Scleroderma associated digital ulcer
1 clinical trial1 recruiting1 country
2 substances studiedView
1 recruiting
Scleroderma associated digital ulcer
Immune system disordersSkin and subcutaneous tissue disorders
Rare diseases archive
Explore rare diseases studied in clinical trials – page 25
619 rare diseases in the catalogue3 136 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)
164Active trials · 20% of the top 10
- 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.
A–Z
619 rare diseases
Serous cystadenocarcinoma ovary
1 clinical trialnone recruiting1 country
Serous cystadenocarcinoma ovary
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Reproductive system and breast disorders
- 1 country
Short stature homeobox gene mutation
1 clinical trialnone recruiting4 countries
2 substances studiedView
Short stature homeobox gene mutation
Cardiac disordersCongenital, familial and genetic disorders
- 4 countries
- Substances:SomatropinVosoritide
Shprintzen-Goldberg syndrome
1 clinical trial1 recruiting1 country
1 substance studiedView
- 1 country
- Substances:Valsartan
Skin angiosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Small intestine leiomyosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
1 recruiting
Small intestine leiomyosarcoma
Blood and lymphatic system disordersGastrointestinal disorders
- 1 country
- Substances:TrabectedinTtf-Ngr
Solitary fibrous tumour
1 clinical trialnone recruiting1 country
1 substance studiedView
Solitary fibrous tumour
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
- 1 country
- Substances:Eribulin
Spina bifida
1 clinical trial1 recruiting1 country1 open to children
1 substance studiedView
1 recruiting1 open to children
Spina bifida
Congenital, familial and genetic disordersNervous system disorders
- 1 country
- Substances:OXYBUTYNIN HYDROCHLORIDE
Squamous cell carcinoma of the vagina
1 clinical trialnone recruiting1 country
2 substances studiedView
Squamous cell carcinoma of the vagina
Gastrointestinal disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 1 country
- Substances:DurvalumabTremelimumab
Stevens-Johnson syndrome
1 clinical trial1 recruiting1 country
- 1 country
Strongyloidiasis
1 clinical trial1 recruiting1 country
1 substance studiedView
- 1 country
- Substances:Ivermectin
Synovial sarcoma metastatic
1 clinical trial1 recruiting1 country
3 substances studiedView
1 recruiting
Synovial sarcoma metastatic
Blood and lymphatic system disordersGastrointestinal disorders
- 1 country
- Substances:PasireotideTrabectedin+1 more
Synovial sarcoma recurrent
1 clinical trial1 recruiting1 country
2 substances studiedView
1 recruiting
Synovial sarcoma recurrent
Blood and lymphatic system disordersGastrointestinal disorders
- 1 country
- Substances:TrabectedinTtf-Ngr
Testicular leiomyosarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Testicular seminoma (pure) stage I
1 clinical trial1 recruiting2 countries
3 substances studiedView
1 recruiting
Testicular seminoma (pure) stage I
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Reproductive system and breast disorders
Testicular seminoma (pure) stage II
1 clinical trialnone recruiting1 country
3 substances studiedView
Testicular seminoma (pure) stage II
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Reproductive system and breast disorders
- 1 country
- Substances:CarboplatinCisplatin+1 more
Thromboangiitis obliterans
1 clinical trialnone recruiting1 country
Thromboangiitis obliterans
Immune system disordersVascular disorders
- 1 country
Thymoma malignant
1 clinical trialnone recruiting4 countries
2 substances studiedView
Thymoma malignant
Blood and lymphatic system disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 4 countries
- Substances:IpilimumabNivolumab
Tongue neoplasm malignant stage unspecified
1 clinical trial1 recruiting1 country
1 substance studiedView
1 recruiting
Tongue neoplasm malignant stage unspecified
Gastrointestinal disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
Undifferentiated sarcoma
1 clinical trial1 recruiting1 country
2 substances studiedView
- 1 country
- Substances:TrabectedinTtf-Ngr
Univentricular heart
1 clinical trial1 recruiting1 country
1 substance studiedView
- 1 country
- Substances:Sildenafil
Vaginal cancer
1 clinical trialnone recruiting2 countries
2 substances studiedView
Vaginal cancer
Gastrointestinal disordersNeoplasms benign, malignant and unspecified (incl cysts and polyps)
- 2 countries
- Substances:AvelumabTipapkinogene Sovacivec
Vulval cancer recurrent
1 clinical trial1 recruiting1 country
1 substance studiedView
1 recruiting
Vulval cancer recurrent
Neoplasms benign, malignant and unspecified (incl cysts and polyps)Reproductive system and breast disorders
- 1 country
- Substances:Bleomycin Sulfate
X-linked lymphoproliferative syndrome
1 clinical trialnone recruiting1 country1 open to children
1 substance studiedView
1 open to children
X-linked lymphoproliferative syndrome
Blood and lymphatic system disordersCongenital, familial and genetic disorders
- 1 country
- Substances:Tadekinig Alfa
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.