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.
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.
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.
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 )
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
Matrix Biomed, Inc.
A Single Patient Compassionate Use of MBM-02 (Tempol) for the Treatment of Prostate Cancer
Conditions: Prostate Cancer
A single patient compassionate use clinical trial for the use of MBM-02 (TEMPOL) for thetreatment of prostate cancer. The single patient will be exposed to orally administeredTEMPOL for up to 12 months at a maximum total daily dose of 800mg.
Amicus Therapeutics
Expanded Access for ATB200/AT2221 for the Treatment of IOPD
Conditions: Pompe Disease Infantile-Onset
This is an expanded access program (EAP) for eligible participants designed to provideaccess to ATB200/AT2221.
ZevraDenmark
Early Access Program With Arimoclomol in US Patients With NPC
Conditions: Niemann-Pick Disease, Type C
NPC is a rare, relentlessly progressive, neurological disease and associated with seriousmorbidity and shortened life expectancy.The purpose of this Expanded Access Program is to provide early access to arimoclomol forpatients with Niemann-Pick Type C disease who, in the opinion and the clinical judgementof the treating physician, may benefit from treatment with arimoclomol.Participants will receive treatment with arimoclomol until their doctor finds it does nothelp them anymore, they withdraw, or the study is stopped for any reason.
Xcovery Holdings, Inc.
Expanded Access to Ensartinib for Participants With ALK+ NSCLC
Conditions: Non-Small Cell Lung Cancer, ALK Gene Rearrangement Positive
This is an open-label, multicenter, intermediate-sized expanded access treatment protocolto the existing IND 111,695 for ensartinib (X-396). The treatment plan is designed toprovide ensartinib to participants with anaplastic lymphoma kinase (ALK)-rearrangednon-small cell lung cancer (NSCLC).
University of Texas, Southwestern Medical Center at Dallas
Intermediate Expanded Access Protocol CNMAu8.EAP03
Conditions: Multiple Sclerosis
This is a single-center intermediate expanded access program to provide access to theinvestigational product, CNM-Au8, up to 25 participants diagnosed with MultipleSclerosis.