Clinical trials: Newborn
Niemann-Pick Disease, Type C
Study of IV VTS-270 for Infantile Liver Disease Associated With Niemann-Pick Disease, Type C
Niemann-Pick disease, type C (NPC) is a lethal, autosomal recessive, lysosomal storage disorder characterized by neurodegeneration in early childhood and death in adolescence.
- Trial name
- NCT03471143
- Testing
- VTS-270
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Washington University School of Medicine
Pompe Disease
Drug-drug Interaction Study
This study evaluates drug-drug interactions between AT2220 (duvoglustat) and recombinant human alpha-glucosidase (rhGAA, also known as alglucosidase alfa) in participants with Pompe Disease.
- Trial name
- NCT01380743
- Testing
- duvoglustat, rhGAA
- Where
- 15 sites in United States, Canada and 2 more
- Volunteers
- 25 people
- Sponsor
- Amicus Therapeutics
Down Syndrome, Subclinical Hypothyroidism
Treatment Trial of Subclinical Hypothyroidism in Down Syndrome
The purpose of this research study is to learn about the effects of treating subclinical hypothyroidism (SCH) with thyroid hormone replacement in children and adolescents with Down syndrome (DS).
- Trial name
- NCT01832753
- Testing
- Levothyroxine
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Children's Hospital of Philadelphia
Cystic Fibrosis
Effect of Sulforaphane in Broccoli Sprouts on Nrf2 Activation
The purpose of this study to investigate the effect of sulforaphane from macerated broccoli sprouts in humans and to evaluate less invasive methods of assessing potential anti-inflammatory drugs in CF.
- Trial name
- NCT01315665
- Testing
- Broccoli sprouts
- Where
- United States
- Volunteers
- 15 people, healthy volunteers welcome
- Sponsor
- University Hospitals Cleveland Medical Center
Treatment of Early Pulmonary Infections With P. Aeruginosa in Cystic Fibrosis Patients
Randomized, Controlled Study of CF Patients Between 3 Months and Less Than 7 Years
This study investigated the efficacy of inhaled TOBI treatment for early infections of P. aeruginosa in paediatric patients with cystic fibrosis.
- Trial name
- EARLY (NCT01082367)
- Testing
- TOBI
- Where
- 19 sites in Canada, Egypt and 8 more
- Volunteers
- 50 people
- Sponsor
- Novartis Pharmaceuticals
Thalassemia, Iron Overload
Combined Chelation Therapy in Patients With Transfusion Dependent Thalassemia and Iron Overload
This is a pilot study looking at the safety and efficacy of giving combination chelation with deferasirox and deferoxamine. The hypothesis is that combination chelation is safe in decreasing overall iron in patients with…
- Trial name
- NCT00901199
- Testing
- Combo Chelation with Deferasirox (Exjade) and Deferoxamine (DFO)
- Where
- United States
- Volunteers
- 22 people
- Sponsor
- Elliott Vichinsky
Adrenoleukodystrophy, Metachromatic Leukodystrophy
HSCT for High Risk Inherited Inborn Errors
Hematopoietic stem cell transplantation has proven effective therapy for individuals with adrenoleukodystrophy (ALD), metachromatic leukodystrophy (MLD) or globoid cell leukodystrophy (GLD, or Krabbe disease).
- Conditions
- Adrenoleukodystrophy, Metachromatic Leukodystrophy, Globoid Cell Leukodystrophy, Tay Sachs Disease
- Trial name
- NCT00383448
- Testing
- Clofarabine, Total body Irradiation, Melphalan
- Where
- United States
- Volunteers
- 38 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Familial Cold Autoinflammatory Syndrome
Study of Safety, Tolerability and Efficacy of DFV890 in Participants With Familial Cold Auto-inflammatory Syndrome (FCAS)
The purpose of this phase II study was to assess the safety, tolerability and efficacy of DFV890 in participants with FCAS.
- Trial name
- DFV890-FCAS (NCT04868968)
- Testing
- DFV890
- Where
- 3 sites in United States, France and 1 more
- Volunteers
- 4 people
- Sponsor
- Novartis Pharmaceuticals
Pregnancy, Neonates
Pregnancy and Neonatal Follow-up of Ongoing Pregnancies Established in Clinical Trial P05693 (P05713)
The objective of follow-up study P05713 is to evaluate whether corifollitropin alfa (Org 36286) treatment for the induction of monofollicular growth in women who underwent ovulation induction (OI) in base study P05693…
- Trial name
- Care (NCT00702338)
- Testing
- corifollitropin alfa, recombinant Follicle Stimulating Hormone (recFSH), human Chorion Gonadotropin (hCG)
- Volunteers
- 1 people
- Sponsor
- Organon and Co
Fragile X Syndrome
Randomized Controlled Study of Donepezil in Fragile X Syndrome
Fragile X syndrome (FraX) is the most common known heritable cause of human intellectual disability. Though recent research has revealed much about the genetic and neurobiological bases of FraX, knowledge about specific and…
- Trial name
- NCT01120626
- Testing
- donepezil, sugar pill
- Where
- United States
- Volunteers
- 45 people
- Sponsor
- Stanford University
Wilson Disease
Single Daily Dosage of Trientine for Maintenance Treatment for Wilson Disease
Hypothesis: The investigators postulate that patients with Wilson disease who are asymptomatic or who have been effectively treated for their symptoms and are in a maintenance phase therapy can be safely and effectively treated…
- Trial name
- NCT01472874
- Testing
- Once a day Trientine
- Where
- United States
- Volunteers
- 8 people
- Sponsor
- Yale University
Hereditary Thyroid Gland Medullary Carcinoma, Locally Advanced Thyroid Gland Medullary Carcinoma
Sorafenib Tosylate in Treating Patients With Metastatic, Locally Advanced, or Recurrent Medullary Thyroid Cancer
This phase II trial studies how well sorafenib tosylate works in treating patients with medullary thyroid cancer that has spread to other parts of the body (metastatic), spread to the tissue surrounding the thyroid (locally…
- Conditions
- Hereditary Thyroid Gland Medullary Carcinoma, Locally Advanced Thyroid Gland Medullary Carcinoma, Multiple Endocrine Neoplasia Type 2A, Multiple Endocrine Neoplasia Type 2B
- Trial name
- NCT00390325
- Testing
- Sorafenib Tosylate
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- National Cancer Institute (NCI)
Stage III Kidney Wilms Tumor, Stage IV Kidney Wilms Tumor
Combination Chemotherapy With or Without Radiation Therapy in Treating Young Patients With Newly Diagnosed Stage III or Stage IV Wilms' Tumor
This phase III trial is studying how well combination chemotherapy with or without radiation therapy works in treating young patients with newly diagnosed stage III or stage IV Wilms' tumor.
