Clinical trials: Newborn
Cystic Fibrosis, Pulmonary
Study of ARO-ENaC in Healthy Volunteers and in Patients With Cystic Fibrosis
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics (PK) of single doses of ARO-ENaC in healthy adult volunteers; and to evaluate the safety, tolerability, PK and efficacy of multiple doses of…
- Trial name
- NCT04375514
- Testing
- ARO-ENaC
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 43 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
Severe Hemophilia A
Pharmacokinetics and Safety of rFVIIIFc Manufactured at 15,000 L (15K) Scale
The primary objective of the study is to compare the pharmacokinetic (PK) of recombinant coagulation factor VIII Fc fusion protein (rFVIIIFc) manufactured at the current scale of 2000 L (2K) to the PK of rFVIIIFc manufactured at…
- Trial name
- Elevate (NCT02502149)
- Testing
- rFVIIIFc
- Where
- 15 sites in United States, Australia and 1 more
- Volunteers
- 24 people
- Sponsor
- Bioverativ Therapeutics Inc.
Severe Hemophilia A
Study to Evaluate the Safety, Pharmacokinetics and Efficacy of Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in Previously Treated Subjects With Severe Hemophilia A
The primary objectives of this study are: to evaluate the safety and tolerability of rFVIIIFc administered as a prophylaxis (Arm 1), weekly (Arm 2), on-demand (Arm 3), and surgical treatment regimen; to evaluate the efficacy of…
- Trial name
- NCT01181128
- Testing
- Factor VIII (rFVIIIFc), Advate®
- Where
- 64 sites in United States, Australia and 17 more
- Volunteers
- 165 people
- Sponsor
- Bioverativ Therapeutics Inc.
Phenylketonuria
Long-Term Extension of Previous rAvPAL-PEG Protocols in Subjects With PKU (PAL-003)
This study is an extension of previous rAvPAL-PEG studies. Administration of rAvPAL-PEG will be continued to assess whether long-term dosing of rAvPAL-PEG is safe and can maintain reduced blood Phe concentrations in PKU subjects.
- Trial name
- NCT00924703
- Testing
- rAvPAL-PEG
- Where
- 14 sites in United States
- Volunteers
- 68 people
- Sponsor
- BioMarin Pharmaceutical
Primary Immunodeficiency
Efficacy, Pharmacokinetics, Safety, and Tolerability of IGSC 20% in Subjects With Primary Immunodeficiency
Approximately 60 subjects will be enrolled in order to have approximately 20 adult subjects and 20 pediatric subjects treated with subcutaneously administered Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography…
- Trial name
- NCT02806986
- Testing
- IGSC 20%
- Where
- 23 sites in Australia, Czechia and 7 more
- Volunteers
- 61 people
- Sponsor
- Grifols Therapeutics LLC
HIV, Pregnancy
Safety and Pharmacokinetics of Dolutegravir in Pregnant HIV Mothers and Their Neonates: A Pilot Study
Aim: To evaluate dolutegravir (DTG) pharmacokinetics in pregnant HIV-infected women Rationale: In developing countries many women present with a new HIV diagnosis in late pregnancy, and are at high risk of transmitting infection…
- Trial name
- DolPHIN1 (NCT02245022)
- Testing
- Dolutegravir 50mg od, Standard of Care
- Where
- 2 sites in South Africa, Uganda
- Volunteers
- 60 people
- Sponsor
- University of Liverpool
Fabry Disease
This Study is Designed to Evaluate PD/PK and Safety of Replagal Manufactured by Two Different Processes.
This study is designed to evaluate safety and PK/PD in Canadian Fabry patients.
- Trial name
- NCT01304277
- Testing
- agalsidase alfa
- Where
- 5 sites in Canada
- Volunteers
- 17 people
- Sponsor
- Shire
Pulmonary Arterial Hypertension
Clinical Study to Evaluate the Effects of Macitentan on Exercise Capacity in Subjects With Eisenmenger Syndrome
Clinical study to assess the efficacy, safety, and tolerability of macitentan in subjects with Eisenmenger Syndrome.
- Trial name
- MAESTRO (NCT01743001)
- Testing
- Macitentan 10 mg
- Where
- 55 sites in United States, Austria and 19 more
- Volunteers
- 226 people
- Sponsor
- Actelion
Congenital Heart Disease
HEARTPrep: A Virtually-delivered Psychosocial Intervention for Mothers Expecting a Baby With Congenital Heart Disease
Congenital heart disease (CHD) is the most common birth defect and is increasingly diagnosed prenatally. Mothers describe their prenatal experiences as highly stressful and traumatic after a diagnosis of CHD.
- Trial name
- HEARTPrep (NCT05129631)
- Testing
- HEARTPrep
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Nemours Children's Clinic
Fetal and Neonatal Alloimmune Thrombocytopenia
Phase 2 Study on the Pharmacokinetics and Safety of RLYB212 in Pregnant Women at Higher Risk for HPA-1a Alloimmunization
The purpose of this Phase 2 study is to assess the pharmacokinetics (PK) and safety of RLYB212 in HPA-1b/b pregnant women at higher risk for HPA-1a alloimmunization and FNAIT.
