Clinical trials: Newborn
Duchenne Muscular Dystrophy
Study to Assess the Safety, Tolerability, and Efficacy of Viltolarsen in Ambulant and Non-Ambulant Boys With DMD (Galactic53)
This is a phase II, open-label study where weekly doses of 80 mg/kg viltolarsen is administered intravenously over a 48-week treatment period to ambulant and non-ambulant DMD patients over the age of 8 years.
- Trial name
- NCT04956289
- Testing
- Viltolarsen
- Where
- 8 sites in United States, China and 4 more
- Volunteers
- 20 people
- Sponsor
- NS Pharma, Inc.
Congenital Hemangioma, Hemangioendothelioma
A Study to Evaluate How Safe and Effective is the Mixture of Lipiodol® Ultra Fluid and Glue When Used for Embolization Procedures
This was a multicenter phase IV Post-Marketing Study. The study is designed to investigate the safety of Lipiodol® Ultra Fluid in association with surgical glues used according to each site medical practice of vascular…
- Conditions
- Congenital Hemangioma, Hemangioendothelioma, Angiosarcoma, Arteriovenous Malformations
- Trial name
- NCT02625389
- Testing
- Lipiodol Ultra Fluid with surgical glues
- Where
- 16 sites in India
- Volunteers
- 132 people
- Sponsor
- Guerbet
Hyperoxaluria
Hydroxyproline Influence on Oxalate Metabolism
Primary hyperoxaluria is an inborn error of metabolism that results in marked overproduction of oxalate by the liver. The excess oxalate causes kidney failure and can cause severe systemic disease due to oxalate deposition in…
- Trial name
- NCT02038543
- Testing
- Hydroxyproline and Leucine
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Mayo Clinic
Mucopolysaccharidosis I, Hurlers Syndrome
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
- Conditions
- Mucopolysaccharidosis I, Hurlers Syndrome, Hurler-Scheie Syndrome
- Trial name
- NCT00912925
- Testing
- rhIDU (recombinant human-Alpha-L-Iduronidase)
- Where
- 4 sites in United States, Canada and 1 more
- Volunteers
- 45 people
- Sponsor
- Genzyme, a Sanofi Company
Intubated Infants Were Intend to Extubation Using Noninvasive Ventilation Strategies
A Trial Comparing Noninvasive Ventilation Strategies in Preterm Infants Following Extubation
Respiratory distress syndrome (RDS) is the main cause of respiratory failure in preterm neonates, its incidence varying from 80% to 25% depending on gestational age.When optimal prenatal care is provided, the best approach to…
- Trial name
- NCT03181958
- Testing
- NHFOV, NCPAP, NIPPV
- Where
- China
- Volunteers
- 1,493 people
- Sponsor
- Daping Hospital and the Research Institute of Surgery of the Third Military Medical University
Duchenne Muscular Dystrophy
A Phase IIa Study of TAS-205 for Duchenne Muscular Dystrophy
The objective of this study is to evaluate the efficacy after 24-week repeated oral doses of TAS-205 in patients with Duchenne Muscular Dystrophy (DMD) in an exploratory manner.
- Trial name
- NCT02752048
- Testing
- TAS-205
- Where
- 11 sites in Japan
- Volunteers
- 36 people
- Sponsor
- Taiho Pharmaceutical Co., Ltd.
Pompe Disease
First-In-Human Study to Evaluate Safety, Tolerability, and PK of Intravenous ATB200 Alone and When Co-Administered With Oral AT2221
This is an international, multi-center, open-label study designed to evaluate if the co-administration of investigational new drugs ATB200 and AT2221 is safe in adults with Pompe disease.
- Trial name
- NCT02675465
- Testing
- ATB200, AT2221
- Where
- 19 sites in United States, Australia and 4 more
- Volunteers
- 29 people
- Sponsor
- Amicus Therapeutics
NF1, Neurofibromatosis
Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and Adults
This study, "A Phase II Study of Cabozantinib (XL l84) for Plexiform Neurofibromas in Subjects with Neurofibromatosis Type I in Children and Adults diagnosed with Neurofibromatosis Type 1 (NF1) and have a type of tumor called a…
- Conditions
- NF1, Neurofibromatosis, Plexiform Neurofibromas
- Trial name
- NF105-CABO (NCT02101736)
- Testing
- Cabozantinib
- Where
- 12 sites in United States
- Volunteers
- 45 people
- Sponsor
- University of Alabama at Birmingham
Congenital Adrenal Hyperplasia
A Ph2b to Evaluate Clinical Efficacy and Safety of Tildacerfont in Adult CAH
An investigation of the efficacy and safety of up to 70 weeks of treatment with Tildacerfont in subjects with classic CAH who have elevated biomarkers at baseline on their current GC regimen.
- Trial name
- NCT04457336
- Where
- 65 sites in United States, Australia and 18 more
- Volunteers
- 96 people
- Sponsor
- Spruce Biosciences
Chronic Pancreatitis
Alpha-1 Antitrypsin (AAT) Enhances Islet Autograft Survival in Patients With Chronic Pancreatitis
Primary objective: To describe and compare the safety and efficacy of treatment with AAT in chronic pancreatitis patients who undergo total pancreatectomy and islet autotransplantation (TP-IAT).
