9 completed, 5 terminated, 4 recruiting, 2 active not recruiting. 13 Phase 2, 5 Phase 1, 4 Phase 3, 2 Not applicable.
Follow Osteopetrosis: new trials by email Literature Review of Osteopetrosis trials Research Report on Osteopetrosis
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Gene-Modified Stem Cell Therapy for Children With Autosomal Recessive Osteopetrosis (ARO) FT024 - Osteopetrosis | Phase 1 / Phase 2 | Recruiting | Fondazione Telethon | 8 | 2026-06-20 | 2026-06-24 |
| Outcomes of Patients With Osteopetrosis Weight-bearing Bone Fractures Open reduction and internal fixation - Osteopetrosis | Not applicable | Completed | Kasr El Aini Hospital | 6 | 2022-01-01 | 2024-07-26 |
| Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC data collection - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Recruiting | Paul Szabolcs | 50 | 2020-08-20 | 2026-01-13 | |
| ACTIMMUNE in Intermediate Osteopetrosis Interferon gamma-1b - Osteopetrosis | Phase 2 | Completed | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | 5 | 2016-02-22 | 2024-10-22 |
| Use of ACTIMMUNE in Patients With ADO2 ACTIMMUNE - Autosomal Dominant Osteopetrosis Type 2 | Phase 2 | Completed | Indiana University | 12 | 2016-01-01 | 2021-01-08 |
| MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Stem Cell Transplantation, IMD Preparative Regimen, Osteopetrosis Only Preparative Regimen - Mucopolysaccharidosis Disorders, Hurler Syndrome | Phase 2 | Active not recruiting | Masonic Cancer Center, University of Minnesota | 149 | 2014-07-10 | 2026-01-07 |
| Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant BPX-501 T cells, Rimiducid - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Active not recruiting | Bellicum Pharmaceuticals | 120 | 2014-04-01 | 2022-07-12 |
| Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant BPX-501 T cells, Rimiducid - Acute Lymphoblastic Leukemia, Leukemia, Acute Myeloid (AML), Child | Phase 1 / Phase 2 | Terminated | Bellicum Pharmaceuticals | 187 | 2014-04-01 | 2023-09-29 |
| Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT Hydroxyurea, Alemtuzumab, Fludarabine - Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes | Phase 2 | Recruiting | Paul Szabolcs | 100 | 2014-02-04 | 2025-12-15 |
| Longitudinal Study of Neurodegenerative Disorders Palliative Care, Hematopoetic Stem Cell Transplantation - MLD, Krabbe Disease | Recruiting | University of Pittsburgh | 1500 | 2012-01-11 | 2026-02-09 | |
| Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen), Stem Cell Transplantation, Cyclosporin, Methotrexate (GVHD prophylaxis) - Osteopetrosis | Phase 2 / Phase 3 | Unknown | Tehran University of Medical Sciences | 10 | 2009-09-01 | 2012-06-04 |
| T-cell Depleted Alternative Donor Transplantation CliniMACS® (T cell depletion) - Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia | Phase 2 | Terminated | Wake Forest University Health Sciences | 53 | 2009-08-01 | 2022-04-22 |
| Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5 Osteopetrosis | Completed | Odense University Hospital | 38 | 2009-01-01 | 2010-09-10 | |
| Allogeneic Transplantation For Severe Osteopetrosis umbilical cord blood transplantation, Campath-1H, Total Lymphoid Irradiation - Severe Osteopetrosis | Phase 2 / Phase 3 | Completed | Masonic Cancer Center, University of Minnesota | 7 | 2008-08-01 | 2019-07-31 |
| Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells Hematopoietic stem cell transplantation - Sickle Cell Disease, Thalassemia | Phase 1 / Phase 2 | Completed | Children's Hospital Los Angeles | 25 | 2008-08-01 | 2016-06-23 |
| Reduced Intensity AlloTransplant For Osteopetrosis Stem Cell or Umbilical Cord Blood Transplantation, Campath, Busulfan, Clofarabine, Total Lymphoid Irradiation - Osteopetrosis | Phase 2 | Terminated | Masonic Cancer Center, University of Minnesota | 3 | 2007-09-01 | 2017-12-28 |
| Stem Cell Transplantation for Children Affected With Osteopetrosis Stem Cell Transplantation, Miltenyi Biotec CliniMACS, Systemic chemotherapy and antibodies - Osteopetrosis | Not applicable | Terminated | St. Jude Children's Research Hospital | 15 | 2004-07-01 | 2017-05-30 |
| rhPTH Therapy for Low Turnover Bone Fragility rhPTH - Osteopetrosis | Phase 1 | Terminated | University of Chicago | 2 | 2003-02-01 | 2016-01-12 |
| Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases Fludarabine, Cyclophosphamide, Busulfan - Bone Marrow Failure, Osteopetrosis | Phase 2 / Phase 3 | Completed | Columbia University | 38 | 2002-09-01 | 2024-10-31 |
| Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis Actimmune Registry - Osteopetrosis | Completed | InterMune | 6 | 2002-01-01 | 2007-11-01 |
Showing 20 of 22. Sign in to see all of them.
