Rare Diseases Clinical Trials
6 recruiting trials for Rare Diseases. Eligibility criteria explained in plain English.
Recruiting Trials
Clinical trial data sourced from the ClinicalTrials.gov registry, maintained by the National Library of Medicine. Always consult your doctor before considering any clinical trial.
Institutional Registry of Rare Diseases
The goal of this observational study is to create a single macro registry system with data collection on common clinical features, grouping the different rare diseases (RD)....
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patients participate in...
Evaluation of HEArt invoLvement in Patients With FABRY Disease
This study evaluates predictors for the incidence of arrhythmias and sudden cardiac death as well as terminal heart failure in patients with Fabry disease.
Epidemiological Study of Treatment Approaches in AChR-Antibody Positive Generalized Myasthenia Gravis in Russia
This is a multicenter, non-interventional, retrospective-prospective, single-arm observational study designed to describe real-world treatment approaches and clinical outcomes...
Longitudinal Study of Ultra-rare Inherited Metabolic and Degenerative Neurological Diseases.
General aim of the study is the improvement of the clinical knowledge of ultra-rare inherited metabolic and degenerative neurological diseases (prevalence less than 5:100,000) in...
Marfan Syndrome (MFS) and Facial Dysmorphism: Non-invasive 3D Assessment
The goal of this study observational prospective study is to define the facial morphological features associated with Marfan syndrome (MFS). The main qustion it aims to answer...
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Frequently Asked Questions
There are currently 6 clinical trials for Rare Diseases, with 6 actively recruiting participants. These include trials across all phases from early-stage Phase 1 to late-stage Phase 3.
To join a clinical trial for Rare Diseases, review the eligibility criteria on the trial detail pages, then talk to your doctor about whether a trial is right for you. Your doctor can help you evaluate the potential benefits and risks.
Phase 3 trials are large-scale studies that test whether a treatment is effective and monitor side effects. There are 0 Phase 3 trials for Rare Diseases, representing treatments closest to potential FDA approval.
Clinical trials follow strict safety protocols overseen by Institutional Review Boards (IRBs) and the FDA. Participants are monitored closely and can withdraw at any time. Always discuss risks and benefits with your healthcare provider before enrolling.
Trial data sourced from the ClinicalTrials.gov API. This site does not provide medical advice — always talk to your doctor about clinical trial participation.