease
- Trial name
- DEGERWD (NCT06209515)
- Where
- Finland
- Volunteers
- 61,292 people
- Sponsor
- Kuopio University Hospital
Medullary Thyroid Carcinoma, Multiple Endocrine Neoplasia Type 2
Study of Molecular Pathways in Medullary Thyroid Carcinoma and Correlation of Molecular Data With Clinical Behavior of the MTC in Individuals Patients
Background: Medullary thyroid carcinoma (MTC) is a rare malignancy, occurring either as a sporadic disease (75% of cases), or in a hereditary pattern as multiple endocrine neoplasia (MEN) type 2 (MEN2A or MEN2B) or familial…
- Trial name
- NCT01424878
- Where
- United States
- Volunteers
- 150 people
- Sponsor
- National Cancer Institute (NCI)
Kartagener Syndrome
Long-term Lung Function and Disease Progression in Children With Early Onset Primary Ciliary Dyskinesia Lung Disease
Primary ciliary dyskinesia (PCD), also known as Kartagener syndrome, is a genetic disorder of the cilia, which are microscopic hair-like cells.
- Trial name
- NCT00722878
- Where
- 6 sites in United States, Canada
- Volunteers
- 48 people
- Sponsor
- University of North Carolina, Chapel Hill