Clinical trials: NPC
Lysosomal Acid Lipase Deficiency, Wolman Disease
Safety, Tolerability, Efficacy, Pharmacokinetics, and Pharmacodynamics of Sebelipase Alfa in Children With Growth Failure Due to Lysosomal Acid Lipase Deficiency
This was an open-label, repeat-dose, intra-participant dose-escalation study of SBC-102 (sebelipase alfa) in children with growth failure due to lysosomal acid lipase (LAL) Deficiency.
- Trial name
- NCT01371825
- Testing
- Sebelipase alfa (SBC-102)
- Where
- 7 sites in United States, Egypt and 3 more
- Volunteers
- 9 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Niemann-Pick Disease, Type C1
Safety and Efficacy of Intravenous Trappsol Cyclo (HPBCD) in Niemann-Pick Type C Patients
This research study is being conducted to find out whether Trappsol® Cyclo™, an experimental treatment for people with Niemann-Pick disease Type C1 (NPC-1) is safe at 3 different dose levels and what effects it has on people who…
- Trial name
- NCT02912793
- Testing
- Hydroxypropyl-beta-cyclodextrin
- Where
- 5 sites in Israel, Sweden and 1 more
- Volunteers
- 12 people
- Sponsor
- Cyclo Therapeutics, Inc.
Niemann-Pick Disease, Type C
Open-label Study of VTS-270 in Participants With Neurologic Manifestations of Niemann-Pick Type C1
This is a multicenter, multinational, open-label study of VTS-270 to evaluate the long-term safety and tolerability of VTS-270 (2-hydroxypropyl-β-cyclodextrin) in participants transitioning from Study VTS301 (Parts A/B…
- Trial name
- NCT03879655
- Testing
- VTS-270
- Where
- Costa Rica
- Volunteers
- 2 people
- Sponsor
- Mandos LLC
Nasopharyngeal Cancer
Randomised Trial of Induction Gemcitabine and Cisplatin Versus Gemcitabine, Cisplatin, Pembrolizumab and Bevacizumab (GPPB) in Nasopharyngeal Cancer
The investigators hypothesize that the addition of bevacizumab and pembrolizumab to induction cisplatin and gemcitabine is tolerable and improves metabolic complete response (mCR), relapse free survival (RFS) and overall survival…
- Trial name
- NCT05305131
- Testing
- cisplatin and gemcitabine, induction chemotherapy cisplatin, gemcitabine, bevacizumab and pembrolizumab.
- Where
- Singapore
- Volunteers
- 50 people
- Sponsor
- National University Hospital, Singapore
Nasopharyngeal Cancer Recurrent
A Study of Toripalimab in Combination With Cisplatin and Gemcitabine in Participants With Recurrent Metastatic Nasopharyngeal Cancer
This study aims to investigate toripalimab with chemotherapy in participants with nasopharyngeal cancer.
- Trial name
- TRANSPARENT (NCT06457503)
- Testing
- Toripalimab, Cisplatin, Gemcitabine
- Where
- 13 sites in United States, Canada
- Volunteers
- 100 people
- Sponsor
- Coherus Oncology, Inc.
Head and Neck Cancer
Antineoplaston Therapy in Treating Patients With Advanced Head and Neck Cancer
Current therapies for advanced Head and Neck Cancer provide very limited benefit to the patient. The anti-cancer properties of Antineoplaston therapy suggest that it may prove beneficial in the treatment of advanced Head and Neck…
- Trial name
- NCT00003489
- Testing
- Antineoplaston therapy (Atengenal + Astugenal)
- Where
- United States
- Volunteers
- 7 people
- Sponsor
- Burzynski Research Institute
Nasopharyngeal Carcinoma
Prospective Study for Molecular Biomarkers of Normal Nasopharyngeal Tissue
The investigators collect nasopharyngeal epithelium samples from healthy individuals and analyze for biomarkers compared with tumor tissues from another clinical research project.
- Trial name
- NCT03048305
- Where
- China
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Sun Yat-sen University
Nasopharyngeal Cancinoma (NPC)
GP Combined with Toripalimab Versus GP Induction Chemotherapy for Advanced Childhood Nasopharyngeal Carcinoma
Nasopharyngeal carcinoma (NPC) has a low incidence rate in children, accounting for only 1-2% of pediatric tumors. However, it is prone to metastasis, and most patients are already in advanced stages at the time of diagnosis.
- Trial name
- NCT06605131
- Testing
- GP+Toripalimab+CCRT, GP+CCRT
- Where
- China
- Volunteers
- 72 people
- Sponsor
- Sun Yat-sen University
Nasopharyngeal Carcinoma
Carboplatin and Docetaxel Followed by Epstein-Barr Virus Cytotoxic T Lymphocytes
Patients have a type of cancer called nasopharyngeal carcinoma (NPC) that has either come back or not gone away after the best known standard treatments.
