Clinical trials: Congenital Abnormality of Kidney
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
X-Linked Hypophosphatemia
A Study to Describe the Lived Experience of XLH for Adolescents at End of Skeletal Growth
An observational, prospective, mixed-methods study involving the integration of quantitative and qualitative data exploring the lived experience of burosumab-treated adolescents with XLH at the end of skeletal growth.
- Trial name
- NCT05181839
- Testing
- Burosumab
- Where
- 14 sites in France, Germany and 3 more
- Volunteers
- 25 people
- Sponsor
- Kyowa Kirin Pharmaceutical Development Ltd
Neuroblastoma, Brain Tumor
Efficacy of Prophylactic Itraconazole in High-Dose Chemotherapy and Autologous Hematopoietic Stem Cell Transplantation
The purpose of this study is to investigate whether the prophylactic use of itraconazole is a better option than empirical use of itraconazole in the management (prevention and treatment) of fungal infection associated with…
- Conditions
- Neuroblastoma, Brain Tumor, Retinoblastoma, Wilms Tumor
- Trial name
- NCT00336531
- Testing
- itraconazole
- Where
- South Korea
- Volunteers
- 100 people
- Sponsor
- Samsung Medical Center
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia
Palliative Care Needs of Children With Rare Diseases and Their Families
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare for future medical decision making.
- Conditions
- Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome
- Trial name
- FACE-Rare (NCT06938542)
- Testing
- Family Centered pediatric palliative care for family caregivers of children with rare diseases.
- Where
- United States
- Volunteers
- 480 people, healthy volunteers welcome
- Sponsor
- Children's National Research Institute
Chronic Kidney Failure, Congenital Heart Defects
Chronic Kidney Disease in Teenagers With Congenital Cardiac Disease
The prevalence of chronic kidney injury (CKD) following cardiac surgery during childhood is not known, but there is evidence of CKD in young adults.
- Trial name
- PRECARDIO (NCT01845402)
- Testing
- blood and urine samples
- Where
- France
- Volunteers
- 120 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Cancer
Valacyclovir in Preventing Cytomegalovirus Infection in Patients Who Are Undergoing Donor Stem Cell Transplantation
RATIONALE: Antivirals such as valacyclovir act against viruses and may be effective in preventing cytomegalovirus. It is not yet known if valacyclovir is effective in preventing cytomegalovirus in patients undergoing stem cell…
- Trial name
- NCT00045292
- Testing
- acyclovir, acyclovir sodium, valacyclovir
- Where
- 6 sites in United States
- Sponsor
- Fred Hutchinson Cancer Center
Desmoplastic Small Round Cell Tumor, Osteosarcoma
Trastuzumab Deruxtecan (DS-8201a) for the Treatment of Recurrent or Refractory Osteosarcoma, Wilms Tumor, and Desmoplastic Small Round Cell Tumor
This phase I/II trial studies the effects of trastuzumab deruxtecan (DS-8201a) in treating patients with osteosarcoma, Wilms tumor (WT) or desmoplastic small round cell tumor (DSRCT) that is newly diagnosed or has come back after…
- Conditions
- Desmoplastic Small Round Cell Tumor, Osteosarcoma, Recurrent Desmoplastic Small Round Cell Tumor, Recurrent Kidney Wilms Tumor
- Trial name
- NCT04616560
- Testing
- Biospecimen Collection, Computed Tomography, Echocardiography Test
- Where
- 32 sites in United States
- Volunteers
- 55 people
- Sponsor
- National Cancer Institute (NCI)
Hemophilia A
Assessment of the Risk of Inhibitor Formation in Previously Treated Patients With Severe Hemophilia A
Most transient inhibitor formation, if any, will develop within the first 4 weeks. The study is to further monitor whether participants with severe Hemophilia A will develop inhibitors or antibodies at the later stage when…
- Trial name
- NCT00621673
- Testing
- Kogenate (BAY 14-2222)
- Where
- 2 sites in United States
- Volunteers
- 1 people
- Sponsor
- Bayer
Cancer
Caspofungin Acetate Compared With Amphotericin B Liposomal in Treating Patients With Persistent Fever and Neutropenia Following Cancer Treatment
RATIONALE: Caspofungin acetate or amphotericin B liposomal may be effective in preventing or controlling fever and neutropenia caused by chemotherapy, bone marrow transplantation, or peripheral stem cell transplantation.
- Trial name
- NCT00008359
- Testing
- caspofungin acetate, liposomal amphotericin B
- Where
- United States
- Sponsor
- Memorial Sloan Kettering Cancer Center
Sickle Cell Disease, Pulmonary Disease
Cooperative Assessment of Late Effects for SCD Curative Therapies
Sickle Cell Disease is one of the most common genetic diseases in the United States, occurring in approximately 1 in 400 births. Approximately 100,000 individuals are diagnosed with SCD in the United States.
