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
Jazz Pharmaceuticals
An Expanded Access Treatment Protocol of Zanidatamab (ZW25) in Patients With HER2-Positive Advanced Biliary Tract Cancer
Conditions: HER2-Positive Advanced Biliary Tract Cancer
This is an intermediate-size Expanded Access Protocol (EAP) for use of zanidatamab (ZW25)in patients with human epidermal growth factor receptor 2 (HER2)-positive advancedbiliary tract cancer (BTC) who are not eligible for other zanidatamab clinical trials,and who in the opinion of the treating oncologist, would potentially benefit fromtreatment with zanidatamab.
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.
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.
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.
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).
Bristol-Myers Squibb
Expanded Access for Apixaban
Conditions:
This is an expanded access designed to provide access to apixaban for eligibleparticipants.
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.
Ellodi Pharmaceuticals, LP
Expanded Access Protocol for Patients With Eosinophilic Esophagitis
Conditions: Eosinophilic Esophagitis
This expanded access program is an open-label, single-arm design where consentingpatients may participate up until APT-1011 is commercially available in the relevantregions or the protocol is terminated by the Sponsor.