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	NCT02666768 (ClinicalTrials.gov)	February 22, 2016	23/6/2015	ACTIMMUNE in Intermediate Osteopetrosis	Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis	Osteopetrosis	Drug: Interferon gamma -1b	Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center	University of Minnesota;Horizon Pharma Ireland, Ltd., Dublin Ireland	Completed	1 Year	N/A	All	5	Phase 2	United States
2	NCT02584608 (ClinicalTrials.gov)	January 1, 2016	20/10/2015	Use of ACTIMMUNE in Patients With ADO2	Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis	Autosomal Dominant Osteopetrosis Type 2	Drug: ACTIMMUNE	Indiana University	Horizon Pharma Ireland, Ltd., Dublin Ireland	Completed	3 Years	65 Years	All	12	Phase 2	United States
3	NCT00043329 (ClinicalTrials.gov)	January 2002	7/8/2002	Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis	Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis	Osteopetrosis	Drug: Actimmune Registry	InterMune	NULL	Completed	N/A	N/A	Both	6		United States
4	NCT00004402 (ClinicalTrials.gov)	November 1999	18/10/1999	Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis		Osteopetrosis	Drug: calcitriol;Drug: interferon gamma	FDA Office of Orphan Products Development	Medical University of South Carolina	Completed	N/A	10 Years	Both	30	Phase 3	NULL

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

NCT02666768
(ClinicalTrials.gov)

February 22, 2016

23/6/2015

ACTIMMUNE in Intermediate Osteopetrosis

Open-label Early Phase 2 Study With a Single Arm of Interferon Gamma-1b Treatment of Osteopetrosis

Osteopetrosis

Drug: Interferon gamma -1b

Lundquist Institute for Biomedical Innovation at Harbor-UCLA Medical Center

University of Minnesota;Horizon Pharma Ireland, Ltd., Dublin Ireland

Completed

1 Year

N/A

All

Phase 2

United States

NCT02584608
(ClinicalTrials.gov)

January 1, 2016

20/10/2015

Use of ACTIMMUNE in Patients With ADO2

Phase 2a Study of Interferon Gamma-1b for the Treatment of Autosomal Dominant Type 2 Osteopetrosis

Autosomal Dominant Osteopetrosis Type 2

Drug: ACTIMMUNE

Indiana University

Horizon Pharma Ireland, Ltd., Dublin Ireland

Completed

3 Years

65 Years

All

Phase 2

United States

NCT00043329
(ClinicalTrials.gov)

January 2002

7/8/2002

Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis

Post-Marketing Surveillance Study of Actimmune (Interferon Gamma-1b) in Patients With Severe Malignant Osteopetrosis

Osteopetrosis

Drug: Actimmune Registry

InterMune

NULL

Completed

N/A

Both

United States

NCT00004402
(ClinicalTrials.gov)

November 1999

18/10/1999

Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis

Osteopetrosis

Drug: calcitriol;Drug: interferon gamma

FDA Office of Orphan Products Development

Medical University of South Carolina

Completed

N/A

10 Years

Both

Phase 3

NULL