- Conditions
- Sickle Cell Disease, Pulmonary Disease, Renal Disease, Heart Disease
- Trial name
- COALESCE (NCT05153967)
- Where
- 5 sites in United States
- Volunteers
- 377 people
- Sponsor
- Vanderbilt University Medical Center
Hyperoxaluria, Primary
An Extension Study to Evaluate the Long-term Efficacy and Safety of Oxabact in Patients With Primary Hyperoxaluria
Open label extension study of Oxabact OC5 in patients with primary hyperoxaluria
- Trial name
- ePHex-OLE (NCT03938272)
- Testing
- Oxabact OC5 - Oxalobacter formigenes Strain HC-1
- Where
- 8 sites in United States, Belgium and 4 more
- Volunteers
- 22 people
- Sponsor
- OxThera
Recurrent Solid Tumor, Recurrent Ewing Sarcoma
Study of Onivyde With Talazoparib or Temozolomide in Children With Recurrent Solid Tumors and Ewing Sarcoma
The phase I portion of this study is designed for children or adolescents and young adults (AYA) with a diagnosis of a solid tumor that has recurred (come back after treatment) or is refractory (never completely went away).
- Conditions
- Recurrent Solid Tumor, Recurrent Ewing Sarcoma, Recurrent Hepatoblastoma, Recurrent Malignant Germ Cell Tumor
- Trial name
- NCT04901702
- Testing
- Onivyde, Talazoparib, Temozolomide
- Where
- 10 sites in United States, Canada
- Volunteers
- 90 people
- Sponsor
- St. Jude Children's Research Hospital
Lymphoma, Childhood Lymphoma
Study of Genistein in Pediatric Oncology Patients (UVA-Gen001)
Toxicities related to pediatric cancer treatment can lead to significant illness, organ damage, treatment delays, increased health care cost, and decrease in quality of life.
- Conditions
- Lymphoma, Childhood Lymphoma, Solid Tumor, Childhood Solid Tumor
- Trial name
- UVA-Gen001 (NCT02624388)
- Testing
- Genistein
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Virginia
Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor
Temozolomide and O6-benzylguanine in Treating Children With Solid Tumors
RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining more than one drug may kill more tumor cells.
- Conditions
- Brain and Central Nervous System Tumors, Childhood Germ Cell Tumor, Extragonadal Germ Cell Tumor, Kidney Cancer
- Trial name
- NCT00020150
- Testing
- O6-benzylguanine, temozolomide
- Where
- United States
- Sponsor
- National Cancer Institute (NCI)
Primary Hyperoxaluria
Study to Evaluate the Efficacy and Safety of Oxabact (OC5) in Patients With Primary Hyperoxaluria
The purpose of this study is to determine if Oxalobacter formigenes is effective at lowering urinary oxalate levels in patients with primary hyperoxaluria.
- Trial name
- NCT02012985
- Testing
- Oxabact OC5 capsules
- Where
- 8 sites in France, Germany and 1 more
- Volunteers
- 28 people
- Sponsor
- OxThera
Sarcoma, Wilm's Tumor
Phase I Study of Ipilimumab (Anti-CTLA-4) in Children and Adolescents With Treatment-Resistant Cancer
This study will examine the safety and efficacy of ipilimumab-an experimental cancer treatment drug used to boost immune response-in children, adolescents, and young adults.
- Conditions
- Sarcoma, Wilm's Tumor, Lymphoma, Neuroblastoma
- Trial name
- NCT01445379
- Testing
- Ipilimumab
- Where
- 3 sites in United States
- Volunteers
- 33 people
- Sponsor
- National Cancer Institute (NCI)
Congenital Heart Disease, Surgery--Complications
A Predictive Score System for AKI Following Pediatric Cardiac Surgery
Acute kidney injury (AKI) has been recognized as a typical post- operative complication among the children undergoing surgical repair of a congenital cardiac defect.
- Conditions
- Congenital Heart Disease, Surgery--Complications, Acute Kidney Injury
- Trial name
- NCT05489263
- Testing
- No intervention
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- China National Center for Cardiovascular Diseases
Neuroblastoma, Sarcoma
A Study of Cabozantinib as a Maintenance Agent to Prevent Progression or Recurrence in High-Risk Pediatric Solid Tumors
This study will expand the types of pediatric cancers being evaluated for response to cabozantinib. The current COG study is restricted to Ewing sarcoma, osteosarcoma, rhabdomyosarcoma, Wilms tumor, and a handful of uncommon…
- Trial name
- NCT05135975
- Testing
- Cabozantinib
- Where
- 6 sites in United States
- Volunteers
- 86 people
- Sponsor
- Nationwide Children's Hospital
Kidney, Polycystic
HALT Progression of Polycystic Kidney Disease Study A
The efficacy of interruption of the renin-angiotensin-aldosterone system (RAAS) on the progression of cystic disease and on the decline in renal function in autosomal dominant kidney disease (ADPKD) will be assessed in two…
- Trial name
- HALT PKD A (NCT00283686)
- Testing
- Lisinopril, Telmisartan, Standard Blood Pressure Control
- Where
- 7 sites in United States
- Volunteers
- 558 people
- Sponsor
- National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK)
Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M), Nasopharyngeal Carcinoma
Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed/Refractory or Metastatic Solid Tumors
There is a significant unmet medical need for effective therapies for pediatric relapsed/refractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug…
- Conditions
- Head and Neck Squamous Cell Carcinoma (HNSCC) - Recurrent/Metastatic (R/M), Nasopharyngeal Carcinoma, Lymphoepithelial Carcinoma, Rhabdomyosarcoma
- Trial name
- MRG003 (NCT07584499)
- Testing
- Becotatug Vedotin
- Where
- China
- Volunteers
- 51 people
- Sponsor
- Sun Yat-sen University