Clinical trials: Fetal Developmental Abnormality
Gout
Pegylated Recombinant Mammalian Uricase (PEG-uricase) as Treatment for Refractory Gout
The purpose of this study is to determine whether PEG-uricase (a chemically modified recombinant mammalian enzyme that degrades uric acid) is effective in controlling hyperuricemia in patients with chronic gout, who cannot…
- Trial name
- NCT00111657
- Testing
- Pegloticase
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- John Sundy
Atopic Dermatitis
Efficacy and Safety Study of QAW039 in the Treatment of Patients With Moderate to Severe Atopic Dermatitis.
The purpose of this study is to determine whether QAW039 is safe and has beneficial effects in people who have moderate to severe atopic dermatitis (AD).
- Trial name
- NCT01785602
- Testing
- QAW039
- Where
- 18 sites in Australia, Austria and 6 more
- Volunteers
- 103 people
- Sponsor
- Novartis Pharmaceuticals
Osteoarthritis, Rheumatoid Arthritis
NexGen TM Tibia Clinical Outcomes Study
The objective of this study is to evaluate clinical performance for the commercially available NexGen TM Monoblock Tibia and NexGen TM Modular Tibia used in primary cementless tibia total knee arthroplasty.
- Conditions
- Osteoarthritis, Rheumatoid Arthritis, Traumatic Arthritis, Polyarthritis
- Trial name
- NCT03272373
- Testing
- NexGen TM Monoblock Tibia, NexGen TM Modular Tibia
- Where
- 7 sites in United States
- Volunteers
- 160 people
- Sponsor
- Zimmer Biomet
Huntington's Disease
Study Evaluating The Safety, Tolerability And Brain Function Of 2 Doses Of PF-0254920 In Subjects With Early Huntington's Disease
This study will evaluate the Safety, Tolerability and Brain Function of 2 doses of PF-0254920 in Subjects with Early Huntington's Disease.
- Trial name
- NCT01806896
- Testing
- PF-02545920
- Where
- France
- Volunteers
- 37 people
- Sponsor
- Pfizer
Phenylketonuria
Fluorodeoxyglucose Positron Emission Tomography (FDG PET) Findings in Patients With Phenylketonuria Before and After KUVAN Therapy
The aim of this pilot study is to determine if there are any changes in brain glucose metabolism in the gray matter of patients with Phenylketonuria (PKU) and whether administration of Sapropterin (KUVAN) therapy can improve such…
- Trial name
- PKU (NCT00986973)
- Testing
- Sapropterin
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Children's Hospital of Philadelphia
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Ondansetron in Preventing Nausea and Vomiting in Patients Undergoing Stem Cell Transplant
RATIONALE: Ondansetron may help lessen or prevent nausea and vomiting in patients undergoing stem cell transplant. PURPOSE: This phase II trial is studying how well ondansetron works in preventing nausea and vomiting in patients…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00795769
- Testing
- ondansetron, survey administration, management of therapy complications
- Where
- United States
- Volunteers
- 49 people
- Sponsor
- Fred Hutchinson Cancer Center
Hypothalamic Obesity
Energy Balance & Weight Loss in Craniopharyngioma-related or Other Hypothalamic Tumors in Hypothalamic Obesity
The proposed multicenter study will test the effect of glucagon-like peptide (GLP)-1 agonist exenatide once weekly extended-release (ExQW, Bydureon®) on clinical outcomes and metabolic parameters in a double-blind…
- Trial name
- ECHO (NCT02664441)
- Testing
- Exenatide
- Where
- 3 sites in United States
- Volunteers
- 42 people
- Sponsor
- Seattle Children's Hospital
Developmental Disabilities, Weight Loss
Weight Management for Adolescents With IDD
The goal of this study is to explore the impact of two different diets (conventional vs. enhanced stop light) and two different delivery systems (face-to-face vs.
- Conditions
- Developmental Disabilities, Weight Loss, Body Weight, Down Syndrome
- Trial name
- IDDAdol (NCT02561754)
- Testing
- Enhanced Stop Light Diet (eSLD), Conventional Diet (CD), Face-to-face (FTF)
- Where
- 2 sites in United States
- Volunteers
- 110 people
- Sponsor
- University of Kansas Medical Center
Graft Versus Host Disease, Leukemia
Donor Peripheral Stem Cell Transplant and Donor Natural Killer Cell Transplant After Total-Body Irradiation, Thiotepa, Fludarabine, and Muromonab-CD3 in Treating Patients With Leukemia or Other Blood Diseases
RATIONALE: Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell and donor natural killer cell transplant helps stop the growth of cancer and abnormal cells and helps stop the patient's immune…
- Conditions
- Graft Versus Host Disease, Leukemia, Myelodysplastic Syndromes
- Trial name
- NCT00450983
- Testing
- muromonab-CD3, natural killer cell therapy, fludarabine phosphate
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Fred Hutchinson Cancer Center
Bronchopulmonary Dysplasia
Study of Inhaled Nitric Oxide (iNO) and Respiratory Outcomes in Late Preterm Infants
The aim of this study is to determine if inhaled nitric oxide will improve short and long term respiratory outcomes in preterm infants.
