There are about 173942 clinical studies being (or have been) conducted in United States. The country of the clinical trial is determined by the location of where the clinical research is being studied. Most studies are often held in multiple locations & countries.
This expanded access protocol is to provide access to the investigational product, SLS-005, to participants with ALS who are not eligible to participate in clinical trials.
This Expanded Access, Phase 3, open label study is intended to provide access to COVAXIN™ (BBV152) to individuals who are at risk or have predisposing conditions that can lead to complications with the current immunization options against SAR-CoV-2 Virus infection.
The objective of the study is to assess the long-term safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy as assessed by a review of adverse events (AEs).
This is an open label, multi-center expanded access treatment protocol evaluating lifileucel (LN-144) in patients with unresectable or metastatic melanoma.
This is an intermediate-size expanded access protocol to provide ONC201 to patients with diffuse intrinsic pontine gliomas who cannot access ONC201 through clinical trials.
A recombinant virus vector constructed from adeno-associated virus (AAV) has been engineered to carry the human aspartoacylase (ASPA) gene expressed from a modified CMV-enhancer chicken β-actin (CB6) promoter. The construct has been shown to produce ASPA in animal models of Canavan disease, which closely match the proposed human study. The proposed clinical trial is an open label, expanded access study administering rAAV9-CB6-AspA gene vector by simultaneous systemic and intracerebroventricular routes to a single human subject (18-24 months of age) with Canavan disease. The subject will also receive immune modulation to transiently ablate B-cells (Rituximab) and modulate T-cell response (Sirolimus) prior to the initial exposure to AAV9. Given the null AspA mutations of the subject and current AAV seronegative status, this regimen will allow for later exposure to the therapeutic vector if needed and block any immuno-toxicity in the CNS. The goal of this study is to measure the safety and efficacy of AAV-mediated gene therapy as a treatment approach for neuronal pathology in Canavan disease. The subject will act as their own control and change from baseline will be assessed in regards to levels of brain NAA, brain water content and morphology, improved clinical status and peripheral levels of NAA. Safety parameters measured in this study will include: serum chemistries and hematology, urinalysis, physical assessments, whole blood assay for vector genomes, immunologic response to ASPA and AAV, as well as reported subject symptom history.
An expanded access program for sotrovimab administered intravenously to participants with COVID-19 illness who meet current authorized/approved criteria for use of sotrovimab.
This individual patient expanded access IND is requested for a patient diagnosed with LMNA-related congenital muscular dystrophy (L-CMD). In this expanded access, the patient will receive the investigational product through 14 intravenous infusions, followed by Follow-Up visit and an End of Study.
This program is intended to provide access to sugemalimab for participants with R/R ENKTL, after their disease failed to respond to prior treatment regimen(s), preceding marketing authorization by the local regulatory agency.
The purpose of this expanded access program is to provide access to the investigational drug verdiperstat in patients with Multiple System Atrophy (MSA). Expanded access allows patients with a serious or a life-threatening disease or condition access to an investigational drug when no satisfactory approved treatment options are available.