276. Achondroplasia
37 clinical trials,   29 drugs   (DrugBank: 6 drugs),   4 drug target genes,   25 drug target pathways

Searched query = "Achondroplasia"
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.

Search in Page e.g. "Phase 3", "Not recruiting", "Japan"
21 trials found
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-001055-40-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 2Australia;United Kingdom
2EUCTR2018-004364-66-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
3NCT03989947
(ClinicalTrials.gov)
June 12, 201922/5/2019An Extension Study to Evaluate Safety and Efficacy of BMN 111 in Children With AchondroplasiaA Phase 2 Open-Label Long-Term Extension Study to Evaluate the Safety and Efficacy of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: Active BMN 111: Daily subcutaneous injection of 15 ug/kg of BMN 111BioMarin PharmaceuticalNULLEnrolling by invitation15 MonthsN/AAll70Phase 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]
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
110Phase 3United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
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]
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
70Phase 2United 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
9NCT03583697
(ClinicalTrials.gov)
May 23, 201814/6/2018A Clinical Trial 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 MonthsAchondroplasiaDrug: BMN 111;Drug: PlaceboBioMarin PharmaceuticalNULLEnrolling by invitationN/A59 MonthsAll70Phase 2United States;Australia;Japan;United Kingdom
10EUCTR2015-003836-11-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
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11NCT03424018
(ClinicalTrials.gov)
December 12, 201728/12/2017An Extension Study to Evaluate the Efficacy and Safety 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 AchondroplasiaAchondroplasiaDrug: BMN 111BioMarin PharmaceuticalNULLActive, not recruiting6 YearsN/AAll119Phase 3United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom
12EUCTR2015-003836-11-ES
(EUCTR)
19/04/201710/03/2017A 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: 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: vorsoritide
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
110Phase 3France;United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
13EUCTR2015-003836-11-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]
BioMarin Pharmaceutical Inc.NULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
110Phase 3United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
14NCT03197766
(ClinicalTrials.gov)
December 12, 201623/5/2017A 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 AchondroplasiaAchondroplasiaDrug: BMN 111;Drug: PlaceboBioMarin PharmaceuticalNULLCompleted5 Years18 YearsAll121Phase 3United States;Australia;Germany;Japan;Spain;Turkey;United Kingdom
15NCT02724228
(ClinicalTrials.gov)
January 26, 201612/2/2016A Study to Evaluate Long-Term Safety, Tolerability, & Efficacy of BMN 111 in Children With Achondroplasia (ACH)A Phase 2, Open-Label, Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: BMN 111BioMarin PharmaceuticalNULLActive, not recruiting7 YearsN/AAll30Phase 2United States;Australia;France;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
16EUCTR2013-004137-32-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]
BioMarin Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
36Phase 2France;United States;Australia;United Kingdom
17NCT02055157
(ClinicalTrials.gov)
January 13, 201418/4/2013A Phase 2 Study of BMN 111 to Evaluate Safety, Tolerability, and Efficacy in Children With AchondroplasiaA Phase 2, Open-label, Sequential Cohort Dose-escalation Study of BMN 111 in Children With AchondroplasiaAchondroplasiaDrug: BMN 111BioMarin PharmaceuticalNULLCompleted5 Years14 YearsAll35Phase 2United States;Australia;France;United Kingdom
18NCT01590446
(ClinicalTrials.gov)
February 201214/3/2012A Study to Evaluate Safety and Tolerability of BMN 111 Administered to Healthy Adult VolunteersA Phase 1, Two-Part, Double-Blind, Placebo-Controlled Study to Evaluate Safety, Tolerability, and Pharmacokinetics of Single and Multiple Doses of BMN 111 Administered to Healthy Adult VolunteersAchondroplasiaDrug: BMN 111;Drug: Normal SalineBioMarin PharmaceuticalNULLCompleted22 Years45 YearsMale74Phase 1United States
19EUCTR2015-004004-30-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
20EUCTR2015-004004-30-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]
BioMarin Pharmaceutical Inc.NULLNA Female: yes
Male: yes
46Phase 2United States;France;Australia;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
21EUCTR2013-004137-32-FR
(EUCTR)
18/06/2015A 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: 18.0;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: TBD
Other descriptive name: MODIFIED RHCNP
BioMarin Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
24Phase 2United States;France;Australia;United Kingdom