- Trial name
- NCT06435845
- Testing
- Anti-(integrin beta-3) human monoclonal antibody
- Where
- 3 sites in Netherlands, Norway and 1 more
- Volunteers
- 1 people
- Sponsor
- Rallybio
Atopic Dermatitis
Vaccine Responses in Tralokinumab-Treated Atopic Dermatitis - ECZTRA 5 (ECZema TRAlokinumab Trial No. 5)
The purpose of this trial is to test if treatment with the trial drug, tralokinumab, can affect the body's immune response to vaccines.
- Trial name
- ECZTRA 5 (NCT03562377)
- Testing
- Tralokinumab, Tdap vaccine, Meningococcal vaccine
- Where
- 51 sites in United States, Canada
- Volunteers
- 215 people
- Sponsor
- LEO Pharma
Congenital Adrenal Hyperplasia, 21-OHD
A Phase 2 Study to Evaluate the Safety, Efficacy and PK of Tildacerfont in Children Aged 2-17 Years With CAH
An investigation of the safety and efficacy of tildacerfont in participants with CAH.
- Trial name
- NCT05128942
- Testing
- Tildacerfont
- Where
- 13 sites in United States
- Volunteers
- 67 people
- Sponsor
- Spruce Biosciences
Atopic Dermatitis
Comparison of the Efficacy and Safety of Two Topical Creams for Pediatric Atopic Dermatitis
This study compares the effectiveness of two topical creams for atopic dermatitis in pediatric subjects. Subjects will be randomly assigned to use one of the two creams twice daily for 6 weeks or until clear.
- Trial name
- NCT00828412
- Testing
- EpiCeram Skin Barrier Emulsion, Desonide Cream 0.05%
- Where
- 5 sites in United States
- Volunteers
- 100 people
- Sponsor
- Promius Pharma, LLC
Cystic Fibrosis
Rollover Study of VX-770 in Cystic Fibrosis Subjects
The primary objective of the study was to evaluate the safety of long-term VX-770 treatment in participants with cystic fibrosis (CF).
- Trial name
- NCT01117012
- Testing
- Ivacaftor
- Where
- 58 sites in United States, Australia and 6 more
- Volunteers
- 192 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Huntington's Disease
A Phase 2, to Evaluating the Safety and Efficacy of Pridopidine Vs Placebo for Symptomatic Treatment in Patients With Huntington's Disease
This is a multicenter, multinational, randomized, parallel-group, double-blind, placebo-controlled, dose range finding study to compare the efficacy and safety of different doses of pridopidine versus placebo in the treatment of…
- Trial name
- NCT02006472
- Testing
- Pridopidine
- Where
- 58 sites in United States, Australia and 10 more
- Volunteers
- 408 people
- Sponsor
- Prilenia
Infant,Premature, Feeding; Difficult, Newborn
Nifty Feeding Cup Versus Generic Medicine Cup Preterm Infants Who Have Difficulty Breastfeeding
The investigators will establish an evidence base for the Nifty Feeding Cup by evaluating its effectiveness and caregiver satisfaction.
- Trial name
- NCT03280381
- Testing
- Nifty feeding cup, Generic medicine cup
- Where
- Ghana
- Volunteers
- 200 people
- Sponsor
- PATH
Kidney, Polycystic, Autosomal Dominant
High Water Intake to Slow Progression of Polycystic Kidney Disease
Polycystic kidney disease (PKD) is a genetic disease that occurs in 1 in 500 individuals and leads to kidney failure in half of all affected. Currently, no treatments exist for PKD.
- Trial name
- NCT00784030
- Testing
- Water
- Where
- United States
- Volunteers
- 23 people, healthy volunteers welcome
- Sponsor
- NYU Langone Health
Neonatal Brachial Plexus Palsy
NMES Efficacy on Patients With NBPP
Neonatal Brachial Plexus Palsy (NBPP), characterized by weakness and sensory loss in the affected arm, results from damage in the prenatal period to the nerves extending from the spine to the arm.
- Trial name
- NCT01999465
- Testing
- Standard NMES device
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- University of Michigan
Endothelial Dysfunction, Hyperhomocysteinemia
Effect of L-Arginine and Pycnogenol on Light to Moderate Hypertension and Endothelial Function
The effect of a combination product (Verum ) with L-arginine, Pycnogenol, vitamin K2, R-(+)-alpha-lipoic acid and vitamins B6, B12 and folic acid is investigated in a double blind placebo-controlled cross-over study.
- Conditions
- Endothelial Dysfunction, Hyperhomocysteinemia, Hypertension Grade I, Subgroup "Borderline" (WHO)
- Trial name
- NCT02392767
- Testing
- Verum
- Where
- Germany
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Dr. Loges & Co. GmbH
Family Relationship, Stress
Impact of Increased Parent Presence in the Neonatal Intensive Care Unit on Parent & Infant Outcomes
The purpose of this pilot study is to compare parent and infant outcomes and unit outcomes pre and post a planned unit-wide intervention aimed at increasing parent presence in the Neonatal Intensive Care Unit (NICU).
- Conditions
- Family Relationship, Stress, Breastfeeding, Bottle Feeding
- Trial name
- NCT02901665
- Testing
- FCC intervention
- Where
- 2 sites in United States
- Volunteers
- 191 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
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