Clinical trials: Newborn Diseases
Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Basophilic Leukemia
Eltrombopag Olamine in Improving Platelet Recovery in Older Patients With Acute Myeloid Leukemia Undergoing Chemotherapy
This phase II trial studies how well eltrombopag olamine works in improving the recovery of platelet counts in older patients with Acute Myeloid Leukemia (AML) undergoing induction (the first treatment given for a disease)…
- Conditions
- Acute Myeloid Leukemia With Multilineage Dysplasia Following Myelodysplastic Syndrome, Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7)
- Trial name
- NCT02071901
- Testing
- eltrombopag olamine
- Where
- United States
- Volunteers
- 31 people
- Sponsor
- Case Comprehensive Cancer Center
Huntington Disease
PHysical Activity and Exercise Outcomes in Huntington's Disease
Huntington's disease (HD) is a genetic, degenerative neurological disease that affects individuals in their third-fourth decade of life and individuals can live 15-20 years with manifest HD.
- Trial name
- PACE-HD (NCT03344601)
- Testing
- physical activity
- Where
- 6 sites in United States, Germany and 1 more
- Volunteers
- 116 people
- Sponsor
- Cardiff University
Tuberous Sclerosis, Neurofibromatoses
Topical Rapamycin Therapy to Alleviate Cutaneous Manifestations of Tuberous Sclerosis Complex (TSC) and Neurofibromatosis I (NF1)
This study is a prospective, randomized, double-blind, placebo-controlled evaluation of the safety of a topically applied formulation of rapamycin to cutaneous fibromatous lesions in subjects with Tuberous Sclerosis Complex (TSC)…
- Conditions
- Tuberous Sclerosis, Neurofibromatoses, Angiofibroma, Neurofibroma
- Trial name
- NCT01031901
- Testing
- Skincerity, Skincerity plus sirolimus/rapamycin, Skinercity plus sirolimus/rapamycin
- Where
- United States
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- The University of Texas Health Science Center, Houston
Glycogen Storage Disease Type I
Dietary Treatment Strategies and Metabolic Control in Glycogen Storage Disease Type I
The present project will specifically assess metabolic effects of dietary interventions with controlled intake of fructose and fructose/galactose in GSDI, with the aim to provide evidence whether relaxed dietary restrictions of…
- Trial name
- GSD-DIET (NCT06852612)
- Testing
- Fructose, Fructose and galactose
- Where
- 2 sites in Switzerland
- Volunteers
- 20 people
- Sponsor
- Insel Gruppe AG, University Hospital Bern
Recessive Dystrophic Epidermolysis Bullosa
A Study of PTR-01 in Recessive Dystrophic Epidermolysis Bullosa
Protocol PTR-01-002 is a 3-part Phase 2, open-label study of PTR-01. While new patients will be enrolled, priority will be given to patients that satisfactorily completed study PTR-01-001.
- Trial name
- NCT04599881
- Testing
- PTR-01
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Phoenix Tissue Repair, Inc.
Fasting, Fed
Use of Breath Acetone aa a Marker of Energy Balance
This study aims at assessing whether breath acetone concentration is a good bio-marker of instantaneous energy balance in healthy subjects.
- Trial name
- ACEX (NCT03390881)
- Testing
- Fasting condition, Sugar condition, Fat condition
- Where
- Switzerland
- Volunteers
- 8 people, healthy volunteers welcome
- Sponsor
- University of Lausanne
Urinary Incontinence
Clinical Study of External Electrical Stimulation for Male Incontinence
The purpose of this research is to evaluate the effectiveness of the Elidah neuromuscular stimulation treatment to reduce incontinence in men post-prostatectomy.
- Trial name
- NCT06620419
- Testing
- Perineal-applied electrical stimulation
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Elidah, Inc.
Hyperinsulinemic Hypoglycemia, Persistent, Congenital Hyperinsulinism
Study of the Use of [18F]-DOPA in Hyperinsulinemic Hypoglycemia
The primary objective of this study is to evaluate the utility of [18F]-DOPA PET to provide improved presurgical planning and distinguish between focal and diffuse forms of HI.
- Conditions
- Hyperinsulinemic Hypoglycemia, Persistent, Congenital Hyperinsulinism, Persistent Hyperinsulinemic Hypoglycemia of Infancy (PHHI)
- Trial name
- [18F]-DOPA (NCT02533219)
- Testing
- 18 F-DOPA
- Where
- United States
- Sponsor
- Baylor College of Medicine
Parkinson's Disease, Parkinsonian Disorders
Gait Analysis in Neurological Disease
The purpose of this study is to investigate whether speed-dependent measures of gait (so called gait signatures) can be identified in patients with neurological conditions that affect gait, particularly in subjects with…
- Conditions
- Parkinson's Disease, Parkinsonian Disorders, Atypical Parkinson Disease, Progressive Supranuclear Palsy
- Trial name
- NCT02994719
- Testing
- Anti-Parkinson medication, Deep Brain Stimulation
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Beth Israel Deaconess Medical Center
0.5-14 Year Old Children With Nephroblastoma
Combination Therapy of Anthracyclines for Children With Nephroblastoma
This study aims to estimate the efficacy and side effects of study drugs in children with nephroblastoma who are treated with combination therapy.
- Trial name
- NCT03892330
- Testing
- Vincristine, Oxytetracycline/ Cyclophosphamide, Liposomal doxorubicin
- Where
- China
- Volunteers
- 120 people
- Sponsor
- Shengjing Hospital
Glycogen Storage Disease Type III
Study to Evaluate Biomarkers and Clinical Manifestations in Individuals With Glycogen Storage Disease Type III (GSD III)
The primary objective of this study is to evaluate potential biomarkers of GSD III.
