Hepatic angiosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Rare diseases archive
Explore rare diseases studied in clinical trials – page 8
204 rare diseases in the catalogue905 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 disorders37%
- Immune system disorders19%
- Congenital, familial and genetic disorders15%
- Nervous system disorders11%
- Musculoskeletal and connective tissue disorders9%
- Eye disorders9%
No. 1 most studied
Myelodysplastic syndrome
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
53Active trials · 18% of the top 10
- recruiting now
- 24
- countries
- 22
- open to children
- 0
- substances tested
- 72
Shares are of these ten diseases' active trials together.
All rare diseases
Browse every rare disease currently studied across European trials.
A–Z
204 rare diseases
Hereditary hypophosphataemic rickets
1 clinical trial1 recruiting3 countries
1 substance studiedView
1 recruiting
Hereditary hypophosphataemic rickets
Congenital, familial and genetic disordersMetabolism and nutrition disorders
- 3 countries
- Substances:Kk8123
Hereditary motor and sensory neuropathy
1 clinical trialnone recruiting4 countries
3 substances studiedView
Hereditary motor and sensory neuropathy
Congenital, familial and genetic disordersNervous system disorders
Immunodeficiency congenital
1 clinical trialnone recruiting5 countries
2 substances studiedView
Immunodeficiency congenital
Congenital, familial and genetic disordersImmune system disorders
- 5 countries
- Substances:AtalurenMavorixafor
Isodicentric chromosome 15 syndrome
1 clinical trialnone recruiting3 countries
1 substance studiedView
Isodicentric chromosome 15 syndrome
Congenital, familial and genetic disordersNervous system disorders
- 3 countries
- Substances:Soticlestat
KBG syndrome
1 clinical trial1 recruiting1 country
1 substance studiedView
1 recruiting
KBG syndrome
Congenital, familial and genetic disordersMusculoskeletal and connective tissue disorders
- 1 country
- Substances:Methylphenidate Hydrochloride
Kearns-Sayre syndrome
1 clinical trial1 recruiting1 country1 open to children
2 substances studiedView
1 recruiting1 open to children
Kearns-Sayre syndrome
Cardiac disordersCongenital, familial and genetic disorders
- 1 country
- Substances:NipocalimabANHYDROUS CALCIUM FOLINATE
Krabbe's disease
1 clinical trialnone recruiting1 country1 open to children
1 substance studiedView
1 open to children
Krabbe's disease
Congenital, familial and genetic disordersMetabolism and nutrition disorders
- 1 country
- Substances:Adeno-Associated Virus Serotype Rh10 Containing The Human Galc Gene
Leiomyosarcoma recurrent
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Loeys-Dietz syndrome
1 clinical trial1 recruiting1 country
1 substance studiedView
- 1 country
- Substances:Valsartan
Lymphangioma
1 clinical trial1 recruiting7 countries
1 substance studiedView
1 recruiting
Lymphangioma
Blood and lymphatic system disordersMusculoskeletal and connective tissue disorders
- 7 countries
- Substances:Alpelisib
Lymphangiosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Marfan's syndrome
1 clinical trial1 recruiting1 country
1 substance studiedView
- 1 country
- Substances:Valsartan
Mast cell activation syndrome
1 clinical trialnone recruiting1 country
2 substances studiedView
Mast cell activation syndrome
Blood and lymphatic system disordersImmune system disorders
Myelodysplastic syndrome with excess blasts
1 clinical trialnone recruiting11 countries
2 substances studiedView
Myelodysplastic syndrome with excess blasts
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 11 countries
- Substances:GilteritinibMidostaurin
Myxofibrosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Ocular melanoma
1 clinical trial1 recruiting2 countries
4 substances studiedView
1 recruiting
Ocular melanoma
Eye disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 2 countries
- Substances:Dacarbazine CitrateRelatlimab+2 more
Optic glioma
1 clinical trialnone recruiting1 country
1 substance studiedView
Optic glioma
Eye disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 1 country
- Substances:Nerve Growth Factor, Recombinant (R100E)
Osteosarcoma recurrent
1 clinical trialnone recruiting1 country
1 substance studiedView
Osteosarcoma recurrent
Musculoskeletal and connective tissue disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 1 country
- Substances:Regorafenib
Paraneoplastic neurological syndrome
1 clinical trial1 recruiting1 country
4 substances studiedView
1 recruiting
Paraneoplastic neurological syndrome
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Nervous system disorders
- 1 country
- Substances:CyclophosphamideMesna+2 more
Pleomorphic leiomyosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
1 recruiting
Pleomorphic leiomyosarcoma
Blood and lymphatic system disordersGastrointestinal disorders
- 1 country
- Substances:TrabectedinTtf-Ngr
Progressive multifocal leukoencephalopathy
1 clinical trial1 recruiting1 country
1 substance studiedView
1 recruiting
Progressive multifocal leukoencephalopathy
Infections and infestationsNervous system disorders
- 1 country
- Substances:Pembrolizumab
Proteus syndrome
1 clinical trialnone recruiting1 country
1 substance studiedView
Proteus syndrome
Congenital, familial and genetic disordersMetabolism and nutrition disorders
- 1 country
- Substances:Miransertib
Rhabdomyosarcoma recurrent
1 clinical trial1 recruiting1 country
2 substances studiedView
1 recruiting
Rhabdomyosarcoma recurrent
Blood and lymphatic system disordersGastrointestinal disorders
- 1 country
- Substances:TrabectedinTtf-Ngr
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.