Condition (MeSH)
Osteopetrosis
ClinicalTrials.gov lists 22 trials for Osteopetrosis or a narrower condition beneath it. Bellicum Pharmaceuticals has the most among industry and government lead sponsors, with 2.
Search trials, drugs, companies and investigators, and follow each one to the others. Individual plan, $249/seat/mo. Card required.
Trials per phase
22 of the 22 trials are tagged with Osteopetrosis itself; the rest are tagged with a narrower condition beneath it.
When did Osteopetrosis trials start? (13)
Shown in this order: most recent first, 1990 onwards. 2026 is partial: trials registered with a 2026 start date so far, not a full year.
Trials over time, by phase
- Phase 1
- Phase 1/2
- Phase 2
- Phase 2/3
- Phase 3
- Not applicable
- Observational
Top of scale: 4
Trials registered with a start date in each year, 1990 onward, stacked by phase (complete years only; the current partial year is in the table's notes). The table below lists the same counts, most recent years first.
| Year | Trials started |
|---|---|
| 2026 (partial year) | 1 |
| 2022 | 1 |
| 2020 | 1 |
| 2016 | 2 |
| 2014 | 4 |
| 2012 | 1 |
| 2009 | 3 |
| 2008 | 2 |
| 2007 | 1 |
| 2004 | 1 |
Showing 10 of 13 earlier years.
No forecast is made for this condition: it averages 0.4 trial starts a year over the five years to September, under the 5 a year the method needs.
Recruitment duration, a model prediction: none of the 22 trials evaluated at the 2026-10-09 snapshot is predicted and still running. Source: ClinicalTrials.gov / AACT (CTTI), public domain.
What phase are Osteopetrosis trials in? (8)
Shown in this order: phase order.
Show the trials by phase as a table
| Phase | Trials |
|---|---|
| Phase 1 | 1 |
| Phase 1/2 | 4 |
| Phase 2 | 6 |
| Phase 2/3 | 3 |
| Phase 3 | 1 |
| Not applicable | 2 |
| Observational | 4 |
| Expanded access | 1 |
How many Osteopetrosis trials are recruiting? (6)
Shown in this order: the order a trial moves through, from not yet recruiting to unknown.
| Status | Trials |
|---|---|
| Recruiting | 4 |
| Active, not recruiting | 2 |
| Completed | 9 |
| Terminated | 5 |
| No longer available | 1 |
Showing 5 of 6 statuses.
Status as registered on ClinicalTrials.gov. Each trial is counted once.
Which lead sponsors run the most Osteopetrosis trials? (3)
Shown in this order: most trials first. Lead sponsors the registry classes as industry, NIH, US federal, other government or network. A hospital or university the registry files as government can appear here. Sponsors it classes as academic or other are shown on the full page, because that class cannot separate an institution from a named investigator.
| Sponsor | Trials |
|---|---|
| Bellicum Pharmaceuticals | 2 |
| FDA Office of Orphan Products Development | 1 |
| InterMune | 1 |
Which drugs are being tested for Osteopetrosis? (3)
Shown in this order: most trials first. One row per drug, matched to a single molecule under all its names, ranked by number of trials.
| Drug | Trials |
|---|---|
| Cytoxan | 2 |
| Busulfan | 2 |
| Rimiducid | 2 |
Interventions as named in the registry (12)
Shown in this order: most trials first.
| Drug | Trials |
|---|---|
| Alemtuzumab s.c. | 3 |
| SQ Fludarabine | 3 |
| Stem Cell Transplantation | 3 |
| BPX-501 T cells | 2 |
| Gamma Interferon 1b | 2 |
Showing 5 of 12 interventions.
These are the names sponsors entered on ClinicalTrials.gov that we have not matched to a single molecule. Some are drugs under a brand or code name; others are procedures, doses or arm labels.
Which serious adverse events do Osteopetrosis trials report? (10)
Shown in this order: most subjects affected first. Subjects affected summed across trials, a cross-trial signal, not a rate.
| Adverse event | Subjects affected | Trials |
|---|---|---|
| Fever | 32 | 1 |
| Graft vs. Host disease | 9 | 1 |
| HHV-6 Reactivation | 9 | 1 |
| Thrombotic microangiopathy | 8 | 2 |
| Pyrexia | 7 | 1 |
Showing 5 of 10 adverse events.
Which publications cover Osteopetrosis? (17)
Shown in this order: major-topic papers first, then newest, then most linked to trials.
Showing 5 of 17 publications.
Prevalence (6)
Shown in this order: jurisdiction, then disease name. Orphanet observation for the mapped ORPHA concept; the mapping relation column says whether that concept is exactly this condition or broader or narrower.
| Orphanet disease | Type | Class | Geography | Jurisdiction | Mapping relation |
|---|---|---|---|---|---|
| Osteopetrosis and related disorders | Annual incidence | 1-9 / 100 000 | Europe | Not stated | Exact |
| Osteopetrosis and related disorders | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Osteopetrosis and related disorders | Point prevalence | Unknown | Worldwide | Not stated | Exact |
| Osteopetrosis and related disorders | Annual incidence | 1-9 / 100 000 | Europe | Not stated | Unspecified |
| Osteopetrosis and related disorders | Annual incidence | 1-9 / 100 000 | Europe | Not stated | Exact |
Showing 5 of 6 prevalence observations.
Rare-disease identity (2)
Shown in this order: exact, broader, narrower, other.
| Orphanet disease | ORPHA code | Relation | Source |
|---|---|---|---|
| Osteopetrosis and related disorders | 2781 | Exact | Orphadata Science knowledge files, Product 1 |
| Osteopetrosis and related disorders | 2781 | Exact | Orphanet Rare Disease Ontology (ORDO) |
Not on this page
Not measured: delivery record.
Checked, with nothing recorded for this condition: sub-conditions in mesh.