Clinical trials: Birth Defects
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia (M7)
Tipifarnib and Etoposide in Treating Older Patients With Newly Diagnosed, Previously Untreated Acute Myeloid Leukemia
This randomized phase II trial is studying the side effects and how well giving tipifarnib together with etoposide works in treating older patients with newly diagnosed, previously untreated acute myeloid leukemia.
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a)
- Trial name
- NCT00602771
- Testing
- tipifarnib, etoposide
- Where
- 5 sites in United States
- Volunteers
- 84 people
- Sponsor
- National Cancer Institute (NCI)
Duchenne Muscular Dystrophy
DART Electrical Impedance Myography (EIM) Trial in Duchenne Muscular Dystrophy (DMD) and Healthy Controls
Physicians seek a method to assess neuromuscular disease that is both non-invasive and quantifiable. Many patients do not tolerate standard current day assessment tools (such as needle electromyogram), and Electrical Impedance…
- Trial name
- NCT01523964
- Testing
- Testing with EIM
- Where
- 6 sites in United States
- Volunteers
- 92 people, healthy volunteers welcome
- Sponsor
- Dart Therapeutics. LLC
Becker Muscular Dystrophy
A Study to Assess Vamorolone in Becker Muscular Dystrophy (BMD)
This Phase II pilot study is a randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, PD, and exploratory clinical efficacy of vamorolone 500mg (250mg for body weight <50 kg) daily…
- Trial name
- NCT05166109
- Testing
- Vamorolone
- Where
- 2 sites in United States, Italy
- Volunteers
- 46 people
- Sponsor
- ReveraGen BioPharma, Inc.
Cystic Fibrosis
A Study to Evaluate the Safety and Efficacy of VX-371 in Subjects With Cystic Fibrosis Who Are Homozygous for the F508del-CFTR Mutation
The purpose of this study is to evaluate the safety and efficacy of treatment with VX-371 in hypertonic saline compared to hypertonic saline alone in subjects with cystic fibrosis (CF) who are ≥12 years of age, homozygous for the…
- Trial name
- NCT02709109
- Testing
- VX-371 + HS, Hypertonic saline, Orkambi
- Where
- 40 sites in United States, France and 2 more
- Volunteers
- 147 people
- Sponsor
- Parion Sciences
Duchenne Muscular Dystrophy
Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)
The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53…
- Trial name
- NCT02740972
- Testing
- NS-065/NCNP-01
- Where
- 7 sites in United States, Canada
- Volunteers
- 16 people
- Sponsor
- NS Pharma, Inc.
Congenital Bleeding Disorder, Haemophilia A
Safety and Efficacy of Turoctocog Alfa in Previously Treated Male Children With Haemophilia A
This trial is conducted in Asia, Europe, and North and South America. The aim of this clinical trial is to investigate the safety and efficacy of turoctocog alfa (recombinant factor VIII, rFVIII (N8)) in male previously treated…
- Trial name
- guardian™ 3 (NCT01138501)
- Testing
- turoctocog alfa
- Where
- 38 sites in United States, Brazil and 11 more
- Volunteers
- 65 people
- Sponsor
- Novo Nordisk A/S
Transient Tachypnea of the Newborn
High Flow Nasal Cannula Versus Bubble Nasal CPAP for the Treatment of Transient Tachypnea of the Newborn in Infants ≥ 35 Weeks Gestation
The primary objective is to determine whether High Flow Nasal Cannula (HFNC) is a superior respiratory modality for neonates ≥36 weeks with transient tachypnea of the newborn (TTN) when compared to the standard of care modality…
- Trial name
- NCT01270581
- Testing
- High Flow Nasal Cannula, Bubble Nasal CPAP
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Icahn School of Medicine at Mount Sinai
Williams Beuren Syndrome
Efficacy of Minoxidil in Children With Williams-Beuren Syndrome
The Williams-Beuren syndrome (WBS) is a sporadic congenital disorder characterized by a multisystem developmental impairment. This syndrome is caused by a microdeletion in chromosome 7q11.23 that encompasses loss of the elastin…
- Trial name
- Williams (NCT00876200)
- Testing
- Minoxidil
- Where
- 18 sites in France
- Volunteers
- 21 people
- Sponsor
- Hospices Civils de Lyon
Sickle Cell Anemia
Transcranial Doppler (TCD) With Transfusions Changing to Hydroxyurea
The primary goal of the Phase III TWiTCH trial is to compare 24 months of alternative therapy (hydroxyurea) to standard therapy (transfusions) for pediatric subjects with sickle cell anemia and abnormally high (≥200 cm/sec)…
- Trial name
- TWiTCH (NCT01425307)
- Testing
- Hydroxyurea
- Volunteers
- 159 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Fabry Disease, Alpha Galactosidase A Deficiency
Study of the Effects of Fabrazyme Treatment on Lactation and Infants
The study was planned for up to 2 years (24 months). Planned full participation for both mother and infant was 24 months, planned full participation of mother and development of infant was 24 months, while planned full…
- Trial name
- NCT00230607
- Testing
- agalsidase beta
- Where
- 5 sites in United States, Austria and 1 more
- Volunteers
- 7 people
- Sponsor
- Genzyme, a Sanofi Company
Gout, Chronic Kidney Diseases
CSP594 Comparative Effectiveness in Gout: Allopurinol vs. Febuxostat
This trial will compare two effective therapies, allopurinol and febuxostat, to lower serum uric acid and therefore prevent further gout attacks. These therapies have never been compared at appropriate doses.
