Clinical trials: Newborn Diseases
Cystic Fibrosis
GLPG2737 on Top of Orkambi in Subjects With Cystic Fibrosis
This is a Phase IIa, multi-center, randomized, double-blind, placebo-controlled, parallel-group study to evaluate GLPG2737 administered orally b.i.d.
- Trial name
- PELICAN (NCT03474042)
- Testing
- GLPG2737
- Where
- 9 sites in Germany
- Volunteers
- 22 people
- Sponsor
- Lakefront Biotherapeutics NV
Cystic Fibrosis Related Diabetes, Cystic Fibrosis
Ivacaftor (Kalydeco) and Insulin in Cystic Fibrosis (CF)
This study is aimed at better understanding the impact of ivacaftor upon insulin and incretin secretion and glucose tolerance in patients with Cystic Fibrosis with a glycine (G551D) mutation.
- Trial name
- NCT02039986
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Children's Hospital of Philadelphia
Child, Osteotomy
Iliac Crest Bone Graft Harvesting for Pediatric Pelvic Osteotomy
The iliac crest is one of the most commonly used bone graft sources, especially in pediatric pelvic osteotomy operations for developmental dysplasia of the hip (DDH) and Legg-Calve-Perthes disease.
- Trial name
- NCT04425369
- Testing
- two-sided approaches for iliac crest bone graft
- Where
- China
- Volunteers
- 47 people
- Sponsor
- Yuxi Su
Cystic Fibrosis, Pancreatic Cystic Fibrosis
Behavioral & Nutritional Treatment to Help CF Preschoolers Grow
The primary objective of this NIH funded clinical trial is to conduct a multi-center, randomized, controlled trial comparing two interventions: a behavioral plus nutrition intervention to a nutrition intervention.
- Trial name
- NCT00241969
- Testing
- Behavioral and Nutrition Treatment, Education and Attention Control
- Where
- 5 sites in United States
- Volunteers
- 78 people
- Sponsor
- Children's Hospital Medical Center, Cincinnati
Pediatric Cancer, Solid Tumors
A Phase I Study of Lyso-thermosensitive Liposomal Doxorubicin and MR-HIFU for Pediatric Refractory Solid Tumors
This study is looking to determine the maximum tolerated dose (MTD) and recommended phase 2 dose (RP2D) of lyso-thermosensitive liposomal doxorubicin (LTLD) administered in combination with MR-HIFU in children with…
- Conditions
- Pediatric Cancer, Solid Tumors, Rhabdomyosarcoma, Ewing Sarcoma
- Trial name
- NCT02536183
- Testing
- Magnetic resonance high intensity focused ultrasound, Lyso-thermosensitive liposomal doxorubicin
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- AeRang Kim
Congenital Heart Disease, Pediatric ALL
HIIT in Youth With Congenital Heart Disease (MedBIKE)
Congenital heart disease (CHD), the most common birth defect, is present in nearly 1% of the population. CHD patients are associated with intense resource utilization and premature death in adulthood.
- Trial name
- NCT04575883
- Testing
- MedBIKE HIIT
- Where
- Canada
- Volunteers
- 44 people
- Sponsor
- University of Alberta
Tourette's Disorder
Safety and Tolerability Study of Once-weekly Oral Aripiprazole in Children and Adolescents With Tourette's Disorder
The goal of the current trial is to obtain long term efficacy, safety and tolerability data of once weekly aripiprazole in children and adolescents with Tourette's Disorder.
- Trial name
- NCT01416441
- Testing
- Aripiprazole
- Where
- 80 sites in United States, Bulgaria and 8 more
- Volunteers
- 170 people
- Sponsor
- Otsuka Pharmaceutical Development & Commercialization, Inc.
Charcot-Marie-Tooth Disease, Type IA
Biomarkers and Validation of Selected Outcome Measures (CMTBiomarker)
CMT is a rare disease for which novel treatments are being developed. Evaluation of intervention efficacy is hampered by slow progression and lack of sensitive outcome measures.
- Trial name
- NCT03386266
- Where
- Germany
- Volunteers
- 156 people
- Sponsor
- University Medical Center Goettingen
Methylmalonic Acidemia, Propionic Acidemia
Study of HST5040 in Subjects With Propionic or Methylmalonic Acidemia
This is an interventional study to assess the safety, PK, and efficacy of HST5040 in 12 subjects - 6 with Methylmalonic Acidemia (MMA) and 6 with Propionic Acidemia (PA).
- Trial name
- HERO (NCT04732429)
- Testing
- HST5040
- Where
- 16 sites in United States, Australia and 1 more
- Volunteers
- 26 people
- Sponsor
- HemoShear Therapeutics
Preeclampsia
PI4 - A Trial Assessing Metformin to Prolong Gestation in Preterm Preeclampsia
Preterm preeclampsia is a severe condition for both the mother and the fetus. Currently, the only treatment available to stop disease progression is termination/delivery of the fetus and placenta.