- Trial name
- CADEN (NCT00953420)
- Testing
- Docetaxel, Carboplatin, Dexamethasone
- Where
- 3 sites in United States
- Volunteers
- 20 people
- Sponsor
- Baylor College of Medicine
Head and Neck Cancer, Pain
Acupuncture or Standard Therapy in Treating Pain and Dysfunction in Patients Who Have Undergone Neck Surgery for Head and Neck Cancer
RATIONALE: Acupuncture may help relieve pain and dysfunction caused by cancer surgery. It is not yet known whether acupuncture is more effective than standard therapy in treating pain and dysfunction in patients who have…
- Trial name
- NCT00090337
- Testing
- acupuncture therapy, standard follow-up care
- Where
- United States
- Volunteers
- 73 people
- Sponsor
- Memorial Sloan Kettering Cancer Center
Head and Neck Cancer
Blood Proteins in Predicting Treatment Benefit in Patients With Recurrent and/or Metastatic Head and Neck Squamous Cell Cancer
RATIONALE: Studying samples of blood in the laboratory may help doctors learn more about cancer. It may also help doctors predict how well patients will respond to treatment.
- Trial name
- NCT00896675
- Testing
- proteomic profiles
- Where
- 3 sites in United States
- Volunteers
- 352 people
- Sponsor
- Vanderbilt-Ingram Cancer Center
Nasopharyngeal Carcinoma
Prospective Comparisons of Clinical Trial and Real-world Outcomes in Nasopharyngeal Carcinoma
Study results from randomized controlled trials (RCTs) usually have been found not adequately inform practice. A RCT is optimized to determine efficacy, while real-world study is conducted in a routine care setting aimed to…
- Trial name
- NCT04108338
- Testing
- Trial setting
- Where
- China
- Volunteers
- 5,448 people
- Sponsor
- Sun Yat-sen University
Nasopharyngeal Cancer, EBV Infections
EBV-Specific CTLs Following CD45 Antibody to Patients With Epstein-Barr Virus (EBV) + Nasopharyngeal Carcinoma (NPC)
To determine the safety of the combination of CD45 monoclonal antibody (Mab) followed by intravenous injection of EBV specific CTL in patients with nasopharyngeal cancer.
- Trial name
- CLANC (NCT00078546)
- Testing
- EBV specific CTL Infusion, Anti CD45 monoclonal antibody
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- Baylor College of Medicine
Niemann-Pick Disease, Type C
Longitudinal Study of Cognition With Niemann-Pick Disease, Type C
Niemann-Pick Disease, Type C (NPC) is a rare neurodegenerative disorder with a wide clinical spectrum and variable age of onset.
- Trial name
- NPC (NCT01899950)
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Mayo Clinic
Wilms Tumor, Fibrosarcoma
Study of High-Dose Chemotherapy With Bone Marrow or Stem Cell Transplant for Rare Poor-Prognosis Cancers
The purpose of this study is to determine whether very high dosages of chemotherapy will improve the chance of surviving cancer.
- Conditions
- Wilms Tumor, Fibrosarcoma, Carcinoma, Round Cell, Nasopharyngeal Cancer
- Trial name
- NCT00141765
- Testing
- Myeloablative Chemotherapy, Stem Cell Rescue
- Where
- United States
- Volunteers
- 25 people
- Sponsor
- University of Michigan Rogel Cancer Center
Nasopharyngeal Cancinoma (NPC), Lymph Node Metastasis
A Deep Learning Model for Diagnosing Lymph Node Metastasis in Nasopharyngeal Carcinoma(NPC)
(I) AI Model for Diagnosing Lymph Node Metastasis We developed an AI model to help diagnose whether a single lymph node in nasopharyngeal cancer has spread. The model uses MRI images of the lymph node and the area around it.
- Trial name
- NPC (NCT06829147)
- Where
- China
- Volunteers
- 500 people
- Sponsor
- Sun Yat-sen University
Gangliosidoses, GM2, Gangliosidosis, GM1
A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of GM1 Gangliosidosis or GM2 Gangliosidosis
An 18-month double-blind, randomized, placebo-controlled, multicenter, Phase 3 study to evaluate the safety and efficacy of oral nizubaglustat (AZ-3102) in late-infantile and juvenile forms of GM1 gangliosidosis or GM2…
- Trial name
- NCT07082543
- Testing
- AZ-3102
- Where
- 27 sites in United States, Argentina and 12 more
- Volunteers
- 75 people
- Sponsor
- Azafaros B.V.
Head and Neck Cancer
Study of Blood and Tissue Samples From Patients With Larynx Cancer, Pharynx Cancer, or Oral Cavity Cancer
RATIONALE: Studying samples of blood and tumor tissue from patients with cancer in the laboratory may help doctors learn more about changes that occur in DNA and identify biomarkers related to cancer.
- Trial name
- CARIO (NCT00963326)
- Testing
- laboratory biomarker analysis
- Where
- 9 sites in France
- Volunteers
- 30 people
- Sponsor
- Centre Oscar Lambret
Spinal Muscular Atrophy, Fragile X Syndrome
Early Check: Expanded Screening in Newborns
Early Check provides voluntary screening of newborns for a selected panel of conditions. The study has three main objectives: 1) develop and implement an approach to identify affected infants, 2) address the impact on infants and…
- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy
- Trial name
- NCT03655223
- Testing
- Confirmatory Testing
- Where
- United States
- Volunteers
- 30,000 people, healthy volunteers welcome
- Sponsor
- RTI International
Nasopharyngeal Cancer
A Study of Nasopharyngeal Carcinoma From Guangdong
This prospective cohort study aims to establish prognostic models, to discover toxicity associated predictors and to validate randomized trials in clinical practice.
- Trial name
- NCT03003182
- Where
- China
- Volunteers
- 2,000 people
- Sponsor
- Sun Yat-sen University