- Trial name
- NCT00379340
- Testing
- 3-Dimensional Conformal Radiation Therapy, Conventional Surgery, Cyclophosphamide
- Where
- 227 sites in United States, Australia and 5 more
- Volunteers
- 395 people
- Sponsor
- Children's Oncology Group
Cystic Fibrosis
Study Investigating a Delayed-Release Pancrelipase in Patients With Exocrine Pancreatic Insufficiency Due to Cystic Fibrosis
This study will assess the effect of pancrelipase delayed release 24,000 unit capsules on fat and nitrogen absorption in subjects with PEI due to Cystic Fibrosis.
- Trial name
- NCT00510484
- Testing
- Pancrelipase Delayed Release
- Where
- 23 sites in United States, Hungary and 3 more
- Volunteers
- 35 people
- Sponsor
- Solvay Pharmaceuticals
Infant, Premature, Diseases, Respiratory Insufficiency Syndrome of Newborn
Impact of CPAP Level on V/Q Mismatch in Premature Infants
Continuous positive airway pressure (CPAP) is used in premature infants to maintain lungs open and facilitate gas exchange. When ventilation/perfusion (V/Q) mismatch is present, areas of the lung that are open for gas exchange do…
- Trial name
- NCT02983825
- Testing
- Continuous positive airway pressure (CPAP); level changes
- Where
- 3 sites in United States
- Volunteers
- 21 people
- Sponsor
- Children's Hospital of Philadelphia
Congenital Diaphragmatic Hernia
Airway Inflammation in Congenital Diaphragmatic Hernia Patients
We propose to examine Congenital Diaphragmatic Hernia (CDH) patients for evidence of airway inflammation as a first step to characterize the reported bronchodilator responsiveness.
- Trial name
- NCT02453750
- Testing
- Hypersaline, Bronchodilator Response
- Where
- Canada
- Volunteers
- 21 people
- Sponsor
- The Hospital for Sick Children
Epidermolysis Bullosa
Biochemical Correction of Severe EB by Allo HSCT and "Off-the-shelf" MSCs
This is an open-label, single institution, phase II study in patients with epidermolysis bullosa (EB). The underlying hypothesis is that the infusion of bone marrow or umbilical cord blood from a healthy unaffected donor will…
- Trial name
- NCT01033552
- Testing
- Cyclophosphamide, Fludarabine, Anti-thymocyte globulin
- Where
- United States
- Volunteers
- 32 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
Craniosynostosis
Comparing MRI to CT on Pediatric Craniosynostosis.
The purpose of this study is to develop and test the effectiveness and diagnostic quality of Zero Echo Time Magnetic Resonance Imaging (ZTE MRI) in comparison to CT.
- Trial name
- NCT04704284
- Testing
- Zero Echo Time Magnetic Resonance Imaging (ZTE MRI)
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- Mayo Clinic
Adult Acute Myeloid Leukemia in Remission, Acute Myeloid Leukemia Arising From Previous Myelodysplastic Syndrome
Fludarabine and Busulfan Followed by Allogeneic Stem Cell Transplant in Treating Older Patients With Acute Myeloid Leukemia in First Complete Remission
This phase II trial studies how well fludarabine and busulfan followed by a donor (allogeneic) stem cell transplant work in treating older patients with acute myeloid leukemia that is in first complete remission.
- Trial name
- NCT00070135
- Testing
- filgrastim, Anti-Thymocyte Globulin, busulfan
- Where
- 21 sites in United States
- Volunteers
- 121 people
- Sponsor
- Alliance for Clinical Trials in Oncology
Chronic Myeloproliferative Disorders, Graft Versus Host Disease
Cyclosporine Eye Drops in Preventing Graft-Versus-Host Disease of the Eye in Patients Who Have Undergone Donor Stem Cell Transplant for Hematologic Cancer or Bone Marrow Failure Disorder
RATIONALE: Cyclosporine eye drops may prevent graft-versus-host disease of the eye in patients who have undergone donor stem cell transplant for hematologic cancer or bone marrow failure disorder.
- Conditions
- Chronic Myeloproliferative Disorders, Graft Versus Host Disease, Leukemia, Lymphoma
- Trial name
- NCT00755040
- Testing
- cyclosporine ophthalmic emulsion
- Where
- 5 sites in United States
- Volunteers
- 164 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
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