DrugEvidence

Drugs / Methotrexate

Methotrexate

Drug

Methotrexate is a cytotoxic antimetabolite drug used across oncology and immune-mediated diseases in indication-specific dosing regimens, often once weekly in non-oncology uses. Labeling requires careful dose scheduling because medication errors can cause fatal toxicity. Initial U.S. approval dates to 1953.

Trials
1437
Recruiting
133
Completed
826
With results
464

Last revised

Pipeline

Phase by status

PhaseRecruitingNot yetBy invitationActiveCompletedTerminatedWithdrawnSuspendedUnknownTotal
Early Phase 1 411712 16
Phase 1 10872948 111
Phase 1/2 64321457 68
Phase 2 531526279741859 524
Phase 2/3 923134112 44
Phase 3 23116238234132 338
Phase 4 158310913638 192
Not applicable 722479222 91
Unknown 63112913 53

Trials started per year

2021
15
2022
41
2023
40
2024
49
2025
44
2026
38

Conditions studied, by phase

ConditionEarly Phase 1Phase 1Phase 1/2Phase 2Phase 2/3Phase 3Phase 4Not applicableUnknownTotal
Arthritis, Rheumatoid 121361893781011 286
Precursor Cell Lymphoblastic Leukemia-Lymphoma 12522106124818144 250
Leukemia 111898641102 186
Lymphoma 181079421113 137
Leukemia, Myeloid, Acute 1712522165 104
Burkitt Lymphoma 1885331324 92
Myelodysplastic Syndromes 1010411116 79
Lymphoma, Non-Hodgkin 554077 64
Graft vs Host Disease 372911535 63
Breast Neoplasms 222312831 60

Most active sponsors

#SponsorTrialsPhase 3
1Children's Oncology Group5731
2M.D. Anderson Cancer Center572
3National Cancer Institute (NCI)4914
4Hoffmann-La Roche4827
5Fred Hutchinson Cancer Center433
6St. Jude Children's Research Hospital387
7Memorial Sloan Kettering Cancer Center211
8Dana-Farber Cancer Institute204
9Bristol-Myers Squibb1910
10Alliance for Clinical Trials in Oncology171

Most studied interventions

#InterventionTrials
1Methotrexate1437
2Cyclophosphamide424
3Cytarabine352
4Vincristine317
5Dexamethasone240
6Doxorubicin240
7Etoposide188
8Mercaptopurine178
9Prednisone174
10Rituximab170

Evidence

Results reported
56.2%464 of 826 completed
Published
19.2%159 of 826 completed
Primary endpoint met
61.5%236 of 384 analyses
Median months to report
16

Documents disclosed

Protocol available
166
Statistical analysis plan
167
PubMed citations
464

Trial profile

Median enrollment
76
Median duration
54.3months
Countries
89
Study sites
14940

Who is enrolled

Adults
1356
Children
537
Older adults
979
Healthy volunteers
52

Median enrollment by phase

Early Phase 1
25
Phase 1
24
Phase 1/2
36
Phase 2
50
Phase 2/3
132
Phase 3
265
Phase 4
100
Not applicable
53
Unknown
222

Where trials run

United States
697
Canada
160
China
150
Germany
119
United Kingdom
119
Australia
117
France
113
Italy
103
Spain
93
Netherlands
78

Busiest cities

#CityCountryTrials
1HoustonUnited States212
2New YorkUnited States186
3BostonUnited States162
4SeattleUnited States160
5ChicagoUnited States158
6PhiladelphiaUnited States156
7DallasUnited States142
8St LouisUnited States141
9ClevelandUnited States135
10Los AngelesUnited States134

Terminations

Terminated
146
Withdrawn
40
Suspended
2
Stopped for recruitment
4.5%64 of 1437 trials

Stated reasons

Other / unclear
79
Recruitment
64
Funding
14
Lack of efficacy
9
Business decision
7
Safety
7
Regulatory
5
COVID-19
3

Reasons are grouped from the sponsor's own wording with a rule-based classifier; the original text is shown below.

Recently stopped

TrialStatusStated reasonStopped
NCT05691478 Suspended Suspended Other - Extend the observation period for current patients 2030-03-20
NCT06015750 Withdrawn Withdrawn Sponsor decision following FDA release from the PMR. 2030-03-13
NCT04961801 Withdrawn Withdrawn Manufacturer AstraZeneca withdrew the study due to lack of funding 2030-01-31
NCT04845035 Withdrawn Withdrawn The PI plans to revise the study design in a new protocol 2029-01-31
NCT07498465 Withdrawn Withdrawn Other - Protocol moved to Withdrawn 2028-11-12
NCT06466798 Withdrawn Withdrawn Inability to find participants meeting eligibility critieria 2027-07-01
NCT06210750 Withdrawn Withdrawn Other - Protocol moved to Disapproved 2026-09-22
NCT06578728 Terminated Terminated The study was terminated due to slow accrual. 2026-06-05
NCT03808610 Terminated Terminated \<75% participation 2026-05-05
NCT03719560 Withdrawn Withdrawn lack of funding 2025-12-31

Trial records