Clinical trials: Neonate
Joint Damage
Effect of Febuxostat on Joint Damage in Hyperuricemic Subjects With Early Gout
This purpose of this study is to assess the effect of febuxostat, once daily (QD), on joint damage in patients with elevated serum urate levels and gout.
- Trial name
- NCT01078389
- Testing
- Febuxostat
- Where
- 56 sites in United States
- Volunteers
- 314 people
- Sponsor
- Takeda
Chronic Iron Overload
Pharmacokinetic Study of Deferiprone in Paediatric Patients
Deferiprone (DFP) is the most extensively studied oral iron chelator to date. It has been authorised in Europe in 1999 for the treatment of iron overload in patients with beta-thalassaemia major when DFO is contraindicated or…
- Trial name
- DEEP-1 (NCT01740713)
- Testing
- Deferiprone, dose level 1, Deferiprone, dose level 2, Deferiprone, dose level 3
- Where
- 7 sites in Cyprus, Egypt and 1 more
- Volunteers
- 23 people
- Sponsor
- Consorzio per Valutazioni Biologiche e Farmacologiche
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)
Respiration; Insufficient or Poor, Newborn
Mask Ventilation With Different Face Masks During Neonatal Resuscitation
Most premature babies have difficulty breathing at birth and need help (what we call resuscitation). The treatment for this is to gently inflate their lungs with a resuscitation device and a facemask.
- Trial name
- NCT01685697
- Testing
- Mask ventilation with a Laerdal face mask, Mask ventilation with a F&P face mask
- Where
- Canada
- Volunteers
- 56 people
- Sponsor
- University of Alberta
Hemophilia A or B With Inhibitors
FEIBA Reconstitution Volume Reduction and Faster Infusion Study (FEIBA STAR)
The purpose of this study is to: 1. To evaluate the tolerability and safety of infusing reduced volume Factor Eight Inhibitor Bypassing Activity (FEIBA) at the standard infusion rate of 2 U/kg/min 2.
- Trial name
- NCT02764489
- Testing
- FEIBA
- Where
- 3 sites in Croatia, North Macedonia and 1 more
- Volunteers
- 45 people
- Sponsor
- Baxalta now part of Shire
Duchenne Muscular Dystrophy
CoQ10 and Prednisone in Non-Ambulatory DMD
This study will help determine if CoQ10 and prednisone, alone and as a combination decrease the decline in cardiopulmonary and skeletal muscle function that occurs in the wheelchair confined phase of DMD.
- Trial name
- NCT00308113
- Testing
- Prednisone, Coenzyme Q10
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Cooperative International Neuromuscular Research Group
Spinal Muscular Atrophy Type 3
Long Term Safety of Amifampridine Phosphate in Spinal Muscular Atrophy 3
A long term safety study of amifampridine phosphate in patients with spinal muscular atrophy (SMA) Type 3.
- Trial name
- SMA3 (NCT03819660)
- Testing
- Amifampridine Phosphate 10 MG Oral Tablet
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Pulmonary Hypertension, Familial Persistent, of the Newborn
A Study To Evaluate Safety And Efficacy Of IV Sildenafil In The Treatment Of Neonates With Persistent Pulmonary Hypertension Of The Newborn
This study will evaluate whether IV sildenafil can reduce the time on inhaled nitric oxide treatment and reduce the failure rate of available treatments for persistent pulmonary hypertension of the newborn.
- Trial name
- NCT01720524
- Testing
- iv sildenafil
- Where
- 42 sites in United States, Belgium and 10 more
- Volunteers
- 59 people
- Sponsor
- Pfizer's Upjohn has merged with Mylan to form Viatris Inc.
Primary Biliary Cholangitis, Cardiovascular Abnormalities
Early Identification of Myocardial Impairment in PBC
Primary biliary cholangitis (PBC) is a chronic inflammatory liver disease leading to cirrhosis. Researches reported patients with PBC may involve abnormalities on skeleton, thyroid and exocrine glands.
- Trial name
- EARLY-MYO-PBC (NCT03545672)
- Testing
- CMR examination
- Where
- China
- Volunteers
- 119 people, healthy volunteers welcome
- Sponsor
- RenJi Hospital
Cystic Fibrosis
Single Dose of pGM169/GL67A in CF Patients
The study objectives are to assess safety, tolerability and gene expression after a single dose of non-viral CFTR gene therapy (pGM169/GL67A) administered to the nose and lungs of patients with cystic fibrosis.
