Clinical trials: Newborn
Cystic Fibrosis
Trial of Aeroquin Versus Tobramycin Inhalation Solution (TIS) in Cystic Fibrosis (CF) Patients
Patients with cystic fibrosis (CF) suffer from chronic infections of the lower respiratory tract that can be caused by one or multiple bacteria, including Pseudomonas aeruginosa, which has been particularly problematic to…
- Trial name
- TIS (NCT01270347)
- Testing
- MP-376 (Levofloxacin Solution for Inhalation), TIS (Tobramycin Inhalation Solution)
- Where
- 130 sites in United States, France and 4 more
- Volunteers
- 282 people
- Sponsor
- Amgen
Fetal Alcohol Spectrum Disorders
Efficacy Trial of the FMF Connect Mobile Health Intervention
The purpose of this study is to test a new smartphone "app" for parents/caregivers of children with fetal alcohol spectrum disorder (FASD). The app is called Families Moving Forward (FMF) Connect.
- Trial name
- NCT05028517
- Testing
- FMF Connect, Coaching
- Where
- United States
- Volunteers
- 129 people, healthy volunteers welcome
- Sponsor
- University of Rochester
Cardiac Transplant, Patent Ductus Arterious
The Pharmacology and Hemodynamics of Dexmedetomidine in Children With Congenital Heart Disease
The purpose of this study is to examine the pharmacokinetics, pharmacodynamics, and pharmacogenomics of dexmedetomidine in the following three pediatric patient populations: patients with bi-directional cavopulmonary anastomosis…
- Conditions
- Cardiac Transplant, Patent Ductus Arterious, Atrial Septal Defect, Bidirectional Cavopulmonary Anastomosis
- Trial name
- NCT00480740
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- Children's National Research Institute
Atopic Dermatitis
Tapinarof for the Treatment of Atopic Dermatitis in Children and Adults
This is a double-blind, randomized, vehicle controlled Phase 3 study to evaluate the efficacy and safety of topical tapinarof cream, 1% compared to vehicle control cream in pediatric and adult subjects with atopic dermatitis.
- Trial name
- NCT05014568
- Testing
- tapinarof cream, 1%, Vehicle cream
- Where
- 67 sites in United States, Canada
- Volunteers
- 407 people
- Sponsor
- Organon and Co
Herpes Simplex Virus, Neonatal Sepsis
A Study to Describe the Pharmacokinetics of Acyclovir in Premature Infants (PTN_Acyclo)
Acyclovir is a drug used to treat herpes simplex virus (HSV) infections in babies. Appropriate dosing of acyclovir is known for adults and children but acyclovir has not been adequately studied in full-term or premature neonates.
- Trial name
- NCT00942084
- Testing
- Acyclovir
- Where
- 3 sites in United States
- Volunteers
- 32 people
- Sponsor
- Phillip Brian Smith
Fetal Growth Retardation
Study Aimed At Improving Height With Genotonorm In Children Born Little And/Or Light With Growth Retardation At The Age
To estimate the percentage of children with serum IGF-1 > 2 standard deviation (compared to a child of the same gender and age and without growth hormone (GH) deficiency) 9 months and 12 months after initiation of GH treatment.
- Trial name
- NCT00174252
- Testing
- Genotonorm (Somatropin)
- Where
- 22 sites in France
- Volunteers
- 57 people
- Sponsor
- Pfizer
Hereditary Haemorrhagic Telangiectasia (HHT)
Diet and Hereditary Haemorrhagic Telangiectasia
Hereditary Haemorrhagic Telangiectasia (HHT) affects 1 in 5,000 people. The purpose of this study is to provide data regarding the diet and nosebleed frequency using a questionnaire. This will be filled in by people with HHT.
- Trial name
- NCT01692015
- Testing
- Questionnaire on dietary history, Questionnaire on nosebleed severity, One week food diary generated by weighing foods
- Where
- United Kingdom
- Volunteers
- 50 people
- Sponsor
- Imperial College London
Down Syndrome
Rivastigmine Study in Adolescents With Down Syndrome
The purpose of this study is to determine if short term use of rivastigmine can improve functional abilities (for example, language, memory, and executive function) in adolescents with Down syndrome.
- Trial name
- DS-Riv (NCT01084135)
- Testing
- Rivastigmine
- Where
- 2 sites in United States
- Volunteers
- 42 people
- Sponsor
- Duke University
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
Hunter Syndrome
A Safety and Dose Ranging Study of Idursulfase (Intrathecal) Administration Via an Intrathecal Drug Delivery Device in Pediatric Patients With Hunter Syndrome Who Have Central Nervous System Involvement and Are Receiving Treatment With Elaprase®
Elaprase (idursulfase), a large molecular protein, is not expected to cross the blood brain barrier at therapeutic levels when administered intravenously.
- Trial name
- NCT00920647
- Testing
- Control, Idursulfase IT (1 mg), Idursulfase IT (10 mg)
- Where
- 3 sites in United States, United Kingdom
- Volunteers
- 16 people
- Sponsor
- Shire
Unresectable or Metastatic Microsatellite Stable (MSS) Solid Tumor, Microsatellite Stable (MSS) Colon Cancer
Study of PI3Kinase Inhibition (Copanlisib) and Anti-PD-1 Antibody Nivolumab in Relapsed/Refractory Solid Tumors With Expansions in Mismatch-repair Proficient (MSS) Colorectal Cancer
A phase I/II study of PI3Kinase inhibition (copanlisib) and anti-PD-1 antibody nivolumab in relapsed/refractory solid tumors with expansions in mismatch-repair proficient (MSS) colorectal cancer.
