Chimerix
Intermediate-size Expanded Access to ONC201 for Patients With H3 K27M-mutant and/or Midline Gliomas
Conditions: Glioma, H3 K27M
This is an intermediate-size expanded access protocol to provide ONC201 (dordaviprone) topatients with H3 K27M-mutant and/or midline gliomas who cannot access ONC201(dordaviprone) through clinical trials.
Pfizer
Expanded Access Protocol for Adults and Pediatric Patients With Sickle Cell Disease Who Have No Alternative Treatment Options
Conditions: Sickle Cell Disease
The objective of this Expanded Access Protocol (EAP) is to provide voxelotor for thetreatment of sickle cell disease (SCD): The study GBT440-041 is the EAP for pediatricpatients aged 6 months to 11 years who have no alternative treatment options and areineligible to participate in a clinical trial of voxelotor and the study C5341057 is theEAP for adults/adolescents 12 years of age and older for patients who cannotsatisfactorily be treated with an authorized medicinal product
Leap Therapeutics, Inc.
Expanded Access Use of DKN-01 for the Treatment of Advanced Solid Tumors
Conditions: Esophageal Neoplasm, Adenocarcinoma of the Gastroesophageal Junction, GastroEsophageal Cancer, Squamous Cell Carcinoma, Gastric Adenocarcinoma, Endometrial Cancer, Uterine Cancer, Ovarian Cancer, Carcinosarcoma, Gastric Cancer
An Expanded Access Protocol for use of DKN-01 for the treatment of advanced solid tumors.
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.
AbbVie
Expanded Access to Telisotuzumab Vedotin
Conditions: Non-small Cell Lung Cancer (NSCLC)
This is an expanded access program (EAP) for eligible participants. This program isdesigned to provide access to Telisotuzumab vedotin prior to approval by the localregulatory agency. Availability will depend on territory eligibility. A medical doctormust decide whether the potential benefit outweighs the risk of receiving aninvestigational therapy based on the individual patient's medical history and programeligibility criteria.
argenx
Efgartigimod Expanded Access for Generalized Myasthenia Gravis
Conditions: Generalized Myasthenia Gravis
This expanded access protocol applies to patients with gMG who are not enrolled in anongoing clinical trial. The aim of the trial is to provide patients with generalizedmyasthenia gravis (gMG), who are ineligible to participate in a clinical trial, access toefgartigimod treatment before regulatory approval.There are country-specific protocols and also individual use EAP. Recruitment for thetreatment protocol in US is now closed (ARGX-113-EAP-2101).
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.
Biogen
Expanded Access Program for Tofersen in Participants With Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
Conditions: Superoxide Dismutase 1-Amyotropic Lateral Sclerosis
The objective of this early access program (EAP) is to provide access to tofersen toeligible participants with amyotrophic lateral sclerosis (ALS) associated with a mutationin the superoxide dismutase 1 (SOD1) gene prior to an alternative access mechanism inorder to address a high unmet medical need in this population.
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.
Celcuity Inc
Expanded Access Protocol for Subjects Previously Treated With Gedatolisib in B2151009
Conditions: Breast Neoplasm Malignant Female
Continued access to treatment for subjects who continue benefit from therapy withgedatolisib in combination with palbociclib, and fulvestrant or letrozole.