Clinical trials: Infant, Newborn, Diseases
Cushing Disease Due to Increased ACTH Secretion
Combination Osilodrostat and Cabergoline in Cushing's Disease
Cushing disease remains a challenging endocrine disorder in which persistent or recurrent hypercortisolism often requires medical therapy after surgery or when surgery is not feasible.
- Trial name
- COSCA-ECD (NCT07603466)
- Testing
- osilodrostat, osilodrostat and cabergoline
- Where
- 5 sites in Iraq
- Volunteers
- 50 people
- Sponsor
- University of Basrah
Flat Feet
Calf Muscle Tightness in Flat Foot Subjects
The purpose of the current work is to determine the prevalence of calf muscle tightness in asymptomatic flat foot subjects.
- Trial name
- NCT04720651
- Volunteers
- 174 people
- Sponsor
- Cairo University
Muscular Dystrophy, Duchenne, Muscular Dystrophies
Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy
This study is a long-term study of ataluren in participants with nonsense mutation Duchenne muscular dystrophy.
- Conditions
- Muscular Dystrophy, Duchenne, Muscular Dystrophies, Muscular Disorders, Atrophic, Muscular Diseases
- Trial name
- NCT03179631
- Testing
- Ataluren
- Where
- 64 sites in United States, Australia and 16 more
- Volunteers
- 360 people
- Sponsor
- PTC Therapeutics
Cystic Fibrosis
Trial to Evaluate Efficacy and Safety of Lenabasum in Cystic Fibrosis
This is a Phase 2 multicenter, double-blind, randomized, placebo-controlled study assessing the efficacy and safety of lenabasum for the treatment of cystic fibrosis in patients 12 years of age or older.
- Trial name
- NCT03451045
- Testing
- Lenabasum 20 mg, Lenabasum 5 mg
- Where
- 105 sites in United States, Austria and 19 more
- Volunteers
- 447 people
- Sponsor
- Corbus Pharmaceuticals Inc.
Alpha-1 Antitrypsin Deficiency
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels [6.0 ×…
- Trial name
- NCT01054339
- Testing
- rAAV1-CB-hAAT
- Where
- 4 sites in United States, Ireland
- Volunteers
- 9 people
- Sponsor
- Beacon Therapeutics
Familial Mediterranean Fever
Assessment of microRNAs Role in Familial Mediterranean Fever FMF Pathophysiology
Familial Mediterranean Fever (FMF) genetic diagnosis is well established for homozygous patients. On the other hand, although heterozygous individuals are theoretically healthy carriers, 1/3 of them will develop clinical symptoms…
- Trial name
- miRinFMF (NCT06033339)
- Testing
- Blood sampling
- Where
- 4 sites in France
- Volunteers
- 40 people, healthy volunteers welcome
- Sponsor
- University Hospital, Montpellier
Leukemia
S0919 Idarubicin, Cytarabine, and Pravastatin in Treating Patients With Relapsed Acute Myeloid Leukemia
RATIONALE: Drugs used in chemotherapy, such as idarubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells or by stopping them from dividing.
- Trial name
- NCT00840177
- Testing
- cytarabine, idarubicin, pravastatin sodium
- Where
- 185 sites in United States
- Volunteers
- 115 people
- Sponsor
- SWOG Cancer Research Network
Infant, Newborn, Diseases
Baby Sickness Evaluation
The goal of this prospective single center feasibility study is to assess the feasibility of using BabyCheck and PO by GPs as an illness severity score for young infants > 6 months of age in order to aid the decision for referral…
- Trial name
- BeSurE (NCT05954975)
- Testing
- BabyCheck and pulse oximetry
- Volunteers
- 1,500 people
- Sponsor
- Ilona C Narayen, MD pHD
Gout
Study of Pegloticase (KRYSTEXXA®) Plus Methotrexate in Patients With Uncontrolled Gout
The overall objective of the study is to assess the efficacy, safety, tolerability, and pharmacokinetics (PK) of the concomitant use of pegloticase with methotrexate (MTX) to enhance the response rate seen with pegloticase alone…
- Trial name
- MIRROR OL (NCT03635957)
- Testing
- Pegloticase, Methotrexate (MTX), Standard Gout Flare Prophylaxis
- Where
- 6 sites in United States
- Volunteers
- 14 people
- Sponsor
- Amgen
Intrauterine Growth Restriction
The Effect of Antenatal Corticosteroid on Umbilical Artery Doppler Velocimetry in IUGR
The purpose of this study is to evaluate the effect of antenatal corticosteroid administration on umbilical artery Doppler velocimetry measurements in pregnancies complicated by IUGR.
