Clinical trials: Effect Neonatal
Severe Combined Immunodeficiency
MND-ADA Transduction of CD34+ Cells From Children With ADA-SCID
Severe combined immune deficiency (SCID) may result from inherited deficiency of the enzyme adenosine deaminase (ADA). Children with ADA-deficient SCID often die from infections in infancy, unless treated with either a bone…
- Trial name
- NCT00794508
- Testing
- ADA gene transfer
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Donald B. Kohn, M.D.
Atopic Dermatitis
Dupilumab Skin Barrier Function Study in Atopic Dermatitis
Primary Objective: Evaluate changes in skin barrier function with transepidermal water loss (TEWL) assessed after skin tape stripping (STS) in pre-defined lesional skin in participants with moderate to severe atopic dermatitis…
- Trial name
- BALISTAD (NCT04447417)
- Testing
- Dupilumab SAR231893
- Where
- 2 sites in United States, Canada
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Sanofi
Marfan Syndrome
Effects of Losartan Versus Atenolol on Aortic and Cardiac Muscle Stiffness in Adults With Marfan Syndrome
Marfan syndrome is an inherited connective tissue disorder with morbidity and mortality from aortic dilation and dissection. The degree of aortic dilation and response to beta-blockade (standard of care) vary in adults with…
- Trial name
- NCT00723801
- Testing
- Atenolol, Losartan
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Brigham and Women's Hospital
Cystic Fibrosis
Effect of Glycine in Cystic Fibrosis
The aim of this study is to evaluate if glycine, orally administered in a daily dose of 0.5 g/kg during 8 weeks, can ameliorate the airway inflammation in children with cystic fibrosis, as compared with placebo.
- Trial name
- NCT01417481
- Testing
- Glycine
- Where
- 3 sites in Mexico
- Volunteers
- 13 people
- Sponsor
- Instituto Nacional de Enfermedades Respiratorias
Cystinuria
Lipoic Acid Supplement for Cystine Stone
This study evaluates how daily alpha lipoic acid supplementation affects cystine kidney stone recurrence. Half of the subjects will receive 1200 mg alpha lipoic acid orally daily for three years, while the other half will receive…
- Trial name
- ALA (NCT02910531)
- Testing
- Alpha lipoic acid
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Thomas Chi, MD
Bronchopulmonary Dysplasia
Effect of Synchronized vs. Continuous HFNC Using NAVA on WOB in Infants With BPD
Patients will be randomized to begin the study with either NAVA-synchronized or continuous HFNC. Each patient will receive two 15-minute trials at different levels of continuous HFNC and two 15-minute trials at corresponding…
- Trial name
- NCT04274192
- Testing
- Synchronized HFNC at 6 liters per minute (LPM), Continuous HFNC at 6 liters per minute (LPM), Synchronized HFNC at 8 liters per minute (LPM)
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- University of Arkansas
Cystic Fibrosis
Pilot Study Testing the Effect of Ivacaftor on Lung Function in Subjects With Cystic Fibrosis and Residual CFTR Function
This study is a multiple within participant crossover study to evaluate the effect of ivacaftor on lung function in participants aged 12 years and older with cystic fibrosis (CF) who have phenotypic or molecular evidence of…
- Trial name
- NCT01685801
- Testing
- Ivacaftor
- Where
- United States
- Volunteers
- 24 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Umbilical Hernia
Analgesic Efficacy After Umbilical Hernia Repair in Children
Umbilical hernia repair is a common painful outpatient procedure performed in children. Often analgesia for this procedure is provided by using local infiltration of the surgical site by the surgeons and perioperative opioids and…
- Trial name
- NCT00578136
- Testing
- Bupivacaine
- Where
- United States
- Volunteers
- 52 people
- Sponsor
- Children's Hospital of Philadelphia
Lactose Intolerance
HYBRID: Hydrogen Breath Test in Lactose Digestion
The purpose of this study is to investigate the effect of a fermented infant formula on lactose digestion in lactose intolerant adults.
- Trial name
- HYBRID (NCT02703987)
- Testing
- Fermented infant milk formula, Non-fermented infant milk formula
- Where
- Singapore
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Danone Asia Pacific Holdings Pte, Ltd.
Phenylketonurias
Open-label, Randomized, 2-way Crossover, Monocentric, Controlled Study to Evaluate the Effect on Daily PHE Fluctuation of PKU GOLIKE Versus SoC in Patients With PKU.
This is an open-label, randomized, 2-way crossover, monocentric controlled study in patients (≥ 16 years old) with phenylketonuria (PKU).
- Trial name
- NCT05827536
- Testing
- PKU GOLIKE, Standard of Care
- Where
- Italy
- Volunteers
- 21 people
- Sponsor
- APR Applied Pharma Research s.a.
Cystic Fibrosis Gastrointestinal Disease
Cystic Fibrosis and Gut Dysmotility: The Effect of Polyethylene Glycol (PEG) on Intestinal Transit
The investigators will recruit 15 patients with cystic fibrosis 18 years of age and older who present with constipation. The investigators will assess baseline motility symptoms with a survey.
