Clinical trials: Neonates
Gaucher Disease
A Phase III Trial to Assess the Safety and Efficacy of Plant Cell Expressed GCD in Patients With Gaucher Disease
Gaucher disease, the most prevalent lysosomal storage disorder, is caused by mutations in the human glucocerebrosidase gene (GCD) leading to reduced activity of the lysosomal enzyme glucocerebrosidase and thereby to the…
- Trial name
- NCT00376168
- Testing
- Plant cell expressed recombinant glucocerebrosidase (prGCD)
- Where
- 11 sites in United States, Canada and 6 more
- Volunteers
- 32 people
- Sponsor
- Pfizer
Muscular Dystrophy, Duchenne
Rimeporide in Patients With Duchenne Muscular Dystrophy
In Duchenne Muscular Dystrophy (DMD) there is an imbalance between the levels of calcium and sodium in the muscles cells which is thought to be important in the damage which occurs overtime.
- Trial name
- RIM4DMD (NCT02710591)
- Testing
- Rimeporide
- Where
- 4 sites in France, Italy and 2 more
- Volunteers
- 20 people
- Sponsor
- EspeRare Foundation
Flat Feet, High Arched Foot
Validation of Plantar Orthoses for Abnormal Plantar Arch Using a New Non-invasive Clinical Imaging System
The goal of this project is to validate a new non-invasive clinical imaging system to evaluate the efficacy of plantar orthotics and to assess the biomechanical efficiency of plantar orthotics for people with flat or high arch…
- Trial name
- NCT05163418
- Testing
- Foot orthosis for flat feet, Foot orthoses for high arches feet
- Where
- Canada
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- TOPMED
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
Gaucher Disease
A Safety and Efficacy Study of Two Dose Levels of Taliglucerase Alfa in Pediatric Subjects With Gaucher Disease
This is a multi-center, double-blind trial to assess the safety and efficacy of taliglucerase alfa in untreated subjects (2 to <18 years old) with Gaucher disease randomly assigned to treatment with one of two doses, 30 or 60…
- Trial name
- NCT01132690
- Testing
- Taliglucerase alfa
- Where
- 3 sites in Israel, Paraguay and 1 more
- Volunteers
- 11 people
- Sponsor
- Pfizer
Atopic Dermatitis
A Study of Baricitinib (LY3009104) in Children and Adolescents With Atopic Dermatitis
The reason for this study is to see if the study drug called baricitinib works and is safe in children and teenage participants with atopic dermatitis.
- Trial name
- BREEZE-AD-PEDS (NCT03952559)
- Testing
- Baricitinib, Topical corticosteroid
- Where
- 78 sites in Argentina, Australia and 15 more
- Volunteers
- 516 people
- Sponsor
- Eli Lilly and Company
Anesthesia; Adverse Effect, Congenital Heart Disease
The Effect of Anesthesia on Neurodevelopmental Outcome (NDO)
The purpose of this study is to assess whether the type of anesthesia, narcotic-based versus inhalational anesthesia administered during cardiopulmonary bypass (CPB) surgery contributes to the wide variation in neurologic…
- Conditions
- Anesthesia; Adverse Effect, Congenital Heart Disease, Neurodevelopmental Disorders
- Trial name
- NDO (NCT03882788)
- Testing
- Isoflurane, Fentanyl (high dose), Fentanyl (low dose)
- Where
- United States
- Volunteers
- 153 people
- Sponsor
- Stanford University
Hunter Syndrome
Collection and Study of Cerebrospinal Fluid in Patients With Hunter Syndrome
The purpose of the study is to collect data on CSF biomarkers in patients with Hunter Syndrome that would serve as reference data for comparison with cognitively impaired patients with Hunter syndrome, patients with other…
- Trial name
- NCT01449240
- Testing
- No treatment
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 10 people
- Sponsor
- Shire
Cystinosis
Cysteamine Eye Drops to Treat Corneal Crystals in Cystinosis
Cystinosis is an inherited disease that results in poor growth and kidney disease, among other things. The damage to the kidneys and other organs is thought to be due to accumulation of cystine inside the cells of various body…
- Trial name
- NCT00001213
- Testing
- Cysteamine
- Where
- United States
- Volunteers
- 328 people
- Sponsor
- National Eye Institute (NEI)
Leukemia, Lymphoma
Donor Stem Cell Transplant in Treating Patients With Relapsed Hematologic Malignancies or Secondary Myelodysplasia Previously Treated With High-Dose Chemotherapy and Autologous Stem Cell Transplant
RATIONALE: Giving chemotherapy, such as busulfan and fludarabine phosphate, before a peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Leukemia, Lymphoma, Lymphoproliferative Disorder, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT01118013
- Testing
- anti-thymocyte globulin, donor lymphocytes, filgrastim
- Where
- 10 sites in United States
- Volunteers
- 6 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Hereditary Angioedema (HAE)
A Double-Blind, Multiple Ascending Dose Study to Assess Safety, Tolerability and Pharmacokinetics of DX-2930 in Hereditary Angioedema Participants
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) profile of multiple subcutaneous administrations of DX-2930 across a range of doses in HAE participants.