- Trial name
- NCT05588986
- Testing
- ultrasonography
- Where
- Turkey (Türkiye)
- Volunteers
- 149 people
- Sponsor
- Merve Demir
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Intraventricular Hemorrhage, Autoregulation
Physiological Disturbances Associated With Neonatal Intraventricular Hemorrhage
Annually, almost 5,000 extremely low birth weight (9 ounces to about 2 lbs) infants born in the US survive with severe bleeding in the brain (intraventricular hemorrhage); this devastating complication of prematurity is…
- Trial name
- PhysDis (NCT00665769)
- Testing
- Hypercapnia, Normocapnia
- Where
- United States
- Volunteers
- 103 people
- Sponsor
- Baylor College of Medicine
Psoriasis, Atopic Dermatitis
LANDSCAPE: Demographics and Treatment Patterns of Patients With Immune-Mediated Inflammatory Skin Diseases in Italian Clinical Practice
This multicenter retrospective observational registry study will collect existing clinical data from Italian centers to describe epidemiology, treatment patterns, clinical evolution, safety, and comorbidities in patients aged 12…
- Conditions
- Psoriasis, Atopic Dermatitis, Vitiligo, Alopecia Areata
- Trial name
- LANDSCAPE (NCT07336641)
- Testing
- Conventional Systemic Therapies, Biologic Therapies
- Where
- Italy
- Volunteers
- 20,000 people
- Sponsor
- Fondazione ISIDE
Mucopolysaccharidosis I
Extension Study Evaluating Long Term Safety and Activity of AGT-181 in Children With MPS I
AGT-181 is a fusion protein containing alpha-L-iduronidase that is intended to deliver the enzyme peripherally and to the brain, when administered intravenously.
- Trial name
- NCT03071341
- Testing
- AGT-181
- Where
- Brazil
- Volunteers
- 19 people
- Sponsor
- ArmaGen, Inc
Congenital Heart Disease, Hypoplastic Left Heart Syndrome
Genetic Determinants of Congenital Heart Disease Outcomes
The purpose of this study is to examine the role of genetic variation in the oxidative stress response on critical perioperative and short-term outcomes after neonatal heart surgery.
- Conditions
- Congenital Heart Disease, Hypoplastic Left Heart Syndrome, Hypoplastic Right Heart Syndrome, d-Transposition of the Great Arteries
- Trial name
- GECHO (NCT01656941)
- Where
- 5 sites in United States, Australia
- Volunteers
- 250 people
- Sponsor
- University of Michigan
Retinitis Pigmentosa (RP)
Subthreshold Micropulse Laser Therapy (SML) in Retinitis Pigmentosa
Introduction Retinitis pigmentosa (RP) is the most common form of genetic retinal degenerative disease. The disease is progressive and leads to blindness and permanent disability within a few years of diagnosis.
- Trial name
- NCT07088705
- Testing
- SML 577 nm, SML 810 nm
- Where
- Poland
- Volunteers
- 60 people
- Sponsor
- Marta P. Wiącek
Wilson's Disease
Cardiac Involvement in Wilson's Disease
Heart damage by copper accumulation has been reported in Wilson's Disease. However, the disease epidemiology is still poorly understood.
- Trial name
- WIL-HEART (NCT05493605)
- Testing
- Delivery of a long-term Holter 21 days Or placement of an implantable holter from the outset so syncope
- Where
- France
- Volunteers
- 150 people
- Sponsor
- Fondation Ophtalmologique Adolphe de Rothschild
Intrauterine Growth Restriction
Intraumbilical Amino Acids and Glucose Supplementation Via Port by Severe IUGR in Human Fetuses
Placental insufficiency is responsible for fetal loss in about 40% of all stillbirths and long term neurological deficits. The mean interval from diagnosis of brain sparing of severe IUGR fetuses to delivery has been recently…
- Trial name
- port-IUGR (NCT02596594)
- Testing
- fetal nutrition port system
- Volunteers
- 14 people
- Sponsor
- Martin-Luther-Universität Halle-Wittenberg
Macular Corneal Dystrophy
Fresh Corneal Lenticule Implantation in Macular Corneal Distrophy With Relex Smile Surgery
The aim of the study is to examine the effect of fresh corneal lenticule implantation as allogenic graft taken from myopic patients to implanted in patients with macular corneal dystrophy using Visumax Femtosecond Laser Smile…
- Trial name
- NCT04642729
- Testing
- Fresh Corneal Lenticule Implantation using Relex-Smile Surgery
- Where
- Kosovo
- Volunteers
- 15 people
- Sponsor
- Eye Hospital Pristina Kosovo
Atopic Dermatitis
Responses to Immunization With Keyhole Limpet Hemocyanin Administered by Scarification and the Intradermal Route
Atopic dermatitis (AD) is a skin disorder in which people often have swelling and skin infections. People with this disease cannot receive the smallpox vaccine because it could cause them to have a fatal reaction known as eczema…
- Trial name
- NCT00614731
- Testing
- KLH carrier-protein
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)