Narcolepsy
19 clinical trials9 recruiting14 countries2 open to children
5 substances studiedView
Rare diseases archive
Explore rare diseases studied in clinical trials – page 3
619 rare diseases in the catalogue3 173 linked trialsUpdated today
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
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
163Active trials · 20% of the top 10
- recruiting now
- 47
- countries
- 25
- open to children
- 0
- substances tested
- 92
Shares are of these ten diseases' active trials together.
All rare diseases
Browse every rare disease currently studied across European trials.
A–Z
619 rare diseases
Neuroendocrine tumour
19 clinical trials11 recruiting10 countries2 open to children
8 substances studiedView
11 recruiting2 open to children
Neuroendocrine tumour
Endocrine disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 10 countries
- Substances:DotatateLanreotide+6 more
Giant cell arteritis
18 clinical trials6 recruiting18 countries
14 substances studiedView
Lupus nephritis
18 clinical trials7 recruiting20 countries1 open to children
25 substances studiedView
7 recruiting1 open to children
Lupus nephritis
Immune system disordersMusculoskeletal and connective tissue disorders
- 20 countries
- Substances:AnifrolumabBelimumab+23 more
Ovarian cancer recurrent
18 clinical trials10 recruiting19 countries
48 substances studiedView
10 recruiting
Ovarian cancer recurrent
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Reproductive system and breast disorders
- 19 countries
- Substances:AbemaciclibAlpelisib+46 more
Ovarian epithelial cancer
18 clinical trials8 recruiting17 countries
32 substances studiedView
8 recruiting
Ovarian epithelial cancer
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Reproductive system and breast disorders
- 17 countries
- Substances:AbemaciclibAtezolizumab+30 more
Clear cell renal cell carcinoma
17 clinical trials8 recruiting17 countries
13 substances studiedView
8 recruiting
Clear cell renal cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Renal and urinary disorders
- 17 countries
- Substances:AcetazolamideBelzutifan+11 more
Marginal zone lymphoma
17 clinical trials3 recruiting19 countries
19 substances studiedView
3 recruiting
Marginal zone lymphoma
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 19 countries
- Substances:AcalabrutinibAnhydrous Caffeine+17 more
Paroxysmal nocturnal haemoglobinuria
17 clinical trials4 recruiting16 countries4 open to children
9 substances studiedView
4 recruiting4 open to children
Paroxysmal nocturnal haemoglobinuria
Blood and lymphatic system disordersRenal and urinary disorders
- 16 countries
- Substances:CemdisiranCrovalimab+7 more
Spinal muscular atrophy
17 clinical trials2 recruiting15 countries6 open to children
7 substances studiedView
2 recruiting6 open to children
Spinal muscular atrophy
Congenital, familial and genetic disordersMusculoskeletal and connective tissue disorders
- 15 countries
- Substances:ApitegromabBIIB115+5 more
Uveal melanoma
17 clinical trials9 recruiting15 countries
15 substances studiedView
9 recruiting
Uveal melanoma
Eye disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 15 countries
- Substances:CrizotinibDacarbazine+13 more
B-cell lymphoma refractory
16 clinical trials6 recruiting16 countries1 open to children
21 substances studiedView
6 recruiting1 open to children
B-cell lymphoma refractory
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 16 countries
- Substances:AcalabrutinibAllopurinol+19 more
B-cell type acute leukaemia
16 clinical trials9 recruiting19 countries2 open to children
10 substances studiedView
9 recruiting2 open to children
B-cell type acute leukaemia
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 19 countries
- Substances:AlemtuzumabBlinatumomab+8 more
Dermatomyositis
16 clinical trials7 recruiting18 countries
19 substances studiedView
- 18 countries
- Substances:AldesleukinAnifrolumab+17 more
Oesophageal squamous cell carcinoma
16 clinical trials9 recruiting18 countries
20 substances studiedView
9 recruiting
Oesophageal squamous cell carcinoma
Gastrointestinal disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 18 countries
- Substances:AtezolizumabAvelumab+18 more
Cutaneous lupus erythematosus
15 clinical trials6 recruiting17 countries
9 substances studiedView
6 recruiting
Cutaneous lupus erythematosus
Immune system disordersMusculoskeletal and connective tissue disorders
- 17 countries
- Substances:AnifrolumabDazukibart+7 more
Ductal adenocarcinoma of pancreas
15 clinical trials8 recruiting13 countries
14 substances studiedView
8 recruiting
Ductal adenocarcinoma of pancreas
Gastrointestinal disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 13 countries
- Substances:AcetazolamideAMG 193+12 more
Gastrointestinal stromal tumour
15 clinical trials7 recruiting17 countries1 open to children
9 substances studiedView
7 recruiting1 open to children
Gastrointestinal stromal tumour
Gastrointestinal disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Diffuse large B-cell lymphoma refractory
14 clinical trials4 recruiting16 countries
29 substances studiedView
4 recruiting
Diffuse large B-cell lymphoma refractory
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 16 countries
- Substances:BGB-16673Brentuximab Vedotin+27 more
Glycogen storage disease type II
14 clinical trials8 recruiting11 countries3 open to children
6 substances studiedView
8 recruiting3 open to children
Glycogen storage disease type II
Cardiac disordersCongenital, familial and genetic disorders
- 11 countries
- Substances:Alglucosidase AlfaAvalglucosidase Alfa+4 more
Huntington's disease
14 clinical trials4 recruiting20 countries
8 substances studiedView
Metastatic renal cell carcinoma
14 clinical trials3 recruiting16 countries
10 substances studiedView
3 recruiting
Metastatic renal cell carcinoma
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Renal and urinary disorders
- 16 countries
- Substances:AxitinibCabozantinib+8 more
Primary biliary cholangitis
14 clinical trials3 recruiting23 countries
7 substances studiedView
- 23 countries
- Substances:BezafibrateElafibranor+5 more
Chronic graft versus host disease
13 clinical trials6 recruiting24 countries1 open to children
12 substances studiedView
6 recruiting1 open to children
Chronic graft versus host disease
Immune system disordersInjury, poisoning and procedural complications
- 24 countries
- Substances:AxatilimabBelumosudil Mesilate+10 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.