DDrare: Database of Drug Development for Rare Diseases

326. 大理石骨病
［臨床試験数：20，薬物数：52（DrugBank：14），標的遺伝子数：16，標的パスウェイ数：78］

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.

Drug = ACTIMMUNE; AP1903; AP1903 A594; Actimmune; Actimmune Registry; BPX-501; BPX-501 T cells; BPX-501 cells; Busulfan; Busulfan, Cyclophosphamide, Thymoglobulin, Fludarabine (Conditioning regimen); CALD HR-D (High-Risk, Regimen C); CALD HR-D (High-Risk, Regimen D); CALD SR-A (Standard-Risk, Regimen A); CALD SR-B (Standard-Risk, Regimen B); CD34+ enriched, T Cell Depleted donor stem cell product; Calcitriol; Campath; Campath, Busulfan, Clofarabine; Campath-1H; Clofarabine; Cyclophosphamide; Cyclophosphamide 30; Cyclophosphamide 40; Cyclosporin; Cyclosporin, Methotrexate (GVHD prophylaxis); Device: Miltenyi Biotec CliniMACS; Fludarabine; Fludarabine monophosphate; Hematopoetic Stem Cell Transplantation; IMD; IMD Preparative Regimen; Interferon Gamma; Interferon gamma; Interferon gamma-1b; Methotrexate; Osteopetrosis Haploidentical Only Preparative Regimen; Osteopetrosis Only Preparative Regimen; Other: Palliative Care; Procedure: Stem Cell Transplantation; Procedure: Stem Cell or Umbilical Cord Blood Transplantation; Procedure: Total Lymphoid Irradiation; Procedure: marrow graft transplantation; Procedure: umbilical cord blood transplantation; RP-L401; Radiation: Total Lymphoid Irradiation; RhPTH; Rimiducid; Rivogenlecleucel; Rivogenleucleucel; Stem Cell Transplantation; Systemic chemotherapy and antibodies; Thymoglobulin

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	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
2	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
3	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

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

NCT01087398
(ClinicalTrials.gov)

September 2009

13/3/2010

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

Phase 2;Phase 3

Iran, Islamic Republic of

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

Phase 2;Phase 3

United States

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

Phase 2;Phase 3

United States