Osteopetrosis clinical trials
The registry lists 23 studies for Osteopetrosis across 6 countries. 4 are recruiting or about to start. Per ClinicalTrials.gov, last updated 2026-06-24.
Status
- Completed
- 9
- Terminated
- 6
- Recruiting
- 4
- Active, not recruiting
- 2
- No longer available
- 1
- Status unknown
- 1
Phase
- Phase 2
- 13
- Phase 1
- 6
- Phase 3
- 4
Where
- United States
- 17
- Italy
- 2
- United Kingdom
- 1
- Egypt
- 1
- Denmark
- 1
- Iran, Islamic Republic of
- 1
Who runs them
Masonic Cancer Center, University of Minnesota (3) · Bellicum Pharmaceuticals (2) · Paul Szabolcs (2) · Children's Hospital Los Angeles (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · St. Jude Children's Research Hospital (1) · Kasr El Aini Hospital (1) · University of Chicago (1)
Studies
Recruiting first, then most recently updated. Showing 23 of 23 — search for more.
- Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO)RecruitingNCT07665021Phase 1/21 country
- Longitudinal Study of Neurodegenerative DisordersRecruitingNCT033332001 country
- Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RICRecruitingNCT045283551 country
- Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCTRecruitingNCT01962415Phase 21 country
- MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisActive, not recruitingNCT02171104Phase 21 country
- Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesCompletedNCT01019876Phase 2/31 country
- ACTIMMUNE in Intermediate OsteopetrosisCompletedNCT02666768Phase 21 country
- Outcomes of Patients With Osteopetrosis Weight-bearing Bone FracturesCompletedNCT06521580Not applicable1 country
- Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell TransplantTerminatedNCT02065869Phase 1/22 countries
- A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant OsteopetrosisTerminatedNCT04525352Phase 11 country
- Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantActive, not recruitingNCT03301168Phase 1/21 country
- T-cell Depleted Alternative Donor TransplantationTerminatedNCT00968864Phase 21 country
- Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell TransplantNo longer availableNCT012000171 country
- Use of ACTIMMUNE in Patients With ADO2CompletedNCT02584608Phase 21 country
- Allogeneic Transplantation For Severe OsteopetrosisCompletedNCT00775931Phase 2/31 country
- Reduced Intensity AlloTransplant For OsteopetrosisTerminatedNCT00638820Phase 21 country
- Stem Cell Transplantation for Children Affected With OsteopetrosisTerminatedNCT00145587Not applicable1 country
- Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood CellsCompletedNCT00730314Phase 1/21 country
- rhPTH Therapy for Low Turnover Bone FragilityTerminatedNCT00145886Phase 11 country
- Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital OsteopetrosisCompletedNCT00004402Phase 3
- Hematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisStatus unknownNCT01087398Phase 2/31 country
- Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5CompletedNCT011990941 country
- Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant OsteopetrosisCompletedNCT000433291 country