Clinical trials: Hormone Replacement
Renal Failure
Renal Replacement Therapy in Neonates
The circumstances for setting up dialysis in the neonatal period are multiple: congenital anomaly of the kidney and urinary tract (CAKUT), secondary acute renal failure whose etiologies are multiple (sepsis, hypovolemia…
- Trial name
- Neo-Renal (NCT05456659)
- Where
- France
- Volunteers
- 72 people
- Sponsor
- University Hospital, Strasbourg, France
Neonatal Abstinence Syndrome
The Alberta Neonatal Abstinence Syndrome Mother-Baby Care ImprovEmeNT Program
Neonatal Abstinence Syndrome (NAS), is a common and costly problem in Alberta that affects approximately 250 babies per year exposed to drugs during pregnancy. Unfortunately, this has become more common in the last 10 years.
- Trial name
- NASCENT (NCT05226624)
- Testing
- Rooming-in care, Baseline standard of care
- Where
- Canada
- Volunteers
- 240 people
- Sponsor
- University of Alberta
Spinal Muscular Atrophy (SMA)
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
The goal of this clinical trial is to learn about the safety, and tolerability of giving an experimental gene therapy called SPX-01 to an infant with spinal muscular atrophy (SMA) type 1.
- Trial name
- NCT07903376
- Testing
- SPX-01
- Where
- Iran
- Volunteers
- 1 people
- Sponsor
- Exir Gene Pharmed
Fabry Disease, Proteinuria
Antiproteinuric Agents and Fabry Disease
Fabry disease is a rare disorder that often has kidney involvement with increased urine protein excretion. Proteinuria is recognized as an important risk factor for progression of chronic kidney disease.
- Trial name
- NCT00343577
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Alabama at Birmingham
Acute Kidney Injury, Renal Replacement Therapy
Use of NGAL for Fluid Dosing and CRRT Initiation in Pediatric AKI
This study follows a group of patients admitted to the PICU who are identified as being at risk for developing acute kidney injury.
- Conditions
- Acute Kidney Injury, Renal Replacement Therapy, Pediatric Intensive Care Units
- Trial name
- Taking Focus 2 (NCT03541785)
- Where
- United States
- Volunteers
- 420 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Metabolic Diseases, Cancer
Transitional Growth Hormone (GH) Use in Growth Hormone Deficient (GHD) Cancer Survivors
Cure rates for childhood malignancies have improved at a remarkable pace.With the increasing cure rate came recognition of the long-term detrimental effects of radiotherapy and chemotherapy, known as "late-effects".
- Trial name
- NCT02629926
- Testing
- GH Replacement (NutropinAq®), No additional treatment
- Where
- United Kingdom
- Volunteers
- 60 people
- Sponsor
- The Leeds Teaching Hospitals NHS Trust
Sepsis, Children, Only
Clinical Features and Outcomes of CBP Versus Non-CBP in Septic Children
The effect of continuous blood purification (CBP) in children is unclear. Also, the timing of early application is still being explored.
- Trial name
- NCT04794595
- Testing
- CBP
- Where
- China
- Volunteers
- 90 people
- Sponsor
- Children's Hospital of Fudan University
Common Variable Immunodeficiency, X-linked Agammaglobulinemia
Study of Subcutaneous Immune Globulin in Patients Requiring IgG Replacement Therapy
The objective of this study is to assess the efficacy, tolerability, safety and pharmacokinetics of IgPro20 in patients with primary humoral immunodeficiency (PID).
- Conditions
- Common Variable Immunodeficiency, X-linked Agammaglobulinemia, Autosomal Recessive Agammaglobulinemia
- Trial name
- NCT00542997
- Testing
- Human Normal Immunoglobulin for Subcutaneous Administration (IGSC)
- Where
- 19 sites in France, Germany and 7 more
- Volunteers
- 51 people
- Sponsor
- CSL Behring
Sly Syndrome, MPS VII
A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)
The primary objective of the study is to evaluate the long-term safety of UX003 in subjects with MPS 7.
- Conditions
- Sly Syndrome, MPS VII, Mucopolysaccharidosis, Mucopolysaccharidosis VII
- Trial name
- NCT02432144
- Testing
- UX003
- Where
- 7 sites in United States, Brazil and 2 more
- Volunteers
- 12 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Acute Kidney Injury in Pediatric Patients
Prismaflex HF20 Set and Prismaflex® System 7.10/7.20 for Acute Continuous Renal Replacement Therapy (CRRT) in Children
The primary objective of this study is to evaluate the efficacy of the Gambro Prismaflex® HF20 Set based on testing the hypothesis that it delivers sufficient renal replacement therapy to effectively treat acute kidney injury…
- Trial name
- NCT02561247
- Testing
- Prismaflex HF20 CRRT Filter, Prismaflex® System 7.10 and 7.20
- Where
- 6 sites in United States
- Volunteers
- 23 people
- Sponsor
- Vantive Health LLC
Tobacco Dependence, Schizophrenia
The Pharmacogenetic Study , Readiness to Change, and Pharmacological Intervention for Smoking Cessation in Schizophrenia
Cigarette smoking represents a major health problem for patients suffering from schizophrenia. Compared to the general population, schizophrenic patients are significantly more likely to be addicted to nicotine.
