Clinical trials: NEWBORN
Alpha 1-Antitrypsin Deficiency
Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)
The purpose of this study is to evaluate the effects of weekly augmentation therapy with ARALAST Fraction IV-1 (Fr IV-1) on epithelial lining fluid (ELF) alpha 1-proteinase inhibitor levels and other ELF analytes and to assess…
- Trial name
- NCT00396006
- Testing
- Alpha1-Proteinase Inhibitor
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 21 people
- Sponsor
- Baxalta now part of Shire
Aniridia, Iris; Deformity
Clinical Evaluation of Morcher Artificial Iris Diaphragms
The purpose of this study is to determine the safety and efficacy of Morcher iris diaphragms in the treatment of congenital and acquired aniridia.
- Trial name
- NCT00812708
- Testing
- Morcher iris diaphragm implantation
- Where
- United States
- Volunteers
- 72 people
- Sponsor
- University of California, Los Angeles
Hemolytic Disease of the Fetus and Newborn
A Study to Evaluate the Safety, Efficacy, Pharmacokinetics and Pharmacodynamics of M281 Administered to Pregnant Women at High Risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (HDFN)
The purpose of this study is to evaluate the safety in mother and neonate/infant of M281 administered to pregnant women who are at high risk for Early Onset Severe Hemolytic Disease of the Fetus and Newborn (EOS-HDFN).
- Trial name
- NCT03842189
- Testing
- M281
- Where
- 19 sites in United States, Australia and 7 more
- Volunteers
- 25 people
- Sponsor
- Janssen Research & Development, LLC
Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner
Using Topical Sirolimus 2% for Patients With Epidermolysis Bullous Simplex (EBS) Study
Epidermolysis bullosa (EB) simplex is a rare orphan disease caused by a mutation in DNA leading to abnormal dominant keratins in the skin.
- Conditions
- Epidermolysis Bullosa Simplex, Epidermolysis Bullosa Simplex Kobner, Weber-Cockayne Syndrome
- Trial name
- NCT02960997
- Testing
- Sirolimus, 2%, Vehicle
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Stanford University
Hyperlipoproteinemia Type II, Hypercholesterolemia, Familial
Study to Assess the Tolerability and Efficacy of Anacetrapib (MK-0859) Co-Administered With Statin in Participants With Heterozygous Familial Hypercholesterolemia (MK-0859-020)
The objective of this study is to evaluate the efficacy and tolerability of adding anacetrapib to ongoing statin therapy in participants with heterozygous familial hypercholesterolemia (HeFH).
- Trial name
- REALIZE (NCT01524289)
- Testing
- Anacetrapib
- Volunteers
- 306 people
- Sponsor
- Merck Sharp & Dohme LLC
Congenital Hypothyroidism, Hypothyroidism
Generic vs. Name-Brand Levothyroxine
This study compares two different brands of thyroxine (thyroid hormone). Currently, pharmacists may be substituting generic formulations of thyroid hormone without your doctor knowing about this.
- Trial name
- NCT00403390
- Testing
- Brand Name Levothyroxine (Synthroid), Generic formulation of Levothyroxine
- Where
- United States
- Volunteers
- 34 people, healthy volunteers welcome
- Sponsor
- Boston Children's Hospital
Huntington Disease
Effect of PBT2 in Patients With Early to Mid Stage Huntington Disease
Huntington disease (HD) is an inherited neurodegenerative disease which affects over 30,000 people in both the United States and Australia.
- Trial name
- Reach2HD (NCT01590888)
- Testing
- PBT2
- Where
- 19 sites in United States, Australia
- Volunteers
- 109 people
- Sponsor
- Prana Biotechnology Limited
Cystic Fibrosis, Mucociliary Clearance Defect
Researching the Effects of Airway Clearance Therapies in Cystic Fibrosis
This is a pilot study investigating the effectiveness and clinical efficacy of airway clearance therapy (ACT) in cystic fibrosis (CF).
