326. Osteopetrosis Clinical trials / Disease details
Clinical trials : 20 / Drugs : 51 - (DrugBank : 14) / Drug target genes : 17 - Drug target pathways : 81
| No. | TrialID | Date_ enrollment | Date_ registration | Public_title | Scientific_title | Condition | Intervention | Primary_ sponsor | Secondary_ sponsor | Recruitment_ Status | Inclusion_ agemin | Inclusion_ agemax | Inclusion_ gender | Target_ size | Phase | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 1 | NCT04525352 (ClinicalTrials.gov) | February 1, 2021 | 11/8/2020 | A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant Osteopetrosis | A Phase I Clinical Trial for Gene Therapy in Infantile Malignant Osteopetrosis (IMO) to Evaluate the Safety and Preliminary Efficacy of Autologous CD34+ Enriched Cells Transduced With a LV Vector Encoding the TCIRG1 Gene | Infantile Malignant Osteopetrosis | Biological: RP-L401 | Rocket Pharmaceuticals Inc. | California Institute for Regenerative Medicine (CIRM) | Active, not recruiting | 1 Month | N/A | All | 2 | Phase 1 | United States |
| 2 | EUCTR2014-000584-41-ES (EUCTR) | 08/06/2017 | 21/03/2017 | Phase II extension study of CaspaCIDe T cells (BPX-501) from an HLA-partially matched family donor after negative selection of TCR aß+T cells in pediatric patients affected by hematological disorders | Phase II extension study of CaspaCIDe T cells (BPX-501) from an HLA-partially matched family donor after negative selection of TCR aß+T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 19.1;Level: HLGT;Classification code 10018849;Term: Haematological disorders NEC;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BPX-501 cells INN or Proposed INN: rivogenleucleucel Other descriptive name: BPX-501 Product Name: AP1903 INN or Proposed INN: Rimiducid Other descriptive name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 175 | Phase 2 | Spain;United Kingdom;Italy | ||
| 3 | EUCTR2016-003226-16-IT (EUCTR) | 03/02/2017 | 17/11/2016 | Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders | Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 19.0;Level: HLGT;Classification code 10018849;Term: Haematological disorders NEC;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: AP1903 Other descriptive name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 175 | Phase 1;Phase 2 | Italy | ||
| 4 | NCT02666768 (ClinicalTrials.gov) | February 22, 2016 | 23/6/2015 | ACTIMMUNE in Intermediate Osteopetrosis | Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis | Osteopetrosis | Drug: Interferon gamma-1b | Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center | University of Minnesota;Horizon Pharma Ireland, Ltd., Dublin Ireland | Completed | 1 Year | N/A | All | 5 | Phase 2 | United States |
| 5 | NCT02584608 (ClinicalTrials.gov) | January 1, 2016 | 20/10/2015 | Use of ACTIMMUNE in Patients With ADO2 | Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis | Autosomal Dominant Osteopetrosis Type 2 | Drug: ACTIMMUNE | Indiana University | Horizon Pharma Ireland, Ltd., Dublin Ireland | Completed | 3 Years | 65 Years | All | 12 | Phase 2 | United States |
| 6 | EUCTR2014-000584-41-IT (EUCTR) | 23/10/2014 | 15/04/2014 | Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders | Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT | Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplasticsyndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopathies) MedDRA version: 16.1;Level: HLGT;Classification code 10018849;Term: Haematological disorders NEC;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BPX-501 cells Product Name: AP1903 | Bellicum Pharmaceuticals, Inc. | NULL | Not Recruiting | Female: yes Male: yes | 30 | Phase 1;Phase 2 | Italy;United Kingdom;Spain | ||
