Clinical trials: Birth Defect
Breast Cancer, Chronic Myeloproliferative Disorders
Aprepitant, Granisetron, & Dexamethasone in Preventing Nausea & Vomiting in Pts. Receiving Cyclophosphamide Before a Stem Cell Transplant
RATIONALE: Antiemetic drugs, such as aprepitant, granisetron, and dexamethasone, may help lessen or prevent nausea and vomiting in patients treated with chemotherapy.
- Conditions
- Breast Cancer, Chronic Myeloproliferative Disorders, Gestational Trophoblastic Tumor, Leukemia
- Trial name
- NCT00293384
- Testing
- Aprepitant, Cyclophosphamide, Dexamethasone
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Chronic Gout Refractory to Conventional Therapy
Re-exposure Study of Pegloticase Intravenous (i.v.) in Symptomatic Gout Patients
The purpose of the study is to evaluate the safety and clinical effect of re-exposure to a 24 week course of treatment of pegloticase i.v. in subjects whose last exposure to pegloticase i.v.
- Trial name
- NCT00675103
- Testing
- pegloticase 8 mg i.v.
- Where
- 4 sites in United States
- Volunteers
- 7 people
- Sponsor
- Savient Pharmaceuticals
Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis
Pilot Study of a Multi-Drug Regimen for Severe Pulmonary Fibrosis in Hermansky-Pudlak Syndrome
This study will examine whether five drugs (pravastatin, Losartan, Zileuton, N-acetylcysteine and erythromycin) used together can slow the course of pulmonary fibrosis (scarring of the lung tissue) in patients with…
- Conditions
- Hermansky-Pudlak Syndrome (HPS), Pulmonary Fibrosis, Oculocutaneous Albinism, Platelet Storage Pool Deficiency
- Trial name
- NCT00467831
- Testing
- Losartan, Zileuton, N-Acetylcysteine
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- National Human Genome Research Institute (NHGRI)
Heart Septal Defects, Atrial, Atrial Septal Defect
Safety and Efficacy Study of Absnow Absorbable ASD Closure System For Treating ASD Patients
The aim of this study is to evaluate the safety and efficacy of Absnow absorbable ASD closure system for treating patients with atrial septal defect.
- Trial name
- NCT03601039
- Testing
- Absnow Absorbable ASD Closure System
- Where
- 14 sites in China
- Volunteers
- 165 people
- Sponsor
- Lifetech Scientific (Shenzhen) Co., Ltd.
Cystic Fibrosis
Study of Ivacaftor in Cystic Fibrosis Subjects Aged 6 to 11 Years With the G551D Mutation
The purpose of this study was to evaluate the efficacy and safety of ivacaftor in subjects with cystic fibrosis aged 6 to 11 years who have the G551D mutation in the cystic fibrosis transmembrane conductance regulator (CFTR)…
- Trial name
- ENVISION (NCT00909727)
- Testing
- Ivacaftor
- Where
- 29 sites in United States, Australia and 5 more
- Volunteers
- 52 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
A Study of Prasugrel in Pediatric Participants With Sickle Cell Disease
The purpose of this study is to determine the correct prasugrel dosage to be given to children with sickle cell disease (SCD).
- Trial name
- Crescent (NCT01476696)
- Testing
- Prasugrel
- Where
- 9 sites in United States
- Volunteers
- 33 people
- Sponsor
- Eli Lilly and Company
Zollinger-Ellison Syndrome, Multiple Endocrine Neoplasia
Treatment of Zollinger-Ellison Syndrome With Prevacid
The purpose of this study is to study the safety and efficacy of high dose Prevacid in the long-term treatment of patients who secrete abnormally large amounts of gastric acid.
- Trial name
- NCT00204373
- Testing
- Lansoprazole (Prevacid)
- Volunteers
- 72 people
- Sponsor
- Charles Mel Wilcox, MD
Phenylketonuria (PKU)
Safety and Efficacy of Self Administered Injections of Pegvaliase (>40mg/Day Dose) in Adults With PKU
This is a Phase 3 open-label extension study enrolling adult patients with PKU who were previously treated with pegvaliase in Studies PAL-003 (NCT00924703) or 165-302 (NCT02468570).
- Trial name
- NCT03694353
- Testing
- Pegvaliase
- Where
- 17 sites in United States
- Volunteers
- 37 people
- Sponsor
- BioMarin Pharmaceutical
Anti-Psychotic, Management of Manifestations of Psychotic Disorders
Assessment of Intra-subject Variability in the Bioavailability of Chlorpromazine Hydrochloride
Cycle Pharmaceuticals Ltd. (Cycle) is developing an oral tablet formulation of Chlorpromazine Hydrochloride and intends to conduct bioequivalence trials to demonstrate its similarity to the RLD.
- Conditions
- Anti-Psychotic, Management of Manifestations of Psychotic Disorders, Treatment of Schizophrenia, Control Nausea and Vomiting
- Trial name
- NCT02943213
- Testing
- Chlorpromazine Hydrochloride
- Where
- South Africa
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Cycle Pharmaceuticals Ltd.
