326. Osteopetrosis
20 clinical trials,   52 drugs   (DrugBank: 14 drugs),   16 drug target genes,   78 drug target pathways

Searched query = "Osteopetrosis"
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
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size
PhaseCountries
1NCT04525352
(ClinicalTrials.gov)
October 202011/8/2020A Trial to Evaluate Safety and Efficacy of RP-L401-0120 in Subjects With Infantile Malignant OsteopetrosisA 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 GeneInfantile Malignant OsteopetrosisBiological: RP-L401Rocket Pharmaceuticals Inc.NULLNot yet recruiting1 MonthN/AAll2Phase 1NULL
2EUCTR2014-000584-41-ES
(EUCTR)
08/06/201721/03/2017Phase 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 disordersPhase 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.NULLNot RecruitingFemale: yes
Male: yes
175Phase 2Spain;United Kingdom;Italy
3EUCTR2016-003226-16-IT
(EUCTR)
03/02/201717/11/2016Follow-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 disordersFollow-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.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
175Phase 1;Phase 2Italy
4NCT02666768
(ClinicalTrials.gov)
February 22, 201623/6/2015ACTIMMUNE in Intermediate OsteopetrosisOpen-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of OsteopetrosisOsteopetrosisDrug: Interferon gamma-1bLundquist Institute for Biomedical Innovation at Harbor-UCLA Medical CenterUniversity of Minnesota;Horizon Pharma Ireland, Ltd., Dublin IrelandCompleted1 YearN/AAll5Phase 2United States
5NCT02584608
(ClinicalTrials.gov)
January 1, 201620/10/2015Use of ACTIMMUNE in Patients With ADO2Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 OsteopetrosisAutosomal Dominant Osteopetrosis Type 2Drug: ACTIMMUNEIndiana UniversityHorizon Pharma Ireland, Ltd., Dublin IrelandCompleted3 Years65 YearsAll12Phase 2United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2014-000584-41-IT
(EUCTR)
23/10/201415/04/2014Phase 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 disordersPhase 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.NULLNot RecruitingFemale: yes
Male: yes
30Phase 1;Phase 2Spain;United Kingdom;Italy
7NCT02171104
(ClinicalTrials.gov)
July 10, 201420/6/2014MT2013-31: Allo HCT for Metabolic Disorders and Severe OsteopetrosisMT2013-31: Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis Following Conditioning With Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATGMucopolysaccharidosis 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 DisordersBiological: 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 MinnesotaNULLRecruitingN/A55 YearsAll100Phase 2United States
8NCT02065869
(ClinicalTrials.gov)
April 201413/2/2014Safety Study of Gene Modified Donor T-cells Following TCR Alpha Beta Depleted Stem Cell TransplantPhase 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 DisordersAcute 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 CellBiological: BPX-501 T cells;Drug: rimiducidBellicum PharmaceuticalsNULLActive, not recruiting1 Month18 YearsAll193Phase 2Italy;United Kingdom;Germany;Spain;United States
9NCT03301168
(ClinicalTrials.gov)
April 201429/9/2017Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell TransplantPhase 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 DisordersAcute Lymphoblastic Leukemia;Leukemia, Acute Myeloid (AML), Child;Lymphoma, Non-Hodgkin;Myelodysplastic Syndromes;Primary Immune Deficiency Disorder;Osteopetrosis;Cytopenia;Hemoglobinopathy in Children;Anemia, AplasticBiological: BPX-501 T cells;Drug: RimiducidBellicum PharmaceuticalsNULLActive, not recruiting1 Month26 YearsAll120Phase 1;Phase 2United States;Italy
10NCT03333200
(ClinicalTrials.gov)
January 11, 201224/4/2017Longitudinal Study of Neurodegenerative DisordersLongitudinal Study of Neurodegenerative DisordersMLD;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 DiseaseOther: Palliative Care;Biological: Hematopoetic Stem Cell TransplantationUniversity of PittsburghNULLRecruitingN/AN/AAll1500United States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT01200017
(ClinicalTrials.gov)
August 20109/9/2010Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell TransplantAn 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 RecipientsAcute Lymphoblastic Leukemia;Acute Myeloid Leukemia;Chronic Myeloid Leukemia;Myelodysplastic Syndrome;Lymphomas;Bone Marrow Failure;Hemoglobinopathy;Immune Deficiency;Osteopetrosis;Cytopenias;Leukocyte Disorders;Anemia Due to Intrinsic Red Cell AbnormalityBiological: CD34+ enriched, T Cell Depleted donor stem cell productChristopher DvorakNULLAvailable2 Months30 YearsAllUnited States
12NCT01087398
(ClinicalTrials.gov)
September 200913/3/2010Hematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisHematopoietic Stem Cell Transplantation for Malignant Infantile OsteopetrosisOsteopetrosisDrug: Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen);Procedure: Stem Cell Transplantation;Drug: Cyclosporin, Methotrexate (GVHD prophylaxis)Tehran University of Medical SciencesNULLRecruitingN/A5 YearsBoth10Phase 2;Phase 3Iran, Islamic Republic of
13NCT00775931
(ClinicalTrials.gov)
August 200816/10/2008Allogeneic Transplantation For Severe OsteopetrosisAllogeneic Hematopoietic Stem Cell Transplantation For Severe OsteopetrosisSevere OsteopetrosisProcedure: umbilical cord blood transplantation;Drug: Campath-1H;Radiation: Total Lymphoid Irradiation;Drug: Cyclophosphamide;Drug: Busulfan;Drug: Fludarabine monophosphate;Procedure: marrow graft transplantationMasonic Cancer Center, University of MinnesotaNULLCompletedN/A45 YearsAll7Phase 2;Phase 3United States
14NCT00638820
(ClinicalTrials.gov)
September 200711/3/2008Reduced Intensity AlloTransplant For OsteopetrosisReduced Intensity Allogeneic Transplantation For Severe Osteopetrosis Incorporating A Second Cd34 Selected GraftOsteopetrosisProcedure: Stem Cell or Umbilical Cord Blood Transplantation;Drug: Campath, Busulfan, Clofarabine;Procedure: Total Lymphoid IrradiationMasonic Cancer Center, University of MinnesotaNULLTerminatedN/A45 YearsAll3Phase 2United States
15NCT00145587
(ClinicalTrials.gov)
July 20041/9/2005Stem Cell Transplantation for Children Affected With OsteopetrosisAllogeneic Hematopoietic Stem Cell Transplantation for Children Affected With Malignant Osteopetrosis: A Pilot StudyOsteopetrosisProcedure: Stem Cell Transplantation;Device: Miltenyi Biotec CliniMACS;Drug: Systemic chemotherapy and antibodiesSt. Jude Children's Research HospitalNULLTerminatedN/AN/AAll15N/AUnited States
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16NCT00145886
(ClinicalTrials.gov)
February 20031/9/2005rhPTH Therapy for Low Turnover Bone FragilityEffect 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 DisordersOsteopetrosisDrug: rhPTHUniversity of ChicagoNULLTerminated20 YearsN/ABoth2Phase 1United States
17NCT01019876
(ClinicalTrials.gov)
June 200223/11/2009Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant DiseasesRisk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Selected Non-Malignant DiseasesBone Marrow Failure;Osteopetrosis;Fanconi Anemia;Severe Combined ImmunodeficiencyDrug: Fludarabine;Drug: Cyclophosphamide;Drug: Cyclophosphamide 40;Drug: Cyclophosphamide 30Columbia UniversityNULLRecruitingN/A30 YearsBoth50Phase 2;Phase 3United States
18NCT00043329
(ClinicalTrials.gov)
January 20027/8/2002Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant OsteopetrosisPost-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant OsteopetrosisOsteopetrosisDrug: Actimmune RegistryInterMuneNULLCompletedN/AN/ABoth6United States
19NCT00004402
(ClinicalTrials.gov)
November 199918/10/1999Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital OsteopetrosisOsteopetrosisDrug: calcitriol;Drug: interferon gammaFDA Office of Orphan Products DevelopmentMedical University of South CarolinaCompletedN/A10 YearsBoth30Phase 3NULL
20EUCTR2014-000584-41-GB
(EUCTR)
15/07/2015Phase I/II study of CaspaCide T cells in children following aß- depleted mis-matched family donor stem cell transplantationPhase 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.NULLNAFemale: yes
Male: yes
175Phase 2Spain;Italy;United Kingdom