- Trial name
- PSIAARP (NCT00495352)
- Testing
- Hihg-doseNRT, Low-dose NRT, bupropion
- Where
- Taiwan
- Volunteers
- 360 people
- Sponsor
- Yu-Li Hospital
MPS IVA, Morquio Syndrome
Gait Analysis in MPS IVA
The purpose of this study is to learn about gait (walking) characteristics in MPS IVA and possible changes in gait with enzyme replacement therapy.
- Trial name
- NCT01920828
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Oregon Health and Science University
Pompe Disease Infantile-Onset
Exercise Capacity in Response to Enzyme Replacement Therapy in Pediatric Pompe Disease.
Our aims are to investigate the acute and long term effect of ERT on exercise capacity; comparing the effect of different ERT dosages (as prescribed by the clinician according to clinical judgment) and assessing the relationship…
- Trial name
- NCT04755751
- Testing
- Cardiopulmonary exercise testing (CPET)
- Where
- Israel
- Volunteers
- 4 people
- Sponsor
- Rambam Health Care Campus
Cystic Fibrosis, Growth Failure
Baby Observational and Nutritional Study
Cystic fibrosis (CF) is a life-shortening disease that causes breathing and digestive problems, but can now be diagnosed at the time of birth. Lung function is very hard to measure in infants, but growth is not.
- Conditions
- Cystic Fibrosis, Growth Failure, Exocrine Pancreatic Insufficiency
- Trial name
- BONUS (NCT01424696)
- Where
- 28 sites in United States
- Volunteers
- 231 people
- Sponsor
- Seattle Children's Hospital
Pompe Disease
Observational Study for Subjects With Pompe Disease Undergoing Immune Modulation Therapies
Hypothesis: the effectiveness of treatment of Pompe Disease with rhGAA enzyme replacement therapy (ERT) is limited at least in part because patients develop antibodies against the provided rhGAA enzyme.
- Trial name
- NCT01451879
- Testing
- Rituximab, Miglustat
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of Florida
Critical Illness, Nutritional Deficiency
Trace Element Repletion Following Severe Burn Injury
Major burn patients are characterized by large exudative losses of Cu, Se and Zn. Trace element (TE) repletion has been shown to improve clinical outcome.
- Conditions
- Critical Illness, Nutritional Deficiency, Trace Element Deficiency, Burn Injury
- Trial name
- NCT03204669
- Testing
- Trace elements replacement
- Where
- Switzerland
- Volunteers
- 139 people
- Sponsor
- Centre Hospitalier Universitaire Vaudois
Immunologic Deficiency Syndromes
Efficacy, Safety and Kinetics Study of Octagam 10% in Primary Immunodeficiency Diseases
Octagam is a human normal immunoglobulin (IGIV) solution for intravenous administration. Octagam 5% is currently registered in more than 60 countries.
- Trial name
- NCT00811174
- Testing
- Octagam 10%
- Where
- Austria
- Volunteers
- 5 people
- Sponsor
- Octapharma
Fabry Disease
Impact of Agalsidase Alfa Therapy on Cardiac funcTION in Patients With Fabry's Cardiomyopathy
Study Design: This is an observational study. No treatment or intervention will be assigned to the subjects. All patients will receive full standard of care concomitant medication for the treatment of their cardiac condition.
- Trial name
- NCT03230591
- Testing
- Echocardiography
- Where
- South Korea
- Volunteers
- 25 people
- Sponsor
- Yonsei University
Acute Kidney Injury
Safety and Efficacy of a Selective Cytopheretic Device (SCD) in Pediatric Patients With Acute Kidney Injury (AKI).
The SCD (Selective Cytopheretic Device) is an extracorporeal device used as an adjunct to renal replacement therapy (RRT) to improve the outcomes of pediatric patients with acute kidney injury (AKI).
- Trial name
- SCD-PED-01 (NCT02820350)
- Testing
- SCD-F40
- Where
- 4 sites in United States
- Volunteers
- 19 people
- Sponsor
- CytoPherx, Inc
Letrozole, Infertility, Female
The Effectiveness of the Letrozole-induced Endometrial Preparation Protocol in Frozen-thawed Embryo Transfer (FET)
The goal of this single center, non-blinded, randomized controlled clinical trial to comparison of pregnancy outcomes and perinatal outcomes in patients undergoing IVF treatment (including ICSI) with FET with letrozole-induce…
- Trial name
- NCT06006091
- Testing
- Letrozole
- Where
- China
- Volunteers
- 858 people
- Sponsor
- International Peace Maternity and Child Health Hospital