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.
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.
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.
DBV Technologies
Expanded Access Protocol of Viaskin® Peanut (DBV712) in Peanut-allergic Children
Conditions: Peanut Allergy
This is an open label expanded access program for male and female patients 2 years orolder, to provide continued desensitization treatment with DBV712 250 mcg.
Matrix Biomed, Inc.
Treatment of Metastatic Cancer in Terminally Diagnosed Patients
Conditions: Metastatic Cancer
The objective is to provide terminally diagnosed patients with a last line of treatmentwhile improving overall quality of life. Tempol can be added to any chemotherapy regimento potentially reduce side effects and overcome chemoresistance.
Neuraptive Therapeutics Inc.
NTX-001 to Repair Peripheral Nerve Transection(s)
Conditions: Peripheral Nerve Transections, Acute or Planned in Upper Extremity and Facial Nerves
The proposed use of NTX-001 for transections of upper extremity and facial peripheralnerves, acutely or planned.
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.
Bristol-Myers Squibb
Expanded Access for Apixaban
Conditions:
This is an expanded access designed to provide access to apixaban for eligibleparticipants.
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.