Clinical trials: Fetal Developmental Abnormality
Gout
Combining Lesinurad With Allopurinol in Inadequate Responders
This study that compare the serum uric acid lowering effects, clinical benefits, and safety of lesinurad in combination with allopurinol to allopurinol alone in subjects with gout who have had an inadequate response to…
- Trial name
- CLEAR 1 (NCT01510158)
- Testing
- Lesinurad, Allopurinol
- Where
- 165 sites in United States
- Volunteers
- 607 people
- Sponsor
- Ardea Biosciences, Inc.
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Dasatinib, Ifosfamide, Carboplatin, and Etoposide in Treating Young Patients With Metastatic or Recurrent Malignant Solid Tumors
RATIONALE: Dasatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Drugs in chemotherapy, such as ifosfamide, carboplatin, and etoposide, work in different ways to stop the growth of…
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00788125
- Testing
- carboplatin, dasatinib, etoposide phosphate
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- City of Hope Medical Center
Congenital Hemophilia A
Pharmacokinetic, Efficacy, and Safety Study of Recombinant Factor VIII Single Chain (rVIII-SingleChain) in Children With Severe Hemophilia A
This is an international, multicenter, open-label study to assess the efficacy, safety, and pharmacokinetic (PK) profile of rVIII-SingleChain in pediatric patients with severe hemophilia A.
- Trial name
- NCT02093897
- Testing
- rVIII-SingleChain
- Where
- 37 sites in United States, Australia and 17 more
- Volunteers
- 84 people
- Sponsor
- CSL Behring
Cardiovascular Diseases
Study of the Effectiveness and Safety of Niaspan ® in the Treatment of Lipid Abnormalities in Latin America
Post-Marketing Observational study of Niaspan® tablet in accordance with each country regulations. This study will be conducted in a prospective, single-arm, multi-center format.
- Trial name
- NCT01200160
- Testing
- Niacin
- Where
- 22 sites in Colombia, Mexico and 1 more
- Volunteers
- 128 people, healthy volunteers welcome
- Sponsor
- Abbott
Atopic Dermatitis
Effect of Pimecrolimus Cream on Cathelicidin Levels in Subjects With Eczema
The purpose of this study is to determine the effect of topical pimecrolimus on the immune system by assessing the levels of antimicrobial peptides in the skin of patients with eczema.
- Trial name
- NCT00946478
- Testing
- Pimecrolimus, Vehicle cream
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- University of California, San Diego
Atopic Dermatitis
Clobetasol Topical Oil for Children With Moderate to Severe Atopic Dermatitis
Open-Label Study designed to evaluate the HPA axis suppression potential of Clobetasol Topical Oil and pharmacokinetic safety / systemic exposure to clobetasol when Clobetasol Topical Oil is applied to pediatric subjects with…
- Trial name
- NCT03847389
- Testing
- Clobetasol propionate 0.05% Topical Oil
- Where
- 6 sites in United States
- Volunteers
- 8 people
- Sponsor
- Hill Dermaceuticals, Inc.
Hereditary Angioedema (HAE)
Efficacy Study of DX-88 (Ecallantide) to Treat Acute Attacks of Hereditary Angioedema (HAE)
The purpose of this study is to evaluate the efficacy and safety of DX-88 (ecallantide) versus placebo in the treatment of moderate to severe acute attacks of hereditary angioedema.
- Trial name
- NCT00457015
- Testing
- ecallantide, Phosphate Buffer Saline (PBS), pH 7.0
- Where
- 45 sites in United States, Canada and 1 more
- Volunteers
- 96 people
- Sponsor
- Shire
Heart Defects, Congenital
Safety and Performance of the COR-VG-001 Conduit in Pediatric Patients for Extracardiac Total Cavopulmonary Connection
The extracardiac Fontan surgery/procedure involves diverting the venous blood from the inferior vena cava to the pulmonary arteries without passing through the morphologic right ventricle.
- Trial name
- EC-TCPC (NCT02377674)
- Testing
- Vascular Graft, Model COR-VG-001
- Where
- Russia
- Volunteers
- 5 people
- Sponsor
- Xeltis
Parkinson's Disease, Gaucher's Disease
Intravenous N-acetylcysteine for the Treatment of Gaucher's Disease and Parkinson's Disease
The investigators are interested in determining if the investigators are able to detect changes in brain chemistry using Magnetic Resonance Spectroscopy (MRS) in individuals with Parkinson's disease (PD), those with Gaucher's…
- Trial name
- NCT01427517
- Testing
- N-acetylcysteine
- Where
- United States
- Volunteers
- 9 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia
Sunitinib in Treating Patients With Idiopathic Myelofibrosis
This phase II trial is studying how well sunitinib works in treating patients with idiopathic myelofibrosis. Sunitinib may stop the growth of abnormal cells by blocking some of the enzymes needed for cell growth and by blocking…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Acute Undifferentiated Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission
- Trial name
- NCT00387426
- Testing
- sunitinib malate
- Where
- United States
- Volunteers
- 14 people
- Sponsor
- National Cancer Institute (NCI)
Down Syndrome
A 4-Week Safety Study of Oral ELND005 in Young Adults With Down Syndrome Without Dementia
This is a prospective, randomized, double-blind, placebo-controlled, parallel-group, three-arm, multicenter study of the safety and PK of ELND005 administered orally for 4 weeks.