- Trial name
- NCT02093923
- Testing
- DX-2930
- Where
- 14 sites in United States, Italy and 1 more
- Volunteers
- 38 people
- Sponsor
- Shire
Fabry Disease
A 12-Week Safety and Pharmacodynamic Study of AT1001 (Migalastat Hydrochloride) in Participants With Fabry Disease
Study to evaluate the safety, tolerability, and pharmacodynamics of migalastat hydrochloride (HCl) (migalastat) and how migalastat works in participants with Fabry disease.
- Trial name
- NCT00283959
- Testing
- migalastat HCl
- Where
- 2 sites in Australia, Brazil
- Volunteers
- 4 people
- Sponsor
- Amicus Therapeutics
Tourettes Syndrome, Tic
N-acetylcysteine (NAC) for Children With Tourette Syndrome
Tourette syndrome is a childhood-onset neuropsychiatric disorder characterized by multiple motor and vocal tics that last for at least a year in duration.
- Trial name
- NCT01172288
- Testing
- N-Acetylcysteine (NAC)
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Yale University
Hypoxic Ischemic Encephalopathy
Topiramate in Neonates Receiving Whole Body Cooling for Hypoxic Ischemic Encephalopathy
The goal is to see whether topiramate (an anti-epileptic agent) improves the outcome of babies with neonatal hypoxic encephalopathy who are receiving whole body cooling.
- Trial name
- NCT01765218
- Testing
- Topiramate
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Kristin R Hoffman
HHT, Hereditary Hemorrhagic Telangiectasia
Intranasal Bevacizumab for HHT-Related Epistaxis
This is a randomized, controlled, double-blind, placebo-controlled trial of intranasal Avastin (bevacizumab) injection versus saline control for control of HHT-related epistaxis when used in conjunction with bipolar…
- Conditions
- HHT, Hereditary Hemorrhagic Telangiectasia, Epistaxis, Nose Bleeds
- Trial name
- NCT02389959
- Testing
- Bevacizumab
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Stanford University
Mevalonate Kinase Deficiency
Canakinumab in Patients With Active Hyper-IgD Syndrome
This pilot study is designed to evaluate the efficacy, the safety, and the pharmacokinetics (PK) / pharmacodynamics (PD) of canakinumab treatment in patients with HIDS.
- Trial name
- NCT01303380
- Testing
- Canakinumab
- Where
- 3 sites in Spain
- Volunteers
- 9 people
- Sponsor
- Novartis Pharmaceuticals
Cystic Fibrosis
EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients
This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.
- Trial name
- NCT02443688
- Testing
- CTX-4430
- Where
- 75 sites in United States, Belgium and 5 more
- Volunteers
- 200 people
- Sponsor
- Celtaxsys, Inc.
Flatfoot
Comparison of Two Different Insole Types in Painful Flexible Flatfoot
The purpose of this study is to compare computer aided design/computer aided manufacturing (CAD/CAM) and semi-custom insole types on pain, quality of life and physical performance and also to decide whether they are necessary in…
- Trial name
- NCT02706327
- Testing
- CAD/CAM Insole, Semi-custom Insole, Control
- Volunteers
- 67 people
- Sponsor
- Eastern Mediterranean University
Haemophilia A
Evaluation of Safety Following Immune Tolerance Induction Treatment With Turoctocog Alfa in Patients With Haemophilia A Following Inhibitor Development in NN7170-4213 Trial
This trial is conducted in Asia, Europe and the United States of America (USA). The aim of the trial is to evaluate safety of immune tolerance induction (ITI) treatment with turoctocog alfa (a recombinant factor VIII) in patients…
- Trial name
- NCT03588741
- Testing
- Turoctocog alfa
- Where
- 14 sites in Austria, Bulgaria and 5 more
- Volunteers
- 1 people
- Sponsor
- Novo Nordisk A/S
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