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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 (only the trials that have ended within the past 5 years are shown).

The information which is displayed is provided by the sponsor, the principal investigator and/or by national drug agencies.

Enter your requested disease name or gene name/symbol to access this information.

The results can be sorted either geographically (by country, region and city, in alphabetical order), or by specificity (from those more focused on a particular disease to those specialised in a group of diseases including the disease you were looking for).


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.