Clinical trials: Congenital Abnormalities
Weight Gain, Body Mass Index
"Effects of High-Calorie Diets VS High-Calorie Formulas on Weight Gain in Children With Congenital Heart Disease "
This study is a randomized controlled trial designed to evaluate the effects of high-calorie diets and specialized nutritional formulas on weight gain and clinical outcomes in underweight children aged 1 to 5 years with…
- Conditions
- Weight Gain, Body Mass Index, Pediatric Nutrition, Congenital Heart Disease
- Trial name
- NCT07552688
- Testing
- High-Calorie Specialized Nutritional Formula, education program, High-Calorie Diet
- Where
- 2 sites in Iraq
- Volunteers
- 75 people
- Sponsor
- Aryan Najmadin Nasradin
Short Stature
IGF-1 Treatment for Individuals With Short Stature Due to PAPP-A2 Deficiency
With this study we want to investigate the pharmacokinetic (PK) effect of a single injection of rhIGF-1 in patients with PAPP-A2 mutations compared to heterozygous carriers and healthy controls.
- Trial name
- NCT02636270
- Testing
- Increlex
- Volunteers
- 7 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Molar-Incisor Hypomineralization
Clinical Efficacies of Different Agents in Molar-Incisor Hypomineralization
This study aims to assess the clinical effectiveness of GC Fuji Triage ® (GC America Inc., Alsip, Illinois) and BeutiSealant (Shofu, Kyoto, Japan) in children aged 8-13 with multiple molars affected by Molar-Incisor…
- Trial name
- NCT06180369
- Testing
- GC Fuji Triage ® GC America Inc., Alsip, Illinois ), BeutiSealant (Shofu, Kyoto, Japonya)
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Marmara University
Cystic Fibrosis
Study of Uridine Triphosphate (UTP) as an Aerosol Spray for Cystic Fibrosis
OBJECTIVES: I. Determine the stability of uridine triphosphate (UTP) and examine the metabolism of exogenous nucleotides on airway epithelial surfaces in patients with cystic fibrosis. II.
- Trial name
- NCT00004705
- Testing
- amiloride, uridine
- Sponsor
- FDA Office of Orphan Products Development
Tuberous Sclerosis, Lymphangioleiomyomatosis
Characterization of Patients With Tuberous Sclerosis Complex, Lymphangioleiomyomatosis and Angiomyolipoma
Tuberous Sclerosis Complex (TSC) is a multisystemic autosomal dominant disease that is characterized by the development of benign neoplasms in brain, kidney, lung, skin and heart.
- Conditions
- Tuberous Sclerosis, Lymphangioleiomyomatosis, Angiomyolipoma
- Trial name
- NCT02325505
- Where
- 3 sites in Brazil
- Volunteers
- 200 people
- Sponsor
- InCor Heart Institute
Atopic Dermatitis, Atopic Eczema
Atopic Dermatitis Biomarker Identification Trial in Omalizumab Usage
This study aims at evaluating the therapeutic potential of omalizumab in atopic dermatitis, and to work out biomarkers predictive of treatment response.
- Trial name
- AMB-WEI-1052-I (NCT01179529)
- Testing
- Omalizumab (Xolair®)
- Where
- Germany
- Sponsor
- Technical University of Munich
Mucopolysaccharidosis IVA, Morquio A Syndrome
Safety and Exercise Study of Two Doses of BMN 110 for Morquio A Syndrome
The primary objective of this study was to evaluate the safety of a 2.0 mg/kg/week and a 4.0 mg/kg/week of BMN 110 in patients with Morquio A syndrome for up to 196 weeks.
- Conditions
- Mucopolysaccharidosis IVA, Morquio A Syndrome, MPS IVA
- Trial name
- NCT01609062
- Testing
- BMN 110
- Where
- 11 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- BioMarin Pharmaceutical
Urea Cycle Disorder
Study of Glycerol Phenylbutyrate & Sodium Phenylbutyrate in Phenylbutyrate Naïve Patients With Urea Cycle Disorders (UCDs)
This is a randomized, controlled, open-label parallel arm study to assess the safety, tolerability, pharmacokinetics and ammonia control, of RAVICTI® as compared to Sodium phenylbutyrate (NaPBA) in urea cycle disorder subjects…
- Trial name
- NCT03335488
- Testing
- RAVICTI, NaPBA
- Where
- 11 sites in United States, Italy and 2 more
- Volunteers
- 16 people
- Sponsor
- Amgen
Developmental Dysplasia of the Hip
Selective Ultrasound Screening for DDH 1991-2006
Early treatment is considered essential for developmental dysplasia of the hip (DDH), but the choice of screening strategy is debated. The investigators evaluated the effect of a selective ultrasound (US) screening programme.
- Trial name
- NCT01866527
- Where
- Norway
- Volunteers
- 81,564 people, healthy volunteers welcome
- Sponsor
- University of Bergen
Hemophilia
Medication Exposure in Older Patients With Constitutional Hemorrhagic Disease
The improvements observed in the care of patients with hemophilia or Willebrand disease have led to an increase in their life expectancy, which today approaches that of the general population.
- Trial name
- M-HEMORRH'AGE (NCT04282486)
- Testing
- Telephone interview
- Where
- France
- Volunteers
- 142 people
- Sponsor
- Hospices Civils de Lyon
Cytomegalovirus Retinitis, HIV Infections
A Phase I/II Open-Labelled Trial of Intravitreal Ganciclovir Salvage Therapy for AIDS Patients With Active CMV Retinitis Who Are Intolerant of Systemic Therapy
AMENDED: 04-12-91 Population of patients changed FROM those who are intolerant of systemic therapy with NON-sight-threatening CMV retinitis TO those AIDS patients intolerant of systemic therapy with CMV retinitis.
