Clinical trials: Neonates
Hemophilia A
A Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Prophylactic Emicizumab Versus no Prophylaxis in Hemophilia A Participants With Inhibitors
This multicenter, open-label study will evaluate the safety, efficacy and pharmacokinetics of prophylactic emicizumab treatment in participants previously treated with episodic or prophylactic bypassing agents.
- Trial name
- HAVEN 1 (NCT02622321)
- Testing
- Emicizumab, rFVIIa, aPCC
- Where
- 44 sites in United States, Australia and 12 more
- Volunteers
- 113 people
- Sponsor
- Hoffmann-La Roche
Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis
An Extension Trial of Inclisiran in Participants With Cardiovascular Disease and High Cholesterol
This clinical study was designed to assess the efficacy, safety, and tolerability of long-term dosing of inclisiran and evolocumab given as subcutaneous injections in participants with high cardiovascular risk and elevated…
- Conditions
- Atherosclerotic Cardiovascular Disease, Symptomatic Atherosclerosis, Type2 Diabetes, Familial Hypercholesterolemia
- Trial name
- ORION-3 (NCT03060577)
- Testing
- Inclisiran, Evolocumab
- Where
- 52 sites in United States, Canada and 3 more
- Volunteers
- 382 people
- Sponsor
- Novartis Pharmaceuticals
Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma
Tazemetostat in Treating Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphoma, or Histiocytic Disorders With EZH2, SMARCB1, or SMARCA4 Gene Mutations (A Pediatric MATCH Treatment Trial)
This phase II Pediatric MATCH trial studies how well tazemetostat works in treating patients with brain tumors, solid tumors, non-Hodgkin lymphoma, or histiocytic disorders that have come back (relapsed) or do not respond to…
- Conditions
- Advanced Malignant Solid Neoplasm, Ann Arbor Stage III Hodgkin Lymphoma, Ann Arbor Stage III Non-Hodgkin Lymphoma, Ann Arbor Stage IV Hodgkin Lymphoma
- Trial name
- NCT03213665
- Testing
- Tazemetostat
- Where
- 117 sites in United States, Puerto Rico
- Volunteers
- 20 people
- Sponsor
- National Cancer Institute (NCI)
Cystic Fibrosis, Motor Neuron Disease
MN4000 for Treatment of CF and MND Patients in the Home Setting
The study was a non-randomized open label pilot study. It was an observational design conducted at one (1) site in the US. All enrolled subjects received treatment with the MN4000.
- Conditions
- Cystic Fibrosis, Motor Neuron Disease, Airway Clearance Impairment
- Trial name
- NCT03797677
- Testing
- MN 4000
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Hill-Rom
Hereditary Angioedema
Open-Label C1 Esterase Inhibitor (C1INH-nf) for the Prevention of Acute Hereditary Angioedema (HAE) Attacks
The study objective was to evaluate the safety and efficacy of prophylactic use of C1INH-nf for the prevention of acute HAE attacks.
- Trial name
- CHANGE 3 (NCT00462709)
- Testing
- C1 esterase inhibitor [human] (C1INH-nf)
- Where
- 48 sites in United States
- Volunteers
- 146 people
- Sponsor
- Shire
Klinefelter Syndrome, XXY Syndrome
Study of Psychological and Motor Effects of Testosterone in Adolescents With XXY/Klinefelter Syndrome
The purpose of this study is to determine if testosterone replacement therapy leads to changes in psychological factors and/or motor skills in adolescent males with 47,XXY (also called Klinefelter syndrome).
- Trial name
- TestoXXY/KS (NCT01585831)
- Testing
- Testosterone gel 1%
- Where
- United States
- Volunteers
- 48 people
- Sponsor
- University of Colorado, Denver
Dyslipidemias, High Cholesterol
Evaluate the Effect of Obicetrapib in Patients With HeFH on Top of Maximum Tolerated Lipid-Modifying Therapies.
