DDrare: Database of Drug Development for Rare Diseases

274. 骨形成不全症
［臨床試験数：78，薬物数：87（DrugBank：19），標的遺伝子数：14，標的パスウェイ数：74］

Searched query = "Osteogenesis Imperfecta"
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 = ACLASTA; AMG 162; AMG 785; ANTI-SCLEROSTIN MONOCLONAL ANTIBODY; Aclasta; Actonel 5mg Film Coated Tablets; Actonel Once a Week 35 mg film-coated tablets; Actonel/Optinate 5mg film-coated tablets; Alendronate; Alendronic acid; Anti-sclerostin monoclonal antibody; BOOST cells; BPS804; Bisphosphonates; Blood sample; Bone marrow transplant; Bonefos; Busulfan; Calcichew tablets; Calcium; Celulas madre mesenquimales troncales adultas alogenicas de médula ósea no expandida; Cyclophosphamide; Cyclosporin; Células mesenquimales troncales adultas alogénicas de médula ósea no expandidas; Células mesenquimales troncales adultas alogénicas de médula ósea no expandidas(ref cruzada PEI nº 12-088); DENOSUMAB; Denosumab; Denosumab - - Immunoglobulin G2 Human Monoclonal Antibody to RANK Ligand; Denosumab - Immunoglobulin G2 Human Monoclonal Antibody to RANK Ligand; Denusomab; Disodium Pamidronate 15mg (15mg strength); Expanded human first trimester fetal liver-derived mesenchymal stem cells; Forsteo; Fresolimumab; GENOTROPIN*1TBF 5,3MG (16UI); H05AA02; High-dose vitamin D (2000 IU per day); Humatrope; Ibandronic acid; Ibandronic acid solution; Mesenchymal Stem Cells; Mesenchymal Stromal Cells; NERIXIA*INFUS IV CONC 2F 100MG; Optinate 5 mg filmcoated tablets; Other: Bone Marrow Cell Transplantation; Other: No active treatment; Other: saline; Pamidronate; Pamidronate (Aredia); Pamidronate sodium; Procedure: Mesenchymal Stem Cell Transplantation; Prolia; REF CRUZADA PEI Nº 12-088; RITUXIMAB; ROMOSOZUMAB; Radiation: Irradiation, Total Body; Risedronate; Risedronate Oral Tablet; Risedronate Sodium; Risedronate sodium; Risedronate sodium (Actonel); Risedronate sodium 2.5mg; Risedronate sodium 5mg; Risedronato sódico 2.5mg; Risedronato sódico 5mg; Risedronic acid; Rituximab; Romosozumab; Setrusumab; Somatropin; Standard-dose vitamin D (400IU per day); Teriparatide; Teriparatide (FORTEO); Teriparatide Pen Injector; VARI; Vitamin D; XGEVA; Xgeva; ZOL446; ZOLEDRONIC ACID; Zoledronic Acid; Zoledronic Acid Kern Pharma 4 mg/100 mL solution for infusion, generic medicinal product; Zoledronic Acid Kern Pharma 4 mg/100 mL solution for infusion, generic medicinal product.; Zoledronic Acid Kern Pharma 4 mg/100 mL solution for infusion,generic medicinal product; Zoledronic acid; Zometa; Ácido risedrónico

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	NCT03706482 (ClinicalTrials.gov)	August 12, 2019	5/10/2018	Boost Brittle Bones Before Birth	An Exploratory, Open Label, Multiple Dose, Multicentre Phase I/II Trial Evaluating Safety and Efficacy of Postnatal or Prenatal and Postnatal Intravenous Administration of Allogeneic Expanded Fetal Mesenchymal Stem Cells for the Treatment of Severe Osteogenesis Imperfecta Compared With a Combination of Historical and Untreated Prospective Controls	Osteogenesis Imperfecta	Biological: BOOST cells	Cecilia Götherström	Karolinska University Hospital;Great Ormond Street Hospital for Children NHS Foundation Trust;University College, London;Universitätsklinikum Köln;UMC Utrecht;Leiden University Medical Center;Lund University	Recruiting	N/A	12 Months	All	210	Phase 1;Phase 2	Sweden
2	EUCTR2015-003699-60-SE (EUCTR)	28/09/2018	23/07/2018	Treatment of severe congenital Brittle bone disease after or before and after birth.	An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of postnatal or prenatal and postnatal intravenous administration of allogeneic expanded fetal mesenchymal stem cells for the treatment of severe Osteogenesis Imperfecta compared with a combination of historical and untreated prospective controls.	Treatment of Osteogenesis Imperfecta (OI) type III and severe type IV.;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]		Karolinska Institutet	NULL	Authorised-recruitment may be ongoing or finished			Female: yes Male: yes	210	Phase 1;Phase 2	Sweden
3	NCT02172885 (ClinicalTrials.gov)	April 2014	12/6/2014	Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta	Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta	Osteogenesis Imperfecta	Biological: Mesenchymal Stem Cells	Hospital de Cruces	Hospital Universitario Getafe;Hospital Infantil Universitario Niño Jesús, Madrid, Spain	Completed	6 Months	12 Years	All	2	Phase 1	Spain

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

NCT03706482
(ClinicalTrials.gov)

August 12, 2019

5/10/2018

Boost Brittle Bones Before Birth

An Exploratory, Open Label, Multiple Dose, Multicentre Phase I/II Trial Evaluating Safety and Efficacy of Postnatal or Prenatal and Postnatal Intravenous Administration of Allogeneic Expanded Fetal Mesenchymal Stem Cells for the Treatment of Severe Osteogenesis Imperfecta Compared With a Combination of Historical and Untreated Prospective Controls

Osteogenesis Imperfecta

Biological: BOOST cells

Cecilia Götherström

Karolinska University Hospital;Great Ormond Street Hospital for Children NHS Foundation Trust;University College, London;Universitätsklinikum Köln;UMC Utrecht;Leiden University Medical Center;Lund University

Recruiting

N/A

12 Months

All

210

Phase 1;Phase 2

Sweden

EUCTR2015-003699-60-SE
(EUCTR)

28/09/2018

23/07/2018

Treatment of severe congenital Brittle bone disease after or before and after birth.

An exploratory, open label, multiple dose, multicentre phase I/II trial evaluating safety and efficacy of postnatal or prenatal and postnatal intravenous administration of allogeneic expanded fetal mesenchymal stem cells for the treatment of severe Osteogenesis Imperfecta compared with a combination of historical and untreated prospective controls.

Treatment of Osteogenesis Imperfecta (OI) type III and severe type IV.;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

Karolinska Institutet

NULL

Authorised-recruitment may be ongoing or finished

Female: yes
Male: yes

210

Phase 1;Phase 2

Sweden

NCT02172885
(ClinicalTrials.gov)

April 2014

12/6/2014

Mesenchymal Stem Cell Based Therapy for the Treatment of Osteogenesis Imperfecta

Osteogenesis Imperfecta

Biological: Mesenchymal Stem Cells

Hospital de Cruces

Hospital Universitario Getafe;Hospital Infantil Universitario Niño Jesús, Madrid, Spain

Completed

6 Months

12 Years

All

Phase 1

Spain