Clinical trials: Newborns
Cystic Fibrosis
Study of Ataluren (PTC124™) in Cystic Fibrosis
Cystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
- Trial name
- NCT00803205
- Testing
- Ataluren
- Where
- 38 sites in United States, Belgium and 9 more
- Volunteers
- 238 people
- Sponsor
- PTC Therapeutics
Duchenne Muscular Dystrophy
Clinical Trial to Evaluate the Efficacy, Safety, and Tolerability of RO7239361 in Ambulatory Boys With Duchenne Muscular Dystrophy
This is a multi-center, randomized, double-blind, placebo-controlled study to assess the efficacy, safety and tolerability of two different weekly doses of RO7239361 in ambulatory boys with Duchenne Muscular Dystrophy (DMD).
- Trial name
- NCT03039686
- Testing
- RO7239361
- Where
- 44 sites in United States, Argentina and 11 more
- Volunteers
- 166 people
- Sponsor
- Hoffmann-La Roche
Fabry Disease
Extension Study of PRX-102 for up to 60 Months
To evaluate the ongoing safety, tolerability, and efficacy parameters of PRX-102 in adult Fabry patients who have successfully completed treatment with PRX-102 in studies PB-102-F01 and PB-102-F02.
- Trial name
- NCT01981720
- Testing
- PRX-102 (pegunigalsidase alfa)
- Where
- 8 sites in United States, Paraguay and 2 more
- Volunteers
- 15 people
- Sponsor
- Protalix
Postoperative Pain
Pain Management for Pectus Excavatum Repair
The objective of this study is to scientifically evaluate two different management strategies for post-operative pain after pectus excavatum repair.
- Trial name
- NCT00413582
- Testing
- Epidural Analgesia, Patient-Controlled IV Analgesia
- Where
- United States
- Volunteers
- 110 people
- Sponsor
- Children's Mercy Hospital Kansas City
Dermatitis, Atopic
An Ascending Multiple Dose Study of VTP-38543 in Adult Participants With Mild to Moderate Atopic Dermatitis
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical efficacy of VTP-38543 administered as a cream, twice-daily, for 28 days in otherwise healthy adult…
- Trial name
- NCT02655679
- Testing
- VTP-38543, Vehicle with Transcutol®P, Vehicle without Transcutol®P
- Where
- 12 sites in United States, Canada
- Volunteers
- 104 people
- Sponsor
- Vitae Pharmaceuticals, Inc.
Neonatal Anemia
Red Blood Cell (RBC) Survival Following Transfusion in Infants
OUR OVERALL HYPOTHESIS is that post-transfusion survival of allogeneic and autologous RBCs can be accurately quantified in anemic human infants using biotin-labeled RBCs combined with mathematical modeling that adjusts for…
- Trial name
- NCT00731588
- Testing
- Transfused Biotin RBCs - Adults Phase I, Transfused Biotin RBCs - Infants Phase II, Transfused Biotin RBCs - Infants Phase III
- Where
- 2 sites in United States
- Volunteers
- 140 people
- Sponsor
- John A Widness
Neonatal Abstinence Syndrome
Methadone Demonstration Project With Neonatal Intensive Care Unit Infants Diagnosed With Neonatal Abstinence Syndrome
This is a non-randomized, un-blinded feasibility study project comparing the Length of Stay (LOS) of Neonatal Intensive Care Unit (NICU) infants diagnosed with Neonatal Abstinence Syndrome (NAS) treated with methadone with…
- Trial name
- NCT03134703
- Testing
- Methadone, Morphine
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- Johns Hopkins All Children's Hospital
Hypercholesterolemia, Familial Hypercholesterolemia
A Study of Enlicitide Decanoate (MK-0616 Oral PCSK9 Inhibitor) in Adults With Heterozygous Familial Hypercholesterolemia (MK-0616-017/CORALreef HeFH)
The goal of this study is to evaluate the efficacy, safety, and tolerability of enlicitide decanoate in adult participants with heterozygous familial hypercholesterolemia.
- Trial name
- NCT05952869
- Testing
- Enlicitide Decanoate
- Where
- 59 sites in United States, Australia and 15 more
- Volunteers
- 303 people
- Sponsor
- Merck Sharp & Dohme LLC
Sickle Cell Disease
A Study Aimed to Assess the Needs of Subjects With Sickle Cell Disease and Healthcare Professionals Managing Sickle Cell Disease Patients in Selected Nigerian Centres
This pilot research is aimed to assess the needs of patients and health workers involved in Sickle Cell Disease (SCD) management in Nigeria.
- Trial name
- NCT04505969
- Testing
- Questionnaire to assess needs
- Where
- 4 sites in Nigeria
- Volunteers
- 192 people
- Sponsor
- Fondazione per la Ricerca Farmacologica Gianni Benzi Onlus
Foramen Ovale, Patent, Echocardiography
Long-term Outcomes After Percutaneous Closure of PFO
International medical centre Medicor from Slovenia has reported 363 successful percutaneous closures of patent foramen ovale (PFO) from October 2006 till October 2023.
