Clinical trials: NEWBORN
Arterio-Venous Fistula, ESRD
VasQ External Support for Arteriovenous Fistula
This is a prospective clinical study of the VasQ external support for arteriovenous fistulas. The device is designed to improve fistula outcomes by optimizing the geometrical configuration of the fistula, influencing…
- Conditions
- Arterio-Venous Fistula, ESRD, Steal Syndrome, Aneurysm
- Trial name
- NCT03242343
- Testing
- VasQ
- Where
- 15 sites in United States
- Volunteers
- 144 people
- Sponsor
- Laminate Medical Technologies
Myeloproliferative Disorders, Leukemia
Umbilical Cord Blood (UCB) Transplant, Fludarabine, Melphalan, and Anti-thymocyte Globulin (ATG) in Treating Patients With Hematologic Cancer
RATIONALE: Giving low doses of chemotherapy before a donor umbilical cord blood transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells.
- Conditions
- Myeloproliferative Disorders, Leukemia, Lymphoma, Multiple Myeloma and Plasma Cell Neoplasm
- Trial name
- NCT00827099
- Testing
- anti-thymocyte globulin, fludarabine phosphate, Melphalan
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- Northside Hospital, Inc.
Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0)
AML Therapy With Irradiated Allogeneic Cells
This pilot clinical trial studies if cells donated by a close genetic relative can help maintain acute myeloid leukemia (AML) complete remission (CR). Eligible patients will receive a standard induction chemotherapy.
- Conditions
- Adult Acute Megakaryoblastic Leukemia (M7), Adult Acute Minimally Differentiated Myeloid Leukemia (M0), Adult Acute Monoblastic Leukemia (M5a), Adult Acute Monocytic Leukemia (M5b)
- Trial name
- NCT02105116
- Testing
- fludarabine phosphate, cytarabine, donor lymphocytes
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Rutgers, The State University of New Jersey
Obesity, Sedentary
Effects of Obesity and Physical Inactivity on Pregnancy Outcomes
Regular maternal physical activity leads to the delivery of lighter, leaner infants. Higher birth weights and childhood obesity are both strong predictors for adult obesity, suggesting that the impact of maternal physical…
- Trial name
- NCT02039414
- Where
- United States
- Volunteers
- 40 people
- Sponsor
- Washington University School of Medicine
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
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
Hereditary Angioedema (HAE)
CINRYZE for the Treatment of Hereditary Angioedema Attacks in Children Under the Age of 12
The objectives of this study were to evaluate: (1) the dose response and (2) the pharmacokinetics (PK) and pharmacodynamics (PD) of intravenous (IV) administration of CINRYZE for the treatment of acute angioedema attacks in…
- Trial name
- NCT01095510
- Testing
- CINRYZE
- Where
- 11 sites in United States, Germany and 1 more
- Volunteers
- 9 people
- Sponsor
- Shire
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
Hemophilia A
Prophylaxis Study of Recombinant Factor VIII Manufactured Protein-Free (rAHF-PFM) in Patients With Hemophilia A
The primary purpose of this randomized, two-arm parallel clinical study in 66 previously treated patients with severe or moderately severe hemophilia A is to compare the rate of bleeding episodes for standard prophylaxis (20-40…
- Trial name
- NCT00243386
- Testing
- Antihemophilic factor, recombinant, manufactured protein-free
- Where
- 29 sites in United States, Austria and 8 more
- Volunteers
- 82 people
- Sponsor
- Baxalta now part of Shire
Prematurity, Necrotizing Enterocolitis
Collaborative Research Group for Necrotizing Enterocolitis
This proposal will test the hypothesis that synthesis and catabolism of epidermal growth factor (EGF), the genotype of the EGF gene, and the microbiome interact to influence EGF expression in infants at risk for necrotizing…
- Trial name
- NCT00828451
- Testing
- [5,5,5-2H3]leucine (stable isotope labeled leucine)
- Where
- United States
- Volunteers
- 29 people
- Sponsor
- Washington University School of Medicine
Retinitis Pigmentosa
A Dose Ranging Study to Evaluate the Safety and Potential Efficacy of rhNGF in Patients With Retinitis Pigmentosa (RP)
The primary objective of the study is to assess the safety and tolerability of two dose regimens of recombinant human nerve growth factor (rhNGF) eye drops solution administered over 6 months versus a vehicle control in patients…
- Trial name
- Lumos (NCT02110225)
- Testing
- rhNGF 60 µg/ml eye drops solution, rhNGF 180 µg/ml eye drops solution
- Where
- 5 sites in Italy
- Volunteers
- 50 people
- Sponsor
- Dompé Farmaceutici S.p.A
Anemia, Aplastic, Amegakaryocytic Thrombocytopenia
Partially Matched Stem Cell Transplantation for Patients With Refractory Severe Aplastic Anemia or Refractory Cytopenias
Due to an overall and disease free survival of 85% to 100%, allogeneic blood or bone marrow stem cell transplantation using an HLA matched sibling donor is the therapy of choice for patients with severe aplastic anemia (SAA).