- Trial name
- NCT02947087
- Testing
- Prolastin-C
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Medical University of South Carolina
Pectus Excavatum
Magnetic Mini-Mover Procedure to Treat Pectus Excavatum
This is a medical research study. The study investigators have developed a method to gradually repair pectus excavatum (sunken chest) deformity by placing a magnet on the sternum (breastbone) and then applying an external…
- Trial name
- 3MP (NCT00466206)
- Testing
- Magnetic Mini-Mover Procedure (3MP), Magnatract (external magnet in a removable external brace), 3MP (Magnetic Mini-Mover Procedure)
- Where
- United States
- Volunteers
- 10 people, healthy volunteers welcome
- Sponsor
- University of California, San Francisco
Herpes Simplex
Acyclovir Herpes Simplex Virus (HSV) Skin, Eye, and Mouth
The purpose of this study is to test whether long-term treatment with oral acyclovir improves the outcome for infants with herpes simplex virus (HSV) disease of the skin, eyes, and mouth (SEM).
- Trial name
- NCT00031447
- Testing
- Acyclovir
- Where
- 28 sites in United States, Canada
- Volunteers
- 29 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Inherited Retinal Dystrophy Due to RPE65 Mutations, Leber Congenital Amaurosis
Safety and Efficacy Study in Subjects With Leber Congenital Amaurosis
The study is a Phase 3, open-label, randomized controlled trial of gene therapy intervention by subretinal administration of AAV2-hRPE65v2 (voretigene neparvovec-rzyl).
- Trial name
- NCT00999609
- Testing
- AAV2-hRPE65v2,voretigene neparvovec-rzyl
- Where
- 2 sites in United States
- Volunteers
- 31 people
- Sponsor
- Spark Therapeutics, Inc.
Primary Hyperoxaluria Type 1 (PH1)
A Study to Evaluate Lumasiran in Children and Adults With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the efficacy and safety of lumasiran in children and adults with primary hyperoxaluria type 1 (PH1).
- Trial name
- ILLUMINATE-A (NCT03681184)
- Testing
- Lumasiran
- Where
- 17 sites in United States, France and 6 more
- Volunteers
- 39 people
- Sponsor
- Alnylam Pharmaceuticals
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)
Cleft Lip, Scarring
Botulinum Toxin to Improve Cosmesis of Primary Cleft Lip Repair
Objective: To determine if the use of botulinum toxin during primary cleft lip repair improves the cosmetic appearance of the cleft lip scar
- Conditions
- Cleft Lip, Scarring, Cleft Palate
- Trial name
- NCT02247193
- Testing
- Botulinum Toxin Type A, Normal Saline Injection
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Alport Syndrome, Kidney Disease
A Prospective Study of Microalbuminuria in Untreated Boys With Alport Syndrome
The goal of the Microalbuminuria in Untreated Boys with Alport Syndrome study is to gather information about critical clinical time points such as when patients with small amounts of protein (microalbuminuria) in their urine…
- Trial name
- MA (NCT00622544)
- Where
- United States
- Volunteers
- 44 people
- Sponsor
- University of Minnesota
Renal Cell Carcinoma (RCC), Urothelial Carcinoma
A Study of DSP-7888 Dosing Emulsion in Combination With Immune Checkpoint Inhibitors in Adult Patients With Advanced Solid Tumors
This is a Phase 1b/2, open-label, multicenter study of DSP-7888 Dosing Emulsion in combination with checkpoint inhibitors (nivolumab or pembrolizumab) in adult patients with solid tumors, that consists of 2 parts: dose search…
- Conditions
- Renal Cell Carcinoma (RCC), Urothelial Carcinoma, Primary Peritoneal Cancer, Platinum-resistant Ovarian Cancer (PROC)
- Trial name
- NCT03311334
- Testing
- DSP-7888 Dosing Emulsion, Nivolumab, Pembrolizumab
- Where
- 19 sites in United States, Canada
- Volunteers
- 47 people
- Sponsor
- Sumitomo Pharma America, Inc.
Atopic Dermatitis
An Open-Label Study Investigating the Effects of Early Skin Barrier Protection on the Development of Atopic Dermatitis
The purpose of this study is to determine whether early use of a bland emollient in newborns, prior to the clinical signs of skin disease, will delay the onset or prevent the development of atopic dermatitis.
- Trial name
- NCT00806221
- Testing
- emollient (Cetaphil cream)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Oregon Health and Science University
Chronic Myeloproliferative Disorders, Fatigue
American Ginseng in Treating Patients With Fatigue Caused by Cancer
RATIONALE: American ginseng may reduce fatigue in patients with cancer. It is not yet known whether American ginseng is more effective than a placebo in treating cancer-related fatigue.
- Conditions
- Chronic Myeloproliferative Disorders, Fatigue, Leukemia, Lymphoma
- Trial name
- NCT00719563
- Testing
- American ginseng
- Where
- 326 sites in United States
- Volunteers
- 364 people
- Sponsor
- Alliance for Clinical Trials in Oncology
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