326. Osteopetrosis Disease details / Clinical trials / Drug dev / DR info
Clinical trials : 18 / Drugs : 43 - (DrugBank : 14) / Drug target genes : 17 - Drug target pathways : 82
Drugs and their primary sponsors and trial info
AP1903
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
2017 Phase 1;Phase 2 EUCTR2016-003226-16-IT Italy;
2014 Phase 1;Phase 2 EUCTR2014-000584-41-IT Italy;Spain;United Kingdom;
AP1903 A594
Bellicum Pharmaceuticals, Inc.
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Actimmune Registry
InterMune
2002 - NCT00043329 United States;
BPX-501 T cells
Bellicum Pharmaceuticals
2014 Phase 2 NCT02065869 Germany;Italy;Spain;United Kingdom;United States;
2014 Phase 1/Phase 2 NCT03301168 Italy;United States;
BPX-501 cells
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2014-000584-41-IT Italy;Spain;United Kingdom;
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Busulfan
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
Tehran University of Medical Sciences
2009 Phase 2/Phase 3 NCT01087398 Iran, Islamic Republic of;
CALD HR-D (High-Risk, Regimen C)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD HR-D (High-Risk, Regimen D)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-A (Standard-Risk, Regimen A)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-B (Standard-Risk, Regimen B)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CD34+ enriched, T Cell Depleted donor stem cell product
Christopher Dvorak
2010 - NCT01200017 United States;
Calcitriol
FDA Office of Orphan Products Development
1999 Phase 3 NCT00004402 -
Campath, Busulfan, Clofarabine
Masonic Cancer Center, University of Minnesota
2007 Phase 2 NCT00638820 United States;
Campath-1H
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Cyclophosphamide
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Cyclophosphamide 30
Columbia University
2002 Phase 2/Phase 3 NCT01019876 United States;
Cyclophosphamide 40
Columbia University
2002 Phase 2/Phase 3 NCT01019876 United States;
Cyclosporin, Methotrexate (GVHD prophylaxis)
Tehran University of Medical Sciences
2009 Phase 2/Phase 3 NCT01087398 Iran, Islamic Republic of;
Fludarabine
Columbia University
2002 Phase 2/Phase 3 NCT01019876 United States;
Fludarabine monophosphate
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Hematopoetic Stem Cell Transplantation
University of Pittsburgh
2012 - NCT03333200 United States;
IMD Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Interferon gamma-1b
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2016 Phase 2 NCT02666768 United States;
Marrow graft transplantation
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Miltenyi Biotec CliniMACS
St. Jude Children's Research Hospital
2004 - NCT00145587 United States;
Osteopetrosis Haploidentical Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Osteopetrosis Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
RP-L401
Rocket Pharmaceuticals Inc.
2021 Phase 1 NCT04525352 United States;
RhPTH
University of Chicago
2003 Phase 1 NCT00145886 United States;
Rimiducid
Bellicum Pharmaceuticals
2014 Phase 2 NCT02065869 Germany;Italy;Spain;United Kingdom;United States;
2014 Phase 1/Phase 2 NCT03301168 Italy;United States;
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Rivogenlecleucel
Bellicum Pharmaceuticals, Inc.
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Rivogenleucleucel
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
Stem Cell Transplantation
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
St. Jude Children's Research Hospital
2004 - NCT00145587 United States;
Tehran University of Medical Sciences
2009 Phase 2/Phase 3 NCT01087398 Iran, Islamic Republic of;
Stem Cell or Umbilical Cord Blood Transplantation
Masonic Cancer Center, University of Minnesota
2007 Phase 2 NCT00638820 United States;
Systemic chemotherapy and antibodies
St. Jude Children's Research Hospital
2004 - NCT00145587 United States;
Total Lymphoid Irradiation
Masonic Cancer Center, University of Minnesota
2007 Phase 2 NCT00638820 United States;
Umbilical cord blood transplantation
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
2017 Phase 1;Phase 2 EUCTR2016-003226-16-IT Italy;
2014 Phase 1;Phase 2 EUCTR2014-000584-41-IT Italy;Spain;United Kingdom;
AP1903 A594
Bellicum Pharmaceuticals, Inc.