- Trial name
- NCT04210427
- Testing
- Polyethylene Glycol 3350, SmartPill Motility System & PillCam Patency Capsule
- Where
- 2 sites in United States
- Volunteers
- 3 people
- Sponsor
- St. Louis University
Leukemia
Prophylactic White Cell Transfusions Versus Therapeutic White Cell Transfusions in Patients With Leukemia
Patients with leukemia often have low white blood cell counts after chemotherapy, which puts them at greater risk for infection.
- Trial name
- NCT01204788
- Testing
- Prophylactic White Cell Transfusion, Therapeutic White Cell Transfusion
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- M.D. Anderson Cancer Center
Cystic Fibrosis
Effect of Andecaliximab on FEV1 in Adults With Cystic Fibrosis
The primary objective of this study is to evaluate the effect of andecaliximab (GS-5745) on pre-bronchodilator forced expiratory volume in 1 second (FEV1) % predicted in adults with cystic fibrosis (CF) after 8 weeks of…
- Trial name
- NCT02759562
- Testing
- Andecaliximab
- Where
- 5 sites in Australia, France and 3 more
- Volunteers
- 6 people
- Sponsor
- Gilead Sciences
Hypospadias
Effect of Steroids on Post-Operative Complications Following Proximal Hypospadias Repair
The primary objective of this study is to determine the efficacy of administering a course of postoperative oral steroids in pediatric patients undergoing proximal hypospadias repair as prevention against complications.
- Trial name
- NCT02162810
- Testing
- Prednisolone
- Where
- United States
- Volunteers
- 28 people, healthy volunteers welcome
- Sponsor
- Francis Schneck
Coarctation of the Aorta
Statins and Endothelial Function in Patients With Coarctation of the Aorta
Coarctation of the aorta (CoA) accounts for approximately 8% of all congenital heart problems. Patients with CoA are well known to have an increased rates of early heart disease, high blood pressure, heart failure and stroke.
- Trial name
- NCT00767572
- Testing
- atorvastatin, Sugar pill
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of California, San Francisco
Dyslipidemias, High Cholesterol
Randomized Study to Evaluate the Effect of Obicetrapib on Top of Maximum Tolerated Lipid-Modifying Therapies
This study will be a placebo-controlled, double-blind, randomized, phase 3 study in participants with underlying heterozygous familial hypercholesterolemia (HeFH) and/or ASCVD to evaluate the efficacy, safety, and tolerability of…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROADWAY (NCT05142722)
- Testing
- Obicetrapib
- Where
- 181 sites in United States, China and 6 more
- Volunteers
- 2,530 people
- Sponsor
- NewAmsterdam Pharma
Ototoxicity
SPI-1005 for Prevention and Treatment of Tobramycin Induced Ototoxicity
The primary objective of this study is to determine the safety and efficacy of SPI-1005 treatment in CF patients with active pulmonary exacerbation that are receiving an IV course of tobramycin, determined by comparing hearing…
- Trial name
- NCT02819856
- Testing
- SPI-1005 Ebselen 200mg Capsule x1, SPI-1005 Ebselen 200mg Capsule x2, SPI-1005 Ebselen 200mg Capsule x3
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Sound Pharmaceuticals, Incorporated
Elevated LDL Cholesterol
A Pharmacodynamic Study to Evaluate the Effect of a Fixed Dose Combination Pill on Low Density Lipoprotein (LDL) Cholesterol
This study evaluates the effect on LDL cholesterol of the 3 drugs given together in the cardiovascular fixed dose combination pill (acetylsalicylic acid, simvastatin, and ramipril) as compared to the effect on LDL cholesterol of…
- Trial name
- NCT01004705
- Testing
- Cardiovascular fixed dose combination pill (acetylsalicylic acid, simvastatin and ramipril),, Simvastatin
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Ferrer Internacional S.A.
Atopic Dermatitis
A Study Investigating the Effect of EDP1815 in the Treatment of Mild, Moderate and Severe Atopic Dermatitis
The purpose of this research study is to determine whether the study drug, EDP1815, is safe and effective in the treatment of atopic dermatitis compared with placebo.
- Trial name
- NCT05121480
- Testing
- EDP1815
- Where
- 59 sites in United States, Australia and 4 more
- Volunteers
- 421 people
- Sponsor
- Evelo Biosciences, Inc.
Cystinosis, Nephropathic Cystinosis
Study in Healthy Adults to Determine the Effect That Food Has on the Absorption and Delivery of the Drug Cystagon™
In order to meet FDA standards of safety and efficacy reporting for most new drugs, food-effect bioavailability (the impact that the presence of food in the digestive tract has on the rate and extent at which a drug is absorbed…
- Trial name
- NCT01432561
- Testing
- Cysteamine bitartrate
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of California, San Diego