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Osteopetrosis clinical trials

The registry lists 22 studies for Osteopetrosis across 6 countries. 4 are recruiting or about to start. 12 of them are mainly about Osteopetrosis (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-03.

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Status

Completed
9
Terminated
5
Recruiting
4
Active, not recruiting
2
Status unknown
1
No longer available
1

Phase

Phase 2
13
Phase 1
5
Phase 3
4

Where

United States
16
Italy
2
Iran, Islamic Republic of
1
Egypt
1
Denmark
1
United Kingdom
1

Who runs them

Masonic Cancer Center, University of Minnesota (3) · Bellicum Pharmaceuticals (2) · Paul Szabolcs (2) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · St. Jude Children's Research Hospital (1) · Wake Forest University Health Sciences (1) · Kasr El Aini Hospital (1) · InterMune (1)

What is being tested here

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

DrugOpenPhase 3All
Fludarabine112
Alemtuzumab102
Hydroxyurea101
Melphalan101
Thiotepa101
Rivogenlecleucel002
Cyclophosphamide022
Interferon gamma-1b002
AP 1903 reagent001
Thymoglobulin011

Drug classes tested here Alkylating agents 1 open · Antimetabolites 1 open

Coming up

0phase 3 studies under way
1study finishes 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 study

Who these studies take

Of the 4 Osteopetrosis studies recruiting now, 1 (25%) set no upper age limit and 3 (75%) stop at 75 or younger. 4 (100%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.

Recruiting studies that take each age, of 4
AgeWomenMen
103 (75%)3 (75%)
183 (75%)3 (75%)
303 (75%)3 (75%)
403 (75%)3 (75%)
503 (75%)3 (75%)
651 (25%)1 (25%)
751 (25%)1 (25%)
851 (25%)1 (25%)

Across all 22 studies, recruiting or not: 68% stop at 75 or younger and 68% at 65; 91% 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 22 of 22. search them.