| 7 | NCT02171104 (ClinicalTrials.gov) | July 10, 2014 | 20/6/2014 | MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis | MT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG | Mucopolysaccharidosis Disorders;Hurler Syndrome;Hunter Syndrome;Maroteaux Lamy Syndrome;Sly Syndrome;Alpha-Mannosidosis;Fucosidosis;Aspartylglucosaminuria;Glycoprotein Metabolic Disorders;Sphingolipidoses;Recessive Leukodystrophies;Globoid Cell Leukodystrophy;Metachromatic Leukodystrophy;Niemann-Pick B;Niemann-Pick C Subtype 2;Sphingomyelin Deficiency;Peroxisomal Disorders;Adrenoleukodystrophy With Cerebral Involvement;Zellweger Syndrome;Neonatal Adrenoleukodystrophy;Infantile Refsum Disease;Acyl-CoA Oxidase Deficiency;D-Bifunctional Enzyme Deficiency;Multifunctional Enzyme Deficiency;Alpha-methylacyl-CoA Racmase Deficiency;Mitochondrial Neurogastrointestingal Encephalopathy;Severe Osteopetrosis;Hereditary Leukoencephalopathy With Axonal Spheroids (HDLS; CSF1R Mutation);Inherited Metabolic Disorders | Biological: Stem Cell Transplantation;Drug: IMD Preparative Regimen;Drug: Osteopetrosis Only Preparative Regimen;Drug: Osteopetrosis Haploidentical Only Preparative Regimen;Drug: cALD SR-A (Standard-Risk, Regimen A);Drug: cALD SR-B (Standard-Risk, Regimen B);Drug: cALD HR-D (High-Risk, Regimen C);Drug: cALD HR-D (High-Risk, Regimen D) | Masonic Cancer Center, University of Minnesota | NULL | Recruiting | N/A | 55 Years | All | 100 | Phase 2 | United States |
| 8 | NCT02065869 (ClinicalTrials.gov) | April 2014 | 13/2/2014 | Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell Transplant | Phase II Extension Study of CaspaCIDe T Cells (BPX-501) From an HLA-partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndrome;Primary Immunodeficiency;Anemia, Aplastic;Osteopetrosis;Hemoglobinopathies;Cytopenia;Fanconi Anemia;Diamond Blackfan Anemia;Thalassemia;Anemia, Sickle Cell | Biological: BPX-501 T cells;Drug: rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 18 Years | All | 193 | Phase 2 | Italy;United Kingdom;Germany;Spain;United States |
| 9 | NCT03301168 (ClinicalTrials.gov) | April 2014 | 29/9/2017 | Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant | Phase I/II Study of CaspaCIDe® T Cells From an HLA-Partially Matched Family Donor After Negative Selection of TCR aß+T Cells in Pediatric Patients Affected by Hematological Disorders | Acute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immune Deficiency Disorder;Osteopetrosis;Cytopenia;Hemoglobinopathy in Children;Anemia, Aplastic | Biological: BPX-501 T cells;Drug: Rimiducid | Bellicum Pharmaceuticals | NULL | Active, not recruiting | 1 Month | 26 Years | All | 120 | Phase 1/Phase 2 | United States;Italy |
| 10 | NCT03333200 (ClinicalTrials.gov) | January 11, 2012 | 24/4/2017 | Longitudinal Study of Neurodegenerative Disorders | Longitudinal Study of Neurodegenerative Disorders | MLD;Krabbe Disease;ALD;MPS I;MPS II;MPS III;Vanishing White Matter Disease;GM3 Gangliosidosis;PKAN;Tay-Sachs Disease;NP Deficiency;Osteopetrosis;Alpha-Mannosidosis;Sandhoff Disease;Niemann-Pick Diseases;MPS IV;Gaucher Disease;GAN;GM1 Gangliosidoses;Morquio Disease;S-Adenosylhomocysteine Hydrolase Deficiency;Batten Disease;Pelizaeus-Merzbacher Disease;Leukodystrophy;Lysosomal Storage Diseases;Purine Nucleoside Phosphorylase Deficiency;Multiple Sulfatase Deficiency Disease | Other: Palliative Care;Biological: Hematopoetic Stem Cell Transplantation | University of Pittsburgh | NULL | Recruiting | N/A | N/A | All | 1500 | United States | |
| 11 | NCT01200017 (ClinicalTrials.gov) | September 2010 | 9/9/2010 | Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant | An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients | Cytopenias;Leukocyte Disorders;Anemia Due to Intrinsic Red Cell Abnormality;Acute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Myelodysplastic Syndrome;Lymphomas;Bone Marrow Failure;Hemoglobinopathy;Immune Deficiency;Osteopetrosis | Biological: CD34+ enriched, T Cell Depleted donor stem cell product | Christopher Dvorak | NULL | No longer available | 2 Months | 30 Years | All | United States | ||