- Trial name
- NCT03711058
- Testing
- Copanlisib, Nivolumab
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Tourette Syndrome
Transcranial Magnetic Stimulation (TMS) for Individuals With Tourette's Syndrome
This study will evaluate the clinical efficacy of 1 Hz repetitive transcranial magnetic stimulation (rTMS) applied to the supplementary motor area (SMA) in Tourette's Syndrome (TS) patients who have not fully responded to…
- Trial name
- TMS (NCT00529308)
- Testing
- Transcranial Magnetic Stimulation (active)
- Where
- 2 sites in United States
- Volunteers
- 20 people
- Sponsor
- Yale University
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Treosulfan, Fludarabine Phosphate, and Total-Body Irradiation Before Donor Stem Cell Transplant in Treating Patients With High-Risk Acute Myeloid Leukemia, Myelodysplastic Syndrome, Acute Lymphoblastic Leukemia
This phase II trial is studying how well giving treosulfan together with fludarabine phosphate and total-body irradiation followed by donor stem cell transplant works in treating patients with high-risk acute myeloid leukemia…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission, Adult Acute Myeloid Leukemia in Remission, Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities
- Trial name
- NCT00860574
- Testing
- treosulfan, fludarabine phosphate, total-body irradiation
- Where
- 3 sites in United States
- Volunteers
- 96 people
- Sponsor
- Fred Hutchinson Cancer Center
Prader-Willi Syndrome
A Study of Oral ARD-101 in Patients With Prader-Willi Syndrome
A Phase 2, Single-Arm, Open-Label Study to Evaluate the Safety and Efficacy of ARD-101 in Patients with Prader-Willi Syndrome
- Trial name
- NCT05153434
- Testing
- ARD-101
- Where
- 2 sites in United States
- Volunteers
- 19 people
- Sponsor
- Aardvark Therapeutics, Inc.
Hunter Syndrome
Study of Intrathecal Idursulfase-IT Administered in Conjunction With Elaprase® in Pediatric Patients With Hunter Syndrome and Early Cognitive Impairment
Study HGT-HIT-094 is a multicenter study designed to determine the effect on clinical parameters of neurodevelopmental status of monthly IT administration of idursulfase-IT 10 mg for 12 months in pediatric patients with Hunter…
- Trial name
- AIM-IT (NCT02055118)
- Testing
- idursulfase-IT, No IT treatment
- Where
- 9 sites in United States, Australia and 5 more
- Volunteers
- 58 people
- Sponsor
- Shire
Hemochromatosis, Cerebrovascular Accident
Stroke With Transfusions Changing to Hydroxyurea
The purpose of this study is to compare standard therapy (transfusions and chelation) with alternative therapy (hydroxyurea and phlebotomy) for the prevention of secondary stroke and management of iron overload in children with…
- Conditions
- Hemochromatosis, Cerebrovascular Accident, Anemia, Sickle Cell, Hematologic Diseases
- Trial name
- NCT00122980
- Testing
- Red Cell Transfusions, Iron Chelation, Hydroxyurea
- Where
- 28 sites in United States
- Volunteers
- 134 people
- Sponsor
- St. Jude Children's Research Hospital
X-linked Hypophosphatemia
An Extension Study of KRN23 in Adults With X-Linked Hypophosphatemia
The primary purpose of this study is to assess the safety and efficacy of repeated subcutaneous (SC) injections of KRN23 in adult subjects with X-Linked Hypophosphatemia (XLH).
- Trial name
- NCT01571596
- Testing
- KRN23
- Where
- 6 sites in United States, Canada
- Volunteers
- 23 people
- Sponsor
- Kyowa Kirin Co., Ltd.
Infantile Colic, Infantile Functional Gastrointestinal Disorders
A Phase I Study to Evaluate the Oral Absorption of Nepadutant in Infants
The purpose of the study is to evaluate the gastrointestinal absorption of nepadutant after single dose as oral solution (and the effect of age on its oral absorption) in infants.
- Trial name
- NCT00655083
- Testing
- Nepadutant
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Menarini Group
Muscular Dystrophies
First Time in Human (FTIH) Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of Single and Repeat Doses of GSK3439171A in Healthy Subjects and to Assess Food Effect
The FTIH study with GSK3439171A will evaluate the safety of GSK3439171A in healthy subjects in order to avoid confounding factors due to the disease or concomitant drugs in patients.
- Trial name
- NCT03627494
- Testing
- GSK3439171A
- Where
- United States
- Volunteers
- 66 people, healthy volunteers welcome
- Sponsor
- GlaxoSmithKline
Pulmonary Hypertension, Sickle Cell Disease
Macitentan in Pulmonary Hypertension of Sickle Cell Disease
This is a pilot study to assess the safety and efficacy of macitentan in patients with pulmonary hypertension of sickle cell disease. This study will enroll approximately 10 subjects.
- Trial name
- MENSCH (NCT02651272)
- Testing
- macitentan
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Boston University
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