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Neurofibromatosis 1 clinical trials

The registry lists 176 studies for Neurofibromatosis 1 across more than 10 countries. 43 are recruiting or about to start. 142 of them are mainly about Neurofibromatosis 1 (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.

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Status

Completed
81
Recruiting
28
Active, not recruiting
22
Status unknown
15
Not yet recruiting
12
Terminated
11

Phase

Phase 2
55
Phase 1
31
Phase 3
6
Phase 4
2
Early Phase 1
1

Where

United States
117
France
14
China
10
United Kingdom
10
Canada
8
Germany
7

Who runs them

National Cancer Institute (NCI) (31) · Massachusetts General Hospital (11) · AstraZeneca (11) · University of Alabama at Birmingham (7) · Shriners Hospitals for Children (3) · Assistance Publique - Hôpitaux de Paris (3) · Children's Hospital Medical Center, Cincinnati (3) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (3)

What is being tested here

Drugs under test in Neurofibromatosis 1 studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
AZD 62446219
Trametinib105
Deoxycholic Acid104
Mirdametinib103
Luvometinib113
RO5126766101
Defactinib101
Abemaciclib101
Cabozantinib101
Azacitidine101

Drug classes tested here Tyrosine kinase inhibitors (TKIs) 2 open · Antimetabolites 2 open · mTOR inhibitors 5 · Antimitotic agents 3 · Angiogenesis inhibitors 3

Coming up

3phase 3 studies under way
21studies finish main data collection within a year

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

Who these studies take

Of the 28 Neurofibromatosis 1 studies recruiting now, 18 (64%) set no upper age limit and 4 (14%) stop at 75 or younger. 19 (68%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 5 (18%) take healthy volunteers.

Recruiting studies that take each age, of 28
AgeWomenMen
1014 (50%)14 (50%)
1826 (93%)26 (93%)
3025 (89%)25 (89%)
4025 (89%)25 (89%)
5025 (89%)25 (89%)
6524 (86%)24 (86%)
7524 (86%)24 (86%)
8523 (82%)23 (82%)

Across all 176 studies, recruiting or not: 48% stop at 75 or younger and 45% at 65; 71% take children; 0% are for women only and 1% for men only; 15% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

How long Neurofibromatosis 1 studies take

Of 24 phase 2 interventional studies under Neurofibromatosis 1, half reached primary completion within 62 months of starting, counting those still running.

Start to primary completionEvery sponsorIndustryNon-industry
Phase 125 mo9–37 · 15 studies——
Phase 262 mo40–95 · 24 studies——
Interventional, no phase20 mo13–52 · 21 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.

Newly registered1

Studies

Recruiting first, then most recently updated. Showing 50 of 176. Browse all 176 → or search them.