Explore our case studies
Gamma scintigraphy demonstrates effective lung deposition of a novel triple combination pressurised metered dose inhaler (pMDI) in COPD patient population.
23/08/2024
A large pharmaceutical Sponsor needed to demonstrate that its novel triple combination pMDI formulation could effectively deliver drugs to the lungs of patients with moderate and severe/ very severe COPD as defined by the European Respiratory Society.
Multiple stakeholders collaborate effectively to deliver HV trial in potential treatment for schizophrenia
11/04/2024
Cardiff University MDI-26478 drug is a potential new treatment for schizophrenia developed by the Medicines Discovery Institute (MDI) at the university. Our scientists worked with their colleagues at the MDI to design a three-part First in Human study. This required us to collaborate with both the Cardiff University Brain Research Imaging Centre (CUBRIC) for neuroimaging […]
Adaptive Trial Design for Novel IPF Small Molecule Therapy
08/01/2024
A small biotech client had developed an asset that was a potential treatment for Idiopathic Pulmonary Fibrosis (IPF). Simbec-Orion was able to provide regulatory advice and support for submission to the MHRA, EMA and FDA, along with a study program that accelerated the delivery of their studies, saved cost and facilitated the transition from Healthy […]
Primary Hyperoxaluria: Umbrella Study Design for FiH Trial Including HV and Patients
08/01/2024
Primary Hyperoxaluria (PH) is an ultra-rare autosomal recessive disease characterised by excessive production of oxalate in the liver. Oxalate is a highly insoluble metabolic end-product that is eliminated mainly by the kidney. Patients with PH are predisposed to the development of multiple and recurrent urinary tract (urolithiasis) and kidney (nephrolithiasis) stones. At present, no therapies […]
Niemann-Pick Type C – Rare Disease Case Study: Surpassing Timelines & Rapid Enrolment In An Ultra-Rare Paediatric Study
16/12/2020
The sponsor needed to produce a new batch of their product with concentrations adapted based on the weight of paediatric patients. An observational study was needed to identify and profile patients before progressing to an interventional study. Patients enrolled were between 2 to 18 years of age with a diagnosis of NP-C.
Non-Hodgkin’s Lymphoma (NHL) and Chronic Lymphocytic Leukaemia (CLL) Case Study: Efficient Enrolment and Dose Escalation Process Ensures Rapid Access To Critical Data
16/12/2020
Our Sponsor needed to ensure first-inpatient study success in Europe, in a highly competitive oncology field amongst sites and patient availability. A minimum enrolment of 42 patients was required to complete the study.
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