Clinical trials: Newborn Diseases
Phenylketonurias, Non Alcoholic Fatty Liver
Walking Program in Fatty Liver Children With Phenylketonuria
phenylketonuria (commonly known as PKU) is an inherited disorder that increases the levels of a substance called phenylalanine in the blood.
- Trial name
- NCT06718842
- Testing
- walking free online supervised program
- Where
- Egypt
- Volunteers
- 40 people
- Sponsor
- Cairo University
Heart Failure, Cardiac Imaging Techniques
MRI to Predict Rejection and Failure in Transplant and Cardiomyopathy Patients
Overall Research Strategy of this protocol is to refine and validate noninvasive CMR imaging sequences with invasive hemodynamic and pathology results to create a comprehensive, noninvasive, radiation-reduced regimen for…
- Conditions
- Heart Failure, Cardiac Imaging Techniques, Congenital Heart Defects
- Trial name
- NCT03822442
- Where
- United States
- Volunteers
- 113 people
- Sponsor
- Children's National Research Institute
Leukemia
Gemcitabine and Mitoxantrone in Treating Patients With Relapsed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as gemcitabine and mitoxantrone, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00268242
- Testing
- Gemcitabine Hydrochloride, Mitoxantrone Hydrochloride
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- The Cleveland Clinic
Ornithine Transcarbamylase Deficiency
Investigation of Brain Nitrogen in Partial Ornithine Transcarbamylase Deficiency (OTCD) Using 1 H MRS, DTI, and fMRI
The purpose of this study is to use various types of MRI and cognitive testing to evaluate changes in the brain and cognitive function that occur in subjects with ornithine transcarbamylase deficiency (OTCD) relative to healthy…
- Trial name
- NCT01569568
- Testing
- MRI scanning, Cognitive testing
- Volunteers
- 49 people, healthy volunteers welcome
- Sponsor
- Andrea Gropman
Infantile Colic
Evaluation of Safety and Efficacy of New Infant Formula in Infantile Colic
The study is conducted to examine the safety and efficacy of a new infant formula and its effects on the gastrointestinal tolerance in infants suffering from colic.
- Trial name
- Coco (NCT01721850)
- Testing
- control formula, intervention formula 1, intervention formula 2
- Where
- 18 sites in Germany, Italy
- Volunteers
- 169 people, healthy volunteers welcome
- Sponsor
- HiPP GmbH & Co. Vertrieb KG
Sickle Cell Anemia
Haploidentical PBMC Transplant for Severe Congenital Anemias
Background: Bone marrow transplantation (BMT), which involves transplanting a donor's marrow stem cells, is capable of curing some congenital anemias.
- Trial name
- NCT00977691
- Testing
- PBSC Transplant, Alemtuzumab, Sirolimus
- Where
- United States
- Volunteers
- 23 people
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Recruitment
Chinese Regional Spinal Muscular Atrophy Patient Registration Study
The primary objectives of this study are to obtain clinically meaningful data on the survival, outcomes, prognosis and treatment effect of all the patients with spinal muscular atrophy (SMA) 5q types 1 to 3 (according to…
- Trial name
- NCT05631418
- Where
- China
- Sponsor
- The Children's Hospital of Zhejiang University School of Medicine
Sickle Cell Disease
A Phase - IIa - IIb, Trial to Study the Safety, Tolerability and Efficacy of Memantine as a Long-term Treatment of SCD
Symptomatic sickle cell disease (SCD) is worldwide the most frequent cause for hereditary hemolytic anemia with recurrent pain crises.
- Trial name
- MeMAGEN (NCT03247218)
- Testing
- Memantine Hydrochloride
- Where
- Israel
- Volunteers
- 40 people
- Sponsor
- HaEmek Medical Center, Israel
Vivax Malaria, G6PD Deficiency
Primaquine for Vivax Malaria in G6PD Intermediate and Deficient Cases.
A non randomized observation study that is aiming to assess the safety and efficacy of high dose primaquine (1mg/kg per day over 7 days) among patients with intermediate (30-70%) G6PD activity and the safety and efficacy of…
- Trial name
- NCT07468513
- Testing
- Patients with G6PD enzyme activities between 30 and <70% of the AMM will be treated with schizontocidal treatment plus high dose primaquine 1mg/kg/day for 7 days, Patients with G6PD enzyme activities <30% of the AMM will be treated with schizontocidal treatment plus 8 weekly primaquine (0.75mg/kg dose).