- Conditions
- Anemia, Aplastic, Amegakaryocytic Thrombocytopenia, Diamond-Blackfan Anemia, Kostmann Syndrome
- Trial name
- NCT00244010
- Testing
- Allogeneic stem cell transplant
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- St. Jude Children's Research Hospital
Fabry Disease, Chronic Kidney Disease, Stage IV (Severe)
Severe Renal Disease Study in Fabry Patients Treated With Fabrazyme
This study was designed to determine appropriate treatment with Fabrazyme at a biweekly dose of either 1 mg/kg or 3 mg/kg in a population of patients with severe renal disease burden.
- Trial name
- NCT00837824
- Testing
- Fabrazyme (agalsidase beta)
- Where
- 9 sites in United States
- Volunteers
- 20 people
- Sponsor
- Genzyme, a Sanofi Company
Atopic Dermatitis
Study Evaluating the Mechanism of Action of PF-04965842 Monotherapy for Moderate-to-severe Atopic Dermatitis
B7451037 is a randomized, double-blind, placebo-controlled, parallel-group, Phase 2a study to investigate the mechanism of action of PF-04965842 by correlating efficacy outcomes with changes from baseline in key skin and blood…
- Trial name
- JADE MOA (NCT03915496)
- Testing
- PF-04965842 200 mg, PF-04965842 100 mg
- Where
- 11 sites in United States, Canada
- Volunteers
- 46 people
- Sponsor
- Pfizer
Tuberous Sclerosis
Adjunctive Ganaxolone Treatment (Part A) in TSC Followed by Long-term Treatment (Part B)
To assess preliminary safety and efficacy of ganaxolone as adjunctive therapy for the treatment of primary seizure types in patients with genetically- or clinically-confirmed TSC-related epilepsy through the end of the 12 week…
- Trial name
- TSC (NCT04285346)
- Testing
- Ganaxolone
- Where
- 7 sites in United States
- Volunteers
- 23 people
- Sponsor
- Marinus Pharmaceuticals
Phenylketonuria
A Phase 3 Substudy to Evaluate Executive Function in Adults With PKU Who Are Participating in the Phase 3 Study, 165-302
A Phase 3 substudy to evaluate executive function in adults with phenylketonuria who are participating in the phase 3 Study, 165-302. Approximately 100 subjects, aged ≥ 18 to ≤ 70 years old, with PKU will be enrolled.
- Trial name
- PRISM303 (NCT02468570)
- Testing
- Administration of CANTAB and Subject Global Assessment
- Where
- 4 sites in United States
- Volunteers
- 9 people
- Sponsor
- BioMarin Pharmaceutical
Leukemia, Myelodysplastic Syndromes
Helical Tomotherapy, Fludarabine Phosphate, and Melphalan Followed By Allo-HSCT in Hematological Malignancies
RATIONALE: Giving chemotherapy drugs, such as fludarabine phosphate and melphalan, and HT before a donor stem cell transplant helps stop the growth of cancer cells.
- Trial name
- NCT00544466
- Testing
- fludarabine phosphate, melphalan, allogeneic hematopoietic stem cell transplantation
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- City of Hope Medical Center
Inherited Mitochondrial Disease, Including Leigh Syndrome
Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease
To evaluate safety, tolerability and efficacy of cysteamine bitartrate delayed-release capsules (RP103) administered at a target maintenance dose of 1.3 g/m²/day in two divided doses, every 12 hours, for up to 6 months in…
- Trial name
- MITO-001 (NCT02023866)
- Testing
- Cysteamine Bitartrate
- Where
- 5 sites in United States
- Volunteers
- 36 people
- Sponsor
- Amgen
Abdominal Surgery, Thoracic Surgery
Parent/Nurse Controlled Analgesia in the Neonatal Intensive Care Unit
The aim of this study is to examine the safety and effectiveness of 2 morphine delivery systems for post-surgical neonates. The investigators hypothesize that this study will be feasible to conduct, and that neonates receiving…
- Trial name
- PNCA (NCT01823497)
- Testing
- Morphine
- Where
- United States
- Volunteers
- 36 people
- Sponsor
- Medical College of Wisconsin
Jaundice, Neonatal
Treatment of Neonatal Jaundice With Filtered Sunlight Phototherapy: Safety and Efficacy in African Neonates
The primary objective of this study is to determine the safety and efficacy of filtered sunlight phototherapy. Sunlight will be filtered by flexible (window-tinting) film.
- Trial name
- NCT01434810
- Testing
- Filtered-sunlight phototherapy, Conventional phototherapy
- Where
- Nigeria
- Volunteers
- 447 people, healthy volunteers welcome
- Sponsor
- University of Minnesota
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