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.
The objective of this treatment protocol is to provide guidance to Treating Physicians who seek access to pegunigalsidase alfa for Fabry patients whose clinical condition, in the opinion of the Treating Physician, requires treatment with enzyme replacement therapy (ERT) with pegunigalsidase alfa and a) cannot be adequately treated with currently approved FDA products and/or b) are not able or willing to participate in any of the on-going clinical trials in the United States.
This is an Intermediate-Size Expanded Access, Open-Label Study for Use of Mino-Lok Therapy (MLT) in Combination with Systemic Antibiotics in the Treatment of Central Line Associated Bloodstream Infection. Mino-Lok may be made available for patients who otherwise do not qualify for the phase 3 clinical trial (NCT02901717 )
Primary Objective: To treat a single patient with gastroparesis who has requested expanded access with tradipitant
The purpose of this research study is to (1) provide access to fenfluramine for patients with intractable epilepsy associated with Dravet syndrome, and (2) evaluate the safety of fenfluramine.
A single patient compassionate use clinical trial for the use of MBM-02 (TEMPOL) for the treatment of prostate cancer. The single patient will be exposed to orally administered TEMPOL for up to 12 months at a maximum total daily dose of 800mg.
This is an expanded access program (EAP) for eligible participants designed to provide access to ATB200/AT2221.
NPC is a rare, relentlessly progressive, neurological disease and associated with serious morbidity and shortened life expectancy. The purpose of this Expanded Access Program is to provide early access to arimoclomol for patients with Niemann-Pick Type C disease who, in the opinion and the clinical judgement of the treating physician, may benefit from treatment with arimoclomol. Participants will receive treatment with arimoclomol until their doctor finds it does not help them anymore, they withdraw, or the study is stopped for any reason.
This is an open-label, multicenter, intermediate-sized expanded access treatment protocol to the existing IND 111,695 for ensartinib (X-396). The treatment plan is designed to provide ensartinib to participants with anaplastic lymphoma kinase (ALK)-rearranged non-small cell lung cancer (NSCLC).
MCT8 deficiency (that is also known as Allan-Herndon-Dudley syndrome) is a rare X-linked inherited disorder of brain development that causes severe intellectual disability and problems with movement.
Forty patients with pancreatic cancer, sarcoma and carcinoma of breast will receive DeltaRex-G intravenously at a dose of 1-4 x 10e11 colony forming units (cfu) or equivalent 0.6-1.8 x 10e10 RV copies per dose one to three times a week. DeltaRex-G may be given alone or with one or more FDA approved cancer therapies/immunotherapies. Based on previous Phase 1/2 US based clinical studies, DeltaRex-G does not suppress the bone marrow or cause serious organ dysfunction, and enhanced immune cell trafficking in tumors may cause the tumors to appear larger or new lesions to appear on CT, PET or MRI. Further, tumor stabilization/regression/remission may occur later during the treatment period. Therefore, DeltaRex-G will be continued regardless of CT, PET or MRI results if the patient has clinical benefit and does not have symptomatic disease progression.