Neurofibromatosis, Neurofibromas
Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform Neurofibromas
This is a second Pilot Study to determine the efficacy of Gleevec® in neurofibromatosis (NF1) patients with plexiform neurofibromas using new response assessment modalities with the secondary goals of assessing Gleevec toxicity…
- Trial name
- 0908-09 (NCT01140360)
- Testing
- Gleevec
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Kent Robertson
Huntington Disease
Open-label Extension Study to Evaluate the Safety and Tolerability of WVE-120102 in Patients With Huntington's Disease
WVE-HDSNP2-002 is an open-label extension (OLE) study to evaluate the safety, tolerability, PK, PD, and clinical effects of WVE-120102 in adult patients with early manifest HD who carry a targeted single nucleotide polymorphism…
- Trial name
- NCT04617860
- Testing
- WVE-120102
- Where
- 20 sites in Australia, Canada and 5 more
- Volunteers
- 36 people
- Sponsor
- Wave Life Sciences USA, Inc.
Cystic Fibrosis
Rollover Study to Evaluate the Safety and Efficacy of Long-term Treatment With Lumacaftor in Combination With Ivacaftor
Study 110 is a Phase 3, multicenter study in subjects aged 6 years and older with cystic fibrosis (CF) who are homozygous for the F508del-CF transmembrane conductance regulator (CFTR) mutation and who participated in Study 109…
- Trial name
- NCT02544451
- Testing
- LUM/IVA
- Where
- 60 sites in United States, Australia and 7 more
- Volunteers
- 246 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Sickle Cell Disease
Comparison of Patient Centered Outcomes for People With Sickle Cell Disease in the Acute Care Setting
The Emergency Department has been the standard location where patients with Sickle Cell Disease (SCD) go to seek care for the treatment of acute painful events.
- Trial name
- ESCAPED (NCT02411396)
- Where
- 3 sites in United States
- Volunteers
- 483 people
- Sponsor
- Johns Hopkins University
Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant
Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) in Homozygous Familial Hypercholesterolemia
The purpose of this study is to evaluate the safety and efficacy of mipomersen (ISIS 301012) in subjects with homozygous familial hypercholesterolemia on lipid-lowering therapy.
- Conditions
- Lipid Metabolism, Inborn Errors, Hypercholesterolemia, Autosomal Dominant, Hyperlipidemias, Metabolic Diseases
- Trial name
- RADICHOL 1 (NCT00607373)
- Testing
- mipomersen
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 51 people
- Sponsor
- Kastle Therapeutics, LLC
Urea Cycle Disorder
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
This is a randomized, controlled, open-label parallel arm study to assess the safety, tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to Sodium phenylbutyrate (NaPBA) in urea cycle disorder subjects…
- Trial name
- NCT03335488
- Testing
- RAVICTI, NaPBA
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 16 people
- Sponsor
- Amgen
Atopic Dermatitis, Healthy Volunteers
Safety Study of Tezepelumab (AMG 157) in Healthy Adults and Adults With Atopic Dermatitis
This study is a single dose escalation study of tezepelumab (AMG 157) in healthy adults (Part A) and adults with moderate to severe atopic dermatitis (Part B).
- Trial name
- NCT00757042
- Testing
- Tezepelumab
- Volunteers
- 78 people, healthy volunteers welcome
- Sponsor
- Amgen
Pulmonary Disease, Transcatheter Pulmonary Valve Replacement (TPVR)
ALTERRA: SAPIEN 3 THV With the Alterra Adaptive Prestent
To demonstrate the safety and effectiveness of the Edwards Alterra Adaptive Prestent in conjunction with the Edwards SAPIEN 3 Transcatheter Heart Valve (THV) System in subjects with a dysfunctional right ventricular outflow…
- Conditions
- Pulmonary Disease, Transcatheter Pulmonary Valve Replacement (TPVR), Tetralogy of Fallot, Congenital Heart Disease
- Trial name
- NCT03130777
- Testing
- Edwards Alterra Adaptive Prestent with SAPIEN 3 THV
- Where
- 14 sites in United States
- Volunteers
- 86 people
- Sponsor
- Edwards Lifesciences
Tuberous Sclerosis Complex
Trial of RAD001 and Neurocognition in Tuberous Sclerosis Complex (TSC)
Tuberous Sclerosis Complex (TSC) is a multi-system disease, usually presenting with seizures, mental retardation and autism, and exhibiting a high variability in clinical findings both among and within families.
- Trial name
- TSC (NCT01289912)
- Testing
- RAD001
- Where
- 2 sites in United States
- Volunteers
- 52 people
- Sponsor
- Mustafa Sahin
Bronchopulmonary Dysplasia, Neurally Adjusted Ventilatory Assist
NAVA vs. CMV Crossover in Severe BPD
This prospective, unblinded, pilot randomized cross-over trial of 2 modes of mechanical ventilation will compare measures of pulmonary mechanics, respiratory gas exchange, and patient comfort between conventional flow triggered…
- Conditions
- Bronchopulmonary Dysplasia, Neurally Adjusted Ventilatory Assist, Mechanical Ventilation
- Trial name
- NCT04821453
- Testing
- NAVA, CMV
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Hemophilia A
Efficacy and Safety Study of a Recombinant Protein-Free Manufactured Factor VIII (rAHF-PFM) in Previously Untreated Hemophilia A Patients
The purpose of this study is to evaluate whether Antihemophilic factor, recombinant, manufactured protein-free (rAHF-PFM) is effective and safe in the treatment of hemophilia A patients who have not been treated with factor VIII…
- Trial name
- NCT00157157
- Testing
- Recombinant Antihemophilic Factor Manufactured and Formulated without Added Human or Animal Proteins (rAHF-PFM)
- Where
- 35 sites in United States, Austria and 8 more
- Volunteers
- 66 people
- Sponsor
- Baxalta now part of Shire