- Trial name
- DS201 (NCT01791725)
- Testing
- ELND005
- Where
- 3 sites in United States
- Volunteers
- 23 people
- Sponsor
- OPKO Health, Inc.
Cystic Fibrosis
Effects of an Antioxidant-Enriched Multivitamin Supplement on Inflammation and Oxidative Stress in Cystic Fibrosis
The purpose of this study will be to evaluate the effects of a modified formulation of AquADEKs (AquADEKs-2) on markers of inflammation, antioxidant levels and oxidative stress.
- Trial name
- AquADEKs-2 (NCT01859390)
- Testing
- AquADEKs-2, control multivitamin
- Where
- 15 sites in United States
- Volunteers
- 73 people
- Sponsor
- University of Colorado, Denver
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
Hemophilia B
Post Marketing Surveillance To Observe Safety and Efficacy Of BeneFIX In Patients With Hemophilia B
To provide safety and effectiveness information of BeneFIX during the post-marketing period as required by Korea FDA regulations, to identify any potential drug related treatment factors in Korean population including: 1) Unknown…
- Trial name
- NCT00484185
- Testing
- BeneFIX (coagulation factor IX (recombinant))
- Where
- South Korea
- Volunteers
- 183 people
- Sponsor
- Pfizer
Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1
Azacitidine With or Without Lenalidomide or Vorinostat in Treating Patients With Higher-Risk Myelodysplastic Syndromes or Chronic Myelomonocytic Leukemia
This randomized phase II/III trial studies how well azacitidine works with or without lenalidomide or vorinostat in treating patients with higher-risk myelodysplastic syndromes or chronic myelomonocytic leukemia.
- Conditions
- Chronic Myelomonocytic Leukemia, Chronic Myelomonocytic Leukemia-1, Chronic Myelomonocytic Leukemia-2, Myelodysplastic Syndrome
- Trial name
- NCT01522976
- Testing
- Azacitidine, Laboratory Biomarker Analysis, Lenalidomide
- Where
- 294 sites in United States, Canada
- Volunteers
- 282 people
- Sponsor
- National Cancer Institute (NCI)
MELAS, LHON
A Dose-escalating Clinical Trial With KH176
Mitochondrial Diseases are rare progressive, multi-system, often early fatal disorders affecting both children and adults. KH176 is a novel chemical entity currently under development for the treatment of inherited mitochondrial…
- Conditions
- MELAS, LHON, Leigh Syndrome, Mitochondrial Disease
- Trial name
- NCT02544217
- Testing
- KH176
- Where
- Belgium
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Khondrion BV
Mucopolysaccharidosis (MPS)
Safety, Tolerability, Ascending Dose and Dose Frequency Study of rhHNS Via an IDDD in MPS IIIA Patients
Sanfilippo syndrome, or Mucopolysaccharidosis (MPS) III, is a rare lysosomal storage disease (LSD) caused by loss in activity of 1 of 4 enzymes necessary for degradation of the glycosaminoglycan (GAG) heparan sulfate (HS) in…
- Trial name
- NCT01155778
- Testing
- Recombinant human heparan N-sulfatase (rhHNS)
- Where
- 2 sites in Netherlands, United Kingdom
- Volunteers
- 12 people
- Sponsor
- Shire
XMEN
Magnesium Supplementation in People With XMEN Syndrome
Background: X-linked immunodeficiency with magnesium defect, Epstein-Barr virus infection, and neoplasia syndrome is called XMEN syndrome. In this genetic condition, the cells have less magnesium than normal.
- Trial name
- NCT02496676
- Testing
- Magnesium L-threonate, Intravenous (IV) magnesium sulfate (MgSO4)
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Nocturnal Enuresis, Anemia, Sickle Cell
Desmopressin for Bedwetting in Children With SCD
This study assesses if using the medication desmopressin will decrease nightime bedwetting in children with sickle cell disease.
- Trial name
- NCT04420585
- Testing
- Desmopressin
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Montefiore Medical Center
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
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