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Discarded Bone Marrow for Hematology Research
The primary objective of this study is to establish a mechanism to obtain discarded bone marrow-containing bone samples from hemoglobinopathy, as well as non-h…
Evaluation Of The Lung Microbiome In NTM Bronchiectasis
A biomarker cohort study design is proposed to study whether specific airway microbiota alterations are associated with pulmonary Non-tuberculous mycobacteria …
Natural History Study of Synucleinopathies
Synucleinopathies are a group of rare diseases associated with worsening neurological deficits and the abnormal accumulation of the protein α-synuclein in the …
Effectiveness and Safety of Sotagliflozin in Slowing Kidney Function Decline in Persons With Type 1 Diabetes and Moderate to Severe Diabetic Kidney Disease
Powerful new drugs that can prevent or delay end stage kidney disease (ESKD) - so called sodium-glucose cotransporter-2 inhibitors (SGLT2i) - are now available…
The Development of Minimally Invasive Nanosensor Technology to Quantify Mitochondrial Function in Human Muscle
Past mitochondrial disease treatment studies have been unsuccessful in determining treatment efficacy, and a major factor has been the lack of validated biomar…
Effect of Hypertonic Saline on Mucociliary Clearance in Non-CF Bronchiectasis
The purpose of this single arm clinical trial is to evaluate the effects of 7% hypertonic saline (HS) delivered by nebulizer on clearance of mucus from the lun…
Tailored Exercise Training Study Among Adults With HFpEF
Heart failure with preserved ejection fraction (HFpEF) is associated with a high morbidity and mortality burden. There are limited pharmacological options avai…
Optimizing Beta Blocker Dosage in Women While Using the Wearable Cardioverter Defibrillator
The primary objective of this pilot study is to document the percentage achievement in effective HR control (average nighttime HR \< 70 bpm) during WCD use in …
A Patient Registry and Natural History Study of Patients with Biallelic HPDL Mutations
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations