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Anemia, Diamond-Blackfan clinical trials

The registry lists 56 studies for Anemia, Diamond-Blackfan across more than 10 countries. 10 are recruiting or about to start. 18 of them are mainly about Anemia, Diamond-Blackfan (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-12.

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Status

Completed
31
Terminated
11
Recruiting
10
Active, not recruiting
2
Withdrawn
1
Status unknown
1

Phase

Phase 2
32
Phase 1
17
Phase 3
4
Phase 4
1

Where

United States
47
United Kingdom
6
Italy
6
Germany
4
Greece
3
Thailand
3

Who runs them

Novartis Pharmaceuticals (5) · National Heart, Lung, and Blood Institute (NHLBI) (5) · Masonic Cancer Center, University of Minnesota (4) · St. Jude Children's Research Hospital (3) · Bellicum Pharmaceuticals (3) · Paul Szabolcs (3) · Northwell Health (3) · UCSF Benioff Children's Hospital Oakland (2)

What is being tested here

Drugs under test in Anemia, Diamond-Blackfan studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted

DrugOpenPhase 3All
Fludarabine229
Alemtuzumab214
Hydroxyurea202
Thiotepa202
Melphalan103
Rituximab102
Deferasirox026
Busulfan025
Fludarabine phosphate015
Cyclophosphamide013

Drug classes tested here Alkylating agents 2 open · Antimetabolites 2 open

Coming up

0phase 3 studies under way
4studies 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 4

Who these studies take

Of the 10 Anemia, Diamond-Blackfan studies recruiting now, 3 (30%) set no upper age limit and 6 (60%) stop at 75 or younger. 10 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (10%) take healthy volunteers.

Recruiting studies that take each age, of 10
AgeWomenMen
1010 (100%)10 (100%)
1810 (100%)10 (100%)
309 (90%)9 (90%)
407 (70%)7 (70%)
506 (60%)6 (60%)
654 (40%)4 (40%)
754 (40%)4 (40%)
854 (40%)4 (40%)

Across all 56 studies, recruiting or not: 63% stop at 75 or younger and 57% at 65; 93% take children; 0% are for women only and 0% for men only; 5% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.

Studies

Recruiting first, then most recently updated. Showing 50 of 56. Browse all 56 → or search them.