Clinical trials: Fetal Developmental Abnormality
Primary Immunodeficiency Diseases (PID)
A Study of Immune Globulin Subcutaneous (Human), 20% Solution (IGSC, 20%) in Japanese Participants With Primary Immunodeficiency Diseases (PID)
In this study, Japanese participants with primary immunodeficiency diseases were treated with Immune Globulin Subcutaneous (Human), 20% solution, (IGSC, 20%).
- Trial name
- NCT04346108
- Testing
- Immune Globulin Intravenous (IGIV), Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%)
- Where
- 8 sites in Japan
- Volunteers
- 17 people
- Sponsor
- Baxalta now part of Shire
Cystic Fibrosis
Study to Validate the Instructions for Use of TOBI® Podhaler™ in Cystic Fibrosis Patients
The purpose of this actual use human factors (HF) study is to validate the approved US TOBI Podhaler Instructions for Use (IFU), by establishing that the IFU effectively communicates the information necessary to achieve safe and…
- Trial name
- NCT02178540
- Testing
- Tobi Podhaler
- Where
- 13 sites in United States
- Volunteers
- 45 people
- Sponsor
- Novartis Pharmaceuticals
Small Fiber Neuropathy, Fabry Disease
Assessment of Small Fiber Neuropathy in Rare Diseases Using Sudoscan
Sudoscan™ (Impeto Medical, Paris France) uses electrochemical skin conductance as a novel noninvasive method to detect sudomotor dysfunction.
- Conditions
- Small Fiber Neuropathy, Fabry Disease, Ehlers Danlos Syndrome, Mitochondrial Disease
- Trial name
- NCT02985710
- Testing
- Sudoscan, Skin biopsy, QSART
- Where
- United States
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- Massachusetts General Hospital
Pectus Excavatum, Funnel Chest
Cryoanalgesia vs. Epidural in the Nuss Procedure
The purpose of this study is to determine whether cryoanalgesia provides better pain control for minimally invasive pectus excavatum repair (the Nuss procedure) than thoracic epidural.
- Trial name
- NCT02721017
- Testing
- Cryoanalgesia, Thoracic epidural (ropivicaine, fentanyl)
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of California, San Francisco
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Lymphoblastic Leukemia in Remission
Haploidentical Donor Bone Marrow Transplant in Treating Patients With High-Risk Hematologic Cancer
This phase II trial studies how well giving fludarabine phosphate, cyclophosphamide, tacrolimus, mycophenolate mofetil and total-body irradiation together with a donor bone marrow transplant works in treating patients with…
- 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
- NCT00049504
- Testing
- cyclophosphamide, fludarabine phosphate, tacrolimus
- Where
- United States
- Volunteers
- 53 people
- Sponsor
- Fred Hutchinson Cancer Center
Sickle Cell Disease
High Dose Vitamin D for Sickle Cell Disease
Vitamin D deficiency (VDD) is very common among African American adolescents and adults in the US, ten times higher than is seen in Caucasians. VDD is also quite common in sickle cell disease (SCD).
- Trial name
- SCD-VitD (NCT01331148)
- Testing
- Vitamin D
- Where
- United States
- Volunteers
- 46 people
- Sponsor
- Wake Forest University Health Sciences
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
Dermatitis, Atopic
A Study of BMS-986166 or Branebrutinib for the Treatment of Participants With Atopic Dermatitis
The purpose of this study is to evaluate the efficacy, safety, and tolerability of BMS-986166 and of branebrutinib, each versus placebo, for the treatment of participants with moderate to severe atopic dermatitis.
- Trial name
- NCT05014438
- Testing
- BMS-986166, Branebrutinib
- Where
- 42 sites in United States, Australia and 5 more
- Volunteers
- 17 people
- Sponsor
- Bristol-Myers Squibb
Polycystic Ovary Syndrome
Study of Pharmacodynamics of LIK066 in Overweight and Obese Women With Polycystic Ovary Syndrome
The purpose of the study was to evaluate whether LIK066 can be developed for the treatment of polycystic ovary syndrome (PCOS) in overweight and obese women.
- Trial name
- PCOS (NCT03152591)
- Testing
- LIK066
- Where
- 5 sites in United States, Germany
- Volunteers
- 29 people
- Sponsor
- Novartis Pharmaceuticals
X-Linked Hypophosphatemia
Study of the Safety, Pharmacodynamics (PD) and Efficacy of KRN23 in Children From 1 to 4 Years Old With X-linked Hypophosphatemia (XLH)
The primary objectives of the study are to: Establish the safety profile of KRN23 for the treatment of XLH in children between 1 and 4 years old Determine the PD effects of KRN23 treatment on serum phosphorus and other PD markers…
- Trial name
- NCT02750618
- Testing
- Burosumab
- Where
- 3 sites in United States
- Volunteers
- 13 people
- Sponsor
- Kyowa Kirin, Inc.
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
The first 1,020 results can be paged through. Add a filter or change the sort order to reach the rest.