AstraZeneca PLC

This is a single arm phase II study of pemigatinib and durvalumab combination in patients with FGFR-2 fusion or rearrangement positive intrahepatic cholangiocarcinoma. Each cycle will be 3 weeks. Pemigatinib is administered at 13.5 mg orally daily 2 weeks on and 1 week off. Durvalumab is administered at 1500 mg intravenously once every 3 weeks. Subjects will require a visit with appropriate laboratory work prior to the start of each cycle. Disease assessment will occur every 9 weeks. Subjects will continue treatment until progression per RECIST 1.1, toxicity or subject/physician decision. A maximum of 24 months (about 35 cycles) of pemigatinib and durvalumab treatment from Cycle 1 Day 1 is allowed.Show more

The MaesTTRo study aims to enroll a global cohort of patients with transthyretin (ATTR) amyloidosis to longitudinally observe the natural course of the disease and describe real-world treatment patterns and outcomes. In addition, information on the effectiveness of ATTR amyloidosis treatments, including eplontersen, which is a ligand-conjugated antisense oligonucleotide gene silencing treatment targeting activity against both the mutant and wild-type TTR protein, will be collected.Show more

This Phase IIb study aims to evaluate the efficacy, safety, and tolerability of 3 doses of AZD8965 treatment compared to placebo in participants with IPF, including those on antifibrotic therapy (nintedanib, pirfenidone, nerandomilast), either alone or in combination, or in those not on antifibrotic therapy.Show more

The overall objective of this DPSS is to describe the occurrence of pregnancy and maternal complications, adverse effects on the developing fetus and neonate, and adverse effects on the infant associated with exposure to eplontersen during pregnancy and/or lactation.

Part 1: To characterize the safety profile of acalabrutinib alone or in combination with rituximab in subjects with R/R FL. Part 2: To characterize the activity of acalabrutinib alone or in combination with rituximab in subjects with R/R MZL, as measured by ORR. Part 3: To characterize the safety of acalabrutinib in combination with rituximab and lenalidomide in subjects with R/R FLShow more

This is a prospective, multicenter, observational study of Chinese pediatric NF1-PN patients treated with selumetinib. The study will be conducted at approximately 12 centers in China and will include approximately 80-100 patients. Treatment centers that have PN diagnosis and/or selumetinib treatment experience will be targeted for recruitment. Patients/caregivers who are eligible and willing to participate will be enrolled into the study. Patients will start selumetinib treatment after enrollment. The study will have a 16-month enrollment period. Patients will be followed up until the end of a 24-month observation period after first dose of selumetinib, or patient death, lost to follow-up, withdrawal of consent, whichever occurs first. Patients will be followed within a 24-month period (starting after first dose received) in the study even if selumetinib is discontinued. The aims of this study are to expand understanding of disease characteristics and treatment pattern of NF1-PN in China in a real-world setting and to evaluate real-world effectiveness and safety of selumetinib for Chinese pediatric patients with NF1-PNShow more

This is a phase 2 study, designed as a European multicentre 6-month double blind random-ized controlled trial (RCT) of AZD0530 versus matched placebo, followed by a 12 month trial comparing open-label extended AZD0530 treatment with historical control data. Study population: Male and female adult patients aged 18 years and older with a diagnosis of FOP who meet the inclusion (active disease) and exclusion criteria will be eligible for participation in this study. The total number of enrolled patients will be 20. Intervention: Patients will be randomized to receive either AZD0530 100mg once daily or matched placebo, taken orally for the first 6 months, immediately followed by an open-label extension in which all patients will receive AZD0530 100mg once daily oral dose for a further 12 months. Endpoints: Endpoints include change in number of active heterotopic bone lesions measured by low-dose whole-body computer tomography (CT)/ \[18F\] NaF Positron Emission Tomography (PET) activityShow more

AstraZeneca was formed in 1999 by the merger of Astra and Zeneca. It entered rare disease with Alexion, a deal announced at $39 billion and completed on July 21, 2021, and Alexion now runs as AstraZeneca's rare disease unit. Alexion's medicines, including Soliris, Ultomiris and Voydeya, are covered on the Alexion Pharmaceuticals page. AstraZeneca's own rare disease medicines include Koselugo (selumetinib) for plexiform neurofibromas in people with NF1 aged 1 and older, Wainua (eplontersen) for the nerve damage of hereditary ATTR amyloidosis in adults, and Fasenra (benralizumab) for adults with EGPA. The FDA is reviewing Ultomiris for IgA nephropathy, efzimfotase alfa for hypophosphatasia and gefurulimab for generalized myasthenia gravis.

AstraZeneca PLC has active programs across 45 rare diseases: Adrenocortical Carcinoma, ATTR Amyloidosis (Transthyretin Amyloidosis), Atypical Hemolytic Uremic Syndrome, Bullous Pemphigoid, Cholangiocarcinoma, Chronic Graft-versus-Host Disease, Cushing Disease, Cystic Fibrosis, Dermatomyositis, Diffuse Intrinsic Pontine Glioma, Dilated Cardiomyopathy, Eosinophilic Esophagitis, Eosinophilic Granulomatosis with Polyangiitis, Essential Thrombocythemia, Fibrodysplasia Ossificans Progressiva, Focal Segmental Glomerulosclerosis, Gastrointestinal Stromal Tumor, Granulomatosis with Polyangiitis, Hereditary Diffuse Gastric Cancer, Hodgkin Lymphoma, Homozygous Familial Hypercholesterolemia, Idiopathic Pulmonary Fibrosis, Immune Thrombocytopenia, Medullary Thyroid Cancer, Membranous Nephropathy, Merkel Cell Carcinoma, Mesothelioma, Multiple Myeloma, Multiple Sclerosis, Multiple System Atrophy, Myasthenia Gravis, Myelodysplastic Syndromes, Myelofibrosis, Neurofibromatosis Type 1, Neuromyelitis Optica, Paraganglioma, Paroxysmal Nocturnal Hemoglobinuria, Polycystic Kidney Disease, Primary Central Nervous System Lymphoma, Pulmonary Arterial Hypertension, Sickle Cell Disease, Sjögren's Syndrome, Systemic Sclerosis, Waldenström Macroglobulinemia, Wild-Type ATTR Cardiac Amyloidosis. These span several therapeutic areas. Each disease page on Trial Friend shows the latest recruiting trials, FDA-approved treatments, and patient resources.

AstraZeneca PLC is a diversified pharmaceutical company headquartered in Cambridge, United Kingdom, founded in 1999. While it operates across multiple therapeutic areas, it maintains a significant rare disease portfolio with 45 active rare disease programs including Adrenocortical Carcinoma, ATTR Amyloidosis (Transthyretin Amyloidosis), Atypical Hemolytic Uremic Syndrome. Many of its rare disease treatments hold orphan drug designations from the FDA.

The clinical trials section on this page shows active and recruiting rare disease trials sponsored by AstraZeneca PLC, pulled live from ClinicalTrials.gov. Each trial card includes the study phase, recruitment status, conditions being studied, number of trial sites, and a direct link to the full study record. You can also explore individual disease pages to see trials from all sponsors for a specific condition.

To explore enrollment, find a recruiting trial in the clinical trials list on this page and click "View full study" to see the full ClinicalTrials.gov record, which includes eligibility criteria, study locations, and contact information for each trial site. Your doctor can also help determine if a specific trial is appropriate for your condition. Not all trials are open at every location, so check the site list for one near you.

AstraZeneca PLC was founded in 1999 and is headquartered in Cambridge, United Kingdom. The company has been active for 27 years. It trades publicly under the ticker symbol AZN.