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Genetic Diseases, Inborn clinical trials

The registry lists 583 studies for Genetic Diseases, Inborn across more than 10 countries. 157 are recruiting or about to start. 148 of them are mainly about Genetic Diseases, Inborn (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12. Counting its 209 narrower conditions too, such as Cystic Fibrosis: 19,083 studies, 3,783 open.

ClinicalTrials.gov finds 17,096 studies for Genetic Diseases, Inborn. How that becomes 583 here

This page counts the studies the registry files under Genetic Diseases, Inborn, and those it files under no condition that list Genetic Diseases, Inbornthemselves. 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. 17,096Found by its condition searchIt reads the conditions a study lists and their synonyms, and also titles and keywords.
  2. −15,059Filed under a narrower condition, counted on its own pageCystic Fibrosis 1,550 · Dermatitis, Atopic 1,509 · Anemia, Sickle Cell 1,083 · Hemophilia A 761 · Muscular Dystrophy, Duchenne 464 · Down Syndrome 425 · 177 more
  3. −1List Genetic Diseases, Inborn as a condition, but the registry did not file them under itMost often written as “Rare Fetal Genetic Diseases” (1). The registry filed them under other conditions only, most often Congenital Abnormalities (1) and CHARGE Syndrome (1).
  4. −1,461Match it another way4 name it in the title and 1,457 by a synonym or other text the search reads.
  5. 575Counted here and found by its search
  6. +8
    Counted here, but its search does not find them
  7. 583The count on this page

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Status

Completed
286
Recruiting
120
Status unknown
46
Active, not recruiting
44
Terminated
32
Not yet recruiting
18

Phase

Phase 2
117
Phase 3
75
Phase 1
71
Phase 4
8
Early Phase 1
2

Where

United States
296
France
94
United Kingdom
84
Canada
77
Italy
64
Germany
63

Who runs them

National Center for Research Resources (NCRR) (31) · Novo Nordisk A/S (19) · BioMarin Pharmaceutical (14) · National Human Genome Research Institute (NHGRI) (12) · Vertex Pharmaceuticals Incorporated (11) · Assistance Publique - Hôpitaux de Paris (10) · National Cancer Institute (NCI) (7) · Regeneron Pharmaceuticals (7)

What is being tested here

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

DrugOpenPhase 3All
Infigratinib325
Exagamglogene autotemcel244
Thalidomide102
Acetylcysteine101
Low-dose Aldesleukin ILT-101101
Aldesleukin101
Nicotinamide-beta-riboside101
Delpacibart zotadirsen111
Anumigilimab101
Senicapoc101

Drug classes tested here Tyrosine kinase inhibitors (TKIs) 3 open · Alkylating agents 5 · Antimetabolites 4 · Corticosteroids 3 · HDAC inhibitors 3

Coming up

19phase 3 studies under way
47studies finish main data collection within a year5 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 120 Genetic Diseases, Inborn studies recruiting now, 60 (50%) set no upper age limit and 45 (38%) stop at 75 or younger. 85 (71%) take children under 18. 2 (2%) are for women only and 4 (3%) for men only; 28 (23%) take healthy volunteers.

Recruiting studies that take each age, of 120
AgeWomenMen
1065 (54%)67 (56%)
1896 (80%)96 (80%)
3094 (78%)94 (78%)
4087 (73%)87 (73%)
5084 (70%)84 (70%)
6582 (68%)80 (67%)
7576 (63%)74 (62%)
8573 (61%)71 (59%)

Across all 583 studies, recruiting or not: 37% stop at 75 or younger and 34% at 65; 65% take children; 4% are for women only and 7% for men only; 18% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Genetic Diseases, Inborn studies take

Of 39 phase 3 interventional studies under Genetic Diseases, Inborn, half reached primary completion within 34 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Phase 16 mo2–20 · 14 studies——
Phase 241 mo19–55 · 25 studies——
Phase 334 mo24–58 · 39 studies34 mo23–58 · 38 studies—
Interventional, no phase41 mo22–60 · 66 studies—41 mo22–60 · 64 studies
Observational110 mo46–193 · 128 studies—124 mo46–… · 94 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 14, 2026.

Started recruiting1

Newly registered4

Studies

Recruiting first, then most recently updated. Showing 50 of 583. Browse all 583 → or search them.