- Trial name
- REACT-CF (NCT03078127)
- Testing
- Baseline (Huff-Cough alone), Oscillatory Positive Expiatory Pressure Device, Whole-Body Vibration Platform
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- University of North Carolina, Chapel Hill
Sickle Cell Anemia
A Phase II Trial of Regadenoson in Sickle Cell Anemia
This research study is a Phase II clinical trial, which tests the safety and effectiveness of an investigational drug called Regadenoson (or Lexiscan) to learn whether the drug works in treating a specific disease, in this case…
- Trial name
- NCT01788631
- Testing
- Regadenoson
- Where
- 12 sites in United States
- Volunteers
- 100 people
- Sponsor
- Dana-Farber Cancer Institute
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
Hemophilia A
Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)
To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To…
- Trial name
- NCT01736475
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method, PEGylated Recombinant Factor VIII
- Where
- 72 sites in United States, Australia and 18 more
- Volunteers
- 159 people
- Sponsor
- Baxalta now part of Shire
Dysautonomia, Familial, Baroreflex Failure Syndrome
Carbidopa for the Treatment of Excessive Blood Pressure Variability
The overall study objectives are to determine whether carbidopa (Lodosyn®) is safe and well tolerated and to assess whether it can inhibit catecholamine-induced paroxysmal hypertension and normalize or reduce the exaggerated…
- Trial name
- CarbiFD (NCT02553265)
- Testing
- Carbidopa Low-Dose, Carbidopa High-Dose
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- NYU Langone Health
Gout
Long-Term Safety of Febuxostat in Subjects With Gout.
The purpose of this study is to evaluate the long-term safety of febuxostat, once daily (QD), in maintaining serum urate levels within clinically acceptable levels in subjects with gout.
- Trial name
- FOCUS (NCT00174941)
- Testing
- Febuxostat
- Volunteers
- 116 people
- Sponsor
- Takeda
Sickle Cell Disease (SCD)
An Indian Multi-centric Phase IV Study to Assess the Safety of Crizanlizumab in Sickle Cell Disease Patients
Sickle cell disease (SCD) is a genetic blood disorder. Crizanlizumab is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD aged 16 years and older.
- Trial name
- NCT04662931
- Testing
- crizanlizumab
- Where
- 7 sites in India
- Volunteers
- 140 people
- Sponsor
- Novartis Pharmaceuticals
Infant Apnea, Neonatal Abstinence Syndrome
Interventions to Help Infants Recover in the Hospital
This pilot project will evaluate independently two non-pharmacological interventions, 1) Neurosensory, Environmental Adaptive Technology (NEATCAP) and 2) Stochastic Vibrotactile Stimulation (SVS), as adjuvant non-pharmacological…
- Conditions
- Infant Apnea, Neonatal Abstinence Syndrome, Sleep
- Trial name
- NCT03881553
- Testing
- NEATCAP, SVS mattress
- Where
- United States
- Volunteers
- 17 people
- Sponsor
- Elisabeth Salisbury, PhD
Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease
Octreotide in Severe Polycystic Liver Disease
This study will evaluate the effect of Octreotide LAR® on the liver volumes of patients with severe polycystic liver disease who are not candidates or decline surgical treatments such as liver cyst fenestration, liver resection…
- Conditions
- Polycystic Kidney, Autosomal Dominant, Polycystic Liver Disease, Hepatomegaly, Liver Diseases
- Trial name
- NCT00426153
- Testing
- Octreotide
- Where
- United States
- Volunteers
- 42 people
- Sponsor
- Mayo Clinic
Pectus Excavatum
Post Operative Pain Control Using Ropivacaine and the ON-Q System in the Adult Pectus Excavatum Patient
Pectus excavatum (PE) is a common chest wall deformity where the sternum is displaced posteriorly. In severe cases, surgery is performed to correct the defect.
- Trial name
- NCT01816477
- Testing
- ON-Q soaker catheter system, Epidural (ropivicaine 0.25%)
- Where
- United States
- Volunteers
- 85 people
- Sponsor
- Mayo Clinic
Neurofibromatosis 2, Meningioma
AZD2014 In NF2 Patients With Progressive or Symptomatic Meningiomas
The goal of this clinical research study is to learn if the study drug AZD2014 can shrink growing or symptomatic meningiomas.
- Trial name
- NCT02831257
- Testing
- AZD2014
- Where
- 2 sites in United States
- Volunteers
- 18 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Telangiectasia, Hereditary Hemorrhagic, Epistaxis
North American Study of Epistaxis in Hereditary Hemorrhagic Telangiectasia (HHT)
The purpose of the NOSE Study is to carefully examine the efficacy and safety of 3 nasal sprays (bevacizumab, estriol, and tranexamic acid), compared to placebo, for the treatment of HHT related nosebleeds.
- Trial name
- NOSE (NCT01408030)
- Testing
- Sterile saline, Bevacizumab, Estriol
- Where
- 6 sites in United States
- Volunteers
- 123 people
- Sponsor
- James Gossage
Rett Syndrome, RTT
A Long-term Safety Study of Cannabidiol Oral Solution (GWP42003-P, CBD-OS) in Patients With Rett Syndrome
This study will be conducted to evaluate the long-term safety of cannabidiol oral solution (GWP42003-P, CBD-OS) in participants with Rett syndrome.
- Trial name
- NCT04252586
- Testing
- GWP42003-P
- Where
- 31 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- Jazz Pharmaceuticals
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