| Expanded Access Program for Participants Who Completed Study 13-601 and Continue to Clinically Benefit From Cerdulatinib (NCT04757259) | Alexion Pharmaceuticals, Inc. | |
| Prochymal Expanded Access Treatment for Pediatric Patients Who Have Failed Steroids for Acute GVHD (NCT00759018) | Mesoblast, Inc. | |
| Expanded Access of Fostamatinib in Patients With Persistent or Chronic Relapsing/Refractory ITP (NCT03363334) | Rigel Pharmaceuticals | |
| Islet Transplant for Type 1 or Surgical Diabetes (NCT01606475) | University of Minnesota | |
| 177Lu-PSMA-617 Managed Access Program for mCRPC Patients (NCT04825652) | Advanced Accelerator Applications | |
| Emergency Administration of EPI-743 to a Single Patient With Leber's Hereditary Optic Neuropathy [LHON] (NCT02300753) | Edison Pharmaceuticals Inc | |
| Post-Trial Access for Guselkumab in Participants With Familial Adenomatous Polyposis (NCT04948398) | Janssen Research & Development, LLC | |
| Compassionate Use for BIA 28-6156 in Patients With Parkinson's Disease (PD) Who Have a Pathogenic Variant in the GBA1 Gene (GBA-PD) and Completed the ACTIVATE Study (NCT07522606) | Bial R&D Investments, S.A. | |
| Expanded Access Program for Ocrelizumab in Participants With Primary Progressive Multiple Sclerosis (NCT02807285) | Genentech, Inc. | |
| Safety and Treatment Outcome Study of PROCHYMAL® (Remestemcel-L) Intravenous Infusion in Subjects With Treatment-resistant Crohn's Disease (NCT01510431) | Mesoblast, Inc. | |
| Expanded Access Use of Sulopenem Etzadroxil/Probenecid for Complicated Urinary Tract Infection (NCT04682834) | Iterum Therapeutics, International Limited | |
| Expanded Access to B-Domain Deleted Recombinant Porcine Factor VIII (OBI-1) in the Treatment of Acquired Hemophilia A Due to Factor VIII Inhibitory Auto-antibodies (NCT01968655) | Baxalta now part of Shire | |
| 68Ga-DOTATOC for Imaging of Neuroendocrine Tumors: Expanded Access Trial (NCT02358018) | Memorial Sloan Kettering Cancer Center | |
| ALIVE Expended Access - Clinical Study (NCT05710042) | BioVentrix | |
| Expanded Access Protocol Using 131I-MIBG Therapy +/- Vorinostat for Refractory Neuroblastoma, Pheochromocytoma, or Paraganglioma (NCT01838187) | Children's Hospital Medical Center, Cincinnati | |
| Expanded Access For Lorlatinib For Patients With Non Small Cell Lung Cancer ALK Positive or ROS1 Positive (NCT03178071) | Pfizer | |
| C11 Sodium Acetate PET/CT Imaging of PCa (NCT02826395) | Jonsson Comprehensive Cancer Center | |
| Prospective Analysis and Comparison of Stress Echo to Real-Time Myocardial Contrast Stress Echo (NCT00575549) | University of Nebraska | |
| EAP_GS010_single Patient (NCT03672968) | GenSight Biologics | |
| Expanded Access Protocol of ATA230 (Third-Party Donor-Derived CMV-CTLs) for the Treatment of CMV Viremia or Disease (NCT03010332) | Atara Biotherapeutics | |
| Expanded Access Study of TJ004309 in Patients With Advanced or Metastatic Cancer (NCT04869501) | I-Mab Biopharma US Limited | |
| Early Access Program of Telaprevir With Peginterferon and Ribavirin in Chronic Hepatitis C Subjects (NCT01508286) | Janssen-Cilag International NV | |
| Expanded Access to Elotuzumab (Empliciti) for Multiple Myeloma (NCT03126617) | Bristol-Myers Squibb | |