- Trial name
- NCT04574830
- Testing
- No Intervention
- Where
- 4 sites in United States
- Volunteers
- 18 people
- Sponsor
- Ultragenyx Pharmaceutical Inc
Multicystic Dysplastic Kidney, Polycystic Kidney Disease
Amnioinfusion for Fetal Renal Failure
The goal of this clinical trial is to learn if serial amnioinfusions can improve the chances of survival for fetuses with severe kidney problems that cause low amniotic fluid (anhydramnios).
- Conditions
- Multicystic Dysplastic Kidney, Polycystic Kidney Disease, Fetal Renal Anomaly, Anhydramnios
- Trial name
- NCT06728228
- Testing
- Amnioinfusion
- Where
- United States
- Volunteers
- 35 people
- Sponsor
- Fetal Care Center, PLLC
Recessive Dystrophic Epidermolysis Bullosa
A Phase 1/2 Trial of PTR-01 in Adult Patients With Recessive Dystrophic Epidermolysis Bullosa (RDEB)
Protocol PTR-01-001 is a Phase 1/2 study of PTR-01. The study is divided into an up to 4-week Screening Period, a 10-week Treatment Period and an 8-week Follow-up Period.
- Trial name
- PTR-01-001 (NCT03752905)
- Testing
- PTR-01, Normal saline
- Where
- 3 sites in United States
- Volunteers
- 12 people
- Sponsor
- Phoenix Tissue Repair, Inc.
Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Basophilic Leukemia
Vorinostat, Cytarabine, and Etoposide in Treating Patients With Relapsed and/or Refractory Acute Leukemia or Myelodysplastic Syndromes or Myeloproliferative Disorders
This phase I trial is studying the side effects and best dose of vorinostat when given together with cytarabine and etoposide in treating patients with relapsed or refractory acute leukemia or myelodysplastic syndromes or…
- Conditions
- Accelerated Phase Chronic Myelogenous Leukemia, Adult Acute Basophilic Leukemia, Adult Acute Eosinophilic Leukemia, Adult Acute Megakaryoblastic Leukemia (M7)
- Trial name
- NCT00357305
- Testing
- vorinostat, cytarabine, etoposide
- Where
- 2 sites in United States
- Volunteers
- 25 people
- Sponsor
- National Cancer Institute (NCI)
Heart Defects, Congenital
Rejuvenated, Washed Packed Red Blood Cells in Pediatric Cardiac Surgery
Blood transfusion is nearly always needed during open heart surgery in children less than 15 kg (35 pounds). The purpose of the red blood cells in the blood is to deliver oxygen to the organs and tissues of the body.
- Trial name
- NCT02485366
- Testing
- Rejuvesol, Cardiac surgery
- Where
- United States
- Sponsor
- Duke University
Neonatal Hyperbilirubinemia, Treatment Decisions
Bilirubin Thresholds in Preterm Infants on Neonatal Intensive CarE Units: The B-NICE Trial
Rationale: Neonatal hyperbilirubinemia is highly prevalent in very preterm infants born <30 weeks. Since 2008, uniform Dutch phototherapy thresholds for preterm infants have been used nationwide, largely based on consensus.
- Conditions
- Neonatal Hyperbilirubinemia, Treatment Decisions, Neurodevelopmental Outcome, Phototherapy
- Trial name
- B-NICE (NCT07674537)
- Testing
- Phototherapy, Exchange Transfusion
- Where
- Netherlands
- Volunteers
- 680 people
- Sponsor
- University Medical Center Groningen
Arteriovenous Malformations
Pilot Study on Trametinib for Surgical Unruptured AVMs
Arteriovenous malformation (AVM) is a tangle of abnormal vessels that can progress through life and cause significant bleeding, deformity, pain, and deficits in day-to-day activities.
- Trial name
- NCT06098872
- Testing
- Trametinib tablet
- Where
- Canada
- Sponsor
- University Health Network, Toronto
Huntington Disease
Symptomatic Therapy for Patients With Huntington's Disease
Based on the previous symptomatic treatment of Chinese HD patients, this study intends to further optimize the treatment regimen of Chinese HD patients so as to further improve the prognosis of patients.
- Trial name
- NCT04071639
- Testing
- Haloperidol 2Mg Tab, Risperidone 1Mg Tab, Zoloft 50Mg Tablet
- Where
- China
- Volunteers
- 60 people
- Sponsor
- Second Affiliated Hospital, Zhejiang University, School of Medicine
Sickle Cell Disease (SCD)
Self-administered Acupressure in Patients With Sickle Cell Disease
The proposed research is to determine the clinical efficacy and neurobiological mechanisms of acupressure analgesia in patients with sickle cell disease (SCD).
- Trial name
- NCT07875777
- Testing
- Verum Acupressure (using AcuWand)
- Where
- 2 sites in United States
- Volunteers
- 300 people
- Sponsor
- University of Cincinnati
Genu Varum, Genu Valgus
IMU-based Assessment of Motor Control in a Population of Young Subjects With Paramorphisms and Dysmorphisms
While various complex pathologies of the developmental age, such as Infantile Cerebral Palsy or Neuromuscular Diseases, are notoriously considered causes of alteration of locomotor development, it is scarcely known whether…
- Conditions
- Genu Varum, Genu Valgus, Flat Feet, Cavus Foot
- Trial name
- MOTOR-CHILD (NCT05763901)
- Testing
- Clinical tests and motor control evaluation using Inertial Measurement Units
- Where
- Italy
- Volunteers
- 151 people
- Sponsor
- Istituto Ortopedico Rizzoli
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