- Trial name
- NCT02579096
- Testing
- allopurinol capsule, 100-800 mg by mouth once daily, febuxostat tablet 40-120 mg by mouth once daily
- Where
- 23 sites in United States
- Volunteers
- 950 people
- Sponsor
- VA Office of Research and Development
Bronchopulmonary Dysplasia
Safety of Sildenafil in Premature Infants
Describe the safety of sildenafil in premature infants at risk of bronchopulmonary dysplasia and determine preliminary effectiveness and pharmacokinetics (PK) of sildenafil.
- Trial name
- SIL02 (NCT03142568)
- Testing
- Sildenafil
- Where
- 15 sites in United States, Canada
- Volunteers
- 109 people
- Sponsor
- University of North Carolina, Chapel Hill
Friedreich Ataxia
Efficacy, Tolerability, and Pharmacokinetics of Multiple Doses of Oral TAK-831 in Adults With Friedreich Ataxia
The purpose of this study is to evaluate the efficacy of TAK-831 versus placebo on upper extremity (arm and hands) motor function and manual dexterity.
- Trial name
- NCT03214588
- Testing
- TAK-831
- Where
- 6 sites in United States
- Volunteers
- 67 people
- Sponsor
- Neurocrine Biosciences
Cystic Fibrosis
Absorptive Clearance After Inhaled Osmotics in Cystic Fibrosis
Blockage of the breathing tubes of the lungs by thick, sticky mucus is a major cause of lung problems for people with cystic fibrosis (CF).
- Trial name
- NCT01223183
- Testing
- hypertonic saline (7%), isotonic saline
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Pittsburgh
Tourette's Disorder
Safety and Tolerability Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to obtain long term efficacy, safety and tolerability data of once weekly aripiprazole in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01416441
- Testing
- Aripiprazole
- Where
- 80 sites in United States, Bulgaria and 8 more
- Volunteers
- 170 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Arrhythmogenic Right Ventricular Dysplasia, Brugada Syndrome
Distribution of Cell-cell Junction Proteins in Arrhythmic Disorders
Every week in the UK, 12 apparently healthy and fit individuals under the age of 35 die suddenly, a tragic event known as sudden cardiac death (SCD).
- Conditions
- Arrhythmogenic Right Ventricular Dysplasia, Brugada Syndrome, Cardiac Channelopathy
- Trial name
- NCT04257994
- Testing
- obtaining a buccal smear sample
- Where
- United Kingdom
- Volunteers
- 26 people
- Sponsor
- St. George's Hospital, London
Acquired Hemophilia A
Study of Modified Recombinant Factor VIII (OBI-1) in Subjects With Acquired Hemophilia A
This study is to test whether the study drug (OBI-1) is safe and effective for the treatment of serious bleeding episodes in people with acquired hemophilia A.
- Trial name
- NCT01178294
- Testing
- OBI-1
- Where
- 12 sites in United States, Canada and 2 more
- Volunteers
- 29 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis
Rollover Study of Ivacaftor in Subjects With Cystic Fibrosis and a Non G551D CFTR Mutation
The purpose of this study is to evaluate the safety of long-term ivacaftor treatment in participants with cystic fibrosis (CF) from Studies 110 (NCT01614457), 111 (NCT01614470), and 113 (NCT01685801).
- Trial name
- KONTINUE (NCT01707290)
- Testing
- Ivacaftor
- Where
- 38 sites in United States, Belgium and 2 more
- Volunteers
- 125 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Leukemia, Myelodysplastic Syndromes
DT388IL3 Fusion Protein in Treating Patients With Acute Myeloid Leukemia or Myelodysplastic Syndromes
RATIONALE: Combinations of biological substances in DT388IL3 fusion protein may be able to carry cancer killing substances directly to the cancer cells.
- Conditions
- Leukemia, Myelodysplastic Syndromes, Blastic Plasmacytoid Dendritic Cell Neoplasm
- Trial name
- NCT00397579
- Testing
- DT388IL3
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Texas Southwestern Medical Center
Gout
MPC-004 for the Treatment of an Acute Gout Flare
This study is a multicenter, randomized, double-blind, placebo-controlled, parallel group, dose-comparison to determine the efficacy and safety of a standard-dose of colchicine (4.8 mg) versus low-dose colchicine (1.8 mg) or…
- Trial name
- AGREE (NCT00506883)
- Testing
- High Dose Colchicine (4.8 mg total dose), Low Dose Colchicine (1.8mg total dose)
- Where
- 83 sites in United States
- Volunteers
- 185 people, healthy volunteers welcome
- Sponsor
- Takeda