| Expanded Access Protocol for Patients With Eosinophilic Esophagitis (NCT05095116) | Ellodi Pharmaceuticals, LP | |
| Expanded Access Use of Stiripentol in Dravet Syndrome or Sodium Channel Mutation Epileptic Encephalopathies (NCT02239276) | Cook Children's Health Care System | |
| Aprotinin US Special Access Protocol (NCT00611845) | Bayer | |
| Expanded Access Use of Itacitinib to Treat a Single Patient With Aplastic Anemia (NCT03906318) | Incyte Corporation | |
| Expanded Access Program With Nivolumab Therapy for Treatment of Advanced/Metastatic SqNSCLC or Non-SqNSCLC After One Prior Systemic Regimen (NCT02475382) | Bristol-Myers Squibb | |
| Use of Pan-vascular Endothelial Growth Factor Receptor (Pan-VEGF) Blockade for the Treatment of Retinopathy of Prematurity (ROP) (Compassionate Use BLOCK-ROP) (NCT00870636) | Children's Hospital Los Angeles | |
| Erwinase Master Treatment Protocol (NCT00590915) | Phoenix Children's Hospital | |
| Low Levels of Electromagnetic Fields to Treat Advanced Cancer (NCT00805337) | Pasche, Boris, M.D. | |
| Compassionate Use of Deferiprone in Patients With PKAN (NCT02635841) | Chiesi Canada Corp | |
| Treatment Use of Domperidone for Gastroparesis (NCT02227927) | Scott Gabbard, MD | |
| Continuation of Drug Supply in Chinese Patients After CAMN107DBR01study Termination (NCT01613053) | Novartis Pharmaceuticals | |
| Treatment of Recurrent GBM With APG-157 Via Expanded Access (NCT05551013) | Aveta Biomics, Inc. | |
| Expanded Access RBS2418 Treatment (NCT05683470) | Riboscience, LLC. | |
| Compassionate Use of Aldoxorubicin in Sarcoma Patients Chemotherapy (NCT02570412) | CytRx | |
| Buphenyl Therapy for Byler's Disease (NCT01784718) | University of Pittsburgh | |
| Emergent Expanded Access for ahSC Augmentation of Nerve Autografts After Severe Peripheral Nerve Injury. (NCT02510079) | W. Dalton Dietrich | |
| This is a Multi-center, Single Arm, Open Label Study Intended to Provide Expanded Access to Plerixafor for Patients With Non-Hodgkin's Lymphoma (NHL), Hodgkin's Disease (HD) or Multiple Myeloma (MM) Who Are to Receive Treatment With an Autologous Peripheral Stem Cell Transplant. (NCT00720603) | Genzyme, a Sanofi Company | |
| Omegaven Expanded Access Protocol (NCT02121769) | St. Luke's Health System, Boise, Idaho | |
| Midostaurin Access Program for Newly Diagnosed FLT3 (ITD or TKD) Mutated AML Adult Patients Eligible for Standard Induction and Consolidation Chemotherapy (NCT02624570) | Novartis Pharmaceuticals | |
| Induction of Donor-Specific Tolerance in Recipients of Live Donor Stem Cell Infusion (Compassionate Use) (NCT02051673) | Talaris Therapeutics Inc. | |
| Expanded Access Program Using IMM-101 for Patients With Advanced Pancreatic Cancer (NCT04137822) | Impatients N.V. trading as myTomorrows | |
| Compassionate Use of the Becker Expander/Breast Implant (NCT01000012) | Mentor Worldwide, LLC | |
| A Randomized, Open-label Phase III Trial of Mapisal® Versus an Urea Hand-foot Cream as Prophylaxis for Capecitabine-induced Hand-foot Syndrome in Patients With Gastrointestinal Tumors or Breast Cancer (NCT01626781) | AIO-Studien-gGmbH | |
| Clinical Use of a Magnetic Anal Sphincter Augmentation Device, Trade Name FENIX (NCT02722707) | Mayo Clinic | |
