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Bone Marrow Failure Disorders clinical trials

The registry lists 91 studies for Bone Marrow Failure Disorders across more than 10 countries. 29 are recruiting or about to start. 17 of them are mainly about Bone Marrow Failure Disorders (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-04. Counting its 8 narrower conditions too, such as Anemia, Aplastic: 591 studies, 145 open.

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Status

Completed
33
Recruiting
23
Active, not recruiting
10
Terminated
7
Status unknown
7
Not yet recruiting
4

Phase

Phase 2
48
Phase 1
20
Phase 3
7
Early Phase 1
4

Where

United States
67
China
6
Spain
3
Australia
2
Sweden
1
Japan
1

Who runs them

National Heart, Lung, and Blood Institute (NHLBI) (9) · Children's Hospital of Philadelphia (5) · Masonic Cancer Center, University of Minnesota (5) · Rocket Pharmaceuticals Inc. (4) · St. Jude Children's Research Hospital (3) · Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins (3) · Fred Hutchinson Cancer Center (3) · Columbia University (2)

What is being tested here

Drugs under test in Bone Marrow Failure Disorders studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Fludarabine2111
Cyclophosphamide217
Cyclosporine207
Busulfan216
Melphalan203
Granulocyte Colony-Stimulating Factor202
Mycophenolic Acid106
Thymoglobulin114
Alemtuzumab102
Mesna102

Drug classes tested here Alkylating agents 4 open · Antimetabolites 3 open · Calcineurin inhibitors 2 open

Coming up

1phase 3 study under way
7studies 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.

All 7

Who these studies take

Of the 23 Bone Marrow Failure Disorders studies recruiting now, 9 (39%) set no upper age limit and 12 (52%) stop at 75 or younger. 19 (83%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 2 (9%) take healthy volunteers.

Recruiting studies that take each age, of 23
AgeWomenMen
1019 (83%)19 (83%)
1822 (96%)22 (96%)
3015 (65%)15 (65%)
4014 (61%)14 (61%)
5014 (61%)14 (61%)
6513 (57%)13 (57%)
7511 (48%)11 (48%)
8511 (48%)11 (48%)

Across all 91 studies, recruiting or not: 52% stop at 75 or younger and 43% at 65; 80% take children; 0% are for women only and 0% for men only; 3% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

What changed recently

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

Started recruiting1

Studies

Recruiting first, then most recently updated. Showing 50 of 91. Browse all 91 → or search them.