- Conditions
- Foramen Ovale, Patent, Echocardiography, Embolic Stroke, Atrial Fibrillation
- Trial name
- NCT05558774
- Where
- Slovenia
- Volunteers
- 364 people
- Sponsor
- MC Medicor
Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency
Comparison of Two Forms of Hydrocortisone in Patients With Congenital Adrenal Hyperplasia
This study will test a new, extended release form of hydrocortisone called Chronocort in patients with congenital adrenal hyperplasia (CAH).
- Conditions
- Congenital Adrenal Hyperplasia, 21-Hydroxylase Deficiency, Adrenogenital Syndrome
- Trial name
- NCT00519818
- Testing
- Chronocort, Cortef
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Neurocrine UK Limited
Neurofibromatosis Type 1
A Randomized Placebo-Controlled Study of Lovastatin in Children With Neurofibromatosis Type 1
The specific aim of this study is to determine whether Lovastatin ™ significantly improves visual spatial learning and/or sustained attention in children with NF1.
- Trial name
- STARS (NCT00853580)
- Testing
- Lovastatin ™
- Where
- 12 sites in United States, Australia
- Volunteers
- 146 people, healthy volunteers welcome
- Sponsor
- University of Alabama at Birmingham
Hemophilia A
Clinical Study to Investigate the Pharmacokinetics, Efficacy, Safety and Immunogenicity of a Recombinant FVIII in Patients With Severe Hemophilia A
This is a clinical study to investigate the pharmacokinetics, efficacy, safety and immunogenicity of human-cl rhFVIII, a newly developed human cell-line derived recombinant FVIII concentrate in previously treated patients with…
- Trial name
- NCT00989196
- Testing
- Human-cl rhFVIII, Kogenate FS
- Where
- 9 sites in United States, Bulgaria and 1 more
- Volunteers
- 22 people
- Sponsor
- Octapharma
Antenatal Care
Group Antenatal Care and Delivery Project
Antenatal care (ANC) has the potential to play a pivotal role in ensuring positive pregnancy outcomes for both mothers and their newborns.
- Trial name
- GRAND (NCT04033003)
- Testing
- Group ANC
- Where
- 14 sites in Ghana
- Volunteers
- 1,761 people
- Sponsor
- University of Michigan
Mucopolysaccharidosis I, Mucopolysaccharidosis II
Clinical Trial of Growth Hormone in MPS I, II, and VI
The purpose of this study is to determine whether growth hormone is a safe and effective treatment for short stature in children with Mucopolysaccharidosis type I, II, and VI.
- Conditions
- Mucopolysaccharidosis I, Mucopolysaccharidosis II, Mucopolysaccharidosis VI
- Trial name
- NCT00748969
- Testing
- Somatropin (DNA origin)
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
Dravet Syndrome (DS), Lennox-Gastaut Syndrome (LGS)
A Study Evaluating Soticlestat in Participants With Dravet Syndrome or Lennox-Gastaut Syndrome Who Have Been Exposed to Fenfluramine
The purpose of this study is to check how soticlestat impacts symptoms of Dravet syndrome [DS] and Lennox-Gastaut syndrome [LGS] in participants who have been exposed to fenfluramine.
- Trial name
- NCT06422377
- Testing
- Soticlestat
- Where
- Denmark
- Volunteers
- 1 people
- Sponsor
- Takeda
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
Enterocolitis, Necrotizing, Premature Infant
NEC Screening Abdominal Radiograph vs Bowel Ultrasound in Preemies
The overall primary objective is to establish the feasibility and pilot the design and delivery of a diagnostic randomized controlled trial (RCT) of BUS (bowel ultrasound) for NEC evaluation which will lead to a successful…
- Trial name
- NCT03963011
- Testing
- Bowel Ultrasound
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Mercy Hospital Kansas City
Neurofibromatoses
Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)
GENERAL OBJECTIVE The general objective is to assess the safety and efficacy of photodynamic therapy (PDT) in the treatment of neurofibromatosis 1 (NF1) tumors in the skin.
- Trial name
- NCT01682811
- Testing
- Part 1 Levulan injection, Part 1 Levulan surface application, Part 1 Levulan surface application twice
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Harry T Whelan, MD
Osteogenesis Imperfecta
Pamidronate to Treat Osteogenesis Imperfecta in Children
This study will evaluate the effect of pamidronate a drug that decreases bone resorption (breakdown) on osteogenesis imperfecta. This is a genetic disorder of collagen, the major protein in bone.
- Trial name
- NCT00005901
- Testing
- Pamidronate (Aredia)
- Where
- United States
- Volunteers
- 34 people
- Sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
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