Clene Nanomedicine
Two Intermediate Expanded Access Protocols (EAP) CNMAu8.EAP01 and CNMAu8.EAP02 for ALS
Conditions: Amyotrophic Lateral Sclerosis
The primary objective of the intermediate expanded access protocol is to provide accessto the investigational product, CNM-Au8, to up to 300 people living with ALS (pALS).No formal clinical hypotheses are being evaluated with concurrent controls.Secondary objectives include assessment of the safety of CNM-Au8 treatment in pALS.Safety will be assessed through the frequency of serious adverse events (SAEs),treatment-emergent adverse events (TEAEs) assessed as 'severe', discontinuations due toTEAEs, and laboratory abnormalities assessed as clinically significant during routineclinical monitoring (as applicable).
Novo Nordisk A/S
Compassionate Use of Concizumab if You Have Haemophilia
Conditions: Congenital Haemophilia
The compassionate use programme will give participants concizumab for free, even thoughit is not yet approved by health authorities. This is because participants need thismedicine to treat their haemophilia properly. The programme will check that participantsare safe and that the medicine works for them. The programme may last for years.Participants will take one injection under their skin every day. Participants will have4-5 visits with the study doctor for the first half year. After that they will have 1visit every half year. At all clinic visits participants will have blood samples taken.Participants will fill in a diary between the visits.A patient is considered to have completed the programme when any of the followingcriteria occurred first: 1) when the patient is included in a clinical trial withconcizumab or 2) up to 6 months after concizumab is commercially available in thepatient's country and approved for the patient (The time span of 6 months should provideample time for the patient to obtain concizumab commercially) or 3) the sponsor decidesto discontinue concizumab clinical development for the patient's population.
Giselle Sholler
An Intermediate Expanded Use Trial of DFMO
Conditions: Neuroblastoma, Medulloblastoma, Typical Teratoid Rhabdoid Tumor, Embryonal Tumor With Abundant Neuropil and True Rosettes, Ependymoblastoma, Medulloepithelioma
To provide DFMO in an expanded use setting to subjects with relapsed rare tumors withincreased LIN28 expression or MYCN amplification or up regulation of ornithinedecarboxylase.
Cook MyoSite
Expanded Access Program for Autologous Muscle Derived Cells (AMDCs)
Conditions:
This is an expanded access program (EAP) for eligible participants designed to provideaccess to autologous muscle derived cells (AMDCs). Treating physicians may submitrequests for expanded access to Cook MyoSite's investigational product by email toMYO-ExpandedAccess@CookMyoSite.com.
Vanda Pharmaceuticals
Single-Patient Expanded Access Protocol for Tradipitant In A Single Patient With Gastroparesis
Conditions: Gastroparesis, Diabetic Gastroparesis
Primary Objective: To treat a single patient with gastroparesis who has requestedexpanded access with tradipitant
Leonard-Meron Biosciences, Inc.
Expanded Access: Mino-Lok Therapy (MLT) for the Treatment of CRBSI/CLABSI
Conditions: Catheter-Related Infections
This is an Intermediate-Size Expanded Access, Open-Label Study for Use of Mino-LokTherapy (MLT) in Combination with Systemic Antibiotics in the Treatment of Central LineAssociated Bloodstream Infection. Mino-Lok may be made available for patients whootherwise do not qualify for the phase 3 clinical trial (NCT02901717 )
Cancer Commons
Expanded Access to Ulixertinib (BVD-523) in Patients with Advanced MAPK Pathway-Altered Malignancies
Conditions: Pancreatic Cancer, Small Bowel Cancer, Colorectal Cancer, Melanoma, Non Small Cell Lung Cancer, Thyroid Cancer, Bladder Cancer, Head and Neck Cancer, Gastric Cancer, Esophageal Cancer, Cholangiocarcinoma, Ovarian Cancer, HepatoCellular Carcinoma, Glioblastoma, MAPK Gene Mutation, KRAS Activating Mutation, BRAF Gene Mutation, NRAS Gene Mutation, HRAS Gene Mutation, MEK Mutation, ERK Mutation
The objective of this expanded access program is to provide ulixertinib (BVD-523) forcompassionate use in advanced cancer patients with MAPK pathway-altered solid tumor(s),including but not limited to KRAS, NRAS, HRAS, BRAF, MEK, and ERK mutations who haveincomplete response to or have exhausted available therapies.Ulixertinib is available for treatment as monotherapy or in combination with otherclinically tolerable agent(s), conditionally approved by the drug manufacturer.
AIM ImmunoTech Inc.
Ampligen in Chronic Fatigue Syndrome
Conditions: Chronic Fatigue Syndrome
This is an open label study of Ampligen in patients with chronic fatigue syndrome.
Eyetech Pharmaceuticals
A Single-Center Trial of High Frequency Pegaptanib for Rapid Restoration of VEGF Levels in Diabetic Retinal Edema
Conditions: Diabetic Macular Edema
Establish the efficacy of initial high frequency loading of intravitreal pegaptanibbi-weekly during the initial treatment period when the VEGF levels are the greatest andthen gradually extending the administration frequency to monthly as homeostasis ensuesfor the treatment of DME, as measured by best-corrected visual acuity.
Jubilant DraxImage Inc.
Expanded Access Protocol Using 131I-MIBG
Conditions: Neuroblastoma, Pheochromocytoma, Paraganglioma
Protocol JDI2007-01 is an Expanded Access Protocol with therapeutic 131I-MIBG forpatients with neuroblastoma or pheochromocytoma / paraganglioma, who otherwise do notqualify for available treatments, or where approved treatment is not commerciallyavailable.