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	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

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

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

Female: yes
Male: yes

175

Phase 2

Spain;Italy;United Kingdom