- Where
- 4 sites in Brazil, Ethiopia and 1 more
- Volunteers
- 100 people
- Sponsor
- Menzies School of Health Research
Dermatitis, Atopic
Comparison Study Between Protopic (Tacrolimus Ointment) and Elidel (Pimecrolimus Cream) in Treating Pediatric Patients With Atopic Dermatitis
Comparison of Protopic Ointment to Elidel Cream to assess the safety and efficacy in pediatric patients with Atopic Dermatitis
- Trial name
- NCT00666159
- Testing
- tacrolimus ointment, pimecrolimus cream
- Where
- 12 sites in United States
- Volunteers
- 226 people
- Sponsor
- Astellas Pharma Inc
Prader-Willi Syndrome
Targeting the Gut Microbiome for Prader-Willi Syndrome Treatment
The gut microbiome has recently emerged as a major contributor to obesity, systemic inflammation, and metabolic disease. Furthermore, intestinal bacteria are crucial players in the gut-brain axis, regulating a broad range of…
- Trial name
- BALPWS (NCT03548480)
- Testing
- Probiotic
- Where
- Spain
- Volunteers
- 39 people
- Sponsor
- Fundació Sant Joan de Déu
Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies
Single Ascending Dose Study in Participants With LCA10
The purpose of this study is to evaluate the safety, tolerability and efficacy of a single escalating doses of EDIT-101 administered via subretinal injection in participants with LCA10 caused by a homozygous or compound…
- Conditions
- Leber Congenital Amaurosis 10, Inherited Retinal Dystrophies, Eye Diseases, Hereditary, Retinal Disease
- Trial name
- NCT03872479
- Testing
- EDIT-101
- Where
- 5 sites in United States
- Volunteers
- 34 people
- Sponsor
- Editas Medicine, Inc.
Locally Advanced Rectal Cancer (LARC), MSS (Microsatellite Stable)
SCRT Based iTNT vs. LCRT Based TNT for MSS Locally Advanced Rectal Cancer
In this prospective, multicentre, randomized phase III trial, 612 locally advanced rectal cancer (LARC, T3-4/N+M0) patients with at least one high-risk features (lower location (≤5cm), cT4, cN2, MRF+, EMVI+, TD+) will be…
- Trial name
- TORCH-STAR (NCT07551479)
- Testing
- short-course radiaotherapy, long-course chemoradiaothearpy, Capecitabine
- Where
- China
- Volunteers
- 612 people
- Sponsor
- Fudan University
Fetal Alcohol Syndrome, Attention Deficit Hyperactivity Disorder (ADHD)
Open-Label Study of the Long Term Tolerability and Safety of Atomoxetine in Children With FASD and ADD/ADHD
Determine if atomoxetine is safe and well tolerated by children with fetal alcohol syndrome.
- Trial name
- B4Z-US-050 (NCT00418262)
- Testing
- Atomoxetine
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Oklahoma
Limb Girdle Muscular Dystrophies
Motor Parameters in Patients With Limb Girdle Muscular Dystrophy
The primary objective of the study is to perform 2-year follow up with motor parameters evolution using instrumental assessments in patients with limb girdle muscular dystrophie, and to identify which motor parameters are…
- Trial name
- EIDY (NCT04772027)
- Where
- France
- Volunteers
- 80 people, healthy volunteers welcome
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Primary Immune Deficiency
Study to Evaluate the Pharmacokinetics, Efficacy, Tolerability, and Safety of Subcutaneous Human Immunoglobulin (Newnorm) in Patients With Primary Immunodeficiency Diseases
Prospective, open-label, single-arm, multicentre Phase 3 study to evaluate the pharmacokinetics, efficacy, tolerability, and safety of subcutaneous human immunoglobulin (Newnorm) in patients with primary immunodeficiency diseases
- Trial name
- NCT04640142
- Testing
- Newnorm
- Where
- 23 sites in United States, Germany and 5 more
- Volunteers
- 50 people
- Sponsor
- Octapharma
Spina Bifida, Myelomeningocele
tSCS in Children With Spina Bifida
A single-center, open-label, investigational pilot trial to explore potential effects of transcutaneous spinal cord stimulation on leg muscle strength and walking in children with myelomeningocele.
- Trial name
- NCT07615686
- Testing
- Transcutaneous stimulator
- Where
- United States
- Volunteers
- 20 people
- Sponsor
- Bailey Petersen
Cystic Fibrosis (CF), Pulmonary Exacerbation
Pharmacokinetics of Antibiotics in Patients With Cystic Fibrosis Trated With Elexacaftor/Tezacaftor/Ivacaftor (ETI)
Cystic fibrosis (CF) is associated with major pharmacokinetic and pharmacodynamic alterations affecting antibiotic exposure, including changes in absorption, distribution, metabolism, and elimination.
- Conditions
- Cystic Fibrosis (CF), Pulmonary Exacerbation, Respiratory Infection Bacterial
- Trial name
- PKCF (NCT07629986)
- Testing
- No intervention assigned
- Where
- France
- Volunteers
- 30 people
- Sponsor
- Fondation Ildys
Genetic Disease
Developing Protocols for Modelling of Genetic Diseases Using Induced Pluripotent Stem Cells
Recent advances have shown that cells from human blood, skin and urine samples can be reprogrammed to become stem cells. These are called induced Pluripotent Stem Cells (iPSCs) and share many characteristics with embryonic stem…
- Trial name
- NCT03612310
- Testing
- Skin biopsy/Urine Collection/Blood Sample Collection
- Where
- United Kingdom
- Volunteers
- 3,000 people
- Sponsor
- Sapna Vyas
Pruritus, Prurigo Nodularis
Study of the Long Term Safety of Serlopitant for the Treatment of Pruritus (Itch)
Study of the long term safety of serlopitant for the treatment of pruritus in adults.
- Conditions
- Pruritus, Prurigo Nodularis, Atopic Dermatitis, Psoriasis
- Trial name
- NCT03540160
- Testing
- 5 mg Serlopitant Tablets
- Where
- 85 sites in United States, Austria and 2 more
- Volunteers
- 558 people
- Sponsor
- Vyne Therapeutics Inc.
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