| Autologous Human Adipose-Derived Mesenchymal Stem Cells in Alzheimer's Disease (NCT04855955) | Celltex Therapeutics Corporation | |
| An Expanded Access IND for the Treatment of Chronic Inflammatory Demyelinating Polyneuropathy (CIDP) (NCT04825626) | Hope Biosciences Research Foundation | |
| Levosimendan Compassionate Use in Pediatric Patients With Advanced Decompensated Heart Failure (NCT02973620) | Tenax Therapeutics, Inc. | |
| Expanded Access Trial of Plant Expressed Recombinant Glucocerebrosidase (prGCD) in Patients With Gaucher Disease (NCT00962260) | Pfizer | |
| Expanded Access for AG-221 (NCT03723057) | Celgene | |
| Treatment of Primary Hyperoxaluria Type 1 With Nedosiran (NCT05993416) | Dicerna Pharmaceuticals, Inc., a Novo Nordisk company | |
| Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism (NCT01461304) | Jerry Vockley, MD, PhD | |
| Individual Patient Compassionate Use of GX-I7 (NCT04289155) | Genexine, Inc. | |
| Expanded Access to Elezanumab (NCT04278235) | AbbVie | |
| Intermediate-size Expanded Access Program (EAP), Mesenchymal Stromal Cells (MSC) for Acute Respiratory Distress Syndrome (ARDS) Due to COVID-19 Infection (NCT04366830) | Mesoblast International Sàrl | |
| Single Patient Use of Selinexor and Dexamethasone for a Patient With Multiple Myeloma (NCT03850704) | Masonic Cancer Center, University of Minnesota | |
| Expanded Treatment Prot of Panobinostat in Combo w/ Bortez and Dex in Pts w/ Relapsed and/or Refractory Multiple Myeloma (NCT02204553) | Novartis Pharmaceuticals | |
| Expanded Access to Bimatoprost (Durysta) (NCT05338606) | AbbVie | |
| Expanded Access Use of ONC201 in a Patient With Diffuse Intrinsic Pontine Gliomas (NCT05392374) | Jazz Pharmaceuticals | |
| Radium-223 Dichloride (BAY88-8223) in Castration-Resistant (Hormone-Refractory) Prostate Cancer Patients With Bone Metastases (NCT01516762) | Bayer | |
| Long-Term Compassionate Use Study for Continued Administration of SCB01A-01 (NCT01151930) | SynCore Biotechnology Co., Ltd. | |
| Individual Patient Expanded Access for AB-PA01, an Investigational Anti-Pseudomonas Aeruginosa Bacteriophage Therapeutic (NCT03395743) | Armata Pharmaceuticals, Inc. | |
| EAP of Apitegromab for Patients With Spinal Muscular Atrophy (NCT06877689) | Scholar Rock, Inc. | |
| Viaskin® Peanut (DBV712) Expanded Access Protocol (NCT04371627) | DBV Technologies | |
| Volanesorsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS) (NCT03544060) | Akcea Therapeutics | |
| Expanded Access for Pembrolizumab (MK-3475) (NCT03311542) | Center Trials & Treatment | |
| An Expanded Access Program to Provide Sugemalimab for the Treatment of Relapsed or Refractory Extranodal Natural Killer/T-Cell Lymphoma (R/R ENKTL) (NCT05131438) | EQRx International, Inc. | |
| Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant (NCT01200017) | Christopher Dvorak | |
| Post Marketing Surveillance Study of Cuprimine (NCT01374282) | Merck Sharp & Dohme LLC | |
| A Phase I Trial of DM-CHOC-PEN in Adolescent and Young Adult (AYA) Subjects With Advanced Cancers (NCT02889445) | DEKK-TEC, Inc. | |
| 3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM) (NCT02012933) | Oregon Health and Science University | |
| Expanded Access Program for Idebenone in Participants With Duchenne Muscular Dystrophy (DMD) (NCT03433807) | Santhera Pharmaceuticals | |
