Phase 2 Study of Bintrafusp alfa in Recurrent/Metastatic Olfactory Neuroblastoma (BARON).Ī Phase II Study of Bevacizumab, Erlotinib and Atezolizumab in Subjects with Advanced Hereditary Leiomyomatosis and Renal Cell Cancer (HLRCC) Associated or Sporadic Papillary Renal Cell Cancer Unraveling the Mechanisms Underlying Primary Sclerosing Cholangitis Through a Multidisciplinary, Integrative Research ApproachĪ Phase 2 Study of Anti-PD-L1 Antibody (Atezolizumab) in Chondrosarcoma and Clear Cell Sarcoma Pilot Assessment To Find Evidence of Gastric Motility Abnormalities in Eosinophilic Gastric DisordersĪ Phase II Trial of the Superenhancer Inhibitor Minnelide in Advanced Refractory Adenosquamous Carcinoma of the Pancreas (ASCP)Ĭlinical and Laboratory Study of Rare Skeletal Disorders Temozolomide and Olaparib For Advanced Pheochromocytoma And ParagangliomaĪ Multi-Center Natural History of Urothelial Cancer and Rare Genitourinary Tract Malignancies The Natural History of Acquired and Inherited Bone Marrow Failure Syndromes.Ī Prospective, Multi-Institutional Phase II Trial Evaluating Temozolomide Vs. Tissue Procurement and Natural History Study of Neuroendocrine Neoplasms (NENs) Including Adrenocortical Carcinoma (ACC) Phase II Study of Olaparib in Subjects with Advanced Pancreatic Acinar Cell CarcinomaĪn Open-label Safety, Pharmacokinetic, and Efficacy Study of Miglustat for the Treatment of CLN3 Disease in Patients 17 Years of Age and Olderĭata Collection of Standard Care of Patients in the EMG Section Intrathecal 2-Hydroxypropyl-Beta-Cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1 Rapid Analysis and Response Evaluation of Combination Anti-Neoplastic Agents in Rare Tumors (RARE CANCER) Trial: RARE 2 Talazoparib and Temozolomide Phase II Study of Growth Hormone Inhibition Using Pegvisomant In Severe Insulin ResistanceĪ Randomized Phase 2 Study of Atezolizumab with or without Selinexor in Alveolar Soft Part Sarcoma (AXIOM)
Natural History and Biospecimen Acquisition Study for Children and Adults with Rare Solid TumorsĪn Open-Label Phase 2 Study to Evaluate PT2977 for the Treatment of Von Hippel-Lindau Disease-Associated Renal Cell Carcinoma.Īn Open-label Phase 2 Study to Evaluate PT2385 for the Treatment of Von Hippel-Lindau Disease-Associated Clear Cell Renal Cell CarcinomaĬlinical, Epidemiologic, and Genetic Studies of Li-Fraumeni SyndromeĬlinical and Molecular Manifestations of Inherited Neurological DisordersĬutaneous Tumorigenesis in Patients with Tuberous Sclerosis Participants currently recruited/enrolledĪ Randomized, Double-Blind, Placebo-Controlled, Multi-Center Study to Evaluate the Efficacy of ManNAc in Subjects with GNE Myopathy
Rare Tumors and Cancer Predisposition in Individuals and Families
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