Natriuretic peptide ( DrugBank: - )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
276軟骨無形成症21

276. 軟骨無形成症


臨床試験数 : 51 薬物数 : 34 - (DrugBank : 6) / 標的遺伝子数 : 4 - 標的パスウェイ数 : 26
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2022-002954-25-DK
(EUCTR)
24/01/202302/12/2022A phase 2b clinical trial to evaluate efficacy and safety of weekly doses of TransCon CNP compared with placebo in participants with achondroplasia aged 2 to 11 years of ageApproaCH: A Phase 2b, Multicenter, Double-Blind, Randomized, Placebo-controlled Trial evaluating Efficacy and Safety of Subcutaneous Doses of TransCon CNP Administered Once Weekly for 52 Weeks in Children with Achondroplasia followed by an Open Label Extension period - ApproaCH Achondroplasia (ACH) in prepubertal children
MedDRA version: 25.0;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 a multi-arm polyethylene glycol carrier molecule through a cleavable linker
Ascendis Pharma Growth Disorders A/SNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
80Phase 2United States;Canada;Spain;Ireland;Australia;Denmark;United Kingdom;New Zealand
2EUCTR2019-002754-22-PT
(EUCTR)
05/02/202118/06/2020A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension PeriodACcomplisH: 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 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period - 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 2United States;Portugal;Canada;Spain;Ireland;Denmark;Austria;Australia;Germany;United Kingdom;Switzerland;New Zealand
3EUCTR2020-001055-40-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
4EUCTR2019-002754-22-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
5EUCTR2019-002754-22-DK
(EUCTR)
02/07/202012/05/2020A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension PeriodACcomplisH: 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 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 25.0;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;Canada;Spain;Ireland;Austria;Australia;Denmark;Germany;United Kingdom;Switzerland;New Zealand
6EUCTR2019-002754-22-AT
(EUCTR)
09/06/202031/10/2019A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension PeriodACcomplisH: 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 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period - ACcomplisH Achondroplasia (ACH) in prepubertal children
MedDRA version: 25.0;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;Canada;Spain;Ireland;Denmark;Australia;Austria;Germany;United Kingdom;Switzerland;New Zealand
7EUCTR2019-002754-22-IE
(EUCTR)
01/11/201903/09/2019A Phase 2 study to evaluate the safety, efficacy and pharmacokinetics of TransCon CNP in prepubertal children with achondroplasia followed by an Open-Label Extension PeriodACcomplisH: 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 52 weeks in prepubertal children with achondroplasia followed by an Open-Label Extension Period. - 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;Canada;Spain;Ireland;Denmark;Austria;Australia;Germany;United Kingdom;Switzerland;New Zealand
8EUCTR2018-004364-66-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
9EUCTR2017-002404-28-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 States;Spain;Turkey;Australia;Germany;Japan;United Kingdom
10EUCTR2017-002404-28-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
11EUCTR2017-002404-28-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
12EUCTR2016-003826-18-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
13JPRN-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
14EUCTR2015-003836-11-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
15EUCTR2015-003836-11-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
16EUCTR2015-003836-11-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
17EUCTR2013-004137-32-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
18EUCTR2013-004137-32-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
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
20EUCTR2019-002754-22-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
21EUCTR2015-004004-30-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