| 12 | NCT01087398 (ClinicalTrials.gov) | September 2009 | 13/3/2010 | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Hematopoietic Stem Cell Transplantation for Malignant Infantile Osteopetrosis | Osteopetrosis | Drug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis) | Tehran University of Medical Sciences | NULL | Recruiting | N/A | 5 Years | Both | 10 | Phase 2/Phase 3 | Iran, Islamic Republic of |
| 13 | NCT00775931 (ClinicalTrials.gov) | August 2008 | 16/10/2008 | Allogeneic Transplantation For Severe Osteopetrosis | Allogeneic Hematopoietic Stem Cell Transplantation For Severe Osteopetrosis | Severe Osteopetrosis | Procedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantation | Masonic Cancer Center, University of Minnesota | NULL | Completed | N/A | 45 Years | All | 7 | Phase 2/Phase 3 | United States |
| 14 | NCT00638820 (ClinicalTrials.gov) | September 2007 | 11/3/2008 | Reduced Intensity AlloTransplant For Osteopetrosis | Reduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected Graft | Osteopetrosis | Procedure: Stem Cell or Umbilical Cord Blood Transplantation;Drug: Campath, Busulfan, Clofarabine;Procedure: Total Lymphoid Irradiation | Masonic Cancer Center, University of Minnesota | NULL | Terminated | N/A | 45 Years | All | 3 | Phase 2 | United States |
| 15 | NCT00145587 (ClinicalTrials.gov) | July 2004 | 1/9/2005 | Stem Cell Transplantation for Children Affected With Osteopetrosis | Allogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot Study | Osteopetrosis | Procedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodies | St. Jude Children's Research Hospital | NULL | Terminated | N/A | N/A | All | 15 | N/A | United States |
| 16 | NCT00145886 (ClinicalTrials.gov) | February 2003 | 1/9/2005 | rhPTH Therapy for Low Turnover Bone Fragility | Effect of 12 Months Treatment With rhPTH on Calcium Balance, Bone Turnover, Bone Mineral Density, and Bone Micro-architecture in Patients With Fractures Associated With Low Bone Turnover and Sclerosing Bone Disorders | Osteopetrosis | Drug: rhPTH | University of Chicago | NULL | Terminated | 20 Years | N/A | Both | 2 | Phase 1 | United States |
| 17 | NCT01019876 (ClinicalTrials.gov) | June 2002 | 23/11/2009 | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases | Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant Diseases | Bone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined Immunodeficiency | Drug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30 | Columbia University | NULL | Recruiting | N/A | 30 Years | Both | 50 | Phase 2/Phase 3 | United States |
| 18 | NCT00043329 (ClinicalTrials.gov) | January 2002 | 7/8/2002 | Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis | Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis | Osteopetrosis | Drug: Actimmune Registry | InterMune | NULL | Completed | N/A | N/A | Both | 6 | United States | |
| 19 | NCT00004402 (ClinicalTrials.gov) | November 1999 | 18/10/1999 | Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis | Osteopetrosis | Drug: calcitriol;Drug: interferon gamma | FDA Office of Orphan Products Development | Medical University of South Carolina | Completed | N/A | 10 Years | Both | 30 | Phase 3 | NULL | |
| 20 | EUCTR2014-000584-41-GB (EUCTR) | 15/07/2015 | Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantation | Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR aß+ T cells in pediatric patients affected by hematological disorders - CaspaCide TCR aß haplo HSCT | Hematological disorders (ALL; AML; Non-Hodgkin lymphoma; Myelodysplastic syndromes; Congenital immune deficiencies; Severe aplastic anemia; Fanconi anemia; Osteopetrosis; Selected cases of hemoglobinopathies) MedDRA version: 20.0;Level: HLGT;Classification code 10018849;Term: Haematological disorders NEC;System Organ Class: 10005329 - Blood and lymphatic system disorders;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15] | Product Name: BPX-501 cells INN or Proposed INN: Rivogenlecleucel Other descriptive name: BPX-501 Product Name: rimiducid INN or Proposed INN: Rimiducid Other descriptive name: AP1903 A594 | Bellicum Pharmaceuticals, Inc. | NULL | NA | Female: yes Male: yes | 175 | Phase 2 | Spain;Italy;United Kingdom |