| Expanded Access for CC-10004 (NCT03740516) | Amgen | |
| Compassionate Use Program to Access Trametinib and Dabrafenib for Patients With BRAF Mutation-positive Metastatic Melanoma (NCT04489433) | Novartis Pharmaceuticals | |
| A Compassionate Use Study of Leronlimab in Breast Cancer (NCT04313075) | CytoDyn, Inc. | |
| BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study (NCT03639844) | Bellicum Pharmaceuticals | |
| Expanded Access [Ga-68] PSMA-11 PET Imaging (NCT04800068) | Mayo Clinic | |
| Vancomycin and Fecal Microbiota Transplant in a Single Patient With Autism Spectrum Disorder (NCT04878718) | ProgenaBiome | |
| Expanded Access Protocol for an Intermediate Size Population - RAVICTI for Byler Disease (NCT02094222) | Robert Squires, Jr. | |
| A Study of Abiraterone Acetate Plus Prednisone in Asymptomatic or Mildly Symptomatic Patients With Metastatic Castration-Resistant Prostate Cancer (NCT01834209) | Janssen Research & Development, LLC | |
| Expanded Access to TAK-113 for Adults With Metastatic Colorectal Cancer (mCRC) (NCT06195514) | Takeda | |
| 131I-Metaiodobenzylguanidine (131I-MIBG) Therapy for Relapsed/Refractory Neuroblastoma (NCT03649438) | University of Texas Southwestern Medical Center | |
| Elranatamab Expanded Access Protocol in Adults With Relapsed/Refractory Multiple Myeloma (NCT05462639) | Pfizer | |
| Efficacy of Parecoxib on Patients With CRPS (NCT01523379) | Ruhr University of Bochum | |
| Compassionate Use of CAR T Cells Targeting the CD19 Antigen and Containing the Inducible Caspase 9 Safety Switch (NCT03594162) | UNC Lineberger Comprehensive Cancer Center | |
| Expanded Access to Entrectinib for Cancers With NTRK1/2/3, ROS1, or ALK Gene Fusions (NCT03066661) | Hoffmann-La Roche | |
| An Expanded Access Program to Axitinib is Available for Patients With Advanced Forms of Kidney Cancer (Ductal; Papillary; Chromophobic; Oncocytic) With Mutations in VHL, PBRM1 / BAP1, SETD2, VEGF) (NCT05941637) | Lynkcell Europe | |
| Use of Domperidone for Treatment of Upper Gastrointestinal Disorders (NCT03128398) | Palo Alto Medical Foundation | |
| Extended Treatment Access Study of MT-3724 for Subjects With Relapsed Non-Hodgkin's B-Cell Lymphoma (NCT02715843) | Molecular Templates, Inc. | |
| Rivaroxaban in Mechanical Valves: RMV Study (NCT02894307) | Hospital Ana Nery | |
| Early Access Program for ALXN1840 in Patients With Wilson Disease (NCT05686564) | Alexion Pharmaceuticals, Inc. | |
| HEMOLEVEN® Expanded Access Program Prevention of Surgical/Postpartum Hemorrhage Severe Inherited Factor XI Deficiency (NCT01701947) | Laboratoire français de Fractionnement et de Biotechnologies | |
| Treatment Protocol of Replagal for Patients With Fabry Disease (NCT01031173) | Shire | |
| Early Patient Access Treatment Use Protocol CA204-220 (NCT02856438) | Bristol-Myers Squibb | |
| Individual Patient Expanded Access IND of Autologous HB-adMSCS for the Treatment of Bilateral Knee Pain. (NCT04604288) | Hope Biosciences Research Foundation | |
| Expanded Access of Imvotamab (IGM-2323) in Patients With R/R NHL (NCT06331832) | IGM Biosciences, Inc. | |
| An Extended Access Program (EAP) for Perampanel (NCT02307578) | Eisai Inc. | |
| AMG 701 Expanded Access Program (NCT05256446) | Amgen | |