- Trial name
- NCT00000673
- Testing
- Ganciclovir
- Where
- 2 sites in United States
- Volunteers
- 38 people
- Sponsor
- National Institute of Allergy and Infectious Diseases (NIAID)
Atopic Dermatitis
Safety, Tolerability and Pharmacokinetic Study in Healthy Subjects by Dermal Application of a LEO 29102
The principal aim of this study is to obtain safety, tolerability and pharmacokinetic data when LEO 29102 is administered cutaneously as single and multiple doses to healthy subjects.
- Trial name
- NCT01423656
- Testing
- LEO 29102
- Where
- United Kingdom
- Volunteers
- 102 people, healthy volunteers welcome
- Sponsor
- LEO Pharma
Hemophilia A
Phase 3/4 Study of a Recombinant Protein-Free Factor VIII (rAHF-PFM): Comparison of Continuous Infusion Versus Intermittent Bolus Infusion in Hemophilia A Subjects Undergoing Major Orthopedic Surgery
The purpose of this study is to compare the hemostatic efficacy and safety of continuous infusion versus intermittent bolus infusion in the peri- and post-operative setting, employing rAHF-PFM, a recombinant antihemophilic factor…
- Trial name
- NCT00357656
- Testing
- Recombinant Protein-Free Factor VIII (rAHF-PFM)
- Where
- 36 sites in United States, Austria and 12 more
- Volunteers
- 85 people
- Sponsor
- Baxalta now part of Shire
Vein of Galen Malformations
Fetal Treatment of Galenic Malformations
This is a prospective, single-arm non-randomized interventional study of fetuses to assess the safety and efficacy of fetal embolization of Vein of Galen Malformation (VOGM).
- Trial name
- NCT04434729
- Testing
- Fetal embolization of vein of Galen malformation using Target XL and XXL Detachable Coils
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Darren Orbach
Neonatal Hypocalcemia, Neonatal Sepsis
Effects of Cord Blood 25-hydroxy-vitamin D Level on Early Neonatal Morbidities
Cholecalciferol (vitamin D3) is a prohormone and its active form is 1,25 dihydroxycholecalciferol. This hormone has effect on both calcium, phosphorus and bone metabolism and also bone morrow, muscle, heart and immune system.
- Trial name
- NCT02147327
- Volunteers
- 120 people
- Sponsor
- Zekai Tahir Burak Women's Health Research and Education Hospital
Hemophilia B
Study To Compare On-Demand Treatment To A Prophylaxis Regimen Of BeneFIX In Subjects With Moderately Severe to Severe Hemophilia B
The purpose of this study will be to determine if a once-weekly prophylaxis regimen of BeneFIX in subjects with moderately severe to severe Hemophilia B is safe and effective.
- Trial name
- NCT01335061
- Testing
- Nonacog alfa
- Where
- 17 sites in Bulgaria, Canada and 7 more
- Volunteers
- 25 people
- Sponsor
- Pfizer
Hepatocellular Carcinoma (HCC), Cholangiocarcinoma
An Open-Label Safety and Tolerability Study of INCB062079 in Subjects With Advanced Hepatocellular Carcinoma and Other Malignancies
The purpose of this study is to evaluate the safety and tolerability, and determine the maximum tolerated dose of INCB062079 in subjects with advanced hepatocellular carcinoma and other malignancies.
- Conditions
- Hepatocellular Carcinoma (HCC), Cholangiocarcinoma, Esophageal Cancer, Nasopharyngeal Cancer
- Trial name
- NCT03144661
- Testing
- INCB062079
- Where
- 6 sites in United States, Belgium
- Volunteers
- 25 people
- Sponsor
- Incyte Corporation
Limb Girdle Muscular Dystrophy, Limb Girdle Muscular Dystrophy Type 2D/R3
Study to Evaluate the Safety, Tolerability, and Efficacy of SRP-9004 Administered by Systemic Infusion in Limb Girdle Muscular Dystrophy Type 2D/R3 Participants in the United States
The primary objective of this study is to evaluate the safety of SRP-9004.
- Trial name
- DISCOVERY (NCT06747273)
- Testing
- SRP-9004
- Where
- 2 sites in United States
- Volunteers
- 4 people
- Sponsor
- Sarepta Therapeutics, Inc.
Thyroid Eye Disease, TED
A Multicenter, Open-label Extension Study Evaluating the Efficacy and Safety of IBI311 in Subjects With Thyroid Eye Disease
A multicenter, open-label extension study evaluating the efficacy and safety of IBI311 in subjects with thyroid eye disease
- Trial name
- NCT06126783
- Testing
- IBI311
- Where
- China
- Volunteers
- 91 people
- Sponsor
- Innovent Biologics (Suzhou) Co. Ltd.
Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22)
GTI-2040 and High-Dose Cytarabine in Treating Patients With Refractory or Relapsed Acute Myeloid Leukemia
This phase I trial is studying the side effects and best dose of GTI-2040 and high-dose cytarabine in treating patients with refractory or relapsed acute myeloid leukemia.
- Conditions
- Adult Acute Myeloid Leukemia With 11q23 (MLL) Abnormalities, Adult Acute Myeloid Leukemia With Inv(16)(p13;q22), Adult Acute Myeloid Leukemia With t(15;17)(q22;q12), Adult Acute Myeloid Leukemia With t(16;16)(p13;q22)
- Trial name
- NCT00070551
- Testing
- GTI-2040, cytarabine, pharmacological study
- Where
- United States
- Volunteers
- 51 people
- Sponsor
- National Cancer Institute (NCI)