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NCT ID: NCT04972487 Approved for marketing - Clinical trials for Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis

Start date: n/a
Phase:
Study type: Expanded Access

The objective of this early access program (EAP) is to provide access to tofersen to eligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism in order to address a high unmet medical need in this population.

NCT ID: NCT05031546 Approved for marketing - Uremic Pruritus Clinical Trials

Intermediate-Size Patient Population Expanded Access Program for Intravenous Difelikefalin

Start date: n/a
Phase:
Study type: Expanded Access

This is an intermediate-size patient population expanded access protocol for the use of intravenous (IV) difelikefalin for the treatment of moderate-to-severe pruritus associated with chronic kidney disease in adult patients undergoing hemodialysis.

NCT ID: NCT05041049 Approved for marketing - Clinical trials for Comparing Visual Reality to High-fidelity Mannequin Stimulation

Virtual Simulation Versus High-Fidelity Mannequin Simulation

Start date: n/a
Phase:
Study type: Expanded Access

Background. Despite remarkable progress, pediatric anesthesia-related mortality remains 2 to 30 times higher than in adults. Non-technical skills rather than a lack of expertise were identified to be the main cause of errors in medicine, particularly in emergency situations. As a consequence, interactive computer-based sessions and high-fidelity simulation have been progressively integrated into pediatric training programs. The setup of a suitable simulation center, however, is expensive and requires space, logistical support and manpower. Virtual reality (VR) is a technological innovation that has been increasingly integrated into clinical practice and medical training. Comparing to the mannequin-based simulation, theoretical advantages of VR are the inherent highly immersive quality, the independent participation with no need of other participants, the ease of multiple repetitions, the lack of time or space constrains, and the reduced costs. Non-technical and operational skills appear to improve significantly after VR training. To date, information on the efficacy of VR in the anesthesia training are limited. Objectives. The primary aim will be to compare the improvement of non-technical skills after one simulation session between VR and high-fidelity mannequin simulation (HFS). Secondary aims will be to compare the overall anesthesia resident non-technical skills performance between VR and HFS, and to determine confidence in the simulation training of each participant. Methods. This is a prospective, randomized, non-inferiority study. Eighteen residents in anesthesia (R1-R5) will be enrolled. Participants of each year will be randomly allocated to be involved in either a VR or a HFS training. The scenario will be the same for both VR and HFS groups. Within two weeks after the training session, all participants will undergo a different, mannequin-based HFS scenario. After each simulation, resident performance will be assessed using the Anaesthetists' Non-Technical Skills scale and the Ottawa Crisis Resource Management Global Rating Scale. Evaluators will also perform a summary evaluation with the Managing Emergencies in Pediatric Anesthesia Global Rating Scale. Resident's satisfaction will be measured with a post-assessment trainee survey scale. Significance. Should the virtual reality demonstrate to be as efficient as mannequin-based simulation, it will be integrated into the residency curriculum and used for resident training and medical education in institutions that have limited access to a simulation center.

NCT ID: NCT05162443 Approved for marketing - Metastatic Cancer Clinical Trials

Expanded Access of Adagrasib (MRTX849) in Patients With Advanced Solid Tumors Who Have a KRAS G12C Mutation

Start date: n/a
Phase:
Study type: Expanded Access

The objective of this EAP is to provide expanded access of adagrasib (MRTX849) to patients with previously treated advanced solid tumors harboring a KRAS G12C mutation.

NCT ID: NCT05183802 Approved for marketing - Obesity Clinical Trials

An Expanded Access Protocol for Setmelanotide for Treatment of Bardet-Biedl Syndrome (BBS)

Start date: n/a
Phase:
Study type: Expanded Access

An open-label, single-arm, multicenter, Expanded Access Protocol [EAP] designed to provide treatment access to setmelanotide (3 mg, administered subcutaneously [SC], once daily) for eligible patients with BBS who have no alternative treatment options. All patients will continue to receive setmelanotide at the discretion of the Treating Physician and while they are deriving clinical benefit.

NCT ID: NCT05286372 Approved for marketing - Clinical trials for Amyotrophic Lateral Sclerosis

An Intermediate Size Expanded Access Protocol of AMX0035 for ALS

Start date: n/a
Phase:
Study type: Expanded Access

The Expanded Access Program will provide access and assess the safety of AMX0035 for the treatment of people living with ALS.

NCT ID: NCT05345678 Approved for marketing - Clinical trials for Unresectable Hepatocellular Carcinoma

Himalaya Early Access Program

Start date: n/a
Phase:
Study type: Expanded Access

To provide early access (i.e., before marketing authorisation) to tremelimumab 300 mg IV administered once on Day 1 of Cycle 1 plus durvalumab 1500 mg IV followed by durvalumab 1500 mg IV Q4W monotherapy in patients with unresectable HCC.

NCT ID: NCT05401214 Approved for marketing - Clinical trials for Metastatic Castration-resistant Prostate Cancer

Pre-approval Access Single Patient Request for Niraparib / Abiraterone Acetate Combination (Nira/AA Combination)

Start date: n/a
Phase:
Study type: Expanded Access

The purpose of this pre-approval access is to provide Niraparib plus Abiraterone Acetate (Nira/AA combination) in the treatment of (a) participant(s) with first line metastatic castration-resistant prostate cancer with specific Homologous Recombination Repair (HRR) gene alterations.

NCT ID: NCT05463939 Approved for marketing - Clinical trials for Relapsed or Refractory Multiple Myeloma

Teclistamab Expanded Access Treatment Protocol for Relapsed or Refractory Multiple Myeloma Patients

Start date: n/a
Phase:
Study type: Expanded Access

The purpose of this expanded access treatment protocol is to provide patients access to teclistamab prior to market authorization. The program is limited to patients with relapsed or refractory multiple myeloma who have exhausted all treatment options available as local standard of care and who are not eligible for a teclistamab clinical trial.

NCT ID: NCT05722912 Approved for marketing - Clinical trials for Graft-versus-host Disease (GVHD)

Expanded Access of Ruxolitinib Cream to Treat a Single Patient With cGVHD

Start date: n/a
Phase:
Study type: Expanded Access

To provide ruxolitinib through an expanded access to treat a single patient with cGVHD