RareTrial

Rare condition

Autosomal dominant osteopetrosis type 1

ORPHA:2783 ↗Gene LRP5Malformation syndrome
Search all Autosomal dominant osteopetrosis type 1 trialsAlert me when a trial opens

0

studies recruiting now

as of 7 Sept 2026

1

studies registered in total

as of 7 Sept 2026

0

countries with a recruiting site

as of 7 Sept 2026

None

recruiting study posted to date

among recruiting studies

Recruiting trials

No recruiting trial found right now.

1 study is registered for Autosomal dominant osteopetrosis type 1, but none was recruiting as of 7 Sept 2026. Here is what is still worth doing.

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Patient organisations

We do not yet list a dedicated organisation for this condition. The directories below are the best route.

NORD ↗GARD ↗Orphanet ORPHA:2783 ↗

About Autosomal dominant osteopetrosis type 1

RareTrial does not yet hold a plain-language description of this condition. The most reliable starting point is Orphanet’s expert page, which lists specialist centres, registries and patient organisations, and the gene involved (LRP5).

Orphanet expert page ↗GARD (NIH) ↗

Treatments being studied

2 approved treatments and 4 in clinical development, from Open Targets (CC BY 4.0). Not medical advice.

Approved: Interferon Gamma-1b (Actimmune)Approved: Bazedoxifene (Bazedoxifene component of ak r215)
Phase 3Calcitriol (Calcijex)
Phase 2Clofarabine (Clolar)
Phase 2Apomine
Phase 2Busulfan (Busilvex)

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Information, not medical advice. Trial listings are shown as recorded on ClinicalTrials.gov; whether any study is right for you is a decision for you and your clinicians, and eligibility is decided by each research team. Disease information from Orphanet (CC BY 4.0).