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Actimmune Registry
InterMune
2002 - NCT00043329 United States;
BPX-501 T cells
Bellicum Pharmaceuticals
2014 Phase 2 NCT02065869 Germany;Italy;Spain;United Kingdom;United States;
2014 Phase 1/Phase 2 NCT03301168 Italy;United States;
BPX-501 cells
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
2014 Phase 1;Phase 2 EUCTR2014-000584-41-IT Italy;Spain;United Kingdom;
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Busulfan
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen)
Tehran University of Medical Sciences
2009 Phase 2/Phase 3 NCT01087398 Iran, Islamic Republic of;
CALD HR-D (High-Risk, Regimen C)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD HR-D (High-Risk, Regimen D)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-A (Standard-Risk, Regimen A)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CALD SR-B (Standard-Risk, Regimen B)
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
CD34+ enriched, T Cell Depleted donor stem cell product
Christopher Dvorak
2010 - NCT01200017 United States;
Calcitriol
FDA Office of Orphan Products Development
1999 Phase 3 NCT00004402 -
Campath, Busulfan, Clofarabine
Masonic Cancer Center, University of Minnesota
2007 Phase 2 NCT00638820 United States;
Campath-1H
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Cyclophosphamide
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Cyclophosphamide 30
Columbia University
2002 Phase 2/Phase 3 NCT01019876 United States;
Cyclophosphamide 40
Columbia University
2002 Phase 2/Phase 3 NCT01019876 United States;
Cyclosporin, Methotrexate (GVHD prophylaxis)
Tehran University of Medical Sciences
2009 Phase 2/Phase 3 NCT01087398 Iran, Islamic Republic of;
Fludarabine
Columbia University
2002 Phase 2/Phase 3 NCT01019876 United States;
Fludarabine monophosphate
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Hematopoetic Stem Cell Transplantation
University of Pittsburgh
2012 - NCT03333200 United States;
IMD Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Interferon gamma-1b
Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center
2016 Phase 2 NCT02666768 United States;
Marrow graft transplantation
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;
Miltenyi Biotec CliniMACS
St. Jude Children's Research Hospital
2004 - NCT00145587 United States;
Osteopetrosis Haploidentical Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
Osteopetrosis Only Preparative Regimen
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
RP-L401
Rocket Pharmaceuticals Inc.
2021 Phase 1 NCT04525352 United States;
RhPTH
University of Chicago
2003 Phase 1 NCT00145886 United States;
Rimiducid
Bellicum Pharmaceuticals
2014 Phase 2 NCT02065869 Germany;Italy;Spain;United Kingdom;United States;
2014 Phase 1/Phase 2 NCT03301168 Italy;United States;
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Rivogenlecleucel
Bellicum Pharmaceuticals, Inc.
- Phase 2 EUCTR2014-000584-41-GB Italy;Spain;United Kingdom;
Rivogenleucleucel
Bellicum Pharmaceuticals, Inc.
2017 Phase 2 EUCTR2014-000584-41-ES Italy;Spain;United Kingdom;
Stem Cell Transplantation
Masonic Cancer Center, University of Minnesota
2014 Phase 2 NCT02171104 United States;
St. Jude Children's Research Hospital
2004 - NCT00145587 United States;
Tehran University of Medical Sciences
2009 Phase 2/Phase 3 NCT01087398 Iran, Islamic Republic of;
Stem Cell or Umbilical Cord Blood Transplantation
Masonic Cancer Center, University of Minnesota
2007 Phase 2 NCT00638820 United States;
Systemic chemotherapy and antibodies
St. Jude Children's Research Hospital
2004 - NCT00145587 United States;
Total Lymphoid Irradiation
Masonic Cancer Center, University of Minnesota
2007 Phase 2 NCT00638820 United States;
Umbilical cord blood transplantation
Masonic Cancer Center, University of Minnesota
2008 Phase 2/Phase 3 NCT00775931 United States;