Anemia, Sickle Cell (4) · Leukemia, Myeloid, Acute (4) · Leukodystrophy, Globoid Cell (4) · Mucopolysaccharidosis I (4) · Myelodysplastic Syndromes (4) · Precursor Cell Lymphoblastic Leukemia-Lymphoma (4) · Primary Immunodeficiency Diseases (4) · alpha-Mannosidosis (4) · Anemia, Diamond-Blackfan (3) · Bone Marrow Failure Disorders (3) · Chediak-Higashi Syndrome (3) · Congenital amegakaryocytic thrombocytopenia (3) · Cytopenia (3) · Gaucher Disease (3)
Bone Diseases (21) · Bone Diseases, Developmental (21) · Musculoskeletal Diseases (21) · Osteochondrodysplasias (21) · Osteosclerosis (21) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (11) · Hemic and Lymphatic Diseases (9) · Genetic Diseases, Inborn (8) · Hematologic Diseases (7) · Immune System Diseases (7) · Lymphatic Diseases (7) · Metabolic Diseases (7) · Nutritional and Metabolic Diseases (7) · Pathologic Processes (7) · Pathological Conditions, Signs and Symptoms (7)
Alemtuzumab (4) · Busulfan (4) · Cyclophosphamide (3) · Stem Cell Transplantation (3) · fludarabine (3) · fludarabine phosphate (3) · AP 1903 reagent (2) · Bone Marrow Transplantation (2) · Cord Blood Stem Cell Transplantation (2) · interferon gamma-1b (2) · thymoglobulin (2) · Antibodies (1) · Antilymphocyte Serum (1) · Calcitriol (1) · Clofarabine (1)
Masonic Cancer Center, University of Minnesota (3) · Bellicum Pharmaceuticals (2) · Paul Szabolcs (2) · Children's Hospital Los Angeles (1) · Christopher Dvorak (1) · Columbia University (1) · FDA Office of Orphan Products Development (1) · Fondazione Telethon (1) · Indiana University (1) · InterMune (1) · Kasr El Aini Hospital (1) · Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center (1) · Odense University Hospital (1) · St. Jude Children's Research Hospital (1) · Tehran University of Medical Sciences (1)
United States (16) · Italy (2) · Denmark (1) · Egypt (1) · Iran (1) · United Kingdom (1)
Paul Orchard, MD (2) · Paul Szabolcs, MD (2) · Amir Ali Hamidieh, MD (1) · Andrew Gilman, MD (1) · Christopher Dvorak, MD (1) · Hisham Abdel-Azim, MD (1) · James Garvin, MD, PhD (1) · Kim Brixen, Professor (1) · Kimberly A Kasow, DO (1) · L. Lyndon Key, Jr. (1) · Lynda E Polgreen, MD, MS (1) · Michael J Econs, MD (1) · Paul Orchard, M.D. (1) · Steven Porter, MD (1)
PMID 20200960, PMID 29296761, PMID 30889272, PMID 32634238, PMID 32750127, PMID 34056870