Knowledge on rare diseases and orphan drugs
COVID-19 & Rare diseasesSearch for a clinical trial
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Orphanet provides information on clinical trials focused on a rare disease or on a group of rare diseases. Trials comprise interventional studies aiming to evaluate a drug (or a combination of drugs or a biological product) to treat (or prevent) a rare disease or a group of rare diseases. Single-centre and national or international multicentric clinical trials are registered. The trials registered in Orphanet can be ongoing, recruiting, or finished.
A collaboration between World Health Organization's ICTRP (International Clinical Trials Registry Platform) (https://www.who.int/ictrp/en/) and Orphanet is in place in order to make clinical trials on rare diseases easily identifiable and findable, thus improving knowledge on rare diseases. This collaboration strengthens Orphanet's position as a reference database by providing an exhaustive rare diseases clinical trials registry for the countries covered by Orphanet network and by IRDiRC, and will assure that ICTRP users can easily identify clinical trials for rare diseases (http://apps.who.int/trialsearch/).
Information on clinical trials may also be provided by the sponsor, the principal investigator and/or by national drug agencies.
Search by disease/gene name: Enter your requested disease name or gene name/symbol to access the list of clinical trials conducted for this disease.
Search by clinical trial title: Enter the complete or incomplete clinical trial title to access the requested trial.
Search by substance/Trade name: Enter the substance or trade name to access the list of concerned clinical trials.
The results can be sorted either geographically (by country, region and city, in alphabetical order), or by specificity (from those more focused on the particular disease you look for to those specialised in a group of diseases including the disease you were looking for).
You can access related Orphanet datasets in an aggregated format through Orphadata after signature of a Data Transfer Agreement/Service Contract.
Warning
The data collection takes place in Orphanet consortium countries and is currently ongoing. Research related resources (research projects, clinical trials, patient registries, biobanks and Variant databases) are also collected and registered in Orphanet database if they are funded by a member agency of the International Rare Diseases Research Consortium (IRDiRC, list of members available here) located in a country for which there is no Orphanet national team.
Information in Orphanet is not intended to replace professional health care. Orphanet cannot be held responsible for harmful, truncated or erroneous use of any information found in the Orphanet database.