Clinical trials: Neonatal Disorder
Cardiac Output, Low
Corticosteroids in Postoperative Critically Ill Neonates With Low Cardiac Output Syndrome With Congenital Heart Disease
Babies having heart surgery often have problems after surgery with their blood pressure and getting enough blood to their bodies.
- Trial name
- Corti (NCT00590018)
- Testing
- Hydrocortisone
- Where
- United States
- Volunteers
- 18 people
- Sponsor
- Baylor College of Medicine
Leukemia, Lymphoma
AlloHCT From Matched Unrelated Donors in Pts w/ Advanced Hematologic Malignancies & Disorders
RATIONALE: Giving chemotherapy with or without total-body irradiation before a donor umbilical cord blood transplant helps stop the growth of cancer or abnormal cells.
- Conditions
- Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00547196
- Testing
- filgrastim, Busulfan, Cyclophosphamide
- Where
- 2 sites in United States
- Volunteers
- 10 people
- Sponsor
- City of Hope Medical Center
Chronic Myeloproliferative Disorders, Leukemia
Sirolimus, Tacrolimus, and Antithymocyte Globulin in Preventing Graft-Versus-Host Disease in Patients With Hematologic Cancer Who Are Undergoing Donor Stem Cell Transplant
RATIONALE: Giving low doses of chemotherapy, monoclonal antibodies, and radiation therapy before a donor peripheral blood stem cell transplant helps stop the growth of cancer cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00691015
- Testing
- rituximab, busulfan, carmustine
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- Barbara Ann Karmanos Cancer Institute
Congenital Heart Disease
Study of Possible Brain Hormone Problems After Open Heart Surgery in Infants
The goal of this study is to evaluate specific hormone levels in children undergoing heart surgery in order to identify patterns associated with any unstable vital signs.
- Trial name
- NCT00850720
- Testing
- Blood draws
- Where
- United States
- Volunteers
- 3 people
- Sponsor
- Emory University
Healthy
Study of PF 04965842 Effect on MATE1/2K Activity in Healthy Participants
This is a Phase 1, randomized, 2 way crossover, open label study of the effect of PF-04965842 on metformin (a probe for MATE1/2K activity) PK in healthy adult participants.
- Trial name
- NCT03796182
- Testing
- Metformin, PF-04965842
- Where
- Belgium
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Pfizer
Retinopathy of Prematurity (ROP)
Aflibercept for Retinopathy of Prematurity - Intravitreal Injection Versus Laser Therapy
The purpose of this study is to demonstrate how well aflibercept works in babies with ROP, comparing it with laser therapy. The study also has the objective to demonstrate how safe aflibercept is when used in babies, and describe…
- Trial name
- FIREFLEYE (NCT04004208)
- Testing
- Eylea (Aflibercept, BAY86-5321), Laser photocoagulation
- Where
- 90 sites in Argentina, Austria and 25 more
- Volunteers
- 113 people
- Sponsor
- Bayer
Phenylketonuria
Study of Phenoptin in Subjects With Phenylketonuria Who Participated in Protocols PKU-004 or PKU-006
The objective of this study is to evaluate the safety of long-term treatment with Phenoptin in subjects with phenylketonuria (PKU) who participated in Phase 3 clinical studies with Phenoptin.
- Trial name
- NCT00332189
- Testing
- sapropterin dihydrochloride
- Where
- 14 sites in United States
- Volunteers
- 111 people
- Sponsor
- BioMarin Pharmaceutical
Cystic Fibrosis
A Study to Evaluate the Efficacy and Safety of VX-661 in Combination With Ivacaftor in Subjects Aged 12 Years and Older With Cystic Fibrosis, Heterozygous for the F508del-CFTR Mutation
Study to evaluate the efficacy of VX-661 in combination with ivacaftor (IVA, VX-770) through Week 12 in participants with cystic fibrosis (CF) who are heterozygous for the F508del mutation on the CF transmembrane conductance…
- Trial name
- NCT02516410
- Testing
- VX-661 plus ivacaftor combination, Ivacaftor
- Where
- 41 sites in United States, Australia and 5 more
- Volunteers
- 168 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial 38833 (P05783)
The objective of this trial is to evaluate whether corifollitropin alfa (MK-8962, Org 36286) treatment for the induction of multifollicular growth in women undergoing Controlled Ovarian Stimulation (COS) prior to in vitro…
- Trial name
- Care (NCT00702520)
- Testing
- Corifollitropin alpha (MK-8962, Org 36286) 100 ug, Corifollitropin alpha (MK-8962, Org 36286) 150 ug, Triptorelin
- Volunteers
- 15 people
- Sponsor
- Organon and Co
Non Malignant Diseases, Immunodeficiencies
Reduced Intensity Conditioning (RIC) Regimen for Patients With Non-malignant Disorders
This Phase II pilot study evaluates the safety and effectiveness of reduced-intensity conditioning (RIC) regimens prior to allogeneic hematopoietic stem cell transplantation (HSCT) in pediatric and young adult patients with…
- Conditions
- Non Malignant Diseases, Immunodeficiencies, Hemoglobinopathies
- Trial name
- RIC (NCT01050855)
- Testing
- Reduced-Intensity Conditioning
- Where
- United States
- Volunteers
- 56 people
- Sponsor
- Children's Hospital of Philadelphia
Congenital Heart Disease
Acute Effects of Inhaled Treprostinil in Fontan Patients
In this pilot study investigators propose to explore the effects of a locally delivered inhaled pulmonary vasodilator (inhaled treprostinil) on exercise performance, pulmonary blood flow, venous pressure response and vascular…
- Trial name
- NCT02769624
- Testing
- Treprostinil
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Cystic Fibrosis, Pneumonia
Population Pharmacokinetics of Prolonged Infusion Meropenem in Cystic Fibrosis (CF) Children
This study will determine the concentrations of the antibiotic meropenem when administered as a 3 hour prolonged infusion in children with cystic fibrosis who are hospitalized with an acute pulmonary exacerbation.
- Conditions
- Cystic Fibrosis, Pneumonia, Pseudomonas Aeruginosa Infection
- Trial name
- NCT01429259
- Testing
- meropenem
- Where
- 7 sites in United States
- Volunteers
- 30 people
- Sponsor
- Joseph Kuti
Hemophilia A
Pharmacokinetic Comparison of Advate rAHF-PFM With Recombinate rAHF in Patients With Severe Hemophilia A
The purpose of this study is to compare the pharmacokinetic parameters and safety of Advate rAHF-PFM versus Recombinate rAHF in well described previously treated patients with severe hemophilia A (factor VIII level < 1%).
- Trial name
- NCT00666406
- Testing
- Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method (rAHF-PFM), Recombinant Factor VIII (rAHF)
- Where
- Germany
- Volunteers
- 9 people
- Sponsor
- Baxalta now part of Shire
Malocclusion, Anxiety
Patient Education Before Orthognathic Surgery
To evaluate the effectiveness in the use of an educational material through: Assessment of knowledge about post-operative orthognathic surgery; assessing levels of anxiety with the survey of Anxiety Inventory-STATE-TRAIT…
- Conditions
- Malocclusion, Anxiety, Micrognathism, Prognathism
- Trial name
- NCT01803204
- Testing
- Booklet - Preoperative Educational
- Where
- Brazil
- Volunteers
- 48 people
- Sponsor
- University of Sao Paulo
Chronic Myeloproliferative Disorders, Leukemia
Combination Chemotherapy and Donor Stem Cell Transplant in Treating Patients With Aplastic Anemia or Hematologic Cancer
RATIONALE: Giving chemotherapy drugs and total-body irradiation before a donor stem cell helps stop the growth of cancer or abnormal cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Chronic Myeloproliferative Disorders, Leukemia, Lymphoma, Myelodysplastic Syndromes
- Trial name
- NCT00003816
- Testing
- anti-thymocyte globulin, busulfan, carboplatin
- Where
- United States
- Volunteers
- 361 people
- Sponsor
- Roswell Park Cancer Institute
Thalassemia
A Study to Determine the Efficacy and Safety of Luspatercept in Adults With Non Transfusion Dependent Beta (β)-Thalassemia
This is a Phase 2, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) versus placebo in adults with non-transfusion dependent beta (β)-thalassemia.
- Trial name
- BEYOND (NCT03342404)
- Testing
- Luspatercept, Best Supportive Care (BSC)
- Where
- 16 sites in United States, Greece and 4 more
- Volunteers
- 145 people
- Sponsor
- Celgene
Vitamin D Deficiency, Thoracic Surgery
Prevention of Vitamin D Deficiency Following Pediatric CHD Surgery: a Phase II Dose Evaluation Randomized Controlled Trial Comparing Usual Care With a High Dose Pre-operative Supplementation Regimen Based on the Institute of Medicine Daily Upper Tolerable Intake Level
Our research group has shown that almost all children with congenital heart disease (CHD) are vitamin D deficient following heart surgery.
- Conditions
- Vitamin D Deficiency, Thoracic Surgery, Pediatric Disorders, Heart Defects, Congenital
- Trial name
- HICCUPS 2 (NCT01838447)
- Testing
- Cholecalciferol
- Where
- Canada
- Volunteers
- 46 people
- Sponsor
- Children's Hospital of Eastern Ontario
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).
- Trial name
- ReNeu (NCT03962543)
- Testing
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- Where
- 43 sites in United States
- Volunteers
- 114 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Gestational Diabetes, Maternal Complication of Pregnancy
CAPO: Continuous Glucose Monitoring in A2 Gestational Diabetes and Pregnancy Outcomes
This study will utilize continuous glucose monitoring in women with A2 gestational diabetes. Women will be randomized to continuous glucose monitoring or routine care with fingersticks to check their blood glucose four times…
- Trial name
- CAPO (NCT04219085)
- Testing
- Continuous Glucose Monitor, Routine Care
- Where
- United States
- Volunteers
- 65 people
- Sponsor
- Yale University
Fabry Disease
Open-Label Extension Study of the Long-Term Effects of Migalastat HCL in Patients With Fabry Disease
This is an open-label extension study intended to provide continued treatment with migalastat hydrochloride (HCl) for participants with Fabry disease who completed treatment of a previous migalastat HCl study.
- Trial name
- NCT02194985
- Testing
- migalastat HCl 150 mg
- Where
- 28 sites in United States, Argentina and 13 more
- Volunteers
- 84 people
- Sponsor
- Amicus Therapeutics