- Trial name
- NCT01748045
- Testing
- inhaled nitric oxide
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Tufts Medical Center
Oculopharyngeal Muscular Dystrophy
Safety Tolerability and Efficacy Study of Cabaletta to Treat Oculopharyngeal Muscular Dystrophy (OPMD) Patients
The Purpose of this study is to assess the Safety, Tolerability and Efficacy of Intravenous Cabaletta® in Oculopharyngeal Muscular Dystrophy (OPMD) Patients.
- Trial name
- HOPEMD (NCT02015481)
- Testing
- Cabaletta
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Bioblast Pharma Ltd.
Neuroendocrine Tumors, Carcinoid Tumors
A Phase II Study of Pertuzumab and Erlotinib for Metastatic or Unresectable Neuroendocrine Tumors
To determine objective response rates (RR) by RECIST guideline version 1.1 for all patients treated with this strategy consisting of initial therapy with pertuzumab as a single agent and then addition of erlotinib for those who…
- Conditions
- Neuroendocrine Tumors, Carcinoid Tumors, Adrenal Gland Tumors, Neuroblastoma
- Trial name
- NCT00947167
- Testing
- pertuzumab, erlotinib
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Pamela L. Kunz
Hypercholesterolemia, Familial Hypercholesterolemia
A Clinical Trial to Assess the Long Term Safety and Tolerability of MK-0653H in Japanese Participants With Hypercholesterolemia (MK-0653H-833)
The study will assess the safety and tolerability of Ezetimibe 10 mg+ Rosuvastatin 2.5 mg and Ezetimibe 10 mg+ Rosuvastatin 5.0 mg for up to 52 weeks in Japanese participants with hypercholesterolemia uncontrolled with…
- Trial name
- NCT02748057
- Testing
- Ezetimibe, Rosuvastatin
- Volunteers
- 135 people
- Sponsor
- Organon and Co
Hypophosphatemia, Familial, Hyperparathyroidism
Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets
The purpose of this study is to determine the effectiveness of paricalcitol, a form of synthetic vitamin D, in lowering parathyroid hormone (PTH) levels and reducing disease symptoms in children and adults with X-linked…
- Trial name
- NCT00417612
- Testing
- Paricalcitol
- Where
- United States
- Volunteers
- 33 people
- Sponsor
- Yale University
Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7)
MS-275 and GM-CSF in Treating Patients With Myelodysplastic Syndrome and/or Relapsed or Refractory Acute Myeloid Leukemia or Acute Lymphocytic Leukemia
This phase II trial is studying how well giving MS-275 together with GM-CSF works in treating patients with myelodysplastic syndrome and/or relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT00462605
- Testing
- entinostat, sargramostim
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- National Cancer Institute (NCI)
Hemophilia A, Hemophilia B
Study of Coagulation Faction VIIa Variant Marzeptacog Alfa (Activated) in Adult Subjects With Hemophilia
This multi-center, open label Phase 1 study will evaluate the pharmacokinetics, pharmacodynamics, and safety of a single IV dose of MarzAA followed by ascending single SC doses of MarzAA in adult subjects with moderate or severe…
- Conditions
- Hemophilia A, Hemophilia B, Hemophilia A With Inhibitor, Hemophilia B With Inhibitor
- Trial name
- NCT04072237
- Testing
- MarzAA (marzeptacog alfa [activated])
- Where
- 5 sites in Bulgaria, Russia
- Volunteers
- 11 people
- Sponsor
- Catalyst Biosciences
Huntington Disease, Irritability
Evaluating the Efficacy of Dextromethorphan/Quinidine in Treating Irritability in Huntington's Disease
The purpose of this study is to assess efficacy and safety of dextromethorphan/quinidine 20mg/10mg (DM/Q 20mg/10mg) in patients with irritability due to Huntington's disease.
- Trial name
- NCT03854019
- Testing
- Dextromethorphan/quinidine 20mg/10mg (DM/Q 20mg/10mg)
- Where
- United States
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Friedreich's Ataxia
Long-Term Safety Extension Study of ACTIMMUNE® (Interferon γ-1b) in Children and Young Adults With Friedreich's Ataxia
The purpose of this long term extension study is to evaluate the long-term safety of ACTIMMUNE® (interferon-γ 1b) in participants with Friedreich's Ataxia (FA).
- Trial name
- STEADFAST (NCT02797080)
- Testing
- interferon γ-1b
- Where
- 4 sites in United States
- Volunteers
- 38 people
- Sponsor
- Amgen
Duchenne Muscular Dystrophy
A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
This study is designed to evaluate the ability of ataluren to increase dystrophin protein levels in muscle cells of participants with nmDMD.
- Trial name
- NCT03648827
- Testing
- Ataluren
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- PTC Therapeutics
Anemia, Sickle Cell
Dexamethasone to Treat Acute Chest Syndrome in People With Sickle Cell Disease
People with sickle cell disease (SCD) may develop acute chest syndrome (ACS), which is a common and serious lung condition that usually requires hospitalization.
- Trial name
- NCT00530270
- Testing
- Dexamethasone
- Where
- 6 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
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