DDrare: Database of Drug Development for Rare Diseases

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-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
2	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
3	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
4	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-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

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

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

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

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