276. 軟骨無形成症
[臨床試験数:37,薬物数:29(DrugBank:6),標的遺伝子数:4,標的パスウェイ数:25

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"
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-001189-13-DK
(EUCTR)
22/12/202007/10/2020Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasiaA PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT 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: Recifercept (proposed INN)
Product Code: PF-07256472
INN or Proposed INN: Recifercept (proposed INN)
Other descriptive name: TA-46
Pfizer Inc., 235 East 42nd Street, New York, NY 10017NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
63Phase 2Portugal;United States;Spain;Belgium;Australia;Denmark;United Kingdom;Italy;Japan
2NCT04638153
(ClinicalTrials.gov)
December 2, 202015/9/2020A Study Of Safety, Tolerability And Effectiveness Of Reciferecept In Children With AchondroplasiaA PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT IN CHILDREN WITH ACHONDROPLASIAAchondroplasiaBiological: ReciferceptPfizerNULLRecruiting3 Months10 YearsAll63Phase 2Belgium;Spain
3NCT04554940
(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
4EUCTR2020-001055-40-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
5EUCTR2019-002754-22-DE
(EUCTR)
28/07/202001/10/2019A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasiaACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: TransCon CNP 3.9 mg CNP-38/vial
Product Code: TransCon CNP
INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Other descriptive name: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Ascendis Pharma Growth Disorders A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Portugal;United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Australia;Denmark;Germany;New Zealand
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
6EUCTR2019-002754-22-DK
(EUCTR)
02/07/202012/05/2020A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasiaACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: TransCon CNP 3.9 mg CNP-38/vial
Product Code: TransCon CNP
INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Other descriptive name: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Ascendis Pharma Growth Disorders A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Portugal;United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Denmark;Australia;Germany;New Zealand
7NCT04085523
(ClinicalTrials.gov)
June 24, 20209/9/2019A Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of TransCon CNP Administered Once Weekly in Prepubertal Children With AchondroplasiaACcomplisH: A Phase 2, Multicenter, Double-blind, Randomized, Placebo-controlled, Dose Escalation Trial Evaluating Safety, Efficacy, and Pharmacokinetics of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 12 Months in Prepubertal Children With AchondroplasiaAchondroplasiaDrug: TransCon CNP;Drug: Placebo for TransCon CNPAscendis Pharma A/SNULLEnrolling by invitation2 Years10 YearsAll60Phase 2United States;Australia
8EUCTR2019-002754-22-AT
(EUCTR)
09/06/202031/10/2019A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasiaACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: TransCon CNP 3.9 mg CNP-38/vial
Product Code: TransCon CNP
INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Other descriptive name: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Ascendis Pharma Growth Disorders A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Portugal;United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Australia;Denmark;Germany;New Zealand
9EUCTR2019-002954-21-GB
(EUCTR)
31/03/202013/05/2020A study to evaluate Infigratinib in children with AchondroplasiaPhase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2 - PROPEL 2 Achondroplasia in Children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: Infigratinib phosphate
Product Code: BGJ398 (also known as BBP-831)
INN or Proposed INN: INFIGRATINIB
QED Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
78Phase 2United States;France;Canada;Spain;Australia;United Kingdom
10NCT04265651
(ClinicalTrials.gov)
March 10, 202029/1/2020Study of Infigratinib in Children With AchondroplasiaPhase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL 2AchondroplasiaDrug: Infigratinib 0.016 mg/kg;Drug: Infigratinib 0.032 mg/kg;Drug: Infigratinib 0.064 mg/kg;Drug: Infigratinib 0.128 mg/kgQED Therapeutics, Inc.NULLRecruiting3 Years11 YearsAll60Phase 2Australia;Spain;United Kingdom
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
11EUCTR2019-002754-22-IE
(EUCTR)
01/11/201903/09/2019A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasiaACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: TransCon CNP 3.9 mg CNP-38/vial
Product Code: TransCon CNP
INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Other descriptive name: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Ascendis Pharma Growth Disorders A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
