276. 軟骨無形成症 Achondroplasia Clinical trials / Disease details


臨床試験数 : 46 薬物数 : 30 - (DrugBank : 6) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 30

  
13 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT04554940
(ClinicalTrials.gov)
October 10, 202014/9/2020A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With AchondroplasiaA Randomized, Controlled, Open-label Clinical Trial With an Open-label Extension to Investigate the Safety of Vosoritide in Infants and Young Children With Achondroplasia at Risk of Requiring Cervicomedullary Decompression SurgeryAchondroplasiaBiological: vosoritideBioMarin PharmaceuticalNULLRecruitingN/A12 MonthsAll20Phase 2Australia;United Kingdom
2EUCTR2020-001055-40-GB
(EUCTR)
29/07/202019/05/2020Open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasiaA randomized, controlled, open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia at risk of requiring cervicomedullary decompression surgery achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
20Phase 2United Kingdom;Australia
3EUCTR2018-004364-66-GB
(EUCTR)
22/10/201911/09/2019A Phase 2 open-label long-term study of BMN 111 in children with AchondroplasiaA Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safetyand Efficacy of BMN 111 in Children with Achondroplasia Achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: Modified recombinant human c-type
Product Code: Natriuretic peptide
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: Modified recombinant human c-type
Product Code: Natriuretic peptide
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
70Phase 2United States;Australia;Japan;United Kingdom
4EUCTR2017-002404-28-DE
(EUCTR)
07/03/201913/12/2018A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia Achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
110 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited Kingdom;Japan;Germany;Australia;Turkey;Spain;United States
5EUCTR2017-002404-28-ES
(EUCTR)
08/11/201823/11/2018A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia Achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
6EUCTR2017-002404-28-GB
(EUCTR)
12/10/201815/06/2018A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 3, Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia Achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
119Phase 3United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
7EUCTR2016-003826-18-GB
(EUCTR)
12/09/201830/05/2018A Phase 2 Study to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with AchondroplasiaA Phase 2 Randomized, Double-Blind, Placebo-Controlled Clinical Trial to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia, Age 0 to < 60 Months Achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
70 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Australia;Japan;United Kingdom
8JPRN-JapicCTI-184167
19/7/201823/10/2018A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With AchondroplasiaA Phase 3 Randomized, Double-Blind,Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia AchondroplasiaIntervention name : modified recombinant human C-type natriuretic peptide
INN of the intervention : Vosoritide
Dosage And administration of the intervention : Subcutaneous injection of 15 micro g/kg of BMN 111 daily
Control intervention name : Placebo
INN of the control intervention : -
Dosage And administration of the control intervention : Subcutaneous injection of placebo daily
BioMarin Pharmaceutical Inc.(ICCC:EPS International Holdings Co. Ltd.)NULLcomplete518BOTH6Phase 3Japan, North America, Europe, Oceania
9EUCTR2015-003836-11-DE
(EUCTR)
14/02/201821/08/2017A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
110Phase 3United States;Spain;Turkey;Australia;Germany;United Kingdom;Japan
10EUCTR2015-003836-11-GB
(EUCTR)
20/01/201720/10/2016A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 3 Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia. achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
110 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
11EUCTR2013-004137-32-GB
(EUCTR)
03/04/201417/02/2014A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children with Achondroplasia achondroplasia
MedDRA version: 19.1;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: Vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
36 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noFrance;United States;Australia;United Kingdom
12EUCTR2015-004004-30-FR
(EUCTR)
10/06/2016A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia achondroplasia
MedDRA version: 19.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850 ;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLNA Female: yes
Male: yes
46 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;France;Australia;United Kingdom
13EUCTR2015-004004-30-GB
(EUCTR)
07/03/2016A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with AchondroplasiaA Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia achondroplasia
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Body processes [G] - Bones and nerves physological processes [G11]
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
Product Name: modified recombinant human C-type natriuretic peptide
Product Code: BMN 111
INN or Proposed INN: vosoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLNAFemale: yes
Male: yes
30Phase 2United States;France;Australia;United Kingdom