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Open-label Extension Study to Evaluate the Long-term Safety and Efficacy of Mexiletine in Paediatric Patients with Myotonic Disorders Who Have Completed the MEX-NM-301 study.

Migraine is a neurological disease characterized by moderate or severe headache, associated with nausea, vomiting, and/or sensitivity to light and sound (International Classification of Headache Disorders, 2018). Migraine can be further categorized according to the frequency of a…

Researchers want to learn more about the use of larotrectinib as a real-world treatment for tropomyosin receptor kinase fusion cancer, also called TRK fusion cancer. In people with TRK fusion cancer, a gene called neurotrophic TRK, (NTRK) joins or "fuses" with another gene. This…

The purpose of the study is to assess if the addition of darolutamide to ADT compared with ADT alone would result in superior clinical efficacy in participants with metastatic hormone-sensitive prostate cancer (mHSPC) by progression-free survival. The researchers want to learn h…

Open-label, single intrathecal injection study of a AAV9 vector carrying the IGHMBP2 gene for IGHMBP2-related diseases.

This is a single arm phase II study that will evaluate the combination of pembrolizumab, docetaxel, and cisplatin or carboplatin (PDC) as single treatment modality in patients with stage II-IVb (T2-4, any N, M0) squamous cell carcinoma of the nasal cavity/paranasal sinuses (PNS S…

This phase II trials studies the effects of yttrium-90 labeled anti-CD25 monoclonal antibody combined with BEAM chemotherapy conditioning in treating patients with Hodgkin lymphoma that does not response to treatment (refractory) or has come back (relapsed). Yttrium-90-labeled an…

This is a single-arm, open-label phase II study with a safety lead-in phase.

This is a phase I/II randomized, masked, clinical trial to determine the safety and efficacy of intravitreal administration of cerliponase alfa.

Biliary drainage and stent placement remains to be the main palliative treatment choice for advanced perihiliar cholangiocarcinoma (pCCA), and the life expectancy is only 4-6 months. Previous single center prospective phase 2 trial showed that hepatic arterial infusion chemothera…

This is a Phase II randomized, controlled, open label breast cancer clinical trial. 66 patients will be enrolled. The drugs being studied are hydroxychloroquine (Plaquenil) and abemaciclib (also Verzenio). This research study is testing whether using these drugs to target the dis…

Some kidney transplant candidates have a very low chance of getting a kidney transplant because their immune systems are "highly sensitized" to most kidney donors. Being "highly sensitized" means that they will likely have to wait a long time (more than 5 years) before an accepta…

Treatments for opioid addiction exist, but effectiveness is compromised when subjects use illicit opiates during treatment. Reuse rates during treatment can be high, and reducing illicit opiate use during treatment has thus recently become a major NIDA policy goal. The 5-minute b…

An open label, dose escalation and dose expansion study to evaluate the safety, tolerability, and anti-tumor activity of STP707 with IV administration in subjects with advanced/metastatic or surgically unresectable solid tumors who are refractory to standard therapy.

The clinical utility trial is designed to evaluate how the results of KidneyIntelX test / platform impacts on the clinical management of type 2 diabetes patients identified as increased risk for rapid kidney function decline within 5-years.

The current trial is designed to evaluate how the results of KidneyIntelX test impacts on the clinical management of type 2 diabetes patients identified as increased risk for rapid kidney function decline within 5-years.

We will determine how best to manage the hepatitis C virus (HCV) epidemic in Pakistan by measuring effectiveness of Pakistan-government sponsored current therapies, emergence of viral resistance, consequences of infection (chiefly liver cancer) and through developing models, base…

Part I (Clinical trial setting): A single-arm phase II study to investigate the efficacy of neoadjuvant atezolizumab (T) + bevacizumab (A) in patients with potentially resectable BCLC stage B/C or high risk resectable hepatocellular carcinoma (HCC) (n = 40) Part II (Biomarker st…

The purpose of this study is to determine the most efficacious and safest dexamethasone dose given intraoperatively during total knee arthroplasty that reduces postoperative opioid consumption and pain, improves postoperative nausea and vomiting, and minimizes postoperative compl…

The goal of this research is to validate novel non-invasive Magnetic resonance imaging (MRI) biomarkers to detect Gastroesophageal varices (GEV) in patients with cirrhosis, including fractional flow change in the portal vein and elevated azygos flow. End-stage liver disease (cir…