- Trial name
- PI4 (NCT06033131)
- Testing
- Metformin ER
- Where
- 18 sites in Finland, Norway and 1 more
- Volunteers
- 294 people
- Sponsor
- Lina Bergman
Genetic Disorders
Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema
The purpose of this single-center study is to explore the efficacy, safety, tolerability and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor in the treatment of acute attacks in patients with hereditary…
- Trial name
- NCT00261053
- Testing
- i.v. recombinant human C1 inhibitor
- Where
- Netherlands
- Volunteers
- 14 people
- Sponsor
- Pharming Technologies B.V.
Patent Foramen Ovale
Internal Microstructure of Patent Foramen Ovale Related to Stroke
This study aims to (1) investigate the internal microstructure of patent foramen ovale related to cryptogenic stroke, transient ischemic attack or migraine and determine the effectiveness of transcatheter closure in these…
- Trial name
- IMPORT (NCT04686253)
- Testing
- not appliable
- Where
- 2 sites in China
- Volunteers
- 1,200 people
- Sponsor
- China National Center for Cardiovascular Diseases
Primary Osteoarthritis, Post-Traumatic Arthritis
AVANTAGE® RELOAD Double Mobility Acetabular Cup -Clinical Study
This is a Post Marketing Clinical Follow Up study (PMCF) on the AVANTAGE RELOAD dual mobility system cup.
- Conditions
- Primary Osteoarthritis, Post-Traumatic Arthritis, Inflammatory Joint Disease, Femoral Neck Fracture
- Trial name
- NCT03357445
- Testing
- Avantage Reload cup
- Where
- 4 sites in France, Slovenia
- Volunteers
- 500 people
- Sponsor
- Zimmer Biomet
Alpha 1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
- Trial name
- NCT04167345
- Testing
- VX-814
- Where
- 28 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Fetal and Neonatal Alloimmune Thrombocytopenia
Phase 2 Study on the Pharmacokinetics and Safety of RLYB212 in Pregnant Women at Higher Risk for HPA-1a Alloimmunization
The purpose of this Phase 2 study is to assess the pharmacokinetics (PK) and safety of RLYB212 in HPA-1b/b pregnant women at higher risk for HPA-1a alloimmunization and FNAIT.
- Trial name
- NCT06435845
- Testing
- Anti-(integrin beta-3) human monoclonal antibody
- Where
- 3 sites in Netherlands, Norway and 1 more
- Volunteers
- 1 people
- Sponsor
- Rallybio
Haemophilia A, Haemophilia A With Inhibitors
A Research Study Investigating Mim8 in Adults and Adolescents With Haemophilia A With or Without Inhibitors
This study is investigating how Mim8 works compared to other medicines in people with haemophilia A, who either have inhibitors or do not have inhibitors.
- Trial name
- NCT05053139
- Testing
- NNC0365-3769 (Mim8)
- Where
- 163 sites in United States, Austria and 32 more
- Volunteers
- 281 people
- Sponsor
- Novo Nordisk A/S
Haematological Malignancy, Acute Leukemia
A Study to Assess a Physical Activity Program in Children, Adolescents and Young Adults Requiring Hematopoietic Stem Cell Allografts
To date, allogeneic haematopoietic stem cell transplantation (aHSCT) is the only curative treatment for many paediatric and young adult haematological pathologies (acute leukaemia, myelodysplastic syndromes, haemoglobinopathies…
- Conditions
- Haematological Malignancy, Acute Leukemia, Myelodysplastic Syndromes, Hemoglobinopathy in Children
- Trial name
- EVAADE (NCT04331483)
- Testing
- Physical activity program
- Where
- France
- Sponsor
- Centre Leon Berard
Metabolism, Inborn Errors
Cardiac Manifestation and Inherited Metabolic Diseases
The aim of this study is to report and describe cardiac manifestations associated with IMD in a cohort of patients followed in a reference center for rare diseases (Lausanne University Hospital, CHUV) from 2017-2020.
- Trial name
- NCT04999566
- Where
- Switzerland
- Volunteers
- 30 people
- Sponsor
- University of Lausanne
Whole Body Imaging, Magnetic Resonance Imaging
Whole Body Magnetic Resonance Imaging With Diffusion Weighted Imaging : Potential Role in Neurofibromatosis
Whole body MRI will be performed in patients with neurofibromatosis Type 1 PURPOSE 1: To determine the total tumor load (neurofibroma) and to diagnose plexiform neurofibromas or malignant peripheral nerve sheath tumors.
- Conditions
- Whole Body Imaging, Magnetic Resonance Imaging, Neurofibromatosis 1, Diffusion Magnetic Resonance Imaging
- Trial name
- NEFIMAR (NCT01777451)
- Testing
- Additional imaging or surgery
- Where
- Belgium
- Volunteers
- 30 people
- Sponsor
- Universitaire Ziekenhuizen KU Leuven
Cystic Fibrosis, Infection
Anti-Inflammatory Pulmonal Therapy of CF-Patients With Amitriptyline and Placebo
Our data indicate that the CFTR-molecule functions as a transporter for sphingosine-1-phosphate and sphingosine or regulates the uptake of these sphingolipids by epithelial cells.
- Conditions
- Cystic Fibrosis, Infection, Pseudomonas Aeruginosa
- Trial name
- NCT00515229
- Testing
- amitriptyline
- Where
- Germany
- Volunteers
- 18 people
- Sponsor
- University Hospital Tuebingen
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