Explore rare diseases studied in clinical trials – page 9
Top 10 most studied rare diseases
Rare diseases with the highest number of active trials right now.
Colours show each disease's body system – lighter shades rank lower.
- Blood and lymphatic system disorders58%
- Gastrointestinal disorders20%
- Neoplasms benign, malignant and unspecified11%
- Hepatobiliary disorders11%
No. 1 most studied
Plasma cell myeloma
- recruiting now
- 50
- countries
- 25
- open to children
- 0
- substances tested
- 141
Shares are of these ten diseases' active trials together.
All rare diseases
Browse every rare disease currently studied across European trials.
619 rare diseases
Mantle cell lymphoma recurrent
5 clinical trialsMantle cell lymphoma recurrent
- 6 countries
- Substances:BGB-16673Fludarabine+10 more
Myxoid liposarcoma
5 clinical trialsMyxoid liposarcoma
Nasopharyngeal cancer
5 clinical trialsNasopharyngeal cancer
- 4 countries
- Substances:Interferon Beta-1ANivolumab
Neuroendocrine carcinoma metastatic
5 clinical trialsNeuroendocrine carcinoma metastatic
- 4 countries
- Substances:Calcium FolinateCarboplatin+11 more
Neuromyelitis optica spectrum disorder
5 clinical trialsNeuromyelitis optica spectrum disorder
- 6 countries
- Substances:ImlifidaseInebilizumab+2 more
Osteosarcoma metastatic
5 clinical trialsOsteosarcoma metastatic
Paraganglion neoplasm
5 clinical trials- 11 countries
- Substances:ExenatideL-Arginine Hydrochloride+3 more
Pemphigoid
5 clinical trials- 15 countries
- Substances:Clobetasol Propionate
Penile cancer
5 clinical trialsPenile cancer
- 4 countries
- Substances:AprepitantAvelumab+7 more
Phaeochromocytoma
5 clinical trialsPhaeochromocytoma
- 11 countries
- Substances:L-Arginine HydrochlorideL-Lysine Hydrochloride+3 more
Porphyria non-acute
5 clinical trials- 12 countries
- Substances:AfamelanotideDersimelagon Phosphate
Pulmonary sarcoidosis
5 clinical trialsPulmonary sarcoidosis
- 9 countries
- Substances:EfzofitimodNamilumab+3 more
Rabies
5 clinical trials- 2 countries
- Substances:EmtricitabineMycobacterium Bovis, Danish Strain 1331
Retinopathy of prematurity
5 clinical trialsRetinopathy of prematurity
- 12 countries
- Substances:AfliberceptOxygen Ph.eur.+1 more
Systemic sclerosis pulmonary
5 clinical trialsSystemic sclerosis pulmonary
- 9 countries
- Substances:AmlitelimabBelimumab+3 more
Warm autoimmune haemolytic anaemia
5 clinical trialsWarm autoimmune haemolytic anaemia
Activated PI3 kinase delta syndrome
4 clinical trialsActivated PI3 kinase delta syndrome
- 5 countries
- Substances:Leniolisib Phosphate
Acute myeloid leukaemia (in remission)
4 clinical trialsAcute myeloid leukaemia (in remission)
- 7 countries
- Substances:AzacitidineClofarabine+5 more
Amyloidosis
4 clinical trialsAmyloidosis
- 7 countries
- Substances:Belantamab MafodotinBortezomib+7 more
Arteriovenous malformation
4 clinical trials- 2 countries
- Substances:Regorafenib
Biliary cancer metastatic
4 clinical trialsBiliary cancer metastatic
Burkitt's lymphoma
4 clinical trialsBurkitt's lymphoma
- 9 countries
- Substances:CyclophosphamideCytarabine+9 more
Clear cell papillary renal cell carcinoma
4 clinical trialsClear cell papillary renal cell carcinoma
- 9 countries
- Substances:DurvalumabLenvatinib+5 more
About rare diseases in clinical trials
A disease is called rare in the EU when it affects fewer than 5 in 10 000 people. Individually uncommon, together they affect millions – and most still have no approved treatment.
Because the number of patients is small, trials in rare diseases often recruit across several countries at once. That is why a trial listed here may be worth looking at even when it runs nowhere near you.