- Trial name
- NCT00789867
- Testing
- pGM169/GL67A
- Where
- United Kingdom
- Volunteers
- 35 people
- Sponsor
- Imperial College London
Thalassemia
Allogeneic Stem Cell Transplantation, Severe Homzygous 0/+Thalassemia or Sever Variants of Beta 0/+ Thalassemia, THALLO
Patients have severe beta-thalassemia or one of the thalassemia variants. Thalassemia is a hereditary disease in which the bone marrow produces abnormal red blood cells that have a shorter life span than normal red blood cells.
- Trial name
- THALLO (NCT00578292)
- Testing
- Busulfan, Fludarabine, Campath 1H
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Baylor College of Medicine
Neonatal Abstinence Syndrome
Comparison Between Methadone and Morphine for Neonatal Opiate Withdrawal
Hypothesis is that the effectiveness of opiate treatment with morphine will result in shorter duration of opiate medication treatment and fewer infants treated with a second drug.
- Trial name
- NAS (NCT01804075)
- Testing
- Methadone, Morphine
- Volunteers
- 31 people
- Sponsor
- Eastern Maine Medical Center
Osteoporosis
1 Year Open-label Extension to CZOL446H2337 Safety and Efficacy Trial of Zoledronic Acid Twice Yearly in Osteoporotic Children Treated With Glucocorticoids
This 1-year open-label extension to CZOL446H2337 is designed to evaluate the safety and efficacy of zoledronic acid twice yearly in osteoporotic children treated with glucocorticoids.
- Trial name
- NCT01197300
- Testing
- Zoledronic acid
- Where
- 10 sites in Australia, Canada and 4 more
- Volunteers
- 25 people
- Sponsor
- Novartis Pharmaceuticals
Acute Respiratory Distress Syndrome
Sigh Ventilation to Increase Ventilator-Free Days in Victims of Trauma at Risk for Acute Respiratory Distress Syndrome
A randomized, concurrent controlled trial to assess if adding sigh breaths to usual invasive mechanical ventilation of victims of trauma who are at risk of developing ARDS will decrease the number of days they require invasive…
- Trial name
- SiVent (NCT02582957)
- Testing
- Sigh breaths
- Where
- 15 sites in United States
- Volunteers
- 524 people
- Sponsor
- University of Minnesota
Mucopolysaccharidosis IVA, Morquio A Syndrome
Efficacy and Safety Study of BMN 110 for Morquio A Syndrome Patients Who Have Limited Ambulation
The primary objective of this study is to evaluate the effect of 2.0 mg/kg/week BMN 110 in a patient population that has limited ambulation, in a period of up to 144 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01697319
- Testing
- BMN 110
- Where
- 7 sites in United States, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- BioMarin Pharmaceutical
Atopic Dermatitis
The Effectiveness of Montelukast on Atopic Dermatitis in Koreans
The purpose of this study is to assess the clinical effectiveness of Montelukast in children (2~6 years old) with atopic dermatitis and identify the pathophysiologic background of Montelukast on the role of modulating the atopic…
- Trial name
- NCT00903357
- Where
- South Korea
- Volunteers
- 54 people
- Sponsor
- Pyun BokYang
Acute Intermittent Porphyria
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).
- Trial name
- NCT02949830
- Testing
- Givosiran
- Where
- 5 sites in United States, Sweden and 1 more
- Volunteers
- 16 people
- Sponsor
- Alnylam Pharmaceuticals
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
Treatment for Relapsed/Refractory AML Based on a High Throughput Drug Sensitivity Assay
This clinical trial uses a laboratory test called a high throughput sensitivity assay in planning treatment for patients with relapsed or refractory acute myeloid leukemia.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT01872819
- Testing
- antitumor drug screening assay, chemotherapy, biological therapy
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- University of Washington
Duchenne Muscular Dystrophy
Tadalafil as Adjuvant Therapy for DMD
This project will assess the vascular responsiveness in leg muscles of boys with Duchenne muscular dystrophy (DMD) to one single dose of tadalafil, a common vasodilator drug, using non-invasive techniques (MRI or Doppler…
- Trial name
- NCT05195775
- Testing
- Tadalafil
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Florida
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
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