60Phase 2Portugal;United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Australia;Denmark;Germany;New Zealand
12EUCTR2018-004364-66-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
13NCT03989947
(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
14EUCTR2017-002404-28-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
15EUCTR2017-002404-28-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
16EUCTR2017-002404-28-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
17EUCTR2016-003826-18-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
18JPRN-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
19NCT03583697
(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
20EUCTR2015-003836-11-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
21NCT03424018
(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
22EUCTR2015-003836-11-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
23EUCTR2015-003836-11-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
24NCT03197766
(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
25NCT02724228
(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
26EUCTR2013-004137-32-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
27NCT02055157
(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
28NCT01435629
(ClinicalTrials.gov)
November 22, 201214/9/2011A Survey Collecting Data on Adult Height in Patients With Achondroplasia Treated With SomatropinOpen-label, Multicenter, Observational, Non-intervention Study to Retrospectively Evaluate the Efficacy of Norditropin® (Adult Height) in Patients With Achondroplasia/Hypochondroplasia Enrolled in the GH-1941 Study [Follow-up Survey]Genetic Disorder;AchondroplasiaDrug: somatropinNovo Nordisk A/SNULLCompletedN/AN/AAll81N/AJapan
29NCT01590446
(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
30NCT01516229
(ClinicalTrials.gov)
May 1, 199719/1/2012Special Survey for Long Term ApplicationSpecific Survey of Norditropin® in Achondroplasia: Survey for Long-term ApplicationGenetic Disorder;AchondroplasiaDrug: somatropinNovo Nordisk A/SNULLCompleted1 Year15 YearsAll395N/AJapan
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
31EUCTR2019-002754-22-PT
(EUCTR)
18/06/2020A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasiaACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: TransCon CNP 3.9 mg CNP-38/vial
Product Code: TransCon CNP
INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Other descriptive name: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Ascendis Pharma Growth Disorders A/SNULLNAFemale: yes
Male: yes
60Phase 2Portugal;United States;Spain;Ireland;Austria;Italy;Switzerland;United Kingdom;Canada;Australia;Denmark;Germany;New Zealand
32EUCTR2015-004004-30-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
33EUCTR2017-000701-21-DE
(EUCTR)
27/04/2017A study looking at growth measurements of children with achondroplasia A Multicenter, Multinational Clinical Assessment Study for Pediatric Patients 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.NULLNot Recruiting Female: yes
Male: yes
500Phase 1France;United States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
34EUCTR2019-002754-22-GB
(EUCTR)
09/09/2019A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasiaACcomplisH: A Phase 2, multicenter, double-blind, randomized, placebo-controlled, dose escalation trial evaluating safety, efficacy, and pharmacokinetics of subcutaneous doses of TransCon CNP administered once weekly for 12 months in prepubertal children with achondroplasia - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 20.0;Level: LLT;Classification code 10000452;Term: Achondroplasia;System Organ Class: 100000004850;Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]
Product Name: TransCon CNP 3.9 mg CNP-38/vial
Product Code: TransCon CNP
INN or Proposed INN: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Other descriptive name: C-TYPE NATRIURETIC PEPTIDE CONJUGATED TO MULTI-ARM POLYETHYLENE GLYCOL CARRIER THROUGH A CLEAVABLE LINKER
Ascendis Pharma Growth Disorders A/SNULLNAFemale: yes
Male: yes
60Phase 2Portugal;United States;Spain;Ireland;Austria;United Kingdom;Italy;Switzerland;Canada;Australia;Denmark;Germany;New Zealand
35EUCTR2020-001189-13-PT
(EUCTR)
29/09/2020Phase 2 study of safety, tolerability, PK and efficacy of recifercept in achondroplasiaA PHASE 2 MULTIPLE DOSE, RANDOMIZED STUDY TO ASSESS THE SAFETY, TOLERABILITY, PHARMACOKINETICS AND EFFICACY OF RECIFERCEPT 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: Recifercept (proposed INN)
Product Code: PF-07256472
INN or Proposed INN: Recifercept (proposed INN)
Other descriptive name: TA-46
Pfizer Inc.NULLNAFemale: yes
Male: yes
63Phase 2United States;Portugal;Canada;Spain;Belgium;Denmark;Australia;United Kingdom;Japan;Italy
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
36EUCTR2015-004004-30-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
37EUCTR2013-004137-32-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