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Rare Diseases clinical trials

The registry lists 592 studies for Rare Diseases across more than 10 countries. 125 are recruiting or about to start. 96 of them are mainly about Rare Diseases (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-10.

ClinicalTrials.gov finds 1,045 studies for Rare Diseases. How that becomes 592 here

This page counts the studies the registry files under Rare Diseases, and those it files under no condition that list Rare Diseasesthemselves. Each study its search finds is placed by how the registry filed it and by the conditions it lists. ClinicalTrials.gov counts as of Nov 8, 2026. How condition pages count

  1. 1,045Found by its condition searchIt reads the conditions a study lists and their synonyms, and also titles and keywords.
  2. −5List Rare Diseases as a condition, but the registry did not file them under itMost often written as “Genetic Rare Disease” (1), “Rare Disease Orodontal” (1), and “Rare Diseases of Genetic Origin” (1). The registry filed 5 of them under no condition at all.
  3. −448Match it another way74 name it in the title, 7 in a keyword, and 367 by a synonym or other text the search reads.
  4. 592The count on this page

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Breast cancer · Recruiting: 2 came in, 3 changed

What changed in your saved searches, Oct 31 – Nov 6:

A study of drug A after surgery for early breast cancer

Came in · now Recruiting

Drug B with hormone therapy in advanced breast cancer

Changed

· Sites: 4 added, including Seoul and Busan

· Enrollment: 300 → 450 participants

Exercise during chemotherapy

Now Active, not recruiting

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Status

Completed
353
Recruiting
92
Status unknown
62
Terminated
26
Not yet recruiting
20
Active, not recruiting
13

Phase

Phase 2
117
Phase 3
69
Phase 1
53
Phase 4
6
Early Phase 1
2

Where

United States
244
France
59
Italy
51
Germany
40
United Kingdom
25
Canada
22

Who runs them

National Center for Research Resources (NCRR) (164) · FDA Office of Orphan Products Development (20) · National Institute of Neurological Disorders and Stroke (NINDS) (13) · University Hospital Tuebingen (13) · National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) (13) · Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD) (13) · Assistance Publique - Hôpitaux de Paris (10) · IRCCS Policlinico S. Donato (10)

What is being tested here

Drugs under test in Rare Diseases studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
S-606001303
Filgrastim113
Cannabidiol112
Pegcetacoplan101
Inebilizumab101
Leucovorin101
Pyrimethamine101
Sulfadiazine101
Cyclophosphamide0110
Immunoglobulins047

Drug classes tested here Alkylating agents 13 · Corticosteroids 8 · Antimetabolites 8 · Androgens 4 · HDAC inhibitors 3

Coming up

4phase 3 studies under way
36studies finish main data collection within a year3 of them phase 3

Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.

The 10 nearest, phase 3 first

Who these studies take

Of the 92 Rare Diseases studies recruiting now, 60 (65%) set no upper age limit and 16 (17%) stop at 75 or younger. 63 (68%) take children under 18. 2 (2%) are for women only and 2 (2%) for men only; 15 (16%) take healthy volunteers.

Recruiting studies that take each age, of 92
AgeWomenMen
1055 (60%)53 (58%)
1885 (92%)84 (91%)
3083 (90%)83 (90%)
4083 (90%)83 (90%)
5081 (88%)80 (87%)
6580 (87%)78 (85%)
7578 (85%)76 (83%)
8575 (82%)73 (79%)

Across all 592 studies, recruiting or not: 40% stop at 75 or younger and 34% at 65; 65% take children; 5% are for women only and 3% for men only; 12% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Rare Diseases studies take

Of 88 observational studies under Rare Diseases, half reached primary completion within 118 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Interventional, no phase37 mo17–60 · 36 studies——
Observational118 mo39–183 · 88 studies——

Each figure is the month by which half the studies had reached primary completion, with the range in which the middle half did. Studies still running count until their last registry update; studies that stopped early are left out. Phase 1 includes early phase 1; observational means prospective or not stated, as retrospective and cross-sectional studies date a window of data rather than a study. How these figures are worked out

What changed recently

From the registry's own updates since Oct 13, 2026.

Started recruiting1

Newly registered3

Studies

Recruiting first, then most recently updated. Showing 50 of 592. Browse all 592 → or search them.