276. 軟骨無形成症 Achondroplasia Clinical trials / Disease details
臨床試験数 : 46 / 薬物数 : 29 - (DrugBank : 6) / 標的遺伝子数 : 5 - 標的パスウェイ数 : 30
| 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 | NCT04554940 (ClinicalTrials.gov) | October 10, 2020 | 14/9/2020 | A Clinical Trial to Evaluate Safety of Vosoritide in At-risk Infants With Achondroplasia | A 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 Surgery | Achondroplasia | Biological: vosoritide | BioMarin Pharmaceutical | NULL | Recruiting | N/A | 12 Months | All | 20 | Phase 2 | Australia;United Kingdom |
| 2 | EUCTR2020-001055-40-GB (EUCTR) | 29/07/2020 | 19/05/2020 | Open-label clinical trial with an open-label extension to investigate the safety of BMN 111 in infants and young children with achondroplasia | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 20 | Phase 2 | United Kingdom;Australia | ||
| 3 | EUCTR2018-004364-66-GB (EUCTR) | 22/10/2019 | 11/09/2019 | A Phase 2 open-label long-term study of BMN 111 in children with Achondroplasia | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 70 | Phase 2 | United States;Australia;Japan;United Kingdom | ||
| 4 | EUCTR2017-002404-28-DE (EUCTR) | 07/03/2019 | 13/12/2018 | A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | Authorised-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): no | United Kingdom;Japan;Germany;Australia;Turkey;Spain;United States | ||
| 5 | EUCTR2017-002404-28-ES (EUCTR) | 08/11/2018 | 23/11/2018 | A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 110 | Phase 3 | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 6 | EUCTR2017-002404-28-GB (EUCTR) | 12/10/2018 | 15/06/2018 | A Phase 3 Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | Authorised-recruitment may be ongoing or finished | Female: yes Male: yes | 119 | Phase 3 | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 7 | EUCTR2016-003826-18-GB (EUCTR) | 12/09/2018 | 30/05/2018 | A Phase 2 Study to Evaluate the Safety and Efficacy of BMN 111 in Infants and Young Children with Achondroplasia | A 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. | NULL | Authorised-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): no | United States;Australia;Japan;United Kingdom | ||
| 8 | JPRN-JapicCTI-184167 | 19/7/2018 | 23/10/2018 | A Study to Evaluate the Efficacy and Safety of BMN 111 in Children With Achondroplasia | A Phase 3 Randomized, Double-Blind,Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of BMN 111 in Children with Achondroplasia | Achondroplasia | Intervention 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.) | NULL | complete | 5 | 18 | BOTH | 6 | Phase 3 | Japan, North America, Europe, Oceania |
| 9 | EUCTR2015-003836-11-DE (EUCTR) | 14/02/2018 | 21/08/2017 | A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | Not Recruiting | Female: yes Male: yes | 110 | Phase 3 | United States;Spain;Turkey;Australia;Germany;United Kingdom;Japan | ||
| 10 | EUCTR2015-003836-11-GB (EUCTR) | 20/01/2017 | 20/10/2016 | A Phase 3 Study to Evaluate the Safety and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | Authorised-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): no | United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom | ||
| 11 | EUCTR2013-004137-32-GB (EUCTR) | 03/04/2014 | 17/02/2014 | A Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | Not 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): no | France;United States;Australia;United Kingdom | ||
| 12 | EUCTR2015-004004-30-FR (EUCTR) | 10/06/2016 | A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | NA | Female: yes Male: yes | 46 | Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): no | United States;France;Australia;United Kingdom | |||
| 13 | EUCTR2015-004004-30-GB (EUCTR) | 07/03/2016 | A Long-term Study to Evaluate Safety, Tolerability, and Efficacy of BMN 111 in Children with Achondroplasia | A 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. | NULL | NA | Female: yes Male: yes | 30 | Phase 2 | United States;France;Australia;United Kingdom |