This study will be a placebo-controlled, double-blind, randomized, phase 3 study to Evaluate the Efficacy, Safety, and Tolerability of Obicetrapib in Participants with a History of Heterozygous Familial Hypercholesterolemia…
- Conditions
- Dyslipidemias, High Cholesterol, Hypercholesterolemia, Familial Hypercholesterolemia
- Trial name
- BROOKLYN (NCT05425745)
- Testing
- Obicetrapib
- Where
- 96 sites in United States, Canada and 8 more
- Volunteers
- 354 people
- Sponsor
- NewAmsterdam Pharma
ALPS, Hypersplenism
Valproic Acid (Depakote[Registered Trademark]) to Treat Autoimmune Lymphoproliferative Syndrome (ALPS)
This study will test whether valproic acid (Depakote[Registered Trademark]) can shrink enlarged lymph glands and spleen in patients with autoimmune lymphoproliferative syndrome (ALPS).
- Conditions
- ALPS, Hypersplenism, Lymphadenopathy
- Trial name
- NCT00605657
- Testing
- Valproic Acid, CT Scan, Blood Sample
- Where
- United States
- Volunteers
- 6 people
- Sponsor
- Koneti Rao
Dermatitis, Atopic
Safety and Efficacy of AN2728 Topical Ointment, 2% in Children, Adolescents, and Adults (Aged 2 Years and Older) With Atopic Dermatitis
The purpose of this study is to investigate the safety and efficacy of AN2728 Topical Ointment, 2% in children, adolescents, and adults (ages 2 years and older) with atopic dermatitis.
- Trial name
- NCT02118792
- Testing
- AN2728 Topical Ointment, 2%, Matching vehicle control
- Where
- United States
- Volunteers
- 764 people
- Sponsor
- Pfizer
Primary Immunodeficiency Disease
Study to Investigate Efficacy, Safety and Pharmacokinetics of BT595 in Subjects With PID
This Phase III clinical study is to test efficacy, safety and pharmacokinetics of BT595 in treating patients with Primary Immunodeficiency (PID)
- Trial name
- NCT02810444
- Testing
- IgG Next Generation (BT595)
- Where
- 19 sites in United States, Germany and 3 more
- Volunteers
- 81 people
- Sponsor
- Biotest
Pompe Disease
Extension Study for Patients Who Have Participated in a BMN 701 Study
This is a Phase 2 open-label, multiple dose study of BMN 701 administered by IV infusion every 2 weeks (qow) to patients with late-onset Pompe disease.
- Trial name
- NCT01435772
- Testing
- BMN 701
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 21 people
- Sponsor
- BioMarin Pharmaceutical
Cerebral Palsy, Spina Bifida
The Effectiveness of Participation-focused Interventions on Body Functions of Youth With Physical Disabilities
Youth with physical disabilities face greater restrictions to participation in community-based activities than their typically developing peers, which can lead to poor health outcomes.
- Conditions
- Cerebral Palsy, Spina Bifida, Muscular Dystrophies, Musculoskeletal Disorder
- Trial name
- NCT03851107
- Testing
- Engagement in 6-week community-based activity program
- Where
- Canada
- Volunteers
- 8 people
- Sponsor
- McGill University
Hemophilia A, Hemophilia B
Study of the Efficacy and Safety PF-06741086 in Adult and Teenage Participants With Severe Hemophilia A or Moderately Severe to Severe Hemophilia B
Treatment with PF-06741086 is anticipated to demonstrate a clinically relevant advantage and/or a major contribution to patient care in comparison to current methods of treatment for hemophilia A or B because it works differently…
- Trial name
- NCT03938792
- Testing
- PF-06741086
- Where
- 62 sites in United States, Bulgaria and 18 more
- Volunteers
- 188 people
- Sponsor
- Pfizer
Electroencephalographic Neonatal Seizures
A Study to Test the Pharmacokinetics, Efficacy, and Safety of Brivaracetam in Newborns With Repeated Electroencephalographic Seizures
The purpose of the study is to evaluate the pharmacokinetics (PK) of brivaracetam (BRV) in neonates who have seizures that are not adequately controlled with previous antiepileptic drug (AED) treatment, and to identify the…
- Trial name
- NCT03325439
- Testing
- Brivaracetam (BRV) intravenous (iv), Brivaracetam (BRV) oral
- Where
- 12 sites in Belgium, Czechia and 5 more
- Volunteers
- 9 people
- Sponsor
- UCB Biopharma SRL
Pregnancy Related
Hospital Waterbirth Trial to Measure Maternal and Neonatal Outcomes
This study aims to explore outcomes of waterbirth in comparison to conventional land birth for low-risk healthy women and neonates in a hospital setting in Milwaukee, WI.
- Trial name
- NCT05175599
- Testing
- Water Birth
- Where
- United States
- Volunteers
- 186 people, healthy volunteers welcome
- Sponsor
- Wake Forest University Health Sciences
Leukemia
S0106 Cytarabine and Daunorubicin w/ or w/o Gemtuzumab Followed By HD Cytarabine and Either Gemtuzumab or Nothing in de Novo AML
RATIONALE: Drugs used in chemotherapy, such as cytarabine and daunorubicin, work in different ways to stop cancer cells from dividing so they stop growing and die.
- Trial name
- NCT00085709
- Testing
- gemtuzumab ozogamicin, observation, Cytosine arabinoside
- Where
- 279 sites in United States, Canada and 1 more
- Volunteers
- 637 people
- Sponsor
- SWOG Cancer Research Network
Regional Saturation of Oxygen (rSO2)
Performance Comparison of Masimo O3 Regional Oximetry Sensors in Neonates
The aim of the study is to compare the performance of the O3 cerebral oximeter and sensor to a 510(k) cleared NIRS (Near-Infrared Spectroscopy) device on neonatal subjects based on data collected from the cutaneously placed…
- Trial name
- NCT03828487
- Testing
- Masimo O3 Neonatal Sensors, 510(k) cleared sensor
- Where
- United States
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- Masimo Corporation
Osteogenesis Imperfecta (OI)
Open-label Extension of Study 20130173 of Denosumab in Children and Young Adults With Osteogenesis Imperfecta
To evaluate long-term safety of denosumab in children/young adults with pediatric osteogenesis imperfecta (OI) who completed the prior study 20130173 (NCT02352753).
- Trial name
- NCT03638128
- Testing
- Denosumab, Alternative osteoporosis medications
- Where
- 22 sites in United States, Australia and 10 more
- Volunteers
- 75 people
- Sponsor
- Amgen
Gastric Adenocarcinoma
Derazantinib Alone or in Combination With Paclitaxel, Ramucirumab or Atezolizumab in Gastric Adenocarcinoma
The purpose of this study was to evaluate the efficacy of derazantinib monotherapy or derazantinib in combination with paclitaxel and ramucirumab in patients with gastric adenocarcinoma (GAC) i.e.
- Trial name
- FIDES-03 (NCT04604132)
- Testing
- Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
- Where
- 81 sites in United States, Argentina and 13 more
- Volunteers
- 47 people
- Sponsor
- Basilea Pharmaceutica
May-Thurner Syndrome, Peripheral Vascular Disease
BARD® The VENOVO™ Venous Stent Study for Treatment of Iliofemoral Occlusive Disease
The BARD® Venovo™ Venous Stent Study is a non-randomized clinical study intended to collect confirmatory evidence of the safety and effectiveness of the Venous Stent for the treatment of iliofemoral occlusive disease.
- Conditions
- May-Thurner Syndrome, Peripheral Vascular Disease, Venous Disease, Iliofemoral Occlusive Disease
- Trial name
- VERNACULAR (NCT02655887)
- Testing
- VENOVO™ Venous Stent
- Where
- 21 sites in United States, Australia and 5 more
- Volunteers